10-Q

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington D.C. 20549

FORM 10-Q

(Mark One)

☒	QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
	For the quarterly period ended June 30, 2021
	OR
☐	TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
	For the transition period from _______ to _______
	Commission file number: 001-36294

uniQure N.V.

(Exact name of Registrant as specified in its charter)

The Netherlands (State or other jurisdiction of incorporation or organization) Not applicable (I.R.S. Employer Identification No.)

Paasheuvelweg 25

1105 BP Amsterdam, The Netherlands

(Address of principal executive offices) (Zip Code)

+31-20-240-6000

(Registrant’s telephone number, including area code)

Securities registered pursuant to Section 12(b) of the Act:

Title of each class:	Trading Symbol(s)	Name of each exchange on which registered
Ordinary Shares, par value €0.05	QURE	The Nasdaq Global Select Market

Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days.    Yes ⌧ No ◻.  

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files).    Yes ⌧ No ◻.  

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, smaller reporting company or an emerging growth company. See the definitions of “large accelerated filer” “accelerated filer” and “smaller reporting company” and “emerging growth company” in Rule 12b-2 of the Exchange Act.

Large accelerated filer  ⌧	Accelerated filer  ◻
Non-accelerated filer ◻	Smaller reporting company ☐ Emerging growth company ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13 (a) of the Exchange Act  ◻

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act.)    Yes ☐ No ⌧

As of July 22, 2021, the registrant had 46,073,373 ordinary shares, par value €0.05, outstanding.

TABLE OF CONTENTS

		Page
	PART I – FINANCIAL INFORMATION
Item 1	Financial Statements	2
Item 2	Management’s Discussion and Analysis of Financial Condition and Results of Operations	19
Item 3	Quantitative and Qualitative Disclosures About Market Risk	41
Item 4	Controls and Procedures	41
	PART II – OTHER INFORMATION
Item 1	Legal Proceedings	42
Item 1A	Risk Factors	43
Item 2	Unregistered Sales of Equity Securities and Use of Proceeds	72
Item 3	Defaults Upon Senior Securities	72
Item 4	Mine Safety Disclosures	72
Item 5	Other Information	72
Item 6	Exhibits	72

SPECIAL CAUTIONARY NOTICE REGARDING FORWARD-LOOKING STATEMENTS

This Quarterly Report on Form 10-Q contains “forward-looking statements” as defined under federal securities laws. Forward-looking statements are based on our current expectations of future events and many of these statements can be identified using terminology such as “believes,” “expects,” “anticipates,” “plans,” “may,” “will,” “projects,” “continues,” “estimates,” “potential,” “opportunity” and similar expressions. These forward-looking statements, include, but are not limited to, statements related to the COVID-19 coronavirus pandemic, our collaboration and license agreements, our cash runway, the advancement of our clinical trials, and the impact of regulatory actions on our regulatory submission timelines.

Forward-looking statements are only predictions based on management’s current views and assumptions and involve risks and uncertainties, and actual results could differ materially from those projected or implied. The most significant factors known to us that could materially adversely affect our business, operations, industry, financial position or future financial performance include those discussed in Part II, Item 1A “Risk Factors,” as well as those discussed in Part I, Item 2 “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and elsewhere in this Quarterly Report on Form 10-Q, as well as other factors which may be identified from time to time in our other filings with the Securities and Exchange Commission (“SEC”), including our most recent Annual Report on Form 10-K filed with the SEC on March 1, 2021, or in the documents where such forward-looking statements appear. You should carefully consider that information before you make an investment decision.

You should not place undue reliance on these forward-looking statements, which speak only as of the date that they were made. Our actual results or experience could differ significantly from those anticipated in the forward-looking statements and from historical results, due to the risks and uncertainties described in this Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, and in our Annual Report on Form 10-K for the year ended December 31, 2020, including in “Part I, Item 1A. Risk Factors,” as well as others that we may consider immaterial or do not anticipate at this time. These cautionary statements should be considered in connection with any written or oral forward-looking statements that we may make in the future or may file or furnish with the SEC. We do not undertake any obligation to release publicly any revisions to these forward-looking statements after completion of the filing of this Quarterly Report on Form 10-Q to reflect later events or circumstances or to reflect the occurrence of unanticipated events. All forward-looking statements attributable to us are expressly qualified in their entirety by these cautionary statements.

In addition, with respect to all of our forward-looking statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.

1

Part I – FINANCIAL INFORMATION

Item 1.Financial Statements

uniQure N.V.

UNAUDITED CONSOLIDATED BALANCE SHEETS

	June 30,		December 31,
	2021		2020
	(in thousands, except share and per share amounts)
Current assets
Cash and cash equivalents	$	677,330	$	244,932
Accounts receivable		2,705		6,618
Prepaid expenses		10,861		4,337
Other current assets and receivables		3,328		3,024
Total current assets		694,224		258,911
Non-current assets
Property, plant and equipment, net of accumulated depreciation of $37.3 million as of June 30, 2021 and $35.2 million as of December 31, 2020, respectively		38,371		32,328
Operating lease right-of-use assets		26,782		26,086
Intangible assets, net		2,637		3,361
Goodwill		525		542
Deferred tax assets		15,965		16,419
Other non-current assets		5,760		2,748
Total non-current assets		90,040		81,484
Total assets	$	784,264	$	340,395
Current liabilities
Accounts payable	$	16,817	$	3,772
Accrued expenses and other current liabilities		23,526		18,038
Current portion of operating lease liabilities		5,752		5,524
Total current liabilities		46,095		27,334
Non-current liabilities
Long-term debt		70,780		35,617
Operating lease liabilities, net of current portion		30,534		30,403
Other non-current liabilities		3,247		3,136
Total non-current liabilities		104,561		69,156
Total liabilities		150,656		96,490
Commitments and contingencies
Shareholders' equity
Ordinary shares, €0.05 par value: 80,000,000 shares authorized as of June 30, 2021 and 60,000,000 shares authorized as of December 31, 2020 and 46,050,250 and 44,777,799 ordinary shares issued and outstanding as of June 30, 2021 and December 31, 2020, respectively		2,788		2,711
Additional paid-in-capital		1,062,234		1,016,018
Accumulated other comprehensive (loss) / income		(4,595)		9,907
Accumulated deficit		(426,819)		(784,731)
Total shareholders' equity		633,608		243,905
Total liabilities and shareholders' equity	$	784,264	$	340,395

The accompanying notes are an integral part of these unaudited consolidated financial statements.

2

uniQure N.V.

UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND

COMPREHENSIVE INCOME (LOSS)

	Three months ended June 30,				Six months ended June 30,
	2021		2020		2021		2020
	(in thousands, except share and per share amounts)				(in thousands, except share and per share amounts)
License revenues		462,400		—		462,400		—
License revenues from related party		—		1,530		—		1,577
Collaboration revenues		1,468		—		1,922		—
Collaboration revenues from related party		—		5		—		62
Total revenues		463,868		1,535		464,322		1,639
Operating expenses:
Cost of contract revenues		(23,178)		—		(23,178)		—
Research and development expenses		(32,747)		(28,401)		(64,777)		(54,414)
Selling, general and administrative expenses		(17,299)		(11,511)		(30,300)		(20,583)
Total operating expenses		(73,224)		(39,912)		(118,255)		(74,997)
Other income		7,590		669		7,942		1,526
Other expense		(226)		(500)		(459)		(839)
Income / (loss) from operations		398,008		(38,208)		353,550		(72,671)
Interest income		37		81		77		903
Interest expense		(1,902)		(970)		(3,453)		(1,945)
Foreign currency gains / (losses), net		6,583		(3,645)		11,209		957
Other non-operating gains, net		—		191		—		2,206
Income / (loss) before income tax expense	$	402,726	$	(42,551)	$	361,383	$	(70,550)
Income tax expense		(3,258)		—		(3,471)		—
Net income / (loss)	$	399,468	$	(42,551)	$	357,912	$	(70,550)
Other comprehensive (loss) / gain:
Foreign currency translation adjustments		(6,942)		4,647		(14,502)		(630)
Total comprehensive gain / (loss)	$	392,526	$	(37,904)	$	343,410	$	(71,180)
Earnings per ordinary share - basic
Basic net income / (loss) per ordinary share	$	8.68	$	(0.96)	$	7.82	$	(1.59)
Earnings per ordinary share - diluted
Diluted net income / (loss) per ordinary share	$	8.51	$	(0.96)	$	7.67	$	(1.59)
Weighted average shares - basic		46,037,900		44,387,463		45,754,766		44,333,460
Weighted average shares - diluted		46,929,870		44,387,463		46,678,835		44,333,460

The accompanying notes are an integral part of these unaudited consolidated financial statements.

3

uniQure N.V.

UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ EQUITY

FOR THE THREE-MONTH PERIOD ENDED JUNE 30

						Accumulated
				Additional		other				Total
	Ordinary shares			paid-in		comprehensive		Accumulated		shareholders’
	No. of shares	Amount		capital		(loss)		deficit		equity
	(in thousands, except share and per share amounts)
Balance at March 31, 2020	44,299,596	$	2,683	$	992,136	$	(11,966)	$	(687,706)	$	295,147
Loss for the period	—		—		—		—		(42,551)		(42,551)
Other comprehensive income	—		—		—		4,647		—		4,647
Exercise of share options	139,178		9		2,461		—		—		2,470
Restricted and performance share units distributed during the period	4,427		—		—		—		—		—
Share-based compensation expense	—		—		5,723		—		—		5,723
Issuance of ordinary shares relating to employee stock purchase plan	1,204		—		69		—		—		69
Balance at June 30, 2020	44,444,405	$	2,692	$	1,000,389	$	(7,319)	$	(730,257)	$	265,505
Balance at March 31, 2021	45,924,729	$	2,780	$	1,049,850	$	2,347	$	(826,287)	$	228,690
Income for the period	—		—		—		—		399,468		399,468
Other comprehensive loss	—		—		—		(6,942)		—		(6,942)
Issuance of ordinary shares	61,845		3		1,862		—		—		1,865
Tax benefit past share issuance cost	—		—		2,977		—		—		2,977
Exercise of share options	55,194		4		515		—		—		519
Restricted share units distributed during the period	8,482		1		(1)		—		—		—
Share-based compensation expense	—		—		7,031		—		—		7,031
Balance at June 30, 2021	46,050,250	$	2,788	$	1,062,234	$	(4,595)	$	(426,819)	$	633,608

The accompanying notes are an integral part of these unaudited consolidated financial statements.

4

uniQure N.V.

UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ EQUITY

FOR THE SIX-MONTH PERIOD ENDED JUNE 30

						Accumulated
				Additional		other				Total
	Ordinary shares			paid-in		comprehensive		Accumulated		shareholders’
	No. of shares	Amount		capital		(loss)		deficit		equity
	(in thousands, except share data)
Balance at December 31, 2019	43,711,954	$	2,651	$	986,803	$	(6,689)	$	(659,707)	$	323,058
Loss for the period	—		—		—		—		(70,550)		(70,550)
Other comprehensive loss	—		—		—		(630)		—		(630)
Exercise of share options	203,940		12		3,390		—		—		3,402
Restricted and performance share units distributed during the period	525,506		29		(29)		—		—		—
Share-based compensation expense	—		—		10,078		—		—		10,078
Issuance of ordinary shares relating to employee stock purchase plan	3,005		—		147		—		—		147
Balance at June 30, 2020	44,444,405	$	2,692	$	1,000,389	$	(7,319)	$	(730,257)	$	265,505
Balance at December 31, 2020	44,777,799	$	2,711	$	1,016,018	$	9,907	$	(784,731)	$	243,905
Income for the period	—		—		—		—		357,912		357,912
Other comprehensive loss	—		—		—		(14,502)		—		(14,502)
Issuance of ordinary shares	921,730		55		29,509		—		—		29,564
Tax benefit past share issuance cost	—		—		2,977		—		—		2,977
Exercise of share options	71,976		5		906		—		—		911
Restricted and performance share units distributed during the period	277,571		17		(17)		—		—		—
Share-based compensation expense	—		—		12,792		—		—		12,792
Issuance of ordinary shares relating to employee stock purchase plan	1,174		—		49		—		—		49
Balance at June 30, 2021	46,050,250	$	2,788	$	1,062,234	$	(4,595)	$	(426,819)	$	633,608

The accompanying notes are an integral part of these unaudited consolidated financial statements.

5

uniQure N.V.

UNAUDITED CONSOLIDATED STATEMENTS OF CASH FLOWS

	Six months ended June 30,
	2021		2020
		(in thousands)
Cash flows from operating activities
Net income / (loss)	$	357,912	$	(70,550)
Adjustments to reconcile net income / (loss) to net cash generated from / used in operating activities:
Depreciation and amortization		3,667		3,505
Share-based compensation expense		12,792		10,078
Change in fair value of derivative financial instruments		-		(2,206)
Unrealized foreign exchange gains, net		(12,279)		(908)
Other non-cash items, net		539		(1,600)
Changes in operating assets and liabilities:
Accounts receivable, prepaid expenses, and other current assets and receivables		(3,355)		1,050
Accounts payable		10,444		(981)
Accrued expenses, other liabilities, and operating leases		5,486		(1,332)
Net cash generated from / (used) in operating activities		375,206		(62,944)
Cash flows from investing activities
Purchases of intangible assets		-		(2,214)
Purchases of property, plant, and equipment		(6,191)		(2,392)
Net cash used in investing activities		(6,191)		(4,606)
Cash flows from financing activities
Proceeds from loan increment, net of debt issuance costs		34,603		-
Proceeds from issuance of ordinary shares		30,899		-
Share issuance costs from issuance of ordinary shares		(1,334)		-
Proceeds from issuance of ordinary shares related to employee stock option and purchase plans		960		3,549
Net cash generated from financing activities		65,128		3,549
Currency effect on cash, cash equivalents and restricted cash		(1,665)		223
Net increase / (decrease) in cash, cash equivalents and restricted cash		432,478		(63,778)
Cash, cash equivalents and restricted cash at beginning of period		247,680		380,726
Cash, cash equivalents and restricted cash at the end of period	$	680,158	$	316,948
Cash and cash equivalents	$	677,330	$	314,265
Restricted cash related to leasehold and other deposits		2,828		2,683
Total cash, cash equivalents and restricted cash	$	680,158	$	316,948
Supplemental cash flow disclosures:
Cash paid for interest	$	(2,943)	$	(2,545)
Non-cash increase in accounts payables and accrued expenses and other current liabilities related to purchases of intangible assets and property, plant, and equipment	$	3,488	$	1,021

The accompanying notes are an integral part of these unaudited consolidated financial statements.

6

1General business information

uniQure (the “Company”) was incorporated on January 9, 2012 as a private company with limited liability (besloten vennootschap met beperkte aansprakelijkheid) under the laws of the Netherlands. The Company is a leader in the field of gene therapy and seeks to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results. The Company’s business was founded in 1998 and was initially operated through its predecessor company, Amsterdam Molecular Therapeutics (AMT) Holding N.V (“AMT”). In 2012, AMT undertook a corporate reorganization, pursuant to which uniQure B.V. acquired the entire business and assets of AMT and completed a share-for-share exchange with the shareholders of AMT. Effective February 10, 2014, in connection with its initial public offering, the Company converted into a public company with limited liability (naamloze vennootschap) and changed its legal name from uniQure B.V. to uniQure N.V.

The Company is registered in the trade register of the Chamber of Commerce (Kamer van Koophandel) in Amsterdam, the Netherlands under number 54385229. The Company’s headquarters are in Amsterdam, the Netherlands, and its registered office is located at Paasheuvelweg 25, Amsterdam 1105 BP, the Netherlands and its telephone number is +31 20 240 6000.

The Company’s ordinary shares are listed on the Nasdaq Global Select Market and trade under the symbol “QURE”.

2Summary of significant accounting policies

2.1Basis of preparation

The Company prepared these unaudited consolidated financial statements in compliance with generally accepted accounting principles in the United States of America (“U.S. GAAP”) and applicable rules and regulations of the Securities and Exchange Commission (“SEC”) regarding interim financial reporting. Any reference in these notes to applicable guidance is meant to refer to authoritative U.S. GAAP as found in the Accounting Standards Codification (“ASC”) and Accounting Standards Update (“ASU”) of the Financial Accounting Standards Board (“FASB”).

The unaudited consolidated financial statements are presented in U.S. dollars, except where otherwise indicated. Transactions denominated in currencies other than U.S. dollars are presented in the transaction currency with the U.S. dollar amount included in parenthesis, converted at the foreign exchange rate as of the transaction date.

2.2Unaudited interim financial information

The interim financial statements and related disclosures are unaudited, have been prepared on the same basis as the annual financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for a fair statement of the financial position, results of operations and changes in financial position for the period presented.

Certain information and footnote disclosures normally included in annual financial statements prepared in accordance with U.S. GAAP have been omitted. The results of operations for the six months ended June 30, 2021, are not necessarily indicative of the results to be expected for the full year ending December 31, 2021 or for any other future year or interim period. The accompanying financial statements should be read in conjunction with the audited financial statements and the related notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, filed with the SEC on March 1, 2021.

2.3Use of estimates

The preparation of the financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities at the date of the financial statements and reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates.

7

2.4Accounting policies

The principal accounting policies applied in the preparation of these unaudited consolidated financial statements are described in the Company’s audited financial statements as of and for the year ended December 31, 2020, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, filed with the SEC on March 1, 2021. There have been no material changes in the Company’s significant accounting policies during the six months ended June 30, 2021 except as noted below.

The Company determined its revenue recognition policies related to the CSL Behring Agreement (defined below) when the CSL Behring Agreement became fully effective on May 6, 2021. Refer to Note 3 (“Collaboration arrangements and concentration of credit risk”) for further detail.

The Company for the first time recognized net income in the three and six months ended June 30, 2021. Net income / (loss) per ordinary share was calculated in accordance with ASC 260, Earnings Per Share. Refer to Note 11 (“Basic and diluted earnings per share”) for further detail.

2.5Recent accounting pronouncements

There have been no new accounting pronouncements or changes to accounting pronouncements during the six months ended June 30, 2021, as compared to the recent accounting pronouncements described in Note 2.3.22 of the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, which could be expected to materially impact the Company’s unaudited consolidated financial statements.

3 Collaboration arrangements and concentration of credit risk

CSL Behring collaboration

On June 24, 2020, (“Signing”), uniQure biopharma B.V., a wholly-owned subsidiary of uniQure N.V., entered into a commercialization and license agreement (the “CSL Behring Agreement”) with CSL Behring LLC, (“CSL Behring”), pursuant to which CSL Behring received exclusive global rights to etranacogene dezaparvovec, the Company’s investigational gene therapy for patients with hemophilia B, (the “Product”). On May 6, 2021, the CSL Behring Agreement became fully effective (“Closing”) upon the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 on May 5, 2021.

Pursuant to the CSL Behring Agreement, the Company has received a $450.0 million upfront cash payment and $12.4 million in other payments related to the transfer of the license, during the three and six months ended June 30, 2021. The Company is eligible to receive up to $1.6 billion in additional payments based on regulatory and commercial milestones. The CSL Behring Agreement also provides that the Company will be eligible to receive tiered double-digit royalties in a range of up to a low-twenties percent of net sales of the Product based on sales thresholds.

On Signing, the Company and CSL Behring entered into a development and commercial supply agreement, pursuant to which, among other things, the Company will supply the Product to CSL Behring at an agreed-upon price commensurate with the stand-alone selling price (“SSP”). The Company will be responsible for supplying development and commercial Product until such time that these capabilities may be transferred to CSL Behring or a designated contract manufacturing organization. The Company will be completing the HOPE-B clinical trial and the validation of the manufacturing process on behalf of CSL Behring, as well as provide further development services if requested by CSL Behring. Activities related to on-demand development services as well as activities related to the completing the HOPE-B clinical trial will be reimbursed by CSL Behring at an agreed full-time-employee rate (“FTE-rate”) and CSL Behring will also reimburse agreed third-party expenses incurred in relation to performing these activities. The validation of the manufacturing process as well as the development and validation of a next generation manufacturing process (“Manufacturing Development Services”) will be reimbursed through a future milestone payment. If completed after certain contractually agreed dates, the milestone payment will be reduced in accordance with a contractually agreed mechanism.

The Company concluded that CSL Behring is a customer in accordance with Topic 606.

8

The Company identified two material performance obligations related to the CSL Behring Agreement:

(i) Sale of the exclusive global rights to the Product (“License”); and

(ii) Generate information to support the regulatory approval of the current and next generation manufacturing process of Product and to provide any such information generated to CSL Behring (“Manufacturing Development”).

These performance obligations are considered distinct from one another, as CSL Behring can benefit from the identified service either on its own or together with other resources that are readily available to CSL Behring, and as the performance obligation is separately identifiable from other performance obligations in the CSL Behring Agreement. The Company continued to develop the Product between Signing and Closing and performed certain reimbursable activities to fulfill the transfer of the global rights (“Additional Covenants” and together with the License the “License Sale”). The Additional Covenants are not considered distinct from the performance obligation to sell the license to CSL Behring as CSL Behring could not benefit from the Additional Covenants on their own, or have these activities be performed with readily available resources.

The Company determined that the fixed upfront payment of $450.0 million and the $12.4 million that the Company received in relation to the Additional Covenants should be allocated to the License Sale. In addition, the Company concluded that variable milestone payments, sales milestone payments and royalties should be allocated to the License Sale performance obligation as well. The Company determined that the sale of the License was completed on May 6, 2021, when it transferred the License and CSL Behring assumed full responsibility for the development and commercialization of the Product. At Closing, the Company evaluated the amount of potential payments and the likelihood that the payments will be received. The Company utilized the most likely amount method to estimate the variable consideration to be included in the transaction price. Since the Company cannot control the achievement of regulatory and first commercial sales milestones, the Company concluded that the potential payments are constrained as of Closing. The Company determined that it will recognize revenue related to these payments only to the extent that it becomes probable that no significant reversal of recognized cumulative revenue will occur thereafter. The Company will include payments related to sales milestones in the transaction price when their achievement becomes probable, and it will include royalties on the sale of Product once these have been earned. The Company recognized $462.4 million of revenues related to the License Sale in the three and six months ended June 30, 2021. The costs associated with fulfilling this performance obligation are presented as “Cost of contract revenue” with the exception of the cost incurred prior to the Closing, which had been presented as “Research and development expenses”.

The Company determined that the variable milestone payment related to Manufacturing Development Services should be allocated to the Manufacturing Development performance obligation. The Company concluded that this milestone payment represents the SSP of the services based on the estimated cost of providing the services including a reasonable margin. The services related to Manufacturing Development will be provided between Closing and the completion of an agreed manufacturing development plan. The variable consideration will be reduced if the Company does not complete the development by pre-agreed dates. The Company utilized the most likely amount method to estimate the variable consideration to be included in the transaction price. Completion of Manufacturing Development is partially dependent on the timing of regulatory submissions by CSL Behring as well as regulatory approvals of the developed manufacturing processes. Since the Company cannot control the timing or outcome of any regulatory decisions, the Company concluded that it would recognize revenue related to this payment when it becomes probable that the milestone has been achieved. The Company did not recognize any revenue related to Manufacturing Development during the three and six months ended June 30, 2021.

The Company recognized $0.4 million of collaboration revenue in the three and six months ended June 30, 2021, compared to nil in the same periods in the prior year.  The Company generates such collaboration revenue from services rendered in relation to completing the HOPE-B clinical trial on behalf of CSL Behring. CSL Behring may request additional development services or to request the Company to support the transfer of manufacturing to a party designated by CSL Behring. These collaboration services will be reimbursed at the pre-agreed FTE-rate. The Company concluded that these rights at Closing do not represent material rights.

9

As of June 30, 2021, the Company has an accounts receivable of $1.3 million from CSL Behring. The Company collected the $2.1 million of accounts receivable related to reimbursable contract fulfillment costs that was outstanding as of December 31, 2020.

Bristol-Myers Squibb collaboration

In May 2015, the Company and Bristol-Myers Squibb (“BMS”) entered into a collaboration and license agreement and various related agreements with BMS (“BMS CLA”).

The initial four-year research term under the collaboration terminated on May 21, 2019. On December 1, 2020, the Company and BMS amended the BMS CLA (“amended BMS CLA”). Under the amended BMS CLA, BMS is limited to four Collaboration Targets. BMS may until November 30, 2021 replace up to two of these four Collaboration Targets with up to two new targets in the field of cardiovascular disease. The Company continues to be eligible to receive research, development, and regulatory milestone payments of up to $217.0 million for each Collaboration Target if defined milestones are achieved.

For as long as any of the four Collaboration Targets are being advanced, BMS may place a purchase order to be supplied with research, clinical and commercial supplies. Subject to the terms of the amended BMS CLA, BMS has the right to terminate the research, clinical and commercial supply relationships, and has certain remedies for failures of supply, up to and including technology transfer for any such failure that otherwise cannot be reasonably resolved. Both BMS and the Company may agree to a technology transfer of manufacturing capabilities pursuant to the terms of the amended BMS CLA.

The amended BMS CLA does not extend the initial four-year research term. BMS may place purchase orders to provide limited services primarily related to analytical and development efforts in respect of the four Collaboration Targets. BMS may request such services for a period not to exceed the earlier of (i) the completion of all activities under a Research Plan and (ii) either (A) three years after the last replacement target has been designated by BMS during the one-year replacement period ending on November 30, 2021, or (B) November 30, 2023 if no replacement targets are designated. BMS continues to reimburse the Company for these services.

The Company evaluated the impact of the amended BMS CLA in relation to its performance obligation to provide access to BMS to its technology and know-how in the field of gene therapy and to participate in joint steering committee and other governing bodies (“License Revenue”).

The Company determined that its remaining performance obligation under the amended BMS CLA was immaterial and recognized the remaining balance of unrecognized License Revenue as of November 30, 2020. The Company includes variable consideration related to any research, development, and regulatory milestone payments, in the transaction price once it is considered probable that including these payments in the transaction price would not result in the reversal of cumulative revenue recognized. Due to the significant uncertainty surrounding the development of gene-therapy product candidates and the dependence on BMS’s performance and decisions, the Company does not generally consider this probable and did not record any License Revenue during the six months ended June 30, 2021.

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4  Fair value measurement

The Company measures certain financial assets and liabilities at fair value, either upon initial recognition or for subsequent accounting or reporting. U.S. GAAP requires disclosure of methodologies used in determining the reported fair values, and establishes a hierarchy of inputs used when available. The three levels of the fair value hierarchy are described below:

● Level 1 – Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company can access at the measurement date.

● Level 2 – Valuations based on quoted prices for similar assets or liabilities in markets that are not active or models for which the inputs are observable, either directly or indirectly.

● Level 3 – Valuations that require inputs that reflect the Company’s own assumptions that are both significant to the fair value measurement and are unobservable.

To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized as Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement.

The carrying amount of cash and cash equivalents, accounts receivable, prepaid expenses, other assets, accounts payable, accrued expenses and other current liabilities reflected in the Consolidated balance sheets approximate their fair values due to their short-term maturities.

The following table sets forth the Company’s assets and liabilities that are required to be measured at fair value on a recurring basis as of June 30, 2021, and December 31, 2020:

	Quoted prices in active markets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)		Total		Classification in Consolidated balance sheets
		(in thousands)
At December 31, 2020
Assets:
Cash, cash equivalents and restricted cash	$	247,680	$	—	$	—	$	247,680	Cash and cash equivalents; other non-current assets
Total assets	$	247,680	$	—	$	—	$	247,680
Liabilities:
Derivative financial instruments		—		—		2,645		2,645	Other non-current liabilities
Total liabilities	$	—	$	—	$	2,645	$	2,645
At June 30, 2021
Assets:
Cash, cash equivalents and restricted cash	$	680,158	$	—	$	—	$	680,158	Cash and cash equivalents; other non-current assets
Total assets	$	680,158	$	—	$	—	$	680,158
Liabilities:
Derivative financial instruments	$	—	$	—	$	2,645	$	2,645	Other non-current liabilities
Total liabilities	$	—	$	—	$	2,645	$	2,645

The Company recognized no gains or losses related to changes in the fair market value of derivative financial instruments during the three and six months ended June 30, 2021.

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Derivative financial instruments

The Company issued derivative financial instruments related to its collaboration with BMS.

In 2015, the Company granted BMS two warrants that were subsequently terminated in connection with the amendment to the BMS CLA on December 1, 2020.

On December 1, 2020, the Company and BMS agreed that upon the consummation of a change of control transaction of uniQure that occurs prior to December 1, 2026 or BMS’ delivery of a target cessation notice for all four Collaboration Targets, the Company (or its third party acquirer) shall pay to BMS a one-time, non-refundable, non-creditable cash payment of $70.0 million, provided that (x) if $70.0 million is greater than five percent (5.0%) of the net proceeds (as contractually defined) from such change of control transaction, the payment shall be an amount equal to five percent of such net proceeds, and (y) if $70.0 million is less than one percent of such net proceeds, the change of control payment shall be an amount equal to one percent of such net proceeds (“CoC-payment”). The Company has not consummated any change of control transaction as of June 30, 2021 that would obligate it to make a CoC-payment.

The Company determined that the CoC-payment should be recorded as a derivative financial liability as of December 1, 2020 and that subsequent changes in the fair market value of this derivative financial liability should be recorded in profit and loss. The fair market value of the derivative financial liability is materially impacted by probability that market participants assign to the likelihood of the occurrence of a change of control transaction that would give rise to a CoC-payment. This probability represents an unobservable input. The Company determined the fair market value of the derivative financial liability by using a present value model based on expected cash flow. The expected cash flows are materially impacted by the probability that market participants assign to the likelihood of the occurrence of a change of control transaction within the biotechnology industry. The Company estimated this unobservable input using the best information available as of June 30, 2021 and December 31, 2020. The Company obtained reasonably available market information that it believed market participants would use in determining the likelihood of the occurrence of a change-of control transaction within the biotechnology industry. Selecting and evaluating market information involves considerable judgement and uncertainty. Based on all such information and its judgment the Company estimated that the fair market value of the derivative financial liability (presented within “Other non-current liabilities”) as of June 30, 2021 and December 31, 2020 was $2.6 million.

5 Accrued expenses and other current liabilities

Accrued expenses and other current liabilities include the following items:

	June 30,		December 31,
	2021		2020
	(in thousands)
Accruals for services provided by vendors-not yet billed	$	16,734	$	8,269
Personnel related accruals and liabilities		6,792		7,687
Contract liability		—		2,082
Total	$	23,526	$	18,038

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6 Long-term debt

On June 14, 2013, the Company entered into a venture debt loan facility with Hercules Capital, Inc. (formerly known as Hercules Technology Growth Capital, Inc.) (“Hercules”), which was amended and restated on June 26, 2014, and again on May 6, 2016 (“2016 Amended Facility”). On December 6, 2018, the Company signed an amendment that both refinanced the then-existing $20.0 million 2016 Amended Facility and allowed the Company to draw down an additional $15.0 million (“2018 Amended Facility”). The 2018 Amended Facility extended the loan’s maturity date from May 1, 2020 until June 1, 2023. The interest-only period was initially extended from November 2018 to January 1, 2021 and was further extended to January 1, 2022 as a result of raising more than $90.0 million in equity financing in September 2019. The interest only period was again further extended to June 1, 2023 as a result of the January 2021 amendment (see below).  The interest rate is adjustable and is the greater of (i) 8.85% and (ii) 8.85% plus the prime rate less 5.50% per annum. Under the 2018 Amended Facility, the Company owes a back-end fee of 4.95% of the outstanding debt. In addition, in May 2020 the Company paid a back-end fee of $1.0 million in relation to the 2016 Amended Facility.

On January 29, 2021, the Company and Hercules amended the 2018 Amended Facility (“2021 Amended Facility”). Pursuant to the 2021 Amended Facility, Hercules agreed to an additional Facility of $100.0 million (“Tranche B”), increasing the aggregate principal amount of the term loan facilities from $35.0 million to up to $135.0 million. On January 29, 2021, the Company drew down $35.0 million of the Tranche B. The Company may draw down the remaining $65.0 million under the Tranche B in a series of one or more advances of not less than $20.0 million each until December 15, 2021. Advances under Tranche B bear interest at a rate equal to the greater of (i) 8.25% or (ii) 8.25% plus the prime rate, less 3.25% per annum. The principal balance and all accrued but unpaid interest on advances under Tranche B is due on June 1, 2023, which date may be extended by the Company by up to two twelve-month periods. Advances under Tranche B may not be prepaid prior to July 29, 2021, following which the Company may prepay all such advances without charge. The Company owes a back-end fee of 4.85% of amounts outstanding under Tranche B. The back-end fee is reduced if prepayment occurs at an earlier date.

The amortized cost (including interest due presented as part of accrued expenses and other current liabilities) of the 2018 Amended Facility and 2021 Amended Facility was $71.3 million as of June 30, 2021, compared to $35.9 million amortized cost for the 2018 Amended Facility as of December 31, 2020, and is recorded net of discount and debt issuance costs. The foreign currency gain on the facilities in the three months ended June 30, 2021, was $0.9 million and the foreign currency loss on the facilities in the six months ended June 30, 2021, was $2.2 million, compared to a foreign currency gain of $0.7 million and $0.0 million during the same periods in 2020 for the 2018 Amended Facility.

Interest expense associated with the 2018 Amended Facility and 2021 Amended Facility during the three and six months ended June 30, 2021, was $1.8 million and $3.4 million, compared to $0.9 million and $1.8 million, respectively, during the same periods in 2020 for the 2018 Amended Facility.

As a covenant in the 2018 Amended Facility and 2021 Amended Facility, the Company has periodic reporting requirements and is required to keep a minimum cash balance deposited in bank accounts in the United States, equivalent to the lesser of (i) 65% of the outstanding balance of principal due or (ii) 100% of worldwide cash and cash equivalents. This restriction on cash and cash equivalents only relates to the location of the cash and cash equivalents, and such cash and cash equivalents can be used at the discretion of the Company. In combination with other covenants, the 2018 Amended Facility and 2021 Amended Facility restricts the Company’s ability to, among other things, incur future indebtedness and obtain additional debt financing, to make investments in securities or in other companies, to transfer assets, to perform certain corporate changes, to make loans to employees, officers, and directors, and to make dividend payments and other distributions to its shareholders. The Company secured the facilities by directly or indirectly pledging its total assets of $784.3 million with the exception of $103.5 million of cash and cash equivalents and other current assets held by uniQure N.V.

The 2018 Amended Facility and 2021 Amended Facility contain provisions that include the occurrence of a material adverse effect, as defined therein, which would entitle Hercules to declare all principal, interest and other amounts owed by the Company immediately due and payable. As of June 30, 2021, the Company was in material compliance with all covenants and provisions.

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7Shareholders’ Equity

On March 1, 2021, the Company entered into a Sales Agreement with SVB Leerink LLC (“SVB Leerink”) with respect to an at-the-market (“ATM”) offering program, under which the Company may, from time to time in its sole discretion, offer and sell through SVB Leerink, acting as agent, its ordinary shares, up to an aggregate offering price of $200.0 million. The Company will pay SVB Leerink a commission equal to 3% of the gross proceeds of the sales price of all ordinary shares sold through it as sales agent under the Sales Agreement. In March and April 2021, the Company issued 921,730 ordinary shares at a weighted average price of $33.52 per share, with net proceeds of $29.6 million, after deducting underwriting discounts and net of offering expenses. The Company defers direct, incremental costs associated to this offering, except for the commission costs to SVB Leerink, which are a reduction to additional paid-in capital, and will deduct these costs from additional paid-in capital in the consolidated balance sheets proportionately to the amount of proceeds raised. During the three and six months ended June 30, 2021, $0.3 million and $1.3 million of direct, incremental costs were deducted from additional paid-in capital.

Following the release of the valuation allowance related to the years 2011 to 2018, the Company recognized the tax benefits of share issuance costs incurred in 2014, 2015, 2017 and 2018. This resulted in an increase of additional paid-in capital of $3.0 million in the three and six months ended June 30, 2021.

8Share-based compensation

The Company’s share-based compensation plans include the 2014 Amended and Restated Share Option Plan (the “2014 Plan”) and inducement grants under Rule 5653(c)(4) of the Nasdaq Global Select Market with terms similar to the 2014 Plan (together the “2014 Plans”). At the annual general meeting of shareholders in June 2021, the Company’s shareholders approved amendments of the 2014 Plan, increasing the shares authorized for issuance by 4,000,000 to a total of 12,601,471.

a) 2014 Plans

Share-based compensation expense recognized by classification included in the Consolidated statements of operations and comprehensive loss in relation to the 2014 Plans for the periods indicated below was as follows:

	Three months ended June 30,				Six months ended June 30,
	2021		2020		2021		2020
	(in thousands)				(in thousands)
Research and development	$	3,286	$	2,889	$	5,960	$	5,271
Selling, general and administrative		3,735		2,825		6,815		4,783
Total	$	7,021	$	5,714	$	12,775	$	10,054

Share-based compensation expense recognized by award type was as follows:

	Three months ended June 30,				Six months ended June 30,
	2021		2020		2021		2020
	(in thousands)				(in thousands)
Award type
Share options	$	3,350	$	2,746	$	6,190	$	4,954
Restricted share units		3,222		2,209		5,782		3,653
Performance share units		449		759		803		1,447
Total	$	7,021	$	5,714	$	12,775	$	10,054

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As of June 30, 2021, the unrecognized share-based compensation expense related to unvested awards under the 2014 Plans were:

	Unrecognized		Weighted average
	share-based		remaining
	compensation		period for
	expense		recognition
	(in thousands)		(in years)
Award type
Share options	$	34,855	2.99
Restricted share units		24,960	2.25
Performance share units		792	0.58
Total	$	60,607	2.66

The Company satisfies the exercise of share options and vesting of Restricted Share Units (“RSUs”) and Performance Share Units (“PSUs”) through newly issued ordinary shares.

Share options

Share options are priced on the date of grant and, except for certain grants made to non-executive directors, vest over a period of four years. The first 25% vests after one year from the initial grant date and the remainder vests in equal quarterly installments over years two, three and four. Certain grants to non-executive directors vest in full after one year. Any options that vest must be exercised by the tenth anniversary of the initial grant date.

The following tables summarize option activity under the Company’s 2014 Plans for the six months ended June 30, 2021:

	Options
	Number of	Weighted average
	ordinary shares	exercise price
Outstanding at December 31, 2020	2,659,279	$	28.13
Granted	914,118	$	36.36
Forfeited	(74,397)	$	43.19
Expired	(9,093)	$	44.14
Exercised	(71,976)	$	12.65
Outstanding at June 30, 2021	3,417,931	$	30.29
Thereof, fully vested and exercisable at June 30, 2021	1,774,059	$	21.68
Thereof, outstanding and expected to vest after June 30, 2021	1,643,872	$	39.58

Total weighted average grant date fair value of options issued during the period (in $ millions)	$	19.4
Proceeds from option sales during the period (in $ millions)	$	0.9

The fair value of each option issued is estimated at the respective grant date using the Hull & White option pricing model with the following weighted-average assumptions:

	Three months ended June 30,		Six months ended June 30,
Assumptions	2021	2020	2021	2020
Expected volatility	75%	70%	75%	70%
Expected terms	10 years	10 years	10 years	10 years
Risk free interest rate	1.72% - 1.85%	0.76% - 0.83%	1.21% - 1.85%	0.76% - 1.44%
Expected dividend yield	0%	0%	0%	0%

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Restricted share units (“RSUs”)

The following table summarizes the RSUs activity for the six months ended June 30, 2021:

	RSU
		Weighted average
	Number of	grant-date fair
	ordinary shares	value
Non-vested at December 31, 2020	467,344	$	43.56
Granted	481,821	$	36.50
Vested	(145,203)	$	39.89
Forfeited	(31,067)	$	42.86
Non-vested at June 30, 2021	772,895	$	39.87
Total weighted average grant date fair value of RSUs granted during the period (in $ millions)		$	17.6

RSUs vest over one to three years. RSUs granted to non-executive directors vest one year from the date of grant.

Performance share units (“PSUs”)

The following table summarizes the PSUs activity for the six months ended June 30, 2021:

	PSU
		Weighted average
	Number of	grant-date fair
	ordinary shares	value
Non-vested at December 31, 2020	212,614	$	42.32
Vested	(132,368)	$	33.09
Forfeited	(2,916)	$	57.56
Non-vested at June 30, 2021	77,330	$	57.56

The PSUs will vest on the third anniversary of the grant, subject to the grantee’s continued employment.

b) Employee Share Purchase Plan (“ESPP”)

In June 2018, the Company’s shareholders adopted and approved an ESPP allowing the Company to issue up to 150,000 ordinary shares. The ESPP is intended to qualify under Section 423 of the Internal Revenue Code of 1986. Under the ESPP, employees are eligible to purchase ordinary shares through payroll deductions, subject to any plan limitations. The purchase price of the ordinary shares on each purchase date is equal to 85% of the lower of the closing market price on the offering date and the closing market price on the purchase date of each three-month offering period. During the six months ended June 30, 2021, 1,174 ordinary shares were issued under the ESPP compared to 3,005 during the same period in 2020. As of June 30, 2021, a total of 130,852 ordinary shares remain available for issuance under the ESPP plan compared to a total of 135,202 as of June 30, 2020.

9 Other income

Other income during the three and six months ended June 30, 2021, was $7.6 million and $7.9 million, compared to $0.7 million and $1.5 million, respectively, during the same periods in 2020.

Other income in 2020 and 2021 includes income from payments received from European authorities to subsidize the Company’s research and development efforts in the Netherlands as well as income from subleasing part of the Amsterdam facility.

In addition, other income during the three and six months ended June 30, 2021 includes $1.3 million of employee retention credit received under the U.S. Coronavirus Aid, Relief, and Economic Security Act, as well as $3.0 million related to 69,899 shares in VectorY B.V. received in conjunction with a settlement agreement entered into in April 2021. No such income was recorded in 2020.

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10 Income taxes

The Company recorded $3.3 million in income tax expense in the three months ended June 30, 2021, and $3.5 million in the six months ended June 30, 2021. The Company recorded no income tax expense during the same periods in 2020.

As of December 31, 2020, the Company had recorded a full valuation allowance against its Dutch net deferred tax assets. On May 6, 2021, the CSL Behring Agreement became effective (refer to Note 3 “Collaboration arrangements and concentration of credit risk”). The Company recorded $462.4 million of license revenue related to closing the transaction. The Company intends to record such revenue in its Dutch tax return related to the 12-month period ended December 31, 2020. As such, the Company expects to file a return showing a taxable profit in the Netherlands in 2020, which would result in the consumption of substantially all of its Dutch net operating losses for the years 2011 to 2018. The Company’s remaining Dutch net operating tax losses carried forward would relate to 2019. The Dutch government on June 4, 2021 enacted legislation, whereby such net operating tax losses can be carried forward indefinitely. The Company expects to continue incurring tax losses for the foreseeable future including 2021. As such, the Company retained its full valuation allowance related to the net deferred assets as of June 30, 2021. The adjustment to the Company’s 2020 filing position resulting in the release and consumption of prior year net operating tax losses carried forward was treated as a discrete event in the three months ended June 30, 2021. The Company allocated part of the release to the tax benefit of share issuance cost incurred in 2014, 2015, 2017 and 2018. This resulted in an increase of additional paid-in capital as well as deferred tax expenses of $3.0 million.

The Company released its valuation allowance against the Company’s deferred tax assets in the United States as of December 31, 2020. The Company recorded $0.2 million and $0.5 million deferred tax expense in relation to its operations in the United States during the three and six months ended June 30, 2021, respectively. The Company recorded a nil net deferred tax expense in the prior year as it had recorded a valuation allowance against its net deferred tax assets in the United States as of June 30, 2020.

The effective income tax rates of respectively -0.8% and -1.0% during the three and six months ended June 30, 2021, is substantially lower than the enacted rated of 25% in the Netherlands as the Company recorded a valuation allowance against its remaining net deferred tax assets in the Netherlands as of December 31, 2020, and June 30, 2021.

11Basic and diluted earnings per share

Basic net income / (loss) per ordinary share is computed by dividing net income / (loss) for the period by the weighted average number of ordinary shares outstanding during the period. Diluted earnings per ordinary share are calculated by adjusting the weighted average number of ordinary shares outstanding, assuming conversion of all potentially dilutive ordinary shares. For the three months and six months ended June 30, 2021, dilutive net income per ordinary share is computed using the treasury method.

The following table provides the calculation of basic and diluted earnings per ordinary share:

	Three months ended June 30,				Six months ended June 30,
	2021		2020		2021		2020
	(in thousands)				(in thousands)
Numerator:
Net income / (loss) attributable to ordinary shares	$	399,468	$	(42,551)	$	357,912	$	(70,550)
		399,468		(42,551)		357,912		(70,550)
Denominator:
Weighted-average number of ordinary shares outstanding - basic		46,037,900		44,387,463		45,754,766		44,333,460
Stock options under 2014 Plans and previous plan		797,165		—		824,101		—
Non-vested RSUs and PSUs		92,681		—		98,514		—
Employee share purchase plan		2,124		—		1,454		—
Weighted-average number of ordinary shares outstanding - diluted		46,929,870		44,387,463		46,678,835		44,333,460

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The following table presents ordinary share equivalents that were excluded from the calculation of diluted net income / (loss) per ordinary share for the three and six months ended June 30, 2021 and 2020 as the effect of their inclusion would have been anti-dilutive.

	Three months ended June 30,				Six months ended June 30,
	2021		2020		2021		2020
	(in thousands)				(in thousands)
Anti-dilutive ordinary share equivalents
Stock options under 2014 Plans and previous plan		2,634,766		2,893,733		2,607,830		2,893,733
Non-vested RSUs and PSUs		757,544		645,636		751,711		645,636
Employee share purchase plan		1,414		254		1,802		254
BMS warrants (derecognized as of December 1, 2020 - see Note 4, "Fair value measurement")		—		8,060,500		—		8,060,500
Total anti-dilutive ordinary share equivalents		3,393,724		11,600,123		3,361,343		11,603,143

The anti-dilutive ordinary shares are presented without giving effect to the application of the treasury method or exercise prices that exceeded the price of the Company’s ordinary shares as of June 30, 2020. In addition, the BMS warrants were not exercisable as of June 30, 2020. This would have resulted in a lower number of potentially dilutive ordinary shares as some stock option grants as well as the BMS warrants would have been excluded.

12Subsequent event

Business combination

On June 22, 2021, the Company announced that it entered into a definitive transaction agreement under which the Company will acquire Corlieve Therapeutics S.A.S. (“Corlieve”, together the “Corlieve Transaction”). The Corlieve Transaction has been approved by the Boards of both companies and does not require the Company’s shareholder approval. The transaction is subject to customary closing conditions as well as review by the French Ministry of Economy, Finance and Recovery (Ministère de l’Economie, des Finances et de la Relance) pursuant to articles L.151-3 and R.151-1 and seq. of the French Code Monétaire et Financier. Currently, the transaction is anticipated to be completed early in the third quarter of 2021.

The Corlieve Transaction strengthens the Company’s pre-clinical program pipeline. Corlieve’s lead gene therapy program employs miRNA silencing technology to target suppression of aberrantly expressed kainate receptors in the hippocampus of patients with temporal lobe epilepsy (“TLE”) and will be known as AMT-260.

Upon closing of the Corlieve Transaction, the Company expects to pay EUR 46.3 million in cash. The Company will fund the acquisition from its cash position. Corlieve’s shareholders are eligible to receive the following additional payments of which up to 25% will be payable in the Company’s ordinary shares at the Company’s election: up to EUR 43.7 million in development milestones through Phase I/II and EUR 160.0 million in milestones associated with Phase III development and the approvals of AMT-260 in the U.S and European Union.

The Corlieve Transaction is expected to be accounted for as a business combination, which requires that assets acquired and liabilities assumed be recognized at their fair value as of the acquisition date.

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Item 2.Management’s Discussion and Analysis of Financial Condition and Results of Operations

The following Management’s Discussion and Analysis of Financial Condition and Results of Operations (“MD&A”) is intended to help the reader understand our results of operations and financial condition. This MD&A is provided as a supplement to, and should be read in conjunction with, our unaudited consolidated financial statements and the accompanying notes thereto and other disclosures included in this Quarterly Report on Form 10-Q, including the disclosures under Part II, Item 1A “Risk Factors,” and our audited financial information and the notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2020, which was filed with the Securities and Exchange Commission ( the “SEC”) on March 1, 2021. Our unaudited consolidated financial statements have been prepared in accordance with generally accepted accounting principles in the U.S. (“U.S. GAAP”) and unless otherwise indicated are presented in U.S. dollars.

Overview

We are a leader in the field of gene therapy and seek to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results. We are advancing a focused pipeline of innovative gene therapies, including product candidates for the treatment of hemophilia B, which effective May 6, 2021, we licensed to CSL Behring pursuant to the CSL Behring Agreement (as defined below), and Huntington’s disease. We believe our validated technology platform and manufacturing capabilities provide us distinct competitive advantages, including the potential to reduce development risk, cost, and time to market. We produce our Adeno-associated virus (“AAV”) -based gene therapies in our own facilities with a proprietary, commercial-scale, current good manufacturing practices (“cGMP”)-compliant, manufacturing process. We believe our Lexington, Massachusetts-based facility is one of the world’s most versatile gene therapy manufacturing facilities.

Business Developments

Below is a summary of our recent significant business developments:

Acquisition of Corlieve Therapeutics

On June 21, 2021, we entered into a share and purchase agreement (“SPA”) to acquire all outstanding shares of Corlieve Therapeutics S.A.S (“Corlieve”). Corlieve’s lead gene therapy program employs miRNA silencing technology to target suppression of aberrantly expressed kainate receptors in the hippocampus of patients with temporal lobe epilepsy (“TLE”). TLE affects approximately 1.3 million people in the U.S. and Europe alone, of which approximately 800,000 patients are unable to adequately control acute seizures with currently approved anti-epileptic therapies. Patients with refractory TLE experience increased morbidity, excess mortality, and poor quality of life. The lead program will be known as AMT-260.

The transaction is subject to customary closing conditions as well as review by the French Ministry of Economy, Finance and Recovery (Ministère de l’Economie, des Finances et de la Relance) pursuant to articles L.151-3 and R.151-1 and seq. of the French Code Monétaire et Financier. Currently, the transaction is anticipated to be completed early in the third quarter of 2021. Upon closing of the Corlieve Transaction we expect to pay EUR 46.3 million in cash to acquire all outstanding ordinary shares of Corlieve. In accordance with the SPA we owe up to EUR 43.7 million in development milestones through Phase I/II and EUR 160.0 million in milestones associated with Phase III development and the approvals of AMT-260 in the U.S and European Union. At our election we can pay up to 25% of such milestone payments in our ordinary shares. The Corlieve transaction is expected to be accounted for as a business combination, which requires that assets acquired and liabilities assumed be recognized at their fair value as of the acquisition date.

Corlieve has an established license and collaboration agreement with REGENXBIO Inc. that includes an exclusive license to AAV9 for the specific genetic target of AMT-260. Under the license and collaboration agreement, REGENXBIO Inc. is eligible to receive milestone payments and royalties on net sales of AMT-260.

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CSL Behring commercialization and license agreement

On June 24, 2020, (“Signing”), uniQure biopharma B.V., a wholly-owned subsidiary of uniQure N.V., entered into a commercialization and license agreement (as amended, the “CSL Behring Agreement”) with CSL Behring LLC (“CSL Behring”) pursuant to which CSL Behring received exclusive global rights to etranacogene dezaparvovec, our investigational gene therapy for patients with hemophilia B (the “Product”).

The effectiveness of the transaction contemplated by the CSL Behring Agreement was contingent on the completion of review under the antitrust laws in the United States, Australia, and the United Kingdom. The transaction had previously been cleared by the Australian and United Kingdom antitrust authorities and became fully effective on May 6, 2021, after the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 expired on May 5, 2021.

As from May 6, 2021, CSL Behring will be responsible for the development and commercialization of the Product. We agreed to complete the validation of the current manufacturing process as well as to complete the development and validation of a next generation manufacturing process. We will be entitled to receive a development milestone payment if we complete these activities in accordance with an agreed development plan and timeline. CSL Behring will also be reimbursing us for our services to facilitate the completion of the HOPE-B clinical trial.

On Signing, we and CSL Behring also entered into a development and commercial supply agreement, pursuant to which, among other things, we will supply the Product to CSL Behring at an agreed-upon price commensurate with the stand-alone selling price (“SSP”). We will be responsible to supply the Product until such time that these capabilities may be transferred to CSL Behring or its designated contract manufacturing organization.

We recorded $462.4 million, including a $450.0 million upfront cash payment following the closing of the CSL Behring Agreement as license revenue in the three and six months ended June 30, 2021. We are eligible to receive more than $0.3 billion in regulatory, development, and first commercial sale milestones, $1.3 billion in additional commercial milestones, and tiered double-digit royalties of up to a low-twenties percentage of net product sales arising from the collaboration. Upon closing, we contractually owed to our licensors $16.7 million of the upfront payment received from CSL Behring. The payments we received significantly increased our cash and cash equivalents position to $677.3 million as of June 30, 2021. This is expected to fund our operations into the first half of 2024 (assuming a full repayment of funds borrowed from Hercules Growth Capital, Inc. under our term loan facilities by 2023, as well as payments we expected to make in relation to our acquisition of Corlieve). We expect that the achievement of the $0.3 billion regulatory, first commercial sales and development milestones would further extend our runway by 12 to 18 months.

Hemophilia B program – Etranacogene dezaparvovec (AMT-061)

Etranacogene dezaparvovec is our lead gene therapy candidate and includes an AAV serotype 5 (“AAV-5”) vector incorporating the functional human Factor IX (“FIX”) Padua variant. We are currently conducting a pivotal study in patients with severe and moderately-severe hemophilia B.

On June 22, 2021, we presented new data from the HOPE-B pivotal study related to the 52-week period post-infusion. Data showed that participants continued to demonstrate durable, sustained increases in FIX activity at 52-weeks post-infusion with a mean FIX activity of 41.5 percent of normal, as measured by a one-stage APTT-based clotting assay, compared to a mean FIX activity of 39.0 percent of normal at 26-weeks of follow-up.

During the 52-week period, a single dose of etranacogene dezaparvovec significantly reduced the annualized rate of bleeding requiring treatment by 80 percent from a prospectively collected 3.39 at baseline to 0.68 bleeding episodes per year (p-value <0.0001). The annualized rate of spontaneous bleeding requiring treatment was also significantly reduced by 85 percent from a prospectively collected 1.16 at baseline to 0.18 bleeds per year during the 52-week period (p-value <0.0001). Usage of FIX replacement therapy (IU/year and infusions/year) in all patients declined 96 percent during the 52-week period, with 52 of 54 patients (96 percent) successfully discontinuing their prophylactic infusions. As previously announced, of the two non-responders, one patient only received a partial dose (less than 10 percent of the dosage) due to an infusion reaction and a second patient had an unusually high pre-existing NAb titer of 3,212, which is expected in less than 1 percent of the general population. We expect all patients to complete their 78-week follow-up visits by the end of the third quarter of 2021, and we expect a BLA to be submitted to the FDA and a MAA to be submitted to EMA in the first quarter of 2022.

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On December 21, 2020, our clinical trials of etranacogene dezaparvovec had been placed on clinical hold by the FDA following a preliminary diagnosis of hepatocellular carcinoma (“HCC”), a form of liver cancer, in one patient in the HOPE-B trial who was treated with etranacogene dezaparvovec.

On March 26, 2021, we submitted to the FDA the results of a comprehensive investigation that found it is highly unlikely the HCC was caused by etranacogene dezaparvovec. On April 23, 2021, the FDA informed us that the clinical hold on our hemophilia B gene therapy program was removed after determining that we had satisfactorily addressed all issues identified.

Etranacogene dezaparvovec has been granted Breakthrough Therapy Designation by the FDA and access to the current priority medicines (“PRIME”) initiative by the EMA.

Huntington’s disease program (AMT-130)

AMT-130 is our novel gene therapy candidate for the treatment of Huntington’s disease. AMT-130 utilizes our proprietary, gene-silencing miQURE platform and incorporates an AAV vector carrying a miRNA specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment. AMT-130 has received orphan drug and Fast Track designations from the FDA and Orphan Medicinal Product Designation from the EMA.

On April 5, 2021 we announced the completion of enrollment of all ten patients in the first dose cohort of our Phase Ib/II clinical trial of AMT-130. On May 27, 2021, we announced that the study’s independent Data Safety Monitoring Board (“DSMB”) recommended we continue patient dosing after reviewing safety data from all 10 patients in the first dose cohort. On June 16, 2021, we announced that the first two patient procedures were completed in the second, higher-dose cohort, which will include 16 patients. The Phase I/II study is a double-blinded, randomized, and controlled clinical trial being conducted in the United States. To date, seven patients have been treated with AMT-130 across both dose cohorts, and five patients have received an imitation surgical procedure that will serve as a control.

On April 5, 2021 we also announced the initiation of a Phase Ib/II clinical trial in Europe. The planned Phase Ib/II study is expected to begin enrolling patients in the second half of 2021. This open-label study will enroll 15 patients with early manifest Huntington’s disease across two dose cohorts. Together with the U.S. study, the European study is intended to establish safety, proof of concept, and the optimal dose of AMT-130 to take forward into Phase III development or into a confirmatory study should an accelerated registration pathway be feasible.

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Research and Development Day

On June 22, 2021, we held a research and development day to outline our vision and strategy of leveraging our AAV engine to deliver gene therapies within our key focus areas. We provided updates on our Fabry (AMT-191) and SCA3 (AMT-150) programs and announced additional new product candidates, including AMT-210 for Parkinson’s disease, AMT-240 for Alzheimer’s disease, and AMT-161 for Amyotrophic Lateral Sclerosis (“ALS”). These new programs are key components of our plan to expand our clinical pipeline.  We also announced our plans to construct a second cGMP manufacturing facility located in Amsterdam, The Netherlands.

Covid pandemic

The coronavirus disease (“Covid”) caused by the severe acute respiratory syndrome coronavirus 2 (“Sars-CoV 2 virus”) was characterized as a pandemic by the World Health Organization (“WHO”) on March 11, 2020. Since then, various, potentially more infectious, variants of the Sars-CoV 2 virus causing Covid have been identified.

Throughout the pandemic, we have been implementing measures to address the impact of Covid on our business. We have implemented a series of protocols governing the operations of our Lexington facility to comply with the requirements of the various orders and guidance from the Commonwealth of Massachusetts and other related orders, guidance, laws, and regulations. We continue to monitor local government rules and recommendations and office protocols will be aligned with these rules and recommendations. Accordingly, we had mandated a work-from-home policy since March 2020 for all non-essential employees at our Amsterdam and Lexington facilities and had implemented additional protocols for our essential employees.

Effective May 29, 2021, Massachusetts has lifted all industry restrictions, with the exceptions of remaining face-covering requirements for all public and private transportation systems and facilities housing vulnerable populations, and capacity has been increased to 100% for all industries. Furthermore, the state of emergency was lifted on June 15, 2021. Implementation of adequate cleaning and hygiene protocols are still encouraged. We have implemented a mandatory Covid PCR testing protocol in our Lexington facility effective February 11, 2021 that requires employees to have tested negative for Covid prior to entering the facility. Also in our Lexington facility, allowed occupancy has been increased to 100% in line with the permitted capacity guidelines and all employees are allowed to work at the Lexington facility subject to social distancing protocols, and a daily health screening and compliance with our Covid PCR testing policy for unvaccinated employees. All employees that are not essential employees, however, remain able to work remotely until December 31, 2021, and we have been operating significantly below 100% capacity. At least until August 1, 2021, for purposes of monitoring Covid surges due to relaxation of requirements, no employee or contractor may enter the Lexington facility unless they are either: (i) fully vaccinated; or (ii) have taken an approved test for Covid and obtained a negative result.

On June 26, 2021, the Dutch government announced updated measures. Since July 1, we allow for an occupancy rate of 50% at our Amsterdam site as well as the limited presence of visitors. We continue to operate our laboratories at our Amsterdam site to comply with social distancing rules and to ensure the health and well-being of our employees under the current circumstances. Employees that can continue to work from home are encouraged to do so through at least August 2021, partly in conjunction with the ongoing expansion of our laboratory space.

The broader implications of Covid, including the implications from the various variants, on our results of operations and overall financial performance remain uncertain. We have experienced increased lead times in the delivery of equipment and disposables that we use to manufacture materials for our various programs. Currently, these have not materially impacted our development timelines and we continue to adapt to the current environment to minimize the effect to our business. However, we may experience more pronounced disruptions in our operations in the future.

Financing

As of December 31, 2020, a $35.0 million term loan was outstanding in accordance with the Second Amended and Restated Loan and Security Agreement (the “2018 Amended Facility”) between us and Hercules Capital, Inc. (“Hercules”).

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On January 29, 2021, we and Hercules amended the 2018 Amended Facility (“2021 Amended Facility”). Pursuant to the 2021 Amended Facility, Hercules agreed to an additional Facility of $100.0 million (“Tranche B”) increasing the aggregate principal amount of the term loan facilities from $35.0 million to up to $135.0 million. On January 29, 2021, we drew down $35.0 million of the Tranche B. We may draw down the remaining $65.0 million under the Tranche B in a series of one or more advances of not less than $20.0 million each until December 15, 2021. Advances under Tranche B bear interest at a rate equal to the greater of (i) 8.25% or (ii) 8.25% plus the prime rate, less 3.25% per annum. The principal balance and all accrued but unpaid interest on advances under Tranche B is due on June 1, 2023, which date may be extended by us by up to two twelve-month periods. Advances under the 2021 Amended Facility may not be prepaid until July 29, 2021, following which we may prepay all such advances without charge.

In addition to Tranche B, the 2021 Amended Facility also extends the interest only payment period of the previously funded $35.0 million term loan from January 1, 2022 to June l, 2023. End of term charges in respect of advances under the 2021 Amended Facility range from 1.65% to 6.85%, depending on the maturity date.

On March 1, 2021, we entered into a Sales Agreement with SVB Leerink LLC (“SVB Leerink”) with respect to an at-the-market (“ATM”) offering program, under which we may, from time to time in our sole discretion, offer and sell through SVB Leerink, acting as agent, our ordinary shares, up to an aggregate offering price of $200.0 million. We will pay SVB Leerink a commission equal to 3% of the gross proceeds of the sales price of all ordinary shares sold through it as a sales agent under the Sales Agreement.

In March and April of 2021, we issued 921,730 ordinary shares at a weighted average price of $33.52 per share, with net proceeds of $29.6 million, after deducting underwriting discounts and net of offering expenses.

Facility

In February 2021 we commenced the expansion of our Amsterdam site to build additional laboratories to support the expansions of our research and development activities as well the construction of a cleanroom designed to be capable of manufacturing cGMP materials at a 500-liter scale. In May 2021, we entered into a sublease agreement to let an additional approximately 1,080 square meters of office space to accommodate the hiring of additional full-time employees. The lease expires in October 2028 and includes an option to break the lease on October 31, 2023.

Organization

On May 17, 2021, Pierre Caloz was appointed as Chief Operating Officer.  Mr. Caloz oversees all manufacturing operations, global CMC development and innovation, supply chain, and facilities.

On June 15, 2021, Christian Klemt was appointed as Chief Financial Officer. Mr. Klemt was our Chief Accounting Officer from August 2017 to June 2021, and he will continue to serve as general manager of our Amsterdam site. Matthew Kapusta, who has been our Chief Executive Officer since December 2016 and had been our Chief Financial Officer from January 2015 to June 2021, will continue to serve as our Chief Executive Officer. In connection with his transition to Chief Financial Officer, Mr. Klemt will also serve as our Principal Financial Officer.

On June 16, 2021, our shareholders voted to approve the reappointment of Mr. David Meek and Ms. Paula Soteropoulos as non-executive directors of the Board of Directors. Mr. Meek has been appointed chairman of the Board. Mr. Philip Astley-Sparke did not stand for reappointment and retired from the Board on June 16, 2021.

Intellectual Property

On May 11, 2021, Pfizer, Inc. filed three petitions seeking Inter Partes Review of U.S. Patent Nos. 9,982,248 (the “’248 Patent”) and 10,465,180 (the “’180 Patent” and together with the ‘248 Patent, the “Patents”). The petitions collectively seek to invalidate all claims of the Patents, which relate to the expression of factor IX protein using a modified “Padua” polypeptide having a leucine in position 338. We are in the process of responding to the petitions.

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Financial Overview

Key components of our results of operations include the following:

	Three months ended June 30,				Six months ended June 30,
	2021		2020		2021		2020
	(in thousands)				(in thousands)
Total revenues	$	463,868	$	1,535	$	464,322	$	1,639
Cost of contract revenue		(23,178)		—		(23,178)		—
Research and development expenses		(32,747)		(28,401)		(64,777)		(54,414)
Selling, general and administrative expenses		(17,299)		(11,511)		(30,300)		(20,583)
Net income / (loss)		399,468		(42,551)		357,912		(70,550)

As of June 30, 2021, and December 31, 2020, we had cash and cash equivalents of $677.3 million and $244.9 million, respectively. We had a net income of $399.5 million and $357.9 million in the three and six months ended June 30, 2021, compared to a net loss of $42.6 million and $70.6 million for the same periods in 2020. As of June 30, 2021, and December 31, 2020, we had accumulated deficits of $426.8 million and $784.7 million, respectively. We recorded a net income in the three and six months ended June 30, 2021 as a result of the closing of the CSL Behring transaction on May 6, 2021.

We anticipate that our expenses will increase substantially as we:

● Advance the clinical development of AMT-130 for our Huntington’s disease gene therapy program;

● Advance multiple research programs related to gene therapy candidates targeting liver-directed and central nervous system (“CNS”) diseases;

● Continue to expand our employee base to support research and development, as well as general and administrative functions;

● Acquire or in-license rights to new therapeutic targets or product candidates;

● Continue to expand, enhance and optimize our technology platform, including our manufacturing capabilities, next-generation viral vectors and promoters, and other enabling technologies; and

● Maintain, expand, and protect our intellectual property portfolio, including in-licensing additional intellectual property rights from third parties.

See “Results of Operations” below for a discussion of the detailed components and analysis of the amounts above.

Critical Accounting Policies and Estimates

In preparing our consolidated financial statements in accordance with U.S. GAAP and pursuant to the rules and regulations promulgated by the SEC we make assumptions, judgments and estimates that can have a significant impact on our net income/loss and affect the reported amounts of certain assets, liabilities, revenue and expenses, and related disclosures. On an ongoing basis, we evaluate our estimates and judgments, including those related to the treatment of the CSL Behring Agreement, our arrangements with Bristol-Myers Squibb (“BMS”), including the amended collaboration and license agreement that we entered into with BMS in December 2020 (the “amended BMS CLA”), share-based payments, corporate income taxes related to valuation allowance and accounting for operating leases under ASC 842. We base our assumptions, judgments and estimates on historical experience and various other factors that we believe to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not clear from other sources. Actual results may differ from these estimates under different assumptions or conditions. In making estimates and judgments, management employs critical accounting policies. During the six months ended June 30, 2021, there were no material changes to our critical accounting policies as reported in our Annual Report on Form 10-K for the year ended December 31, 2020, which was filed with the SEC on March 1, 2021, with the exception of determining the accounting treatment in accordance with ASC 606 related to the CSL Behring Agreement as discussed below.

We believe that the assumptions, judgments, and estimates related to the treatment of the CSL Behring Agreement, the amended BMS CLA, share-based payments, corporate income taxes related to valuation allowance, and accounting for operating leases under ASC 842 to be our critical accounting policies.

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The preparation of our consolidated financial statements for the three- and six- month period ended June 30, 2021, required us to analyze the accounting treatment of the CSL Behring Agreement that we entered into on June 24, 2020 (“Signing”). The effectiveness of the transaction had been contingent on completion of review under antitrust laws in the United States, Australia, and the United Kingdom. The transaction had previously been cleared by the Australian and United Kingdom antitrust authorities and became fully effective on May 6, 2021 (“Closing”), after the waiting period under the HSR Act expired on May 5, 2021.    

We identified two material performance obligations related to the CSL Behring Agreement:

(i) Sale of the exclusive global rights to the Product (“License”); and

(ii) Generate information to support the regulatory approval of the current and next generation manufacturing process of Product and to provide any such information generated to CSL Behring (“Manufacturing Development”).

We continued to develop the Product between Signing and Closing and performed certain reimbursable activities to fulfill the transfer of the global rights (“Additional Covenants” and together with the License the “License Sale”). We determined that the fixed upfront payment of $450.0 million and the $12.4 million that we received in relation to the Additional Covenants should be allocated to the License Sale. In addition, we concluded that variable milestone payments, sales milestone payments and royalties should be allocated to the License Sale performance obligation as well. We determined that the sale of the License was completed on May 6, 2021, when we transferred the License and CSL Behring assumed full responsibility for the development and commercialization of the Product. At Closing we evaluated the amount of potential payments and the likelihood that the payments will be received. We utilized the most likely amount method to estimate the variable consideration to be included in the transaction price. Since we cannot control the achievement of regulatory and first commercial sales milestones, we concluded that the potential payments are constrained as of Closing. We determined that we would recognize revenue related to these payments, only to the extent that it becomes probable that no significant reversal of recognized cumulative revenue will occur thereafter. We will include payments related to sales milestones in the transaction price when their achievement becomes probable, and we will include royalties on the sale of Product once these have been earned. We recognized $462.4 million of revenues related to the License Sale in the three and six months ended June 30, 2021

We determined that the variable milestone payment related to Manufacturing Development Services should be allocated to the Manufacturing Development performance obligation. We concluded that this milestone payment represents the SSP of the services based on the estimated cost of providing the services including a reasonable margin. The services related to Manufacturing Development will be provided between Closing and the completion of an agreed manufacturing development plan. The variable consideration will be reduced if we do not complete the development by pre-agreed dates. We utilized the most likely amount method to estimate the variable consideration to be included in the transaction price. Completion of Manufacturing Development is partially dependent on the timing of regulatory submissions by CSL Behring as well as regulatory approvals of the developed manufacturing processes. Since we cannot control the timing or outcome of any regulatory decisions, we concluded that we would recognize revenue related to this payment when it becomes probable that the milestone has been achieved. We did not recognize any revenue related to Manufacturing Development during the three and six months ended June 30, 2021.

We recognized $0.4 million of collaboration revenue in the three and six months ended June 30, 2021, compared to nil in the same periods in the prior year. We generate such collaboration revenue from services rendered in relation to completing the HOPE-B clinical trial on behalf of CSL Behring. CSL Behring may request additional development services or to request us to support the transfer of manufacturing to a party designated by CSL Behring. These collaboration services will be reimbursed at the pre-agreed full-time-employee rate (“FTE-rate”). We concluded that these rights at Closing do not represent material rights.

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We also analyzed the impact of Closing on our net deferred tax asset in the Netherlands. Prior to Closing we had determined that the potential Closing would represent insufficient positive evidence to overcome the significant negative evidence of having been loss-making during the past three years. Accordingly, we had therefore recorded a full valuation allowance related to our net deferred tax assets. We determined that in accordance with Dutch tax law the upfront payment as well as payments related to the Additional Covenants, to the extend such activities were conducted in the 12 months ended December 31, 2020, can more likely than not be included in the Dutch tax return for the 12-month period ended December 31, 2020. Including this income in our 2020 tax position will consume substantially all of our Dutch net operating losses for the years 2011 to 2018. We treated the adjustment to our 2020 filing position resulting in the release of the valuation allowance related to our Dutch net operating losses for the years 2011 to 2018 as a discrete event in the three months ended June 30, 2021. We allocated part of the release to the tax benefit of share issuance cost incurred in 2014, 2015, 2017 and 2018. This resulted in an increase of additional paid- in capital as well as the recognition of deferred tax expenses of $3.0 million during the three and six months ended June 30, 2021. Based on changes to Dutch tax law enacted in June 2021 our remaining net operating tax loss related to 2019 can be carried forward indefinitely. We continue to expect incurring tax losses for the foreseeable future including 2021. As such, we retain our full valuation allowance related to the remaining net deferred asset as of June 30, 2021.

Revenues

We recognize revenue when the customer obtains control of promised goods or services, in an amount that reflects the consideration which we expect to receive in exchange for those goods or services. To determine revenue recognition for arrangements that we determine are within the scope of ASC 606, we perform the following five steps: (i) identify the contract with the customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) we satisfy a performance obligation. We only apply the five-step model to contracts when it is probable that we will collect the consideration we are entitled to in exchange for the goods or services we transfer to the customer. At contract inception, once the contract is determined to be within the scope of ASC 606, we assess the goods or services promised within each contract and determine those that are performance obligations and assess whether each promised good or service is distinct. We then recognize as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied.

We estimate the transaction price based on the amount expected to be received for transferring the promised goods or services in the contract. The consideration may include fixed consideration or variable consideration. At inception of each arrangement that includes variable consideration, we evaluate the amount of potential payments and the likelihood that the payments will be received. We utilize either the most likely amount method or expected amount method to estimate the amount expected to be received based on which method best predicts the amount expected to be received. The amount of variable consideration included in the transaction price may be constrained and is included in the transaction price only to the extent that it is probable that a significant reversal in the amount of cumulative revenue recognized will not occur in a future period.

We allocate the transaction price based on the estimated SSP of each of the performance obligations. Variable consideration is allocated specifically to one or more performance obligations in a contract when the terms of the variable consideration relate to the satisfaction of the performance obligation and the resulting amounts allocated are consistent with the amounts that we expect to receive for the satisfaction of each performance obligation. We recognize revenue when or as control of the performance obligation transfers to the customer. For performance obligations which consist of licenses and other promises, we utilize judgement to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time.

CSL Behring    

Following the Closing of the CSL Behring agreement, we recognize license revenue related to the License Sale of the global rights to the Product. We evaluated that our performance obligation related to the License Sale was satisfied on Closing and recognized $462.4 million in license revenue related to a $450.0 million upfront payment and $12.4 million of payments in relation to Additional Covenants. We will recognize additional license revenue in relation to the License Sale when it becomes probable that regulatory and sales milestone events will be achieved as well as when royalties on sales of Product have been earned.

We recognize collaboration revenues associated with Manufacturing Development. We will recognize collaboration revenue related to a contractual development milestone when it becomes probable the milestone will be achieved.

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We recognize collaboration revenues associated with Development Services that will be reimbursed by CSL Behring relating to clinical development activities. These services are provided by our employees. Collaboration revenue related to these contracted services is recognized when the performance obligations are satisfied.

BMS

In May 2015, we and Bristol-Myers Squibb (“BMS”) entered into a collaboration and license agreement and various related agreements with BMS (“BMS CLA”). We have recognized license revenues associated with the amortization of the non-refundable upfront payment and target designation fees we received from BMS in accordance with the BMS CLA. We evaluated our outstanding performance obligation following the amendment of the BMS CLA on December 1, 2020 (“amended BMS CLA”) and determined that our remaining performance obligation related to license revenues is immaterial. We updated our measure of progress accordingly and amortized the remaining balance of unrecognized revenue as of December 1, 2020. In accordance with the amended BMS CLA, we continue to be eligible to receive research, development, and regulatory milestone payments as well as sales milestone payments and royalties for each of the four active Collaboration Targets if defined milestones are achieved in relation to the license to our technology and know-how. We will recognize revenue from these payments when earned or as sales occur.

We recognize collaboration revenues associated with Collaboration Target-specific pre-clinical analytical development and process development activities that are reimbursable by BMS under the BMS CLA and the amended BMS CLA as well as other related agreements. Collaboration revenue related to these contracted services is recognized when performance obligations are satisfied.

Cost of Contract Revenues

We expense contract fulfillment costs associated with license revenue recognized under the CSL Behring Agreement as costs of contract revenues.

Research and development expenses

We expense research and development (“R&D”) expenses as incurred. Our R&D expenses generally consist of costs incurred for the development of our target candidates, which include:

● Employee-related expenses, including salaries, benefits, travel and share-based compensation expense;

● Costs incurred for laboratory research, preclinical and nonclinical studies, clinical trials, statistical analysis and report writing, and regulatory compliance costs incurred with clinical research organizations and other third-party vendors;

● Costs incurred to conduct consistency and comparability studies;

● Costs incurred for the development and improvement of our manufacturing processes and methods;

● Costs associated with our research activities for our next-generation vector and promoter platform;

● Costs associated with the rendering of collaboration services as well as the continued development of Product between Signing and Closing; and

● Facilities, depreciation, and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, insurance, and other supplies.

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Our research and development expenses may vary substantially from period to period based on the timing of our research and development activities, including manufacturing campaigns, regulatory submissions and enrollment of patients in clinical trials. The successful development of our product candidates is highly uncertain. Estimating the nature, timing or cost of the development of any of our product candidates involves considerable judgement due to numerous risks and uncertainties associated with developing gene therapies, including the uncertainty of:

● the scope, rate of progress and expense of our research and development activities;

● our ability to successfully manufacture and scale-up production;

● clinical trial protocols, speed of enrollment and resulting data;

● the effectiveness and safety of our product candidates;

● the timing of regulatory approvals; and

● our ability to agree to ongoing development budgets with collaborators who reimburse the costs of our development programs.

A change in the outcome of any of these variables with respect to our product candidates that we may develop, including as a result of the Covid pandemic, could mean a significant change in the expenses and timing associated with the development of such product candidate.

Selling, general and administrative expenses

Our general and administrative expenses consist principally of employee, office, consulting, legal and other professional and administrative expenses. We incur expenses associated with operating as a public company, including expenses for personnel, legal, accounting and audit fees, board of directors’ costs, directors' and officers' liability insurance premiums, Nasdaq listing fees, expenses related to investor relations and fees related to business development and maintaining our patent and license portfolio. Our selling costs include employee expenses, as well as professional fees related to the preparation of a commercial launch of etranacogene dezaparvovec and advisory fees related to obtaining the CSL Behring Agreement.

Other items, net

Our other income primarily consists of payments received to subsidize our research and development efforts, income from the subleasing of our Amsterdam facility, the recognition of the equity stake received in VectorY B.V., as well as an employee retention credit under the U.S. Coronavirus Aid, Relief, and Economic Security Act (“CARES Act”).

Our other expense primarily consists of expenses we incur in relation to our subleasing income.

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Results of Operations

Comparison of the three months ended June 30, 2021 and 2020

The following table presents a comparison of our results of operations for the three months ended June 30, 2021 and 2020.

	Three months ended June 30,
	2021		2020		2021 vs 2020
	(in thousands)
Total revenues	$	463,868	$	1,535	$	462,333
Operating expenses:
Cost of contract revenues		(23,178)		—		(23,178)
Research and development expenses		(32,747)		(28,401)		(4,346)
Selling, general and administrative expenses		(17,299)		(11,511)		(5,788)
Total operating expenses		(73,224)		(39,912)		(33,312)
Other income		7,590		669		6,921
Other expense		(226)		(500)		274
Income / (loss) from operations		398,008		(38,208)		436,216
Other non-operating items, net		4,718		(4,343)		9,061
Net income / (loss) before income tax expense	$	402,726	$	(42,551)	$	445,277
Income tax expense		(3,258)		-		(3,258)
Net income / (loss)	$	399,468	$	(42,551)	$	442,019

Revenue

Our revenue for the three months ended June 30, 2021 and 2020 was as follows:

	Three months ended June 30,
	2021		2020		2021 vs 2020
	(in thousands)
License revenue	$	462,400	$	1,530	$	460,870
Collaboration revenue		1,468		5		1,463
Total revenues	$	463,868	$	1,535	$	462,333

We recognized $462.4 million in license revenue related to the License Sale on Closing of the CSL Behring Agreement in May 2021. We did not recognize any such license revenue related to the CSL Behring Agreement in the three months ended June 30, 2020.

We recognized $1.5 million in license revenue related to upfront payments and target designation fees received from BMS in 2015 under the BMS CLA for the three months ended June 30, 2020. We did not recognize any license revenue under the amended BMS CLA during the three months ended June 30, 2021.

We recognized $1.5 million collaboration revenue in the three months ended June 30, 2021, compared to $0.0 million for the same period in 2020. Since May 6, 2021, we recognize collaboration revenue related to services we provide in accordance with the CSL Behring Agreement. Our collaboration revenue increased further as a result of increased services provided to BMS following our December 2020 amendment of the BMS CLA.

Cost of contract revenues

We recognized $23.2 million in cost of contract revenues for the three months ended June 30, 2021, compared to nil the same period in 2020. We recorded $16.7 million of expenses related to payments owed to our licensors on Closing as well as $6.5 million of other external expenses incurred to fulfill the License Sale to CSL Behring.

29

Research and development expenses

Research and development expenses for the three months ended June 30, 2021 were $32.7 million, compared to $28.4 million for the same period in 2020. Other research and development expenses are separately classified in the table below. These are not allocated as they are deployed across multiple projects under development.

	Three months ended June 30,
	2021		2020		2021 vs 2020
	(in thousands)
Etranacogene dezaparvovec (AMT-060/061)	$	2,332	$	4,673	$	(2,341)
Huntington's disease (AMT-130)		2,472		2,095		377
Programs in preclinical development and platform related expenses		2,425		1,770		655
Total direct research and development expenses	$	7,229	$	8,538	$	(1,309)
Employee and contractor-related expenses		13,082		9,990		3,092
Facility expenses		4,397		4,185		212
Disposables		3,601		2,501		1,100
Share-based compensation expense		3,286		2,894		392
Other expenses		1,152		293		859
Total other research and development expenses	$	25,518	$	19,863	$	5,655
Total research and development expenses	$	32,747	$	28,401	$	4,346

Direct research and development expenses

Etranacogene dezaparvovec (AMT-060/061)

In the three months ended June 30, 2021 and 2020, the external costs for our hemophilia B program were primarily related to the execution of our Phase III clinical trial. We enrolled patients into a six-month lead in phase between January 2018 and September 2019 and dosed a total of 54 patients between January 2019 and March 2020. We also incur costs related to the long-term follow-up of patients in our Phase I/II clinical trial of AMT-060 and our Phase IIb clinical trial of etranacogene dezaparvovec. During 2020 and up to the Closing of the CSL Behring Agreement we also incurred costs related to the preparation of a Biologics License Application (“BLA”) and marketing authorization application (“MAA”) and for commercialization of the Product. After the Closing, CSL Behring will be responsible for the clinical and regulatory development and commercialization of the Product. Direct research and development expenses related to clinical development incurred between Closing and June 30, 2021 are presented net of reimbursements due from CSL Behring.

Huntington disease (AMT-130)

In the three months ended June 30, 2021, and June 30, 2020, our external costs for the development of Huntington’s disease were primarily related to the execution of our Phase I/II clinical trial in the United States. In addition, our external costs in the three months ended June 30, 2021, included expenses related to the preparation of our Phase I/II clinical trial in Europe that we announced in April 2021.

Preclinical programs & platform development

In the three months ended June 30, 2021, we incurred $2.4 million of costs primarily related to our preclinical activities primarily associated with product candidates, SCA3 (AMT-150) and Fabry disease (AMT-190 and AMT-191), as well as various other research programs and technology innovation projects, compared to costs of $1.8 million in the same period in 2020, which also included costs related to our product candidate for Hemophilia A (AMT-180) that was deprioritized in June 2020.

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Other research & development expenses

● We incurred $13.1 million in personnel and contractor related expenses in the three months ended June 30, 2021, compared to $10.0 million for the same period in 2020. Our costs during the three months ended June 30, 2021 increased by $3.1 million as a result of the recruitment of personnel to support the development of our product candidates;

● We incurred $4.4 million in operating expenses and depreciation expenses related to our rented facilities in the three months ended June 30, 2021, compared to $4.2 million in the same period in 2020; and

● We incurred $3.3 million in share-based compensation expenses in the three months ended June 30, 2021, compared to $2.9 million for the same period in 2020 primarily driven by increase in awards granted, including those to newly recruited personnel.

Selling, general and administrative expenses

Selling, general and administrative expenses for the three months ended June 30, 2021 were $17.3 million, compared to $11.5 million for the same period in 2020.

● We incurred $4.5 million in financial advisory fees in relation to our licensing transaction with CSL Behring in the three months ended June 30, 2021, compared to nil in the same period in 2020;

● We incurred $4.2 million in personnel and contractor related expenses in the three months ended June 30, 2021, compared to $2.9 million in the same period in 2020. The increase in the three months ended June 30, 2021, relates to the recruitment of personnel;

● We incurred $3.7 million in share-based compensation expenses in the three months ended June 30, 2021, compared to $2.8 million in the same period in 2020 primarily driven by increase in awards granted, including those to newly recruited personnel; and

● We incurred $2.0 million in professional fees in the three months ended June 30, 2021, compared to $3.0 million in the same period in 2020. We regularly incur accounting, audit and legal fees associated with operating as a public company.

Other items, net

We recognized $3.0 million in other income in relation to the equity stake received in VectorY B.V. in conjunction with the Settlement Agreement in the three months ended June 30, 2021, compared to no such income for the same period in 2020.

We recognized $1.3 million in other income of employee retention credit under the U.S. CARES Act in the three months ended June 30, 2021, compared to no such income for the same period in 2020.

We recognized $3.0 million in other income from payments received from European authorities to subsidize our research and development efforts in the Netherlands in the three months ended June 30, 2021, compared to $0.3 million for the same period in 2020.

Other non-operating items, net

We recognize interest income associated with our cash and cash equivalents.

We hold monetary items and enter into transactions in foreign currencies, predominantly in euros and U.S. dollars. We recognize foreign exchange results related to changes in these foreign currencies.

We issued warrants to BMS in 2015. We recognize changes in the fair value of these warrants within other non-operating income / (expense).  Following the termination of the BMS warrants on December 1, 2020, we no longer recognize changes in the fair value of these warrants within other non-operating (expense) / income. As of the same date, we recognized a derivative financial liability related to a contingent payment due to BMS upon the consummation of a change of control transaction (“CoC-payment”) as described elsewhere in the Quarterly Report on Form 10-Q.

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Our other non-operating items, net, for the three months ended June 30, 2021 and 2020 were as follows:

	Three months ended June 30,
	2021		2020		2021 vs 2020
	(in thousands)
Interest income	$	37	$	81	$	(44)
Interest expense - Hercules long-term debt		(1,902)		(970)		(932)
Foreign currency gains / (losses), net		6,583		(3,645)		10,228
Other non-operating gains		—		191		(191)
Total other non-operating income / (loss), net	$	4,718	$	(4,343)	$	9,061

We recognized a net foreign currency gain, related to our borrowings from Hercules and our cash and cash equivalents as well as loans between entities within the uniQure group, of $6.6 million during the three months ended June 30, 2021, compared to a net loss of $3.6 million during the same period in 2020.

In the three months ended June 30, 2021, we recognized income of nil within other non-operating gains as there was no change in the fair value of the BMS derivative financial liability for the CoC-payment, compared to income of $0.2 million during the same period in 2020 related to changes in the fair value of the BMS warrants from the BMS CLA.  

Income tax expense

We recognized $3.3 million of deferred tax expense in the three months ended June 30, 2021, and nil in the same period ended 2020.  

32

Comparison of the six months ended June 30, 2021 and 2020

The following table presents a comparison of the six months ended June 30, 2021 and 2020.

	Six months ended June 30,
	2021		2020		2021 vs 2020
	(in thousands)
Total revenues	$	464,322	$	1,639	$	462,683
Operating expenses:
Cost of contract revenues		(23,178)		—		(23,178)
Research and development expenses		(64,777)		(54,414)		(10,363)
Selling, general and administrative expenses		(30,300)		(20,583)		(9,717)
Total operating expenses		(118,255)		(74,997)		(43,258)
Other income		7,942		1,526		6,416
Other expense		(459)		(839)		380
Income / (loss) from operations		353,550		(72,671)		426,221
Non-operating items, net		7,833		2,121		5,712
Income / (loss) before income tax expense	$	361,383	$	(70,550)	$	431,933
Income tax expense		(3,471)		—		(3,471)
Net income / (loss)	$	357,912	$	(70,550)	$	428,462

Revenue

Our revenue for the six months ended June 30, 2021 and 2020 was as follows:

	Six months ended June 30,
	2021		2020		2021 vs 2020
	(in thousands)
License revenue	$	462,400	$	1,577	$	460,823
Collaboration revenue		1,922		62		1,860
Total revenues	$	464,322	$	1,639	$	462,683

We recognized $462.4 million license revenue related to the License Sale on Closing of the CSL Behring Agreement in May 2021. We did not recognize any such license revenue related to the CSL Behring Agreement in the six months ended June 30, 2020.

We recognized $1.6 million license revenue related to upfront payments and target designation fees received from BMS in 2015 under the BMS CLA for the six months ended June 30, 2020. We did not recognize any license revenue under the amended BMS CLA during the six months ended June 30, 2021.

We recognized $1.9 million collaboration revenue in the six months ended June 30, 2021, compared to $0.1 million for the same period in 2020. The increase in collaboration revenue primarily relates to the increased activities under the amended BMS CLA and the CSL Behring Agreement.

Cost of contract revenues

We recognized $23.2 million in cost of contract revenues for the six months ended June 30, 2021 compared to nil the same period in 2020. We recorded $16.7 million of expenses related to payments owed to our licensors on Closing as well as $6.5 million of other external expenses incurred to fulfill the License Sale to CSL Behring.

33

Research and development

Research and development expenses for the six months ended June 30, 2021 were $64.8 million, compared to $54.4 million for the same period in 2020. Other research and development expenses are separately classified in the table below. These are not allocated as they are deployed across multiple projects under development.

	Six months ended June 30,
	2021		2020		2021 vs 2020
	(in thousands)
Etranacogene dezaparvovec (AMT-060/061)	$	5,979	$	9,213	$	(3,234)
Huntington's disease (AMT-130)		4,443		3,155		1,288
Programs in preclinical development and platform related expenses		4,714		3,225		1,489
Total direct research and development expenses	$	15,136	$	15,593	$	(457)
Employee and contractor-related expenses		24,677		19,338		5,339
Facility expenses		9,021		8,201		820
Disposables		6,945		4,911		2,034
Share-based compensation expense		5,960		5,289		671
Other expenses		3,038		1,082		1,956
Total other research and development expenses	$	49,641	$	38,821	$	10,820
Total research and development expenses	$	64,777	$	54,414	$	10,363

Direct research and development expenses

Etranacogene dezaparvovec (AMT-060/061)

In the six months ended June 30, 2021 and 2020, the external costs for our hemophilia B program were primarily related to the execution of our Phase III clinical trial. We enrolled patients into a six-month lead in phase between January 2018 and September 2019 and dosed a total of 54 patients between January 2019 and March 2020. We also incur costs related to the long-term follow-up of patients in our Phase I/II clinical trial of AMT-060 and our Phase IIb clinical trial of etranacogene dezaparvovec. During 2020 and up to the Closing of the CSL Behring Agreement, we also incurred costs related to the preparation of a BLA and MAA and for commercialization of the Product. After the Closing, CSL Behring will be responsible for the clinical and regulatory development and commercialization of the Product. Direct research and development expenses related to clinical development incurred between Closing and June 30, 2021, are presented net of reimbursements due from CSL Behring.

Huntington disease (AMT-130)

In the six months ended June 30, 2021 and June 30, 2020, our external costs for the development of Huntington’s disease were primarily related to the execution of our Phase I/II clinical trial in the United States. In addition, our external costs in the six months ended June 30, 2021, included expenses related to the preparation of our Phase I/II clinical trial in Europe that we announced in April 2021.

Preclinical programs & platform development

In the six months ended June 30, 2021, we incurred $4.7 million of costs primarily related to our preclinical activities primarily associated with product candidates, SCA3 (AMT-150) and Fabry disease (AMT-190 and AMT-191), as well as various other research programs and technology innovation projects, compared to costs of $3.2 million in the same period in 2020, which also included costs related to our product candidate for Hemophilia A (AMT-180) that was deprioritized in June 2020.

34

Other research & development expenses

● We incurred $24.7 million in personnel and contractor related expenses in the six months ended June 30, 2021, compared to $19.3 million for the same period in 2020. Our costs during the six months ended June 30, 2021 increased by $5.4 million as a result of the recruitment of personnel to support the development of our product candidates;

● We incurred $9.0 million in operating expenses and depreciation expenses related to our rented facilities in the six months ended June 30, 2021, compared to $8.2 million in the same period in 2020 as a result of expanding our organization; and

● We incurred $6.0 million in share-based compensation expenses in the six months ended June 30, 2021, compared to $5.3 million for the same period in 2020 primarily driven by increase in awards granted, including those to newly recruited personnel.

Selling, general and administrative expenses

Selling, general and administrative expenses for the six months ended June 30, 2021 were $30.3 million, compared to $20.6 million for the same period in 2020.

● We incurred $8.1 million in personnel and contractor related expenses in the six months ended June 30, 2021, compared to $6.1 million in the same period in 2020. The increase in the six months ended June 30, 2021, relates to the recruitment of personnel;

● We incurred $6.8 million in share-based compensation expenses in the six months ended June 30, 2021, compared to $4.8 million in the same period in 2020 primarily driven by increase in awards granted, including those to newly recruited personnel;

● We incurred $5.0 million in professional fees in the six months ended June 30, 2021, compared to $4.2 million in the same period in 2020. We regularly incur accounting, audit and legal fees associated with operating as a public company; and

● We incurred $4.5 million in financial advisory fees in relation to our licensing transaction with CSL Behring in the six months ended June 30, 2021, compared to nil in the same period in 2020.

Other items, net

We recognized $3.0 million in other income in relation to the equity stake received in VectorY B.V. in conjunction with the Settlement Agreement in the six months ended June 30, 2021, compared to no such income for the same period in 2020.

We recognized $1.3 million in other income of employee retention credit under the U.S. CARES Act in the six months ended June 30, 2021, compared to no such income for the same period in 2020.

We recognized $3.0 million in income from payments received from European authorities to subsidize our research and development efforts in the Netherlands in the six months ended June 30, 2021, compared $0.5 million for the same period in 2020.

Other non-operating items, net

We recognize interest income associated with our cash and cash equivalents.

We hold monetary items and enter into transactions in foreign currencies, predominantly in euros and U.S. dollars. We recognize foreign exchange results related to changes in these foreign currencies.

35

We issued warrants to BMS in 2015. We recognize changes in the fair value of these warrants within other non-operating income / (expense).  Following the termination of the BMS warrants on December 1, 2020, we no longer recognize changes in the fair value of these warrants within other non-operating (expense) / income. As of the same date, we recognized a derivative financial liability related to a contingent payment due to BMS upon the consummation of a CoC-payment as described elsewhere in the Quarterly Report on Form 10-Q.

			Six months ended June 30,
	2021			2020		2021 vs 2020
			(in thousands)
Interest income	$	77		$	903	$	(826)
Interest expense		(3,453)			(1,945)		(1,508)
Foreign currency gains, net		11,209			957		10,252
Other non-operating gains, net		—			2,206		(2,206)
Total non-operating income, net	$	7,833		$	2,121	$	5,712

We recognized a net foreign currency gain, related to our borrowings from Hercules and our cash and cash equivalents as well as loans between entities within the uniQure group, of $11.2 million during the six months ended June 30, 2021, compared to a net gain of $1.0 million during the same period in 2020.

In the six months ended June 30, 2021, we recognized income of nil within other non-operating items as there was no change in the fair value of the BMS derivative financial liability for the CoC-payment, compared to income of $2.2 million during the same period in 2020 related to changes in the fair value of the BMS warrants from the BMS CLA.  

Income tax expense

We recognized $3.5 million of deferred tax expense for the six months ended June 30, 2021 and nil in the same period ended 2020.  

Financial Position, Liquidity and Capital Resources

As of June 30, 2021, we had cash, cash equivalents and restricted cash of $680.2 million which include payments received from CSL Behring following the Closing. This is expected to fund our operations into the first half of 2024 (assuming a full repayment of funds borrowed from Hercules under our term loan facility by 2023, as well as payments we expected to make in relation to our acquisition of Corlieve). We expect that the achievement of $0.3 billion of regulatory, first commercial sale, and development milestones would further extend our runway by 12 to 18 months.

	Six months ended June 30,
	2021		2020
	(in thousands)
Cash, cash equivalents and restricted cash at the beginning of the period	$	247,680	$	380,726
Net cash generated from / (used in) operating activities		375,206		(62,944)
Net cash used in investing activities		(6,191)		(4,606)
Net cash generated from financing activities		65,128		3,549
Foreign exchange impact		(1,665)		223
Cash, cash equivalents and restricted cash at the end of period	$	680,158	$	316,948

We had previously incurred losses and cumulative negative cash flows from operations since our business was founded by our predecessor entity AMT Therapeutics (“AMT”) Holding N.V. in 1998. As a result of receiving the upfront payment upon Closing of the CSL Behring Agreement, we generated $375.2 million cash flows from operations during the six months ended June 30, 2021. We recorded net income of $399.5 million and $357.9 during the three and six months ended June 30, 2021, compared to a net loss of $42.6 million and $70.6 million during the same period in 2020. As of June 30, 2021, we had an accumulated deficit of $426.8 million.

36

Sources of liquidity

From our first institutional venture capital financing in 2006 through May 2021, we funded our operations primarily through private and public placements of equity securities and convertible and other debt securities as well as payments from our collaboration partners. In May 2021, we received a $462.4 million cash payment due from CSL Behring upon Closing. In addition, we could potentially receive up to $0.3 billion in regulatory, first commercial sales and development milestone payments from CSL Behring.

On March 1, 2021, we entered into a Sales Agreement with SVB Leerink with respect to an ATM offering program, under which we may, from time to time in our sole discretion, offer and sell through SVB Leerink, acting as agent, our ordinary shares, up to an aggregate offering price of $200.0 million. We will pay SVB Leerink a commission equal to 3% of the gross proceeds of the sales price of all ordinary shares sold through it as a sales agent under the Sales Agreement. In the six months ended June 30, 2021, we issued 921,730 million ordinary shares for net proceeds of $29.6 million.

On December 6, 2018, we signed the 2018 Amended Facility with Hercules that both refinanced our then-existing $20.0 million credit facility and provided us with an additional unconditional commitment of $15.0 million as well as a conditional commitment of $15 million that expired on June 30, 2020. At signing, we drew down an additional $15.0 million, for a total outstanding amount of $35.0 million.

The 2018 Amended Facility extended the loan’s maturity date until June 1, 2023. The interest-only period was initially extended from November 2018 to January 1, 2021. The interest-only period was further extended to January 1, 2022 as a result of raising more than $90.0 million in equity financing in September 2019. The interest-only period was again further extended to June 1, 2023 as a result of the 2021 Amended Facility.  The variable interest rate of the 2018 Amended Facility is equal to the greater of (i) 8.85% or (ii) 8.85% plus the prime rate less 5.50%. Under the 2018 Amended Facility, we paid a facility fee equal to 0.50% at signing of the $35.0 million loan outstanding and will owe a back-end fee of 4.95% of the outstanding debt. In addition, in May 2020, we paid a back-end fee of $1.0 million in relation to the 2016 Amended Facility.

On January 29, 2021, we and Hercules entered into the 2021 Amended Facility. Pursuant to the 2021 Amended Facility, Hercules agreed to an additional Facility of $100.0 million (“Tranche B”), increasing the aggregate principal amount of the term loan facilities from $35.0 million to up to $135.0 million. On January 29, 2021, we drew down $35.0 million of the Tranche B. We may draw down the remaining $65.0 million under the Tranche B in a series of one or more advances of not less than $20.0 million each until December 15, 2021. Advances under the Tranche B bear interest at a rate equal to the greater of (i) 8.25% or (ii) 8.25% plus the prime rate, less 3.25% per annum. The principal balance and all accrued but unpaid interest on advances under the Tranche B is due on June 1, 2023, which date may be extended by us by up to two twelve-month periods. Advances under the Tranche B may not be prepaid until six-months after the Closing Date, following which we may prepay all such advances without charge. As of June 30, 2021, $70.0 million was outstanding under the 2018 Amended Facility and the 2021 Amended Facility (December 31, 2020: $35.0 million under the 2018 Amended Facility).

We are subject to the same covenants under our 2018 Amended Facility and 2021 Amended Facility and may become subject to covenants under any future indebtedness that could limit our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends, which could adversely impact our ability to conduct our business. In addition, our pledge of assets as collateral to secure our obligations under the 2018 Amended Facility and 2021 Amended Facility may limit our ability to obtain debt financing. To the extent we need to finance our cash needs through equity offerings or debt financings, such financing may be subject to unfavorable terms including without limitation, the negotiation and execution of definitive documentation, as well as credit and debt market conditions, and we may not be able to obtain such financing on terms acceptable to us or at all. If financing is not available when needed, including through debt or equity financings, or is available only on unfavorable terms, we may be unable to meet our cash needs. If we raise additional funds through collaborations, strategic alliances or marketing, distribution, or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves, which could have a material adverse effect on our business, financial conditions, results of operations and cash flows.

37

Net Cash generated from / used in operating activities

Net cash generated from operating activities was $375.2 million for the six months ended June 30, 2021 and consisted of net income of $357.9 million adjusted for non-cash items, including depreciation and amortization expense of $3.7 million, share-based compensation expense of $12.8 million, unrealized foreign exchange gains of $12.3 million and other non-cash items, net, of $0.5 million. Net cash generated from operating activities also included favorable changes in operating assets and liabilities of $12.6 million. These changes primarily related to a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $3.4 million primarily related to an increase in various prepayments and a net increase in accounts payable, accrued expenses, other liabilities, and operating leases of $15.9 million primarily related to an increase in amounts to be paid to licensors upon the CSL Behring Closing. Net income primarily consisted of $462.4 million license revenue recognized on Closing.

Net cash used in operating activities was $62.9 million for the six months ended June 30, 2020 and consisted of a net loss of $70.6 million adjusted for non-cash items, including depreciation and amortization expense of $3.5 million, share-based compensation expense of $10.1 million, fair value gains on derivative financial instruments of $2.2 million, unrealized foreign exchange gain of $0.9 million, and a decrease in other non-cash items, net of $1.6 million. Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $1.3 million. These changes primarily related to a net decrease in accounts receivable and accrued income, prepaid expenses, and other current assets of $1.1 million and a net decrease in accounts payable, accrued expenses and other liabilities of $2.3 million.

Net cash used in investing activities

In the six months ended June 30, 2021, we used $6.2 million in our investing activities compared to $4.6 million for the same period in 2020.

	Six months ended June 30,
	2021		2020
	(in thousands)
Build out of Amsterdam site	$	(4,470)	$	(1,816)
Build out of Lexington site		(1,721)		(576)
Acquisition of licenses, patents, and other rights		—		(2,214)
Total investments	$	(6,191)	$	(4,606)

The build out of the Amsterdam site consumed $4.5 million cash during the six months ended June 30, 2021, compared to $1.8 million for the same period in 2020. The increase is a result of the construction of additional laboratories to support the expansions of our research and development activities as well as the construction of a cleanroom designed to be capable of manufacturing cGMP materials at a 500-liter scale.

Net cash generated from financing activities

In the six months ended June 30, 2021, we generated $65.1 million in our financing activities compared to $3.5 million for the same period in 2020.

	Six months ended June 30,
	2021		2020
	(in thousands)
Cash flows from financing activities
Proceeds from loan increment, net of debt issuance costs	$	34,603	$	-
Proceeds from issuance of ordinary shares, net of issuance costs		29,565		-
Proceeds from issuance of shares related to employee stock option and purchase plans		960		3,549
Net cash generated from financing activities	$	65,128	$	3,549

In January 2021, we received $34.6 million net proceeds from the Hercules 2021 Amended Facility.  

We received net proceeds of $29.6 million associated with our ATM offering in March and April 2021.

During the six months ended June 30, 2021, we received $1.0 million from the exercise of options to purchase ordinary shares in relation to our share incentive plans compared to $3.5 million for the same period in 2020.

38

Funding requirements

We believe our cash and cash equivalents as of June 30, 2021, will fund our operations into the first half of 2024 (assuming a full repayment of funds borrowed from Hercules under our term loan facilities by 2023, as well as payments we expected to make in relation to our acquisition of Corlieve). We expect that the achievement of the $0.3 billion in regulatory, first commercial sales and development milestones would further extend our runway by 12 to 18 months. Our future capital requirements will depend on many factors, including but not limited to:

● achieving the milestones and royalties as defined within the CSL Behring Agreement;

● payments we will make in relation to the contemplated acquisition of Corlieve;

● the scope, timing, results, and costs of our current and planned clinical trials, including those for AMT-130 in Huntington’s disease;

● the extent to which we acquire or in-license other businesses, products, product candidates or technologies;

● the amount and timing of revenue, if any, we receive from commercial sales of any product candidates for which we, or our collaboration partner, receives marketing approval in the future;

● the amount and timing of revenue, if any, we receive from manufacturing products for CSL Behring.

● the scope, timing, results and costs of preclinical development and laboratory testing of our additional product candidates;

● the need for additional resources and related recruitment costs to support the preclinical and clinical development of our product candidates;

● the need for any additional tests, studies, or trials beyond those originally anticipated to confirm the safety or efficacy of our product candidates and technologies;

● the cost, timing and outcome of regulatory reviews associated with our product candidates;

● our ability to enter into collaboration arrangements in the future;

● the costs and timing of preparing, filing, expanding, acquiring, licensing, maintaining, enforcing, and prosecuting patents and patent applications, as well as defending any intellectual property-related claims;

● the repayments of the principal and other fees associated with our venture debt loan with Hercules, which following the January 29, 2021 amendment will be due in June 2023;

● the costs associated with maintaining quality compliance and optimizing our manufacturing processes, including the operating costs associated with our Lexington, Massachusetts manufacturing facility;

● the costs associated with increasing the scale and capacity of our manufacturing capabilities; and

● the costs associated with process validation and inspection readiness of etranacogene dezaparvovec.

Contractual obligations and commitments

The table below sets forth our contractual obligations and commercial commitments as of June 30, 2021, that are expected to have an impact on liquidity and cash flows in future periods.

	Less than 1		Between 1		Between 3
	year		and 3 years		and 5 years		Over 5 years		Total
	(in thousands)
Debt obligations (including $15.6 million interest payments)	$	6,068	$	79,498	$	—	$	—	$	85,566
Operating lease obligations		5,862		12,712		13,576		25,954		58,104
Total	$	11,930	$	92,210	$	13,576	$	25,954	$	143,670

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We also have obligations to make future payments to third parties that become due and payable on the achievement of certain development, regulatory and commercial milestones (such as the start of a clinical trial, filing of a BLA, approval by the FDA or product launch). We have not included these commitments on our balance sheet or in the table above because the achievement and timing of these milestones is not fixed and determinable. We also have obligations to make future payments that become due and payable upon the collection of milestone payments from CSL Behring. We have not included these commitments on our balance sheet or in the table above because the achievement and timing of these milestones is not fixed and determinable.

We enter into contracts in the normal course of business with clinical research organizations (“CROs”) for preclinical research studies and clinical trials, research supplies and other services and products for operating purposes. These contracts generally provide for termination on notice, and therefore are cancelable contracts and not included in the table of contractual obligations and commitments.

Off-Balance Sheet Arrangements

As of June 30, 2021, we did not have any off-balance sheet arrangements as defined in Item 303(a)(4) of Regulation S-K.

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Item 3.Quantitative and Qualitative Disclosures about Market Risk

We are exposed to a variety of financial risks in the normal course of our business, including market risk (including currency, price and interest rate risk), credit risk and liquidity risk. Our overall risk management program focuses on preservation of capital and the unpredictability of financial markets and has sought to minimize potential adverse effects on our financial performance and position.

Our market risks and exposures to such market risks during the six months ended June 30, 2021, have not materially changed from our market risks and our exposure to market risk discussed in Part II, Item 7A of our Annual Report on Form 10-K for the year ended December 31, 2020, which was filed with the SEC on March 1, 2021.

Item 4.Controls and Procedures

Evaluation of Disclosure Controls and Procedures

Our management, with the participation of our chief executive officer (“CEO”) and chief financial officer (“CFO”), evaluated the effectiveness of our disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended (the “Exchange Act”)) as of June 30, 2021. Based on such evaluation, our CEO and CFO concluded that as of June 30, 2021, our disclosure controls and procedures were effective to ensure that information required to be disclosed by it in reports the Company files or submits under the Exchange Act is recorded, processed, summarized and reported within the time periods specified in the rules and forms of the SEC, and that such material information is accumulated and communicated to the Company’s management, including its Principal Executive Officer and Principal Financial Officer, to allow timely decisions regarding required disclosure. Because of the inherent limitations in all control systems, any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving the desired control objectives, and management necessarily is required to apply its judgment in evaluating the cost-benefit relationship of possible controls and procedures. Furthermore, the Company’s controls and procedures can be circumvented by the individual acts of some persons, by collusion of two or more people or by management override of such control, and misstatements due to error or fraud may occur and not be detected on a timely basis.

Changes in Internal Control over Financial Reporting

During the second quarter of 2021, there were no changes in our internal control over financial reporting (as defined in Rule 13a-15(f) under the Exchange Act) that materially affected, or are reasonably likely to materially affect, our internal control over financial reporting except for additional controls established in relation to the revenue recognition under the CSL Behring Agreement and for calculating diluted earnings per ordinary share.  

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Part II – OTHER INFORMATION

Item 1.Legal Proceedings

On or about February 22, 2021, Dr. Konstantinova, VectorY B.V., and Forbion International Management B.V. commenced a summary proceeding in the Netherlands primarily seeking an order: (i) allowing VectorY and Dr. Konstantinova to continue their employment relationship; (ii) suspending the non-competition agreement between uniQure biopharma B.V. and Dr. Konstantinova; and (iii) precluding any monetary penalties pursuant to that non-competition agreement. The complaint also sought payment of the costs of legal proceedings and a monetary monthly payment to Dr. Konstantinova in lieu of a promise by uniQure biopharma B.V. to release Dr. Konstantinova from her obligations under the non-competition agreement.

On April 16, 2021, we settled all matters related to the dispute described above (the “Settlement”). In connection with the Settlement, we received, among other things, preference shares in VectorY representing 5% of the fully diluted share capital in VectorY. In addition, we and certain related Forbion entities entered into a Cooperation Agreement.

Under the terms of the Cooperation Agreement, we and the Forbion entities agreed to certain non-disparagement provisions, and the Forbion entities agreed, among other things, for a period of two years from April  16, 2021:

1. To vote all of their ordinary shares in uniQure N.V. (1) in favor of the re-election of any persons serving on the Board of Directors of the Company (the “Board”) as of the date of the Cooperation Agreement and nominated by the Board for re-election; (2) against any nominees to serve on the Board who have not been recommended by the Board, and (3) with respect to all other matters, other than certain defined exempt matters, in accordance with the Board’s recommendations as identified in our notice of general meeting or any supplement thereto. 

2. Not to make any announcement or proposal with respect to, or offer, seek, propose, or indicate an interest in (A) any form of business combination or acquisition or other transaction relating to assets or securities of the uniQure N.V. or any of its subsidiaries, (B) any form of restructuring, recapitalization, or similar transaction with respect to the uniQure N.V. or any of its subsidiaries or (C) any form of tender or exchange offer for the ordinary shares of the uniQure N.V. 

3. Not to make, engage in, assist with, or in any way participate in, directly or indirectly, any solicitation of proxies or written consents to vote (or withhold the vote of) any voting securities of uniQure N.V. 

4. Not to take certain other specified actions aimed at changing or influencing the Board, management, or control of the uniQure N.V. 

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Item 1A.Risk Factors

An investment in our ordinary shares involves a high degree of risk. You should carefully consider the following information about these risks, together with the other information appearing elsewhere in this Quarterly Report on Form 10-Q, including our financial statements and related notes thereto, and the risk factors discussed in Part I, Item 1A “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2020, filed with the SEC on March 1, 2021, before deciding to invest in our ordinary shares. We operate in a dynamic and rapidly changing industry that involves numerous risks and uncertainties. The risks and uncertainties described below are not the only ones we face. Other risks and uncertainties, including those that we do not currently consider material, may impair our business. If any of the risks discussed below actually occur, our business, financial condition, operating results or cash flows could be materially adversely affected. This could cause the value of our securities to decline, and you may lose all or part of your investment.

Summary Risk Factors

The following is a summary of the principal risks associated with an investment in our ordinary shares:

● We may encounter substantial delays in, and impediments to the progress of our clinical trials or fail to demonstrate the safety and efficacy of our product candidates.

● Our business and operations have been, and may continue to be, materially and adversely affected by the ongoing Covid pandemic.

● We may not be successful in our efforts to use our gene therapy technology platform to build a pipeline of additional product candidates.

● We may not be successful in our efforts to in-license or acquire product candidates that align with our research and development strategy.

● Our manufacturing facility is subject to significant government regulations and approvals. If we fail to comply with these regulations or to maintain these approvals our business could be materially harmed.

● Our resources might be adversely affected if we are unable to meet our product development and supply needs and obligations, including our ability to complete the validation of our existing manufacturing processes as well as to develop larger scale manufacturing processes, which could adversely affect our ability to sufficiently meet our future production needs or regulatory filing timelines.

● We cannot predict when or if we will obtain marketing approval to commercialize a product candidate.

● We are exposed to a number of external factors such as competition, insurance coverage of and pricing and reimbursement for our product candidates that may adversely affect our product revenue and that may cause our business to suffer.

● We rely on licenses of intellectual property from third parties, and such licenses may not provide adequate rights or may not be available in the future on commercially reasonable terms or at all, and our licensors may be unable to obtain and maintain patent protection for the technology or products that we license from them.

● If we are unable to obtain and maintain patent protection for our technology and products, or if the scope of the patent protection is not sufficiently broad, our ability to successfully commercialize our products may be impaired.

● Our reliance on third parties may require us to share our trade secrets, which could increase the possibility that a competitor will discover them or that our trade secrets will be misappropriated or disclosed.

● We will likely need to raise additional funding, which may not be available on acceptable terms, or at all. Failure to obtain capital when needed may force us to delay, limit or terminate our product development efforts or other operations which could have a material adverse effect on our business, financial condition, results of operations, and cash flows.

● Our relationships with customers and third-party payers will be subject to applicable anti-kickback, anti-bribery, fraud and abuse and other laws and regulations, which, if we are found in violation thereof, could expose us to criminal sanctions, civil penalties, contractual damages, reputational harm and diminished profits and future earnings.

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● We are subject to laws governing data protection in the different jurisdictions in which we operate. The implementation of such data protection regimes is complex, and should we fail to fully comply, we may be subject to penalties that may have an adverse effect on our business, financial condition, and results of operations.

● Our internal computer systems, or those of our collaborators or other contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of our product development programs.

● If we fail to maintain an effective system of internal controls, we may be unable to accurately report our results of operations or prevent fraud or fail to meet our reporting obligations, and investor confidence and the market price of our ordinary shares may be materially and adversely affected.

Risks Related to the Current Covid Pandemic

Our business and operations have been, and may continue to be, materially and adversely affected by the ongoing Covid pandemic.

The ongoing outbreak of Covid originated in Wuhan, China, in December 2019 and has since spread to multiple countries, including the United States and the Netherlands. On March 11, 2020, the WHO declared the outbreak a pandemic. The Covid pandemic is affecting the United States and global economies and has affected and may continue to affect our operations and those of third parties on which we rely. The Covid pandemic has caused and may continue to cause disruptions in our raw material supply, our commercial-scale manufacturing capabilities for AAV-based gene therapies, the development of our product candidates, employee productivity and the conduct of current and future clinical trials. In addition, the Covid pandemic has affected and may continue to affect the operations of the FDA, EMA, and other health authorities, which could result in delays of reviews and approvals, including with respect to our product candidates.

As evidenced by the postponement of procedures for two patients in our Phase I/II clinical study of AMT-130, the evolving Covid pandemic has impacted the pace of enrollment and procedures in our clinical trials, as well as caused challenges in scheduling follow-up visits and managing other aspects of our clinical trials. We may be affected by similar delays as patients may avoid or may not be able to travel to healthcare facilities and physicians’ offices unless due to a health emergency and clinical trial staff can no longer get to the clinic. Such facilities and offices have been and may continue to be required to focus limited resources on non-clinical trial matters, including treatment of Covid patients, thereby decreasing availability, in whole or in part, for clinical trial services. In addition, employee disruptions and remote working environments related to the Covid pandemic, and federal, state, and local public health measures designed to mitigate the spread of the virus, have impacted and could continue to negatively impact the efficiency and pace with which we work and develop our product candidates and our manufacturing capabilities. Further, while the potential economic impact brought by, and the duration of, the Covid pandemic is difficult to assess or predict, the impact of the Covid pandemic on global financial markets may reduce our ability to access capital, which could negatively impact our short-term and long-term liquidity. The ultimate impact of the Covid pandemic is highly uncertain and subject to change. We do not yet know the full extent of potential delays or impacts on our business, financing, or clinical trial activities or on healthcare systems or the global economy as a whole. However, these negative effects could have a material impact on our liquidity, capital resources, operations, and business and those of the third parties on whom we rely.

Risks Related to the Development of Our Product Candidates

None of our product candidates have been approved for commercial sale and they might never receive regulatory approval or become commercially viable. We have never generated any significant revenue from product sales and may never be profitable.

All our product candidates are in research or development. We have not generated any revenues from the sale of products or manufacturing of our product for a licensee and do not expect to generate any such revenue before 2022. Our product candidates, including AMT-130 and any of our other potential product candidates, will require extensive preclinical and/or clinical testing, manufacture development and regulatory approval prior to commercial use. Our research and development efforts may not be successful. Even if our clinical development efforts result in positive data, our product candidates may not receive regulatory approval or be successfully introduced and marketed at prices that would permit us to operate profitably.

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We may encounter substantial delays in and impediments to the progress of our clinical trials or fail to demonstrate the safety and efficacy of our product candidates.

Clinical and non-clinical development is expensive, time-consuming, and uncertain as to outcome. Our product candidates are in different stages of clinical or preclinical development, and there is a significant risk of failure or delay in each of these programs. We cannot guarantee that any preclinical tests or clinical trials will be completed as planned or completed on schedule, if at all. A failure of one or more preclinical tests or clinical trials can occur at any stage of testing. Events that may prevent successful or timely completion of clinical development, as well as product candidate approval, include, but are not limited to:

● occurrence of serious adverse events associated with a product candidate that are viewed to outweigh its potential benefits;

● delays in reaching a consensus with regulatory agencies on study design;

● delays in reaching agreement on acceptable terms with prospective clinical research organizations (“CROs”) and clinical trial sites;

● delays in receiving regulatory authorization to conduct the clinical trials or a regulatory authority decision that the clinical trial should not proceed;

● delays in obtaining or failure to obtain required IRB and IBC approval at each clinical trial site;

● requirements of regulatory authorities, IRBs, or IBCs to modify a study in such a way that it makes the study impracticable to conduct;

● regulatory authority requirements to perform additional or unanticipated clinical trials;

● regulatory authority refusal to accept data from foreign clinical study sites;

● disagreements with regulatory authorities regarding our study design, including endpoints, our chosen indication, or our interpretation of data from preclinical studies and clinical trials or a finding that a product candidate’s benefits do not outweigh its safety risks;

● delays in obtaining or failure to obtain required approvals from a DSMB or other required approvals;

● imposition of a clinical hold by regulatory agencies after an inspection of our clinical trial operations or trial sites;

● suspension or termination of clinical research for various reasons, including noncompliance with regulatory requirements or a finding that the participants are being exposed to unacceptable health risks, undesirable side effects, or other unexpected characteristics (alone or in combination with other products) of the product candidate, or due to findings of undesirable effects caused by a chemically or mechanistically similar therapeutic or therapeutic candidate;

● failure by CROs, other third parties or us to adhere to clinical trial requirements or otherwise properly manage the clinical trial process, including meeting applicable timelines, properly documenting case files, including the retention of proper case files, and properly monitoring and auditing clinical sites;

● failure of sites or clinical investigators to perform in accordance with Good Clinical Practice or applicable regulatory guidelines in other countries;

● failure of patients to abide by clinical trial requirements;

● difficulty or delays in patient recruiting into clinical trials or in the addition of new investigators;

● the impact of the COVID-19 pandemic on the healthcare system or any clinical trial sites;

● delays or deviations in the testing, validation, manufacturing, and delivery of our product candidates to the clinical sites;

● delays in having patients complete participation in a study or return for post-treatment follow-up;

● clinical trial sites or patients dropping out of a study;

● the number of patients required for clinical trials of our product candidates being larger than we anticipate;

● clinical trials producing negative or inconclusive results, or our studies failing to reach the necessary level of statistical significance, requiring that we conduct additional clinical trials or abandon product development programs;

● interruptions in manufacturing clinical supply of our product candidates or issues with manufacturing product candidates that meet the necessary quality requirements;

● unanticipated clinical trial costs or insufficient funding, including to pay substantial application user fees;

● occurrence of serious adverse events or other undesirable side effects associated with a product candidate that are viewed to outweigh its potential benefits;

● disagreements with regulatory authorities regarding the interpretation of our clinical trial data and results, or the emergency of new information about or impacting our product candidates;

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● determinations that there are issues with our manufacturing facility or process; or

● changes in regulatory requirements and guidance, as well as new, revised, postponed, or frozen regulatory requirements, especially in light of the change in the United States administration, that require amending or submitting new clinical protocols, undertaking additional new tests or analyses, or submitting new types or amounts of clinical data.

Before obtaining marketing approval from regulatory authorities for the sale of our product candidates, we must conduct extensive clinical trials to demonstrate the safety and efficacy of the product candidates in humans. Such trials and regulatory review and approval take many years. It is impossible to predict when or if any of our clinical trials will demonstrate that product candidates are effective or safe in humans.

If the results of our clinical trials are inconclusive, or fail to meet the level of statistical significance required for approval or if there are safety concerns or adverse events associated with our product candidates, we may:

● be delayed in or altogether prevented from obtaining marketing approval for our product candidates;

● obtain approval for indications or patient populations that are not as broad as intended or desired;

● obtain approval with labeling that includes significant use or distribution restrictions or safety warnings;

● be subject to changes with the way the product is administered;

● be required to perform additional clinical trials to support approval or be subject to additional post-marketing testing requirements;

● have regulatory authorities withdraw their approval of the product or impose restrictions on its distribution in the form of a modified risk evaluation and mitigation strategy;

● be subject to the addition of labeling statements, such as warnings or contraindications;

● be sued; or

● experience damage to our reputation.

Because of the nature of the gene therapies we are developing, regulators may also require us to demonstrate long-term gene expression, clinical efficacy and safety, which may require additional or longer clinical trials, and which may not be able to be demonstrated to the regulatory authorities’ standards.

Our ability to recruit patients for our trials is often reliant on third parties, such as clinical trial sites. Clinical trial sites may not have the adequate infrastructure established to handle gene therapy products or may have difficulty finding eligible patients to enroll into a trial.

In addition, we, or any collaborators we may have may not be able to locate and enroll enough eligible patients to participate in these trials as required by the FDA, the EMA or similar regulatory authorities outside the United States and the European Union. This may result in our failure to initiate or continue clinical trials for our product candidates or may cause us to abandon one or more clinical trials altogether. Because our programs are focused on the treatment of patients with rare or orphan or ultra-orphan diseases, our ability to enroll eligible patients in these trials may be limited or slower than we anticipate considering the small patient populations involved and the specific age range required for treatment eligibility in some indications. In addition, our potential competitors, including major pharmaceutical, specialty pharmaceutical and biotechnology companies, academic institutions and governmental agencies and public and private research institutions, may seek to develop competing therapies, which would further limit the small patient pool available for our studies. Also, patients may be reluctant to enroll in gene therapy trials where there are other therapeutic alternatives available or that may become available, which may be for various reasons including uncertainty about the safety or effectiveness of a new therapeutic such as a gene therapy and the possibility that treatment with a gene therapy therapeutic could preclude future gene therapy treatments due to the formation of antibodies following and in response to the treatment.

Any inability to successfully initiate or complete preclinical and clinical development could result in additional costs to us or impair our ability to receive marketing approval, to generate revenues from product sales or obtain regulatory and commercialization milestones and royalties. In addition, if we make manufacturing or formulation changes to our product candidates, including changes in the vector or manufacturing process used, we may need to conduct additional studies to bridge our modified product candidates to earlier versions. It is also possible that any such manufacturing of formulation changes may have an adverse impact on the performance of the product candidate. Clinical trial delays could also shorten any periods during which we may have the exclusive right to commercialize our product candidates or allow our competitors to bring products to market before we do, which could impair our ability to successfully commercialize our product candidates and may materially harm our business, financial condition, and results of operations.

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Our progress in early-stage clinical trials may not be indicative of long-term efficacy in late-stage clinical trials, and our progress in trials for one product candidate may not be indicative of progress in trials for other product candidates.

Study designs and results from previous studies are not necessarily predictive of our future clinical study designs or results, and initial, top-line, or interim results may not be confirmed upon full analysis of the complete study data. Our product candidates may fail to show the required level of safety and efficacy in later stages of clinical development despite having successfully advanced through initial clinical studies. Changes to product candidates may also impact their performance in subsequent studies.

By example, our initial clinical trials in hemophilia B were conducted with AMT-060. Following these studies, we made modifications to AMT-060, substituting two nucleotides in the coding sequence for FIX. This modified product candidate is etranacogene dezaparvovec. In 2017, we announced our plans to advance etranacogene dezaparvovec, which includes an AAV5 vector carrying the FIX-Padua transgene, into a pivotal study. While we believe etranacogene dezaparvovec and AMT-060, our product candidate that was previously studied in a Phase I/II study, have been demonstrated to be materially comparable in nonclinical studies and manufacturing quality assessments, it is possible that ongoing or future clinical studies of etranacogene dezaparvovec may show unexpected differences from AMT-060. Should these differences have an unfavorable impact on clinical outcomes, or should they not have their intended effect of increasing the product candidate’s FIX activity, they may adversely impact our ability to achieve regulatory approval or market acceptance of etranacogene dezaparvovec. We may also need to conduct additional or longer-term studies, which may delay regulatory submissions or approvals and which the regulatory authorities may ultimately not accept or approve.

In our Phase I/II clinical study of AMT-060, we screened patients for pre-existing anti-AAV5 antibodies to determine their eligibility for the trial. Three of the ten patients screened for the study tested positive for anti-AAV5 antibodies on reanalysis using a more sensitive antibody assay. Since we did not observe any ill-effects or correlation between the level of anti-AAV5 antibodies and clinical outcomes, patients who have anti-AAV5 antibodies are permitted to enroll in our planned pivotal study of etranacogene dezaparvovec. Since we only have been able to test a limited number of patients and have limited clinical and pre-clinical data, it is possible that ongoing or future clinical studies may not confirm these results, and if so, negatively impact the outcome of our study.

In advance of treating patients in the pivotal study of etranacogene dezaparvovec, we conducted a short study to confirm the dose expected to be used in the pivotal trial. The dose-confirmation study enrolled three patients, who were administered a single dose of 2x10¹³ gc/kg. We have relied on the short-term data from this study, including FIX activity and safety outcomes during the weeks following administration of etranacogene dezaparvovec, to confirm the dose to be used in the pivotal study. Following the results of this study, our Data Monitoring Committee confirmed the dose of 2x10¹³ gc/kg for administration in the pivotal study. Given the limited number of patients and short follow-up period, data from this study may differ materially from the future results of our planned pivotal study of etranacogene dezaparvovec.

A number of companies in the pharmaceutical and biotechnology industries have suffered significant setbacks in later-stage clinical trials even after achieving promising results in early-stage clinical trials. If a larger population of patients does not experience positive results during clinical trials, if these results are not reproducible or if our products show diminishing activity over time, our product candidates may not receive approval from the FDA or EMA. Data obtained from preclinical and clinical activities are subject to varying interpretations, which may delay, limit, or prevent regulatory approval. In addition, we may encounter regulatory delays or rejections because of many factors, including changes in regulatory policy during the period of product development. Failure to confirm favorable results from earlier trials by demonstrating the safety and effectiveness of our products in later-stage clinical trials with larger patient populations could have a material adverse effect on our business, financial condition, and results of operations.

Additionally, where there are differences in the early-stage and late-stage trials, such as the differences between AMT-060 and AMT-061, regulatory authorities may require additional or longer-term data in late-stage trials, which may delay regulatory submissions or approvals and which the regulatory authorities may ultimately not accept or approve.

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Fast track product, breakthrough therapy, priority review, or Regenerative Medicine Advanced Therapy (“RMAT”) designation by the FDA, or access to the PRIME scheme by the EMA, for our product candidates may not lead to faster development or regulatory review or approval process, and it does not increase the likelihood that our product candidates will receive marketing approval.

We have obtained and may in the future seek one or more of fast track designation, breakthrough therapy designation, RMAT designation, PRIME scheme access or priority review designation for our product candidates. A fast track product designation is designed to facilitate the clinical development and expedite the review of drugs intended to treat a serious or life-threatening condition and which demonstrate the potential to address an unmet medical need. A breakthrough therapy is defined as a drug that is intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition, where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. A RMAT designation is designed to accelerate approval for regenerative advanced therapies. Priority review designation is intended to speed the FDA marketing application review timeframe for drugs that treat a serious condition and, if approved, would provide a significant improvement in safety or effectiveness. PRIME is a scheme provided by the EMA, similar to the FDA’s breakthrough therapy designation, to enhance support for the development of medicines that target an unmet medical need.

For drugs and biologics that have been designated as fast track products, RMAT, or breakthrough therapies, or granted access to the PRIME scheme, interaction and communication between the regulatory agency and the sponsor of the trial can help to identify the most efficient path for clinical development. Sponsors of fast track products, RMAT products, or breakthrough therapies may also be able to submit marketing applications on a rolling basis, meaning that the FDA may review portions of a marketing application before the sponsor submits the complete application to the FDA, if the sponsor pays the user fee upon submission of the first portion of the marketing application and the FDA approves a schedule for the submission of the remaining sections. For products that receive a priority review designation, the FDA's marketing application review goal is shortened to six months, as opposed to ten months under standard review.

Designation as a fast track product, breakthrough therapy, RMAT, PRIME, or priority review product is within the discretion of the regulatory agency. Accordingly, even if we believe one of our product candidates meets the relevant criteria, the agency may disagree and instead determine not to make such designation. In any event, the receipt of such a designation for a product candidate may not result in a faster development process, review or approval compared to drugs considered for approval under conventional regulatory procedures and does not assure ultimate marketing approval by the agency. In addition, the FDA may later decide that the products no longer meet the applicable conditions for qualification as either a fast track product, RMAT, or a breakthrough therapy or, for priority review products, decide that the period for FDA review or approval will not be shortened.

      We may not be successful in our efforts to use our gene therapy technology platform to build a pipeline of additional product candidates.

An element of our strategy is to use our gene therapy technology platform to expand our product pipeline and to progress these candidates through preclinical and clinical development ourselves or together with collaborators. Although we currently have a pipeline of programs at various stages of development, we may not be able to identify or develop product candidates that are safe and effective. Even if we are successful in continuing to build our pipeline, the potential product candidates that we identify may not be suitable for clinical development. Research programs to identify new product candidates require substantial technical, financial, and human resources. We or any collaborators may focus our efforts and resources on potential programs or product candidates that ultimately prove to be unsuccessful. If we do not continue to successfully develop and commercialize product candidates based upon our technology, we may face difficulty in obtaining product revenues in future periods, which could result in significant harm to our business, results of operations and financial position and materially adversely affect our share price.

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Our strategy of obtaining rights to key technologies through in-licenses may not be successful.

We seek to expand our product pipeline from time to time in part by in-licensing the rights to key technologies, including those related to gene delivery, genes, and gene cassettes. The future growth of our business will depend in significant part on our ability to in-license or otherwise acquire the rights to additional product candidates or technologies, particularly through our collaborations with academic research institutions. However, we may be unable to in-license or acquire the rights to any such product candidates or technologies from third parties on acceptable terms or at all. The in-licensing and acquisition of these technologies is a competitive area, and many more established companies are also pursuing strategies to license or acquire product candidates or technologies that we may consider attractive. These established companies may have a competitive advantage over us due to their size, cash resources and greater clinical development and commercialization capabilities. In addition, companies that perceive us to be competitors may be unwilling to license rights to us. Furthermore, we may be unable to identify suitable product candidates or technologies within our areas of focus. If we are unable to successfully obtain rights to suitable product candidates or technologies, our business, financial condition, and prospects could suffer.

Negative public opinion and increased regulatory scrutiny of gene therapy and genetic research may damage public perception of our product candidates or adversely affect our ability to conduct our business or obtain marketing approvals for our product candidates.

Public perception may be influenced by claims that gene therapy is unsafe, and gene therapy may not gain the acceptance of the public or the medical community. The risk of cancer remains a concern for gene therapy, and we cannot assure that it will not occur in any of our planned or future clinical studies. In addition, there is the potential risk of delayed adverse events following exposure to gene therapy products due to persistent biological activity of the genetic material or other components of products used to carry the genetic material.

A small number of patients have experienced serious adverse events during our clinical trials of either AMT-060 (our first-generation hemophilia B gene therapy) or etranacogene dezaparvovec. In each instance of a serious adverse event, whether or not attributed to one of our product candidates, the issues resolved without delay in the respective clinical trial. However, adverse events in our clinical trials or those conducted by other parties (even if not ultimately attributable to our product candidates), and the resulting publicity, could result in delay, a hold or termination of our clinical trials, increased governmental regulation, unfavorable public perception, failure of the medical community to accept and prescribe gene therapy treatments, potential regulatory delays in the testing or approval of our product candidates, stricter labeling requirements for those product candidates that are approved and a decrease in demand for any such product candidates. If any of these events should occur, it may have a material adverse effect on our business, financial condition and results of operations.

Certain of our product candidates may require medical devices for product administration and/or diagnostics, resulting in our product candidates being deemed combination products. This may result in the need to comply with additional regulatory requirements. If we are unable to meet these regulatory requirements, we may be delayed or not be able to obtain product approval.

Certain of our product candidates, such as AMT-130, require medical devices, such as a stereotactic, magnetic resonance imaging guided catheter, for product administration.  Other of our product candidates may also require the use of a companion diagnostic device to confirm the presence of specific genetic or other biomarkers.  This may result in our product candidates being deemed to be combination products, potentially necessitating compliance with the FDA’s investigational device regulations, separate marketing application submissions for the medical device component, a demonstration that our product candidates are safe and effective when used in combination with the medical devices, cross labeling with the medical device, and compliance with certain of the FDA’s device regulations.  If we are not able to comply with the FDA’s device regulations, if we are not able to effectively partner with the applicable medical device manufacturers, if we or any partners are not able to obtain any required FDA clearances or approvals of the applicable medical devices, or if we are not able to demonstrate that our product candidates are safe and efficacious when used with the applicable medical devices, we may be delayed in or may never obtain FDA approval for our product candidates, which would materially harm our business.

Moreover, certain of our delivery modalities, such as direct delivery of product candidates to the brain, may require significant physician ability and skill.  If physicians are not able to effectively deliver our product candidates to the applicable site of action or if delivery modalities are too difficult, we may never be able to obtain approval for our product candidates, may be delayed in obtaining approval, or, following approval, physicians may not adopt our product candidates, any of which may materially harm our business.

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Risks Related to Our Manufacturing

Our manufacturing facility is subject to significant government regulations and approvals. If we fail to comply with these regulations or maintain these approvals our business will be materially harmed.

Our manufacturing facility in Lexington is subject to ongoing regulation and periodic inspection by the FDA, EMA, and other regulatory bodies to ensure compliance with current cGMP. Moreover, before approving a BLA for any product candidate, the FDA will inspect our manufacturing facility and processes. Any failure to follow and document our adherence to such cGMP regulations or other regulatory requirements may lead to significant delays in the availability of products for commercial sale or clinical study, may result in the termination of or a hold on a clinical study, or may delay or prevent filing or approval of marketing applications for our products.

Failure to comply with applicable regulations could also result in the FDA, EMA, or other applicable authorities taking various actions, including levying fines and other civil penalties; imposing consent decrees or injunctions; requiring us to suspend or put on hold one or more of our clinical trials; suspending or withdrawing regulatory approvals; delaying or refusing to approve pending applications or supplements to approved applications; requiring us to suspend manufacturing activities or product sales, imports or exports; requiring us to communicate with physicians and other customers about concerns related to actual or potential safety, efficacy, and other issues involving our products; mandating or recommending product recalls or seizing products; imposing operating restrictions; and seeking criminal prosecutions, among other outcomes. Poor control of production processes can also lead to the introduction of adventitious agents or other contaminants, or to inadvertent changes in the properties or stability of a product candidate that may not be detectable in final product testing and that could have an adverse effect on clinical studies, or patient safety or efficacy. Moreover, if our manufacturing facility is not able to follow regulatory requirements, we may need to implement costly and time-consuming remedial actions. Any of the foregoing could materially harm our business, financial condition, and results of operations.

Moreover, if we are not able to manufacture a sufficient amount of our product candidates for clinical studies or eventual commercialization, our development program and eventual commercial prospects will be harmed.  If we cannot produce an adequate amount of our product candidates in compliance with the applicable regulatory requirements, we may need to contract with a third party to do so, in which case third party manufacturers may not be available or available on favorable terms.  The addition of a new manufacturer may also require FDA approvals, which we may not be able to obtain.

Gene therapies are complex and difficult to manufacture. We could experience capacity, production or technology transfer problems that result in delays in our development or commercialization schedules or otherwise adversely affect our business.

The insect-cell based manufacturing process we use to produce our products and product candidates is highly complex and in the normal course is subject to variation or production difficulties. Issues with any of our manufacturing processes, even minor deviations from the normal process, could result in insufficient yield, product deficiencies or manufacturing failures that result in adverse patient reactions, lot failures, insufficient inventory, product recalls and product liability claims. Additionally, we may not be able to scale up some or all of our manufacturing processes that may result in delays in regulatory approvals or otherwise adversely affect our ability to manufacture sufficient amounts of our products.

Many factors common to the manufacturing of most biologics and drugs could also cause production interruptions, including raw materials shortages, raw material failures, growth media failures, equipment malfunctions, facility contamination, labor problems, natural disasters, disruption in utility services, terrorist activities, or cases of force majeure and acts of god (including the effects of the Covid pandemic) beyond our control. We also may encounter problems in hiring and retaining the experienced specialized personnel needed to operate our manufacturing process, which could result in delays in our production or difficulties in maintaining compliance with applicable regulatory requirements.

Any problems in our manufacturing processes or facilities could make us a less attractive collaborator for academic research institutions and other parties, which could limit our access to additional attractive development programs, result in delays in our clinical development or marketing schedules and materially harm our business.

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Our use of viruses, chemicals and other hazardous materials requires us to comply with regulatory requirements and exposes us to significant potential liabilities.

Our development and manufacturing processes involve the use of viruses, chemicals, other (potentially) hazardous materials and produce waste products. Accordingly, we are subject to national, federal, state, and local laws and regulations in the United States and the Netherlands governing the use, manufacture, distribution, storage, handling, treatment, and disposal of these materials. In addition to ensuring the safe handling of these materials, applicable requirements require increased safeguards and security measures for many of these agents, including controlling access and screening of entities and personnel who have access to them, and establishing a comprehensive national database of registered entities. In the event of an accident or failure to comply with environmental, occupational health and safety and export control laws and regulations, we could be held liable for damages that result, and any such liability could exceed our assets and resources, and could result in material harm to our business, financial condition, and results of operations.

Our resources might be adversely affected if we are unable to validate our manufacturing processes or develop new processes to meet our product supply needs and obligations.

The manufacture of our AAV gene therapies, including etranacogene dezaparvovec, is complex and requires significant expertise.  Even with the relevant experience and expertise, manufacturers of gene therapy products often encounter difficulties in production, particularly in scaling out and validating initial production, and ensuring that the product meets required specifications. These problems include difficulties with production costs and yields, quality control, including stability and potency of the product, quality assurance testing, operator error, shortages of qualified personnel, as well as compliance with strictly enforced federal, state and foreign regulations.  In the past, we have manufactured certain batches of etranacogene dezaparvovec, and other product candidates, intended for nonclinical, clinical and process validation purposes that have not met all of our pre-specified quality parameters.  To meet our expected future production needs and our regulatory filing timelines for etranacogene dezaparvovec, as well as other gene therapy product candidates, we will need to complete the validation of our existing manufacturing processes as well as to develop larger scale manufacturing processes. If we are unable to consistently manufacture etranacogene dezaparvovec, or other gene therapy product candidates, in accordance with our pre-specified quality parameters and applicable regulatory standards, it could adversely impact our ability to validate our manufacturing processes, to meet our production needs, to file our BLA or other regulatory submissions, to develop our other proprietary programs, to conserve our cash, or to receive financial payments pursuant to our agreements with third parties, including with CSL Behring in return for supplying etranacogene dezaparvovec following regulatory approval.

Risks Related to Regulatory Approval of Our Products

We cannot predict when or if we will obtain marketing approval to commercialize a product candidate.

The development and commercialization of our product candidates, including their design, testing, manufacture, safety, efficacy, purity, recordkeeping, labeling, storage, approval, advertising, promotion, sale, and distribution, are subject to comprehensive regulation by the FDA and other regulatory agencies in the United States, the EMA, and other regulatory agencies of the member states of the European Union, and similar regulatory authorities in other jurisdictions. Failure to obtain marketing approval for a product candidate in a specific jurisdiction will prevent us from commercializing the product candidate in that jurisdiction.

The process of obtaining marketing approval for our product candidates in the United States, the European Union, and other countries is expensive and may take many years, if approval is obtained at all. Changes in marketing approval policies during the development period, changes in or the enactment of additional statutes or regulations, or changes in regulatory review for each submitted product application, may cause delays in the approval or rejection of an application. Regulatory authorities have substantial discretion in the approval process and may refuse to accept any application, may decide that our data are insufficient for approval, may require additional preclinical, clinical, or other studies and may not complete their review in a timely manner. Further, any marketing approval we ultimately obtain may be for only limited indications or be subject to stringent labeling or other restrictions or post-approval commitments that render the approved product not commercially viable.

If we experience delays in obtaining marketing approval for any of our product candidates in the United States, the European Union, or other countries, the commercial prospects of our other product candidates may be harmed and our ability to generate revenues will be materially impaired.

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The risks associated with the marketing approval process are heightened by the status of our products as gene therapies.

We believe that all our current product candidates will be viewed as gene therapy products by the applicable regulatory authorities. While there are a number of gene therapy product candidates under development, in the United States, the FDA has only approved a limited number of gene therapy products, to date. Accordingly, regulators, like the FDA, may have limited experience with the review and approval of marketing applications for gene therapy products.

Both the FDA and the EMA have demonstrated caution in their regulation of gene therapy treatments, and ethical and legal concerns about gene therapy and genetic testing may result in additional regulations or restrictions on the development and commercialization of our product candidates that are difficult to predict. The FDA and the EMA have issued various guidance documents pertaining to gene therapy products, with which we likely must comply to gain regulatory approval of any of our product candidates in the United States or European Union, respectively. The close regulatory scrutiny of gene therapy products may result in delays and increased costs and may ultimately lead to the failure to obtain approval for any gene therapy product.

Regulatory requirements affecting gene therapy have changed frequently and continue to evolve, and agencies at both the U.S. federal and state level, as well as congressional committees and foreign governments, have sometimes expressed interest in further regulating biotechnology. In the United States, there have been a number of recent changes relating to gene therapy development. By example, FDA issued a number of new guidance documents on human gene therapy development, one of which was specific to human gene therapy for hemophilia and another of which was specific to rare diseases. Moreover, the European Commission conducted a public consultation in early 2013 on the application of EU legislation that governs advanced therapy medicinal products, including gene therapy products, which could result in changes in the data we need to submit to the EMA for our product candidates to gain regulatory approval or change the requirements for tracking, handling and distribution of the products which may be associated with increased costs. In addition, divergent scientific opinions among the various bodies involved in the review process may result in delays, require additional resources, and ultimately result in rejection. The FDA, EMA, and other regulatory authorities will likely continue to revise and further update their approaches to gene therapies in the coming years. These regulatory agencies, committees and advisory groups and the new regulations and guidelines they promulgate may lengthen the regulatory review process, require us to perform additional studies, increase our development costs, lead to changes in regulatory positions and interpretations, delay or prevent approval and commercialization of our product candidates or lead to significant post-approval limitations or restrictions. Delay or failure to obtain, or unexpected costs in obtaining, the regulatory approval necessary to bring a potential product to market could decrease our ability to generate sufficient product revenues to maintain our business.

Our failure to obtain or maintain orphan product exclusivity for any of our product candidates for which we seek this status could limit our commercial opportunity, and if our competitors are able to obtain orphan product exclusivity before we do, we may not be able to obtain approval for our competing products for a significant period.

Regulatory authorities in some jurisdictions, including the United States and the European Union, may designate drugs for relatively small patient populations as orphan drugs. While certain of our product candidates have received orphan drug designation, there is no guarantee that we will be able to receive such designations in the future. The FDA may grant orphan designation to multiple sponsors for the same compound or active molecule and for the same indication. If another sponsor receives FDA approval for such product before we do, we would be prevented from launching our product in the United States for the orphan indication for a period of at least seven years unless we can demonstrate clinical superiority.

Moreover, while orphan drug designation neither shortens the development or regulatory review time, nor gives the product candidate advantages in the regulatory review or approval process, generally, if a product with an orphan drug designation subsequently receives the first marketing approval for the relevant indication, the product is entitled to a period of market exclusivity, which precludes the FDA or the EMA from approving another marketing application for the same drug for the same indication for that period. The FDA and the EMA, however, may subsequently approve a similar drug or same drug, in the case of the United States, for the same indication during the first product's market exclusivity period if the FDA or the EMA concludes that the later drug is clinically superior in that it is shown to be safer or more effective or makes a major contribution to patient care. Orphan exclusivity in the United States also does not prevent the FDA from approving another product that is considered to be the same as our product candidates for a different indication or a different product for the same orphan indication.  If another product that is the same as ours is approved for a different indication, it is possible that third-party payors will reimburse for products off-label even if not indicated for the orphan condition.

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Orphan drug exclusivity may be lost if the FDA or the EMA determines that the request for designation was materially defective, or if the manufacturer is unable to assure sufficient quantity of the drug to meet the needs of patients with the rare disease or condition or if the incidence and prevalence of patients who are eligible to receive the drug in these markets materially increase. The inability to obtain or failure to maintain adequate product exclusivity for our product candidates could have a material adverse effect on our business prospects, results of operations and financial condition.

Additionally, regulatory criteria with respect to orphan products is evolving, especially in the area of gene therapy.  By example, in the United States, whether two gene therapies are considered to be the same for the purpose of determining clinical superiority is subject to change, and depends on a number of factors, including the expressed transgene, the vector, and other product or product candidate features. Accordingly, whether any of our product candidates will be deemed to be the same as another product or product candidate is uncertain.

As appropriate, we intend to seek all available periods of regulatory exclusivity for our product candidates. However, there is no guarantee that we will be granted these periods of regulatory exclusivity or that we will be able to maintain these periods of exclusivity.

The FDA grants product sponsors certain periods of regulatory exclusivity, during which the agency may not approve, and in certain instances, may not accept, certain marketing applications for competing drugs. For example, biologic product sponsors may be eligible for twelve years of exclusivity from the date of approval, seven years of exclusivity for drugs that are designated to be orphan drugs, and/or a six-month period of exclusivity added to any existing exclusivity period for the submission of FDA requested pediatric data. While we intend to apply for all periods of market exclusivity that we may be eligible for, there is no guarantee that we will be granted any such periods of market exclusivity. By example, regulatory authorities may determine that our product candidates are not eligible for periods of regulatory exclusivity for various reasons, including a determination by the FDA that a BLA approval does not constitute a first licensure of the product. Additionally, under certain circumstances, the FDA may revoke the period of market exclusivity. Thus, there is no guarantee that we will be able to maintain a period of market exclusivity, even if granted. In the case of orphan designation, other benefits, such as tax credits and exemption from user fees may be available. If we are not able to obtain or maintain orphan drug designation or any period of market exclusivity to which we may be entitled, we could be materially harmed, as we will potentially be subject to greater market competition and may lose the benefits associated with programs. It is also possible that periods of exclusivity will not adequately protect our product candidates from competition. For instance, even if we receive twelve years of exclusivity from the FDA, other applicants will still be able to submit and receive approvals for versions of our product candidates through a full BLA.

If we do not obtain or maintain periods of market exclusivity, we may face competition sooner than otherwise anticipated.  For instance, in the United States, this could mean that a competing biosimilar product may be able to submit an application to the FDA and obtain approval. This may require that we undertake costly and time-consuming patent litigation, to the extent available, or defend actions brought by the biosimilar applicant for declaratory judgement. If a biosimilar product does enter the market, it is possible that it could be substituted for one of our product candidates, especially if it is available at a lower price.

It is also possible that, at the time we obtain approval of our product candidates, regulatory laws and policies around exclusivities may have changed. For instance, there have been efforts to decrease the United States period of exclusivity to a shorter timeframe. Future proposed budgets, international trade agreements and other arrangements or proposals may affect periods of exclusivity.

Risks Related to Commercialization

If we are unable to successfully commercialize our product candidates or experience significant delays in doing so, our business could be materially harmed.

Our ability to generate product revenues will depend on the successful development and eventual commercialization of our product candidates. The success of our product candidates will depend on many factors, including:

● successful execution of our contractual relationship with CSL Behring for the commercialization of etranacogene dezaparvovec;

● successful completion of preclinical studies and clinical trials, and other work required by regulators;

● receipt and maintenance of marketing approvals from applicable regulatory authorities;

● our ability to timely manufacture sufficient quantities of our products according to required quality specifications;

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● obtaining and maintaining patent and trade secret protection and non-patent, orphan drug exclusivity for our product candidates;

● obtaining and maintaining regulatory approvals using our manufacturing facility in Lexington, Massachusetts;

● launch and commercialization of our products, if approved, whether alone or in collaboration with others;

● identifying and engaging effective distributors or resellers on acceptable terms in jurisdictions where we plan to utilize third parties for the marketing and sales of our product candidates;

● acceptance of our products, if approved, by patients, the medical community, and third-party payers;

● effectively competing with existing therapies and gene therapies based on safety and efficacy profiles;

● the strength of our marketing and distribution;

● achieve optimal pricing based on durability of expression, safety, and efficacy;

● the ultimate content of the regulatory authority approved label, including the approved clinical indications, and any limitations or warnings;

● any distribution or use restrictions imposed by regulatory authorities;

● the interaction of our products with any other medicines that patients may be taking or the restriction on the use of our products with other medicines;

● the standard of care at the time of product approval;

● the relative convenience and ease of administration of our products;

● obtaining and maintaining healthcare coverage and adequate reimbursement;

● any price concessions, rebates, or discounts we may need to provide;

● complying with any applicable post-approval requirements and maintaining a continued acceptable overall safety profile; and

● obtaining adequate reimbursement for the total patient population and each subgroup to sustain a viable commercial business model in U.S. and EU markets.

By example, even if our product candidates are approved, they may be subject to limitations that make commercialization difficult. There may be limitations on the indicated uses and populations for which the products may be marketed. They may also be subject to other conditions of approval, may contain significant safety warnings, including boxed warnings, contraindications, and precautions, may not be approved with label statements necessary or desirable for successful commercialization, or may contain requirements for costly post-market testing and surveillance, or other requirements, including the submission of a risk evaluation and mitigation strategy, or REMS, to monitor the safety or efficacy of the products. Failure to achieve or implement any of the above elements could result in significant delays or an inability to successfully commercialize our product candidates, which could materially harm our business.

The affected populations for our gene therapies may be smaller than we or third parties currently project, which may affect the size of our addressable markets.

Our projections of the number of people who have the diseases we are seeking to treat, as well as the subset of people with these diseases who have the potential to benefit from treatment with our therapies, are estimates based on our knowledge and understanding of these diseases. The total addressable market opportunities for these therapies will ultimately depend upon many factors, including the diagnosis and treatment criteria included in the final label, if approved for sale in specified indications, acceptance by the medical community, patient consent, patient access and product pricing and reimbursement.

Prevalence estimates are frequently based on information and assumptions that are not exact and may not be appropriate, and the methodology is forward-looking and speculative. The use of such data involves risks and uncertainties and is subject to change based on various factors. Our estimates may prove to be incorrect and new studies may change the estimated incidence or prevalence of the diseases we seek to address. The number of patients with the diseases we are targeting may turn out to be lower than expected or may not be otherwise amenable to treatment with our products, reimbursement may not be sufficient to sustain a viable business for all sub populations being studied, or new patients may become increasingly difficult to identify or access, any of which could adversely affect our results of operations and our business.

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The addressable markets for AAV-based gene therapies may be impacted by the prevalence of neutralizing antibodies to the capsids, which are an integral component of our gene therapy constructs. Patients that have pre-existing antibodies to a particular capsid may not be eligible for administration of a gene therapy that includes this particular capsid. For example, etranacogene dezaparvovec, our gene therapy candidate for hemophilia B patients, incorporates an AAV5 capsid. In our Phase I/II clinical study of AMT-060, we screened patients for pre-existing anti-AAV5 antibodies to determine their eligibility for the trial. Three of the ten patients screened for the study tested positive for anti-AAV5 antibodies on reanalysis. Although we did not observe any ill-effects or correlation between the level of anti-AAV5 antibodies and clinical outcomes in these three patients, suggesting that patients who have anti-AAV5 antibodies may still be eligible for AAV5-based gene therapies, since we only have been able to test a limited number of patients and have limited clinical and pre-clinical data, we do not know if future clinical studies will confirm these results. This may limit the addressable market for etranacogene dezaparvovec and any future revenues derived from the sale of the product, if approved.

Any approved gene therapy we seek to offer may fail to achieve the degree of market acceptance by physicians, patients, third party payers and others in the medical community necessary for commercial success.

Doctors may be reluctant to accept a gene therapy as a treatment option or, where available, choose to continue to rely on existing treatments. The degree of market acceptance of any of our product candidates that receive marketing approval in the future will depend on many factors, including:

● the efficacy and potential advantages of our therapies compared with alternative treatments;

● our ability to convince payers of the long-term cost-effectiveness of our therapies and, consequently, the availability of third-party coverage and adequate reimbursement;

● the cost of treatment with gene therapies, including ours, in comparison to traditional chemical and small-molecule treatments;

● the limitations on use and label requirements imposed by regulators;

● the convenience and ease of administration of our gene therapies compared with alternative treatments;

● the willingness of the target patient population to try new therapies, especially a gene therapy, and of physicians to administer these therapies;

● the strength of marketing and distribution support;

● the prevalence and severity of any side effects;

● limited access to site of service that can perform the product preparation and administer the infusion; and

● any restrictions by regulators on the use of our products.

A failure to gain market acceptance for any of the above reasons, or any reasons at all, by a gene therapy for which we receive regulatory approval would likely hinder our ability to recapture our substantial investments in that and other gene therapies and could have a material adverse effect on our business, financial condition, and results of operation.

If we are unable to expand our commercialization capabilities or enter into agreements with third parties to market and sell any of our product candidates for which we obtain marketing approval, we may be unable to generate any product revenue.

To successfully commercialize any products that may result from our development programs, we need to continue to expand our commercialization capabilities, either on our own or with others. The development of our own market development effort is, and will continue to be, expensive and time-consuming and could delay any product launch. Moreover, we cannot be certain that we will be able to successfully develop this capability.

We may enter into collaborations regarding our other product candidates with other entities to utilize their established marketing and distribution capabilities, but we may be unable to enter into such agreements on favorable terms, if at all. If any current or future collaborators do not commit sufficient resources to commercialize our products, or we are unable to develop the necessary capabilities on our own, we will be unable to generate sufficient product revenue to sustain our business. We compete with many companies that currently have extensive, experienced and well-funded medical affairs, marketing, and sales operations to recruit, hire, train and retain marketing and sales personnel. We also may face competition in any search for third parties to assist us with the sales and marketing efforts of our product candidates. Without an internal team or the support of a third party to perform marketing and sales functions, we may be unable to compete successfully against these more established companies.

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If the market opportunities for our product candidates are smaller than we believe they are, our product revenues may be adversely affected, and our business may suffer.

We focus our research and product development on treatments for severe genetic and orphan diseases. Our understanding of both the number of people who have these diseases, as well as the subset of people with these diseases who have the potential to benefit from treatment with our product candidates, are based on estimates. These estimates may prove to be incorrect and new studies may reduce the estimated incidence or prevalence of these diseases. The number of patients in the United States, the EU and elsewhere may turn out to be lower than expected, may not be otherwise amenable to treatment with our products or patients may become increasingly difficult to identify and access, any of which could adversely affect our business, financial condition, results of operations and prospects.

Further, there are several factors that could contribute to making the actual number of patients who receive other potential products less than the potentially addressable market. These include the lack of widespread availability of, and limited reimbursement for, new therapies in many underdeveloped markets. Further, the severity of the progression of a disease up to the time of treatment, especially in certain degenerative conditions, could diminish the therapeutic benefit conferred by a gene therapy. Lastly, certain patients’ immune systems might prohibit the successful delivery of certain gene therapy products to the target tissue, thereby limiting the treatment outcomes.

Our gene therapy approach utilizes vectors derived from viruses, which may be perceived as unsafe or may result in unforeseen adverse events. Negative public opinion and increased regulatory scrutiny of gene therapy may damage public perception of the safety of our product and product candidates and adversely affect our ability to conduct our business or obtain regulatory approvals for our product candidates.

Gene therapy remains a novel technology. Public perception may be influenced by claims that gene therapy is unsafe, and gene therapy may not gain the acceptance of the public or the medical community. In particular, our success will depend upon physicians who specialize in the treatment of genetic diseases targeted by our product and product candidates, if approved, prescribing treatments that involve the use of our product and product candidates, if approved, in lieu of, or in addition to, existing treatments with which they are familiar and for which greater clinical data may be available. More restrictive government regulations or negative public opinion would have an adverse effect on our business, financial condition, results of operations and prospects and may delay or impair the development and commercialization of our product candidates or demand for any products we may develop. For example, earlier gene therapy trials led to several well-publicized adverse events, including cases of leukemia and death seen in other trials using other vectors. Serious adverse events in our clinical trials, or other clinical trials involving gene therapy products or our competitors’ products, even if not ultimately attributable to the relevant product candidates, and the resulting publicity, could result in increased government regulation, unfavorable public perception, potential regulatory delays in the testing or approval of our product candidates, stricter labeling requirements for those product candidates that are approved and a decrease in demand for any products for which we obtain marketing approval.

Ethical, legal, and social issues may reduce demand for any gene therapy products for which we obtain marketing approval.

Prior to receiving certain gene therapies, patients may be required to undergo genetic testing. Genetic testing has raised concerns regarding the appropriate utilization and the confidentiality of information provided by genetic testing. Genetic tests for assessing a person’s likelihood of developing a chronic disease have focused public attention on the need to protect the privacy of genetic information. For example, concerns have been expressed that insurance carriers and employers may use these tests to discriminate on the basis of genetic information, resulting in barriers to the acceptance of genetic tests by consumers. This could lead to governmental authorities restricting genetic testing or calling for limits on or regulating the use of genetic testing, particularly for diseases for which there is no known cure. Any of these scenarios could decrease demand for any products for which we obtain marketing approval.

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If we obtain approval to commercialize any of our product candidates outside of the United States, a variety of risks associated with international operations could materially adversely affect our business.

We expect that we will be subject to additional risks in commercializing any of our product candidates outside the United States, including:

● different regulatory requirements for approval of drugs and biologics in foreign countries;

● reduced protection for intellectual property rights;

● unexpected changes in tariffs, trade barriers and regulatory requirements which may make it more difficult or expensive to export or import products and supplies to or from the United States;

● economic weakness, including inflation, or political instability in particular foreign economies and markets;

● compliance with tax, employment, immigration, and labor laws for employees living or traveling abroad;

● foreign currency fluctuations, which could result in increased operating expenses and reduced revenues, and other obligations incident to doing business in another country;

● workforce uncertainty in countries where labor unrest is more common than in the United States;

● production shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad; and

● business interruptions resulting from geopolitical actions, including war and terrorism or natural disasters including earthquakes, typhoons, floods, and fires.

We face substantial competition, and others may discover, develop, or commercialize competing products before or more successfully than we do.

The development and commercialization of new biotechnology and biopharmaceutical products, including gene therapies, is highly competitive. We may face intense competition with respect to our product candidates, as well as with respect to any product candidates that we may seek to develop or commercialize in the future, from large and specialty pharmaceutical companies and biotechnology companies worldwide, who currently market and sell products or are pursuing the development of products for the treatment of many of the disease indications for which we are developing our product candidates. Potential competitors also include academic institutions, government agencies and other public and private research organizations that conduct research, seek patent protection, and establish collaborative arrangements for research, development, manufacturing, and commercialization. In recent years, there has been a significant increase in commercial and scientific interest and financial investment in gene therapy as a therapeutic approach, which has intensified the competition in this area.

We are aware of numerous companies focused on developing gene therapies in various indications, including Applied Genetic Technologies Corp., Abbvie, Abeona Therapeutics, Adverum Biotechnologies, Ally Therapeutics, Apic Bio, Asklepios BioPharmaceutical, Astellas, AVROBIO, Bayer, Biogen, BioMarin, bluebird bio, CRISPR Therapeutics, Editas Medicine, Expression Therapeutics, Fate, Freeline Therapeutics, Generation Bio, Genethon, GlaxoSmithKline, Homology Medicines, Intellia Therapeutics, Johnson & Johnson, Krystal Biotech, Lexeo Therapeutics, LogicBio Therapeutics, Lysogene, MeiraGTx, Milo Biotechnology, Mustang Bio, Novartis, Orchard Therapeutics, Oxford Biomedica, Passage Bio, Pfizer, REGENXBIO, Renova Therapeutics, Roche, Rocket Pharmaceuticals, Sangamo Therapeutics, Sanofi, Selecta Biosciences, Sarepta Therapeutics, Sio Therapeutics, Solid Biosciences, SwanBio, Takeda, Taysha Gene Therapies, Ultragenyx, Vivet Therapeutics, and Voyager Therapeutics, as well as several companies addressing other methods for modifying genes and regulating gene expression. We may also face competition with respect to the treatment of some of the diseases that we are seeking to target with our gene therapies from protein, nucleic acid, antisense, RNAi and other pharmaceuticals under development or commercialized at pharmaceutical and biotechnology companies such as Alnylam Pharmaceuticals, Bayer, BioMarin, CSL Behring, Dicerna Pharmaceuticals, Ionis Pharmaceuticals, Novartis, Novo Nordisk, Pfizer, Translate Bio, Roche, Sanofi, Sobi, Takeda, WaVe Life Sciences, and numerous other pharmaceutical and biotechnology firms.

Our commercial opportunity could be reduced or eliminated if our competitors develop and commercialize products that are safer, more effective, have fewer or less severe side effects, are more convenient or are less expensive than the products that we develop. Our competitors also may obtain FDA, EMA, or other regulatory approval for their products more rapidly than we do, which could result in our competitors establishing a strong market position before we are able to enter the market. A competitor approval may also prevent us from entering the market if the competitor receives any regulatory exclusivities that block our product candidates. Because we expect that gene therapy patients may generally require only a single administration, we believe that the first gene therapy product to enter the market for a particular indication will likely enjoy a significant commercial advantage and may also obtain market exclusivity under applicable orphan drug regimes.

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Many of the companies with which we are competing or may compete in the future have significantly greater financial resources and expertise than we do in research and development, manufacturing, preclinical testing, conducting clinical trials, obtaining regulatory approvals, and marketing approved products. Mergers and acquisitions in the pharmaceutical and biotechnology industries may result in more resources being concentrated among a smaller number of our competitors. Smaller and other early-stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large and established companies. These third parties compete with us in recruiting and retaining qualified scientific and management personnel, establishing clinical trial sites and patient registration for clinical trials, as well as in acquiring technologies complementary to, or necessary for, our programs.

If we do not achieve our projected development goals in the timeframes we announce and expect, the commercialization of our product candidates may be delayed and, as a result, our stock price may decline.

For planning purposes, we estimate the timing of the accomplishment of various scientific, clinical, regulatory, and other product development goals, or development milestones. These development milestones may include the commencement or completion of scientific studies, clinical trials, the submission of regulatory filings, and approval for commercial sale. From time to time, we publicly announce the expected timing of some of these milestones. All these milestones are based on a variety of assumptions. The actual timing of these milestones can vary dramatically compared to our estimates, in many cases for reasons beyond our control. If we do not meet these milestones, including those that are publicly announced, the commercialization of our products may be delayed and, as a result, our stock price may decline.

Risks Related to Our Dependence on Third Parties

We rely, and expect to continue to rely, on third parties to conduct, supervise, and monitor our preclinical studies and clinical trials, and those third parties may not perform satisfactorily, including failing to meet deadlines for the completion of such trials or failing to comply with regulatory requirements.

We rely on third parties, study sites, and others to conduct, supervise, and monitor our preclinical and clinical trials for our product candidates and do not currently plan to independently conduct clinical or preclinical trials of any other potential product candidates. We expect to continue to rely on third parties, such as CROs, clinical data management organizations, medical and scientific institutions, and clinical and preclinical investigators, to conduct our preclinical studies and clinical trials.

While we have agreements governing the activities of such third parties, we have limited influence and control over their actual performance and activities. For instance, our third-party service providers are not our employees, and except for remedies available to us under our agreements with such third parties we cannot control whether or not they devote sufficient time and resources to our ongoing clinical, non-clinical, and preclinical programs. If these third parties do not successfully carry out their contractual duties, meet expected deadlines or conduct our preclinical studies or clinical trials in accordance with regulatory requirements or our stated protocols, if they need to be replaced or if the quality or accuracy of the data they obtain is compromised due to the failure to adhere to our protocols, regulatory requirements or for other reasons, our trials may be repeated, extended, delayed, or terminated, we may not be able to obtain, or may be delayed in obtaining, marketing approvals for our product candidates, we may not be able to, or may be delayed in our efforts to, successfully commercialize our product candidates, or we or they may be subject to regulatory enforcement actions. As a result, our results of operations and the commercial prospects for our product candidates would be harmed, our costs could increase and our ability to generate revenues could be delayed. To the extent we are unable to successfully identify and manage the performance of third-party service providers in the future, our business may be materially and adversely affected. Our third-party service providers may also have relationships with other entities, some of which may be our competitors, for whom they may also be conducting trials or other therapeutic development activities that could harm our competitive position.

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Our reliance on these third-parties for development activities will reduce our control over these activities. Nevertheless, we are responsible for ensuring that each of our studies is conducted in accordance with the applicable protocol, legal, regulatory, and scientific standards, and our reliance on third parties does not relieve us of our regulatory responsibilities. For example, we will remain responsible for ensuring that each of our trials is conducted in accordance with the general investigational plan and protocols for the trial. We must also ensure that our preclinical trials are conducted in accordance with GLPs, as appropriate. Moreover, the FDA and comparable foreign regulatory authorities require us to comply with GCPs for conducting, recording, and reporting the results of clinical trials to assure that data and reported results are credible and accurate and that the rights, integrity, and confidentiality of trial participants are protected. Regulatory authorities enforce these requirements through periodic inspections of trial sponsors, clinical and preclinical investigators, and trial sites. If we or any of our third-party service providers fail to comply with applicable GCPs or other regulatory requirements, we or they may be subject to enforcement or other legal actions, the data generated in our trials may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us to perform additional studies.

In addition, we will be required to report certain financial interests of our third-party investigators if these relationships exceed certain financial thresholds or meet other criteria. The FDA or comparable foreign regulatory authorities may question the integrity of the data from those clinical trials conducted by investigators who may have conflicts of interest.

We cannot assure that upon inspection by a given regulatory authority, such regulatory authority will determine that any of our trials complies with the applicable regulatory requirements. In addition, our clinical trials must be conducted with product candidates that were produced under GMP conditions. Failure to comply with these regulations may require us to repeat clinical trials, which would delay the regulatory approval process. We also are required to register certain clinical trials and post the results of certain completed clinical trials on a government-sponsored database, ClinicalTrials.gov, within specified timeframes. Failure to do so can result in enforcement actions and adverse publicity.

Agreements with third parties conducting or otherwise assisting with our clinical or preclinical studies might terminate for a variety of reasons, including a failure to perform by the third parties. If any of our relationships with these third parties terminate, we may not be able to enter into arrangements with alternative providers or to do so on commercially reasonable terms. Switching or adding additional third parties involves additional cost and requires management time and focus. In addition, there is a natural transition period when a new third party commences work. As a result, if we need to enter into alternative arrangements, it could delay our product development activities and adversely affect our business. Though we carefully manage our relationships with our third parties, there can be no assurance that we will not encounter challenges or delays in the future or that these delays or challenges will not have a material adverse impact on our business, financial condition and prospects, and results of operations.

We also rely on other third parties to store and distribute our products for the clinical and preclinical trials that we conduct. Any performance failure on the part of our distributors could delay development, marketing approval, or commercialization of our product candidates, producing additional losses and depriving us of potential product revenue.

We rely on third parties for important aspects of our development programs. If these parties do not perform successfully or if we are unable to enter into or maintain key collaboration or other contractual arrangements, our business could be adversely affected.

We have in the past entered into, and expect in the future to enter into, collaborations with other companies and academic research institutions with respect to important elements of our development programs.

Any collaboration may pose several risks, including the following:

● collaborators have significant discretion in determining the efforts and resources that they will apply to these collaborations;

● we may have limited or no control over the design or conduct of clinical trials sponsored by collaborators;

● we may be hampered from entering into collaboration arrangements if we are unable to obtain consent from our licensors to enter into sublicensing arrangements of technology we have in-licensed;

● if any collaborator does not conduct the clinical trials they sponsor in accordance with regulatory requirements or stated protocols, we will not be able to rely on the data produced in such trials in our further development efforts;

● collaborators may not perform their obligations as expected;

● collaborators may also have relationships with other entities, some of which may be our competitors;

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● collaborators may not pursue development and commercialization of any product candidates or may elect not to continue or renew development or commercialization programs based on clinical trial results, changes in the collaborators' strategic focus or available funding, or external factors, such as an acquisition, that divert resources or create competing priorities;

● collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial, or abandon a product candidate, repeat or conduct new clinical trials or require a new formulation of a product candidate for clinical testing;

● collaborators could develop, independently or with third parties, products that compete directly or indirectly with our products or product candidates, if, for instance, the collaborators believe that competitive products are more likely to be successfully developed or can be commercialized under terms that are more economically attractive than ours;

● our collaboration arrangements may impose restrictions on our ability to undertake other development efforts that may appear to be attractive to us;

● product candidates discovered in collaboration with us may be viewed by our collaborators as competitive with their own product candidates or products, which may cause collaborators to cease to devote resources to the commercialization of our product candidates;

● a collaborator with marketing and distribution rights that achieves regulatory approval may not commit sufficient resources to the marketing and distribution of such product or products;

● disagreements with collaborators, including over proprietary rights, contract interpretation or the preferred course of development, could cause delays or termination of the research, development or commercialization of product candidates, lead to additional responsibilities for us, delay or impede reimbursement of certain expenses or result in litigation or arbitration, any of which would be time-consuming and expensive;

● collaborators may not properly maintain or defend our intellectual property rights or may use our proprietary information in such a way as to invite litigation that could jeopardize or invalidate our rights or expose us to potential litigation;

● collaborators may infringe the intellectual property rights of third parties, which may expose us to litigation and potential liability; and

● collaborations may in some cases be terminated for the convenience of the collaborator and, if terminated, we could be required to expend additional funds to pursue further development or commercialization of the applicable product or product candidates.

If any collaboration does not result in the successful development and commercialization of products or if a collaborator were to terminate an agreement with us, we may not receive future research funding or milestone or royalty payments under that collaboration, and we may lose access to important technologies and capabilities of the collaboration. All the risks relating to product development, regulatory approval and commercialization described herein also apply to the activities of any development collaborators.

Risks Related to Our Intellectual Property

We rely on licenses of intellectual property from third parties, and such licenses may not provide adequate rights or may not be available in the future on commercially reasonable terms or at all, and our licensors may be unable to obtain and maintain patent protection for the technology or products that we license from them.

We currently are heavily reliant upon licenses of proprietary technology from third parties that is important or necessary to the development of our technology and products, including technology related to our manufacturing process, our vector platform, our gene cassettes and the therapeutic genes of interest we are using. These and other licenses may not provide adequate rights to use such technology in all relevant fields of use. Licenses to additional third-party technology that may be required for our development programs may not be available in the future or may not be available on commercially reasonable terms, which could have a material adverse effect on our business and financial condition.

In some circumstances, we may not have the right to control the preparation, filing and prosecution of patent applications, or to maintain the patents, covering technology that we license from third parties. In addition, some of our agreements with our licensors require us to obtain consent from the licensor before we can enforce patent rights, and our licensor may withhold such consent or may not provide it on a timely basis. Therefore, we cannot be certain that these patents and applications will be prosecuted and enforced in a manner consistent with the best interests of our business. In addition, if third parties who license patents to us fail to maintain such patents, or lose rights to those patents, the rights we have licensed may be reduced or eliminated.

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Our intellectual property licenses with third parties may be subject to disagreements over contract interpretation, which could narrow the scope of our rights to the relevant intellectual property or technology or increase our financial or other obligations to our licensors.

The agreements under which we license intellectual property or technology from third parties are complex, and certain provisions in such agreements may be susceptible to multiple interpretations. The resolution of any contract interpretation disagreement that may arise could narrow what we believe to be the scope of our rights to the relevant intellectual property or technology, or increase what we believe to be our financial or other obligations under the relevant agreement, either of which could have a material adverse effect on our business and financial condition.

If we fail to comply with our obligations in our intellectual property licenses with third parties, we could lose rights that are important to our business.

Our licensing arrangements with third parties may impose diligence, development and commercialization timelines, milestone payment, royalty, insurance, and other obligations on us. If we fail to comply with these obligations, our counterparties may have the right to terminate these agreements either in part or in whole, in which case we might not be able to develop, manufacture or market any product that is covered by these agreements or may face other penalties under the agreements. Such an occurrence could materially adversely affect the value of the product candidate being developed under any such agreement. Termination of these agreements or reduction or elimination of our rights under these agreements may result in our having to negotiate new or amended agreements with less favorable terms or cause us to lose our rights under these agreements, including our rights to important intellectual property or technology.

If we are unable to obtain and maintain patent protection for our technology and products, or if the scope of the patent protection is not sufficiently broad, our ability to successfully commercialize our products may be impaired.

We rely, in part, upon a combination of forms of intellectual property, including in-licensed and owned patents to protect our intellectual property. Our success depends in a large part on our ability to obtain and maintain this protection in the United States, the European Union, and other countries, in part by filing patent applications related to our novel technologies and product candidates. Our patents may not provide us with any meaningful commercial protection, prevent competitors from competing with us or otherwise provide us with any competitive advantage. For example, patents we own currently are and may become subject to future patent opposition or similar proceedings, which may result in loss of scope of some claims or the entire patent. Our competitors may be able to circumvent our owned or licensed patents by developing similar or alternative technologies or products in a non-infringing manner.

Successful challenges to our patents may result in loss of exclusivity or freedom to operate or in patent claims being narrowed, invalidated, or held unenforceable, in whole or in part, which could limit our ability to stop others from using or commercializing similar or identical technology and products, or limit the duration of the patent protection of our technology and products.

The patent prosecution process is expensive, time-consuming, and uncertain, and we may not be able to file and prosecute all necessary or desirable patent applications at a reasonable cost or in a timely manner. It is also possible that we will fail to identify patentable aspects of our research and development output before it is too late to obtain patent protection. Additionally, given the amount of time required for the development, testing and regulatory review of new product candidates, patents protecting such candidates might expire before or shortly after such candidates are commercialized. As a result, our owned and licensed patent portfolio may not provide us with sufficient rights to exclude others from commercializing products similar or identical to ours.

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The patent position of biotechnology and pharmaceutical companies generally is highly uncertain, involves complex legal and factual questions and has in recent years been the subject of much litigation. In addition, the laws of foreign countries may not protect our rights to the same extent as the laws of the United States. For example, EU patent law with respect to the patentability of methods of treatment of the human body is more limited than U.S. law. Publications of discoveries in the scientific literature often lag the actual discoveries, and patent applications in the United States and other jurisdictions are typically not published until 18 months after their priority date, or in some cases at all. Therefore, we cannot know with certainty whether we were the first to make the inventions or that we were the first to file for patent protection of the inventions claimed in our owned or licensed patents or pending patent applications. As a result, the issuance, scope, validity, enforceability, and commercial value of our patent rights are highly uncertain. Our pending and future patent applications may not result in patents being issued that protect our technology or products, in whole or in part, or which effectively prevent others from commercializing competitive technologies and products. Changes in either the patent laws or interpretation of the patent laws in the European Union, the United States or other countries may diminish the value of our patents or narrow the scope of our patent protection. Our inability to obtain and maintain appropriate patent protection for any one of our products could have a material adverse effect on our business, financial condition, and results of operations.

We may become involved in lawsuits to protect or enforce our patents or other intellectual property, or third parties may assert their intellectual property rights against us, which could be expensive, time consuming and unsuccessful.

Competitors may infringe our owned or licensed patents or other intellectual property. To counter infringement or unauthorized use, we may be required to file infringement claims, which can be expensive and time consuming. An adverse result in any litigation proceeding could put one or more of our patents at risk of being invalidated, maintained in more narrowly amended form or interpreted narrowly.

Even if resolved in our favor, litigation or other legal proceedings relating to intellectual property claims may cause us to incur significant expenses, increase our operating losses, reduce available resources, and could distract our technical and management personnel from their normal responsibilities. In addition, there could be public announcements of the results of hearings, motions or other interim proceedings or developments, which could have an adverse effect on the price of our ordinary shares.

Third parties may initiate legal proceedings alleging that we are infringing their intellectual property rights, the outcome of which would be uncertain and could have a material adverse effect on the success of our business. For example, outside of the United States two of the patents we own are subject to patent opposition. If these or future oppositions are successful or if we are found to otherwise infringe a third party's intellectual property rights, we could be required to obtain a license from such third party to continue developing and marketing our products and technology. We may not be able to obtain the required license on commercially reasonable terms or at all. Even if we could obtain a license, it could be non-exclusive, thereby giving our competitors access to the same technologies licensed to us. We could be forced, including by court order, to cease commercializing the infringing technology or product or otherwise to cease using the relevant intellectual property. In addition, we could be found liable for monetary damages, including treble damages and attorneys' fees if we are found to have willfully infringed a patent. A finding of infringement could prevent us from commercializing our product candidates or force us to cease or materially modify some of our business operations, which could materially harm our business. Claims that we have misappropriated the confidential information or trade secrets of third parties could have a similar negative impact on our business.

For example, we are aware of patents owned by third parties that relate to some aspects of our programs that are still in development. In some cases, because we have not determined the final methods of manufacture, the method of administration or the therapeutic compositions for these programs, we cannot determine whether rights under such third-party patents will be needed. In addition, in some cases, we believe that the claims of these patents are invalid or not infringed or will expire before commercialization. However, if such patents are needed and found to be valid and infringed, we could be required to obtain licenses, which might not be available on commercially reasonable terms, or to cease or delay commercializing certain product candidates, or to change our programs to avoid infringement.

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If we are unable to protect the confidentiality of our proprietary information and know-how, the value of our technology and products could be adversely affected.

In addition to seeking patent protection, we also rely on other proprietary rights, including protection of trade secrets, know-how and confidential and proprietary information. To maintain the confidentiality of our trade secrets and proprietary information, we enter into confidentiality agreements with our employees, consultants, collaborators and other third parties who have access to our trade secrets. Our agreements with employees also provide that any inventions conceived by the individual while rendering services to us will be our exclusive property. However, we may not obtain these agreements in all circumstances, and individuals with whom we have these agreements may not comply with their terms. The assignment of intellectual property rights may not be self-executing, or the assignment agreements may be breached, and we may be forced to bring claims against third parties, or defend claims that they may bring against us, to determine the ownership of what we regard as our intellectual property. In addition, in the event of unauthorized use or disclosure of our trade secrets or proprietary information, these agreements, even if obtained, may not provide meaningful protection, particularly for our trade secrets or other confidential information. To the extent that our employees, consultants, or contractors use technology or know-how owned by third parties in their work for us, disputes may arise between us and those third parties as to the rights in related inventions.

Adequate remedies may not exist in the event of unauthorized use or disclosure of our confidential information including a breach of our confidentiality agreements. Enforcing a claim that a party illegally disclosed or misappropriated a trade secret is difficult, expensive, and time consuming, and the outcome is unpredictable. In addition, some courts in and outside of the United States are less willing or unwilling to protect trade secrets. If any of our trade secrets were to be lawfully obtained or independently developed by a competitor or other third party, we would have no right to prevent them from using that technology or information to compete with us. The disclosure of our trade secrets or the independent development of our trade secrets by a competitor or other third party would impair our competitive position and may materially harm our business, financial condition, results of operations, stock price and prospects.

Our reliance on third parties may require us to share our trade secrets, which could increase the possibility that a competitor will discover them or that our trade secrets will be misappropriated or disclosed.

Because we collaborate from time to time with various organizations and academic research institutions on the advancement of our gene therapy platform, we must, at times, share trade secrets with them. We seek to protect our proprietary technology in part by entering into confidentiality agreements and, if applicable, materials transfer agreements, collaborative research agreements, consulting agreements or other similar agreements with our collaborators, advisors, and consultants prior to beginning research or disclosing proprietary information. These agreements typically limit the rights of the third parties to use or disclose our confidential information, such as trade secrets. Despite the contractual provisions employed when working with third parties, the need to share trade secrets and other confidential information increases the risk that such trade secrets become known by our competitors, are inadvertently incorporated into the technology of others, or are disclosed or used in violation of these agreements. Given that our proprietary position is based, in part, on our know-how and trade secrets, a competitor's discovery of our trade secrets or other unauthorized use or disclosure would impair our competitive position and may have a material adverse effect on our business.

In addition, these agreements typically restrict the ability of our collaborators, advisors, and consultants to publish data potentially relating to our trade secrets. Our academic collaborators typically have rights to publish data, if we are notified in advance and may delay publication for a specified time to secure our intellectual property rights arising from the collaboration. In other cases, publication rights are controlled exclusively by us, although in some cases we may share these rights with other parties. We also conduct joint research and development programs that may require us to share trade secrets under the terms of our research and development partnerships or similar agreements.

Some courts inside and outside the United States are less willing or unwilling to protect trade secrets. If any of our trade secrets were to be lawfully obtained or independently developed by a competitor, we would have no right to prevent them, or those with whom they communicate, from using that technology or information to compete with us.  

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Risks Related to Pricing and Reimbursement

We face uncertainty related to insurance coverage of, and pricing and reimbursement for product candidates for which we may receive marketing approval.

We anticipate that the cost of treatment using our product candidates will be significant. We expect that most patients and their families will not be capable of paying for our products themselves. There will be no commercially viable market for our product candidates without reimbursement from third party payers, such as government health administration authorities, private health insurers and other organizations. Even if there is a commercially viable market, if the level of third-party reimbursement is below our expectations, most patients may not be able to afford treatment with our products and our revenues and gross margins will be adversely affected, and our business will be harmed.

Government authorities and other third-party payers, such as private health insurers and health maintenance organizations, decide for which medications they will pay and, subsequently, establish reimbursement levels. Reimbursement systems vary significantly by country and by region, and reimbursement approvals must be obtained on a country-by-country basis. Government authorities and third-party payers have attempted to control costs by limiting coverage and the amount of reimbursement for particular medications and procedures and negotiating or requiring payment of manufacturer rebates. Increasingly, third party payers require drug companies to provide them with predetermined discounts from list prices, are exerting influence on decisions regarding the use of particular treatments and are limiting covered indications. Additionally, in the United States and some foreign jurisdictions, pending or potential legislative and regulatory changes regarding the healthcare system and insurance coverage could result in more rigorous coverage criteria and downward pressure on drug prices, and may affect our ability to profitably sell any products for which we obtain marketing approval. For example, on November 27, 2020, CMS issued an interim final rule implementing a Most Favored Nation payment model under which reimbursement for certain Medicare Part B drugs and biologicals will be based on a price that reflects the lowest per capita GDP-adjusted price of any non-U.S. member country of the OECD with a GDP per capita that is at least sixty percent of the U.S. GDP per capita.

The pricing review period and pricing negotiations for new medicines take considerable time and have uncertain results. Pricing review and negotiation usually begins only after the receipt of regulatory marketing approval, and some authorities require approval of the sale price of a product before it can be marketed. In some markets, particularly the countries of the European Union, prescription pharmaceutical pricing remains subject to continuing direct governmental control and to drug reimbursement programs even after initial approval is granted and price reductions may be imposed. Prices of medical products may also be subject to varying price control mechanisms or limitations as part of national health systems if products are considered not cost-effective or where a drug company's profits are deemed excessive. In addition, pricing and reimbursement decisions in certain countries can lead to mandatory price reductions or additional reimbursement restrictions in other countries. Because of these restrictions, any product candidates for which we may obtain marketing approval may be subject to price regulations that delay or prohibit our or our partners' commercial launch of the product in a particular jurisdiction. In addition, we or any collaborator may elect to reduce the price of our products to increase the likelihood of obtaining reimbursement approvals. If countries impose prices, which are not sufficient to allow us or any collaborator to generate a profit, we or any collaborator may refuse to launch the product in such countries or withdraw the product from the market. If pricing is set at unsatisfactory levels, or if the price decreases, our business could be harmed, possibly materially. If we fail to obtain and sustain an adequate level of coverage and reimbursement for our products by third party payers, our ability to market and sell our products could be adversely affected and our business could be harmed.

Due to the generally limited addressable market for our target orphan indications and the potential for our therapies to offer therapeutic benefit in a single administration, we face uncertainty related to pricing and reimbursement for these product candidates.

The relatively small market size for orphan indications and the potential for long-term therapeutic benefit from a single administration present challenges to pricing review and negotiation of our product candidates for which we may obtain marketing authorization. Most of our product candidates target rare diseases with relatively small patient populations. If we are unable to obtain adequate levels of reimbursement relative to these small markets, our ability to support our development and commercial infrastructure and to successfully market and sell our product candidates for which we may obtain marketing approval could be adversely affected.

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We also anticipate that many or all our gene therapy product candidates may provide long-term, and potentially curative benefit, with a single administration. This is a different paradigm than that of other pharmaceutical therapies, which often require an extended course of treatment or frequent administration. As a result, governments and other payers may be reluctant to provide the significant level of reimbursement that we seek at the time of administration of our gene therapies or may seek to tie reimbursement to clinical evidence of continuing therapeutic benefit over time. Additionally, there may be situations in which our product candidates will need to be administered more than once, which may further complicate the pricing and reimbursement for these treatments. In addition, considering the anticipated cost of these therapies, governments and other payers may be particularly restrictive in making coverage decisions. These factors could limit our commercial success and materially harm our business.  

Risks Related to Our Financial Position and Need for Additional Capital

We have incurred significant losses in prior years and expect to incur losses over the next several years and may never achieve or maintain profitability.

We had a net gain of $357.9 million in the six months ended June 30, 2021, and a net loss of $125.0 in the full year 2020 and $124.2 million in the full year 2019. As of June 30, 2021, we had an accumulated deficit of $426.8 million. In the past, we have financed our operations primarily through the sale of equity securities and convertible debt, venture loans, upfront payments from our collaboration partners and, to a lesser extent, subsidies and grants from governmental agencies and fees for services. We expect to finance our operations in 2021 and 2022 primarily from the $462.4 million payments we collected from CSL Behring in May 2021. We have devoted substantially all our financial resources and efforts to research and development, including preclinical studies and clinical trials. We expect to continue to incur significant expenses and losses over the next several years, and our net losses may fluctuate significantly from quarter to quarter and year to year. Our profit in the three and six months ended June 30, 2021 was materially impacted by the amount of license revenue that we recognized as a result of the Closing of the transaction under the CSL Behring Agreement.

We anticipate that our expenses will increase substantially as we:

● Advance the clinical development of AMT-130 for our Huntington’s disease gene therapy program;

● Advance multiple research programs related to gene therapy candidates targeting liver-directed and CNS diseases;

● Continue to expand our employee base to support research and development, as well as general and administrative functions;

● Acquire or in-license rights to new therapeutic targets or product candidates;

● Continue to expand, enhance, and optimize our technology platform, including our manufacturing capabilities, next-generation viral vectors and promoters, and other enabling technologies; and

● Maintain, expand, and protect our intellectual property portfolio, including in-licensing additional intellectual property rights from third parties.

We may never succeed in these activities and, even if we do, may never generate revenues that are sufficient to achieve or sustain profitability. Our failure to become and remain profitable would depress the value of our company and could impair our ability to expand our business, maintain our research and development efforts, diversify our product offerings, or even continue our operations.

We will likely need to raise additional funding, which may not be available on acceptable terms, or at all. Failure to obtain capital when needed may force us to delay, limit or terminate our product development efforts or other operations which could have a material adverse effect on our business, financial condition, results of operations and cash flows.

We expect to incur significant expenses in connection with our on-going activities and that we will likely need to obtain substantial additional funding in connection with our continuing operations. In addition, we have based our estimate of our financing requirements on assumptions that may prove to be wrong, and we could use our capital resources sooner than we currently expect.

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Adequate capital may not be available to us when needed or may not be available on acceptable terms. Our ability to obtain debt financing may be limited by covenants we have made the 2018 Amended Facility and our 2021 Amended Facility with Hercules and our pledge to Hercules of substantially all our assets as collateral. If we raise additional capital through the sale of equity or convertible debt securities, our shareholders' ownership interest could be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of holders of our ordinary shares.

If we raise additional funds through collaborations, strategic alliances, or marketing, distribution, or licensing arrangements with third parties, we may have to issue additional equity, relinquish valuable rights to our technologies, future revenue streams, products, or product candidates, or grant licenses on terms that may not be favorable to us. If we are unable to raise capital when needed or on attractive terms, we could be forced to delay, reduce, or eliminate our research and development programs or any future commercialization efforts, which would have a negative impact on our financial condition, results of operations and cash flows.

Our existing and any future indebtedness could adversely affect our ability to operate our business.

As of June 30, 2021, we had $70.0 million of outstanding principal of borrowings under the 2018 Amended Facility and 2021 Amended Facility, which following our January 2021 amendment we are required to repay in June 2023. We could in the future incur additional debt obligations beyond our borrowings from Hercules. Our existing loan obligations, together with other similar obligations that we may incur in the future, could have significant adverse consequences, including:

● requiring us to dedicate a portion of our cash resources to the payment of interest and principal, reducing money available to fund working capital, capital expenditures, research and development and other general corporate purposes;

● increasing our vulnerability to adverse changes in general economic, industry and market conditions;

● subjecting us to restrictive covenants that may reduce our ability to take certain corporate actions or obtain further debt or equity financing;

● limiting our flexibility in planning for, or reacting to, changes in our business and the industry in which we compete; and

● placing us at a disadvantage compared to our competitors that have less debt or better debt servicing options.

We may not have sufficient funds, and may be unable to arrange for additional financing, to pay the amounts due under our existing loan obligations. Failure to make payments or comply with other covenants under our existing debt could result in an event of default and acceleration of amounts due. Under the 2018 Amended Facility as well as the 2021 Amended Facility, the occurrence of an event that would reasonably be expected to have a material adverse effect on our business, operations, assets, or condition is an event of default. If an event of default occurs and the lender accelerates the amounts due, we may not be able to make accelerated payments, and the lender could seek to enforce security interests in the collateral securing such indebtedness, which includes substantially all our assets.

Risks Related to Other Legal Compliance Matters

Our relationships with customers and third-party payers will be subject to applicable anti-kickback, anti-bribery, fraud and abuse and other laws and regulations, which, if we are found in violation thereof, could expose us to criminal sanctions, civil penalties, contractual damages, reputational harm and diminished profits and future earnings.

Healthcare providers, physicians and third-party payers will play a primary role in the recommendation and prescription of any products for which we obtain marketing approval. Our future arrangements with third party payers and customers may expose us to broadly applicable anti-bribery laws, including the Foreign Corrupt Practices Act, as well as fraud and abuse and other healthcare laws and regulations that may constrain the business or financial arrangements and relationships through which we would be able to market, sell and distribute any products for which we obtain marketing approval.

Efforts to ensure that our business arrangements with third parties will comply with applicable laws and regulations could involve substantial costs. If our operations, or the activities of our collaborators, distributors or other third-party agents are found to be in violation of any of these laws or any other governmental regulations that may apply to us, we may be subject to significant civil, criminal, and administrative penalties, damages, fines, imprisonment, exclusion of products from government funded healthcare programs and the curtailment or restructuring of our operations. The costs associated with any of these actions could be substantial and could cause irreparable harm to our reputation or otherwise have a material adverse effect on our business, financial condition, and results of operations.

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We are subject to laws governing data protection in the different jurisdictions in which we operate. The implementation of such data protection regimes is complex, and should we fail to fully comply, we may be subject to penalties that may have an adverse effect on our business, financial condition, and results of operations.

Many national and state laws govern the privacy and security of health information and other personal and private information. They often differ from each other in significant ways. For instance, the EU has adopted a comprehensive data protection law called the General Data Protection Regulation (“GDPR”) that took effect in May 2018. The GDPR, together with the national legislation of the EU member states governing the processing of personal data, impose strict obligations and restrictions on the ability to collect, analyze and transfer personal data, including health data from clinical trials and adverse event reporting. In particular, these obligations and restrictions concern the consent of the individuals to whom the personal data relates, the information provided to the individuals, the transfer of personal data out of the EU, security breach notifications, security and confidentiality of the personal data, and imposition of substantial potential fines for breaches of the data protection obligations. The GDPR imposes penalties for non-compliance of up to the greater of EUR 20.0 million or 4% of worldwide revenue. Data protection authorities from the different EU member states may interpret the GDPR and national laws differently and impose additional requirements, which add to the complexity of processing personal data in the EU. Guidance on implementation and compliance practices are often updated or otherwise revised. The significant costs of compliance with, risk of regulatory enforcement actions under, and other burdens imposed by the GDPR as well as under other regulatory schemes throughout the world related to privacy and security of health information and other personal and private data could have an adverse impact on our business, financial condition, and results of operations.

Product liability lawsuits could cause us to incur substantial liabilities and to limit commercialization of our therapies.

We face an inherent risk of product liability related to the testing of our product candidates in human clinical trials and in connection with product sales. If we cannot successfully defend ourselves against claims that our product candidates or products caused injuries, we will incur substantial liabilities. Regardless of merit or eventual outcome, liability claims may result in:

● decreased demand for any product candidates or products that we develop or sell;

● injury to our reputation and significant negative media attention;

● negative publicity or public opinion surrounding gene therapy;

● withdrawal of clinical trial participants or sites, or discontinuation of development programs;

● significant costs to defend the related litigation;

● substantial monetary awards to trial participants or patients;

● loss of revenue;

● initiation of investigations, and enforcement actions by regulators; and product recalls, withdrawals, revocation of approvals, or labeling, marketing, or promotional restrictions;

● reduced resources of our management to pursue our business strategy; and

● the inability to further develop or commercialize any products that we develop.

Dependent upon the country where the clinical trial is conducted, we currently hold coverages ranging from EUR 500,000 to EUR 6,500,000 per occurrence and per clinical trial. Such coverage may not be adequate to cover all liabilities that we may incur. We may need to increase our insurance coverage as we expand our clinical trials. In addition, insurance coverage is increasingly expensive. We may not be able to maintain insurance coverage at a reasonable cost or in an amount adequate to satisfy any liability that may arise. In the event insurance coverage is insufficient to cover liabilities that we may incur, it could have a material adverse effect on our business, financial condition, and results of operations.

Healthcare legislative and regulatory reform measures may have a material adverse effect on our financial operations.

Our industry is highly regulated and changes in law may adversely impact our business, operations, or financial results. The Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation Act, or the PPACA, is a sweeping measure intended to, among other things, expand healthcare coverage within the United States, primarily through the imposition of health insurance mandates on employers and individuals and expansion of the Medicaid program. Several provisions of the law may affect us and increase certain of our costs.

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In addition, other legislative changes have been adopted since the PPACA was enacted. These changes include aggregate reductions in Medicare payments to providers of 2% per fiscal year, which went into effect on April 1, 2013 and, following passage of the Bipartisan Budget Act of 2018, will remain in effect through 2027 unless additional Congressional action is taken. In January 2013, President Obama signed into law the American Taxpayer Relief Act of 2012, which, among other things, further reduced Medicare payments to several types of providers and increased the statute of limitations period for the government to recover overpayments to providers from three to five years. These laws may result in additional reductions in Medicare and other healthcare funding, which could have a material adverse effect on our customers and, accordingly, our financial operations.

We anticipate that the PPACA, as well as other healthcare reform measures that may be adopted in the future, may result in more rigorous coverage criteria and additional downward pressure on the reimbursement our customers may receive for our products. Further, there have been, and there may continue to be, judicial and Congressional challenges to certain aspects of the PPACA. For example, the U.S. Tax Cuts and Jobs Act of 2017 includes a provision repealing, effective January 1, 2019, the tax-based shared responsibility payment imposed by the Affordable Care Act on certain individuals who fail to maintain qualifying health coverage for all or part of a year that is commonly referred to as the "individual mandate". Additional legislative and regulatory changes to the PPACA, its implementing regulations and guidance and its policies, remain possible in the 117th U.S. Congress and under the Biden Administration. However, it remains unclear how any new legislation or regulation might affect the prices we may obtain for any of our product candidates for which regulatory approval is obtained. Any reduction in reimbursement from Medicare and other government programs may result in a similar reduction in payments from private payers. The implementation of cost containment measures or other healthcare reforms may prevent us from being able to generate revenue, attain profitability or commercialize our products.

Our internal computer systems, or those of our collaborators or other contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of our product development programs.

Our internal computer systems and those of our current and any future collaborators and other contractors or consultants are vulnerable to damage from computer viruses, unauthorized access, natural disasters, terrorism, war and telecommunication and electrical failures. The size and complexity of our information technology systems, and those of our collaborators, contractors and consultants, and the large amounts of confidential information stored on those systems, make such systems vulnerable to service interruptions or to security breaches from inadvertent or intentional actions by our employees, third-party vendors and/or business partners, or from cyber-attacks by malicious third parties. Cyber-attacks are increasing in their frequency, sophistication, and intensity, and have become increasingly difficult to detect. Cyber-attacks could include the deployment of harmful malware, ransomware, denial-of-service attacks, social engineering, and other means to affect service reliability and threaten the confidentiality, integrity, and availability of information. Cyber-attacks also could include phishing attempts or e-mail fraud to cause payments or information to be transmitted to an unintended recipient. The increased number of employees working remotely due to Covid might increase our vulnerability to the above risk.

While we have not experienced a system failure, accident, cyber-attack, or security breach that has resulted in a material interruption in our operations to date, we have experienced and addressed recent system failures, cyber-attacks, and security breaches. In the future, such events could result in a material disruption of our development programs and our business operations, whether due to a loss of our trade secrets, data, or other proprietary information or other similar disruptions. Additionally, any such event that leads to unauthorized access, use or disclosure of personal information, including personal information regarding our patients or employees, could harm our reputation, cause us not to comply with federal and/or state breach notification laws and foreign law equivalents and otherwise subject us to liability under laws and regulations that protect the privacy and security of personal information. Security breaches and other inappropriate access can be difficult to detect, and any delay in identifying them may lead to increased harm of the type described above. While we have implemented security measures to protect our information technology systems and infrastructure, there can be no assurance that such measures will prevent service interruptions or security breaches that could adversely affect our business and the further development and commercialization of our product and product candidates could be delayed.

Risks Related to Employee Matters and Managing Growth

Our future success depends on our ability to retain key executives and technical staff and to attract, retain and motivate qualified personnel.

We are highly dependent on hiring, training, retaining and motivating key personnel to lead our research and development, clinical operations and manufacturing efforts. Although we have entered into employment agreements with our key personnel, each of them may terminate their employment on short notice. We do not maintain key person insurance for any of our senior management or employees.

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The loss of the services of our key employees could impede the achievement of our research and development objectives and seriously harm our ability to successfully implement our business strategy. Furthermore, replacing senior management and key employees may be difficult and may take an extended period because of the limited number of individuals in our industry with the breadth and depth of skills and experience required to successfully develop gene therapy products. Competition to hire from this limited pool is intense, and we may be unable to hire, train, retain or motivate these key personnel on acceptable terms.

If we are unable to continue to attract and retain high quality personnel, our ability to pursue our growth strategy will be limited.

Risks Related to Our Ordinary Shares

The price of our ordinary shares has been and may in the future be volatile and fluctuate substantially.

Our share price has been and may in the future be volatile. From the start of trading of our ordinary shares on the Nasdaq Global Select Market on February 4, 2014 through July 22, 2021, the sale price of our ordinary shares ranged from a high of $82.49 to a low of $4.72. The closing price on July 22, 2021, was $26.49 per ordinary share. The stock market in general and the market for smaller biopharmaceutical companies in particular, have experienced extreme volatility that has often been unrelated to the operating performance of particular companies. The market price for our ordinary shares may be influenced by many factors, including:

● the success of competitive products or technologies;

● results of clinical trials of our product candidates or those of our competitors;

● public perception of gene therapy;

● regulatory delays and greater government regulation of potential products due to adverse events;

● regulatory or legal developments in the European Union, the United States, and other countries;

● developments or disputes concerning patent applications, issued patents or other proprietary rights;

● the recruitment or departure of key personnel;

● the level of expenses related to any of our product candidates or clinical development programs;

● the results of our efforts to discover, develop, acquire or in-license additional product candidates or products;

● actual or anticipated changes in estimates as to financial results, development timelines or recommendations by securities analysts;

● variations in our financial results or those of companies that are perceived to be similar to us;

● changes in the structure of healthcare payment systems;

● market conditions in the pharmaceutical and biotechnology sectors;

● mergers, acquisitions, licensing, and collaboration activity among our peer companies in the pharmaceutical and biotechnology sectors; and

● general economic, industry and market conditions.

Our directors, executive officers and major shareholders, if they choose to act together, will continue to have a significant degree of control with respect to matters submitted to shareholders for approval.

Our directors, executive officers and major shareholders holding more than 5% of our outstanding ordinary shares, in the aggregate, beneficially own approximately 54.9% of our issued shares (including such shares to be issued in relation to exercisable options to purchase ordinary shares) as at June 30, 2021. As a result, if these shareholders were to choose to act together, they may be able, as a practical matter, to control many matters submitted to our shareholders for approval, as well as our management and affairs. For example, these persons, if they choose to act together, could control the election of the board directors and the approval of any merger, consolidation or sale of all or substantially all our assets. These shareholders may have interests that differ from those of other of our shareholders and conflicts of interest may arise.

Provisions of our articles of association or Dutch corporate law might deter acquisition bids for us that might be considered favorable and prevent or frustrate any attempt to replace our board.

Certain provisions of our articles of association may make it more difficult for a third party to acquire control of us or effect a change in our board. These provisions include:

● staggered terms of our directors;

● a provision that our directors may only be removed at a general meeting of shareholders by a two-thirds majority of votes cast representing more than half of the issued share capital of the Company; and

● a requirement that certain matters, including an amendment of our articles of association, may only be brought to our shareholders for a vote upon a proposal by our board.

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We do not expect to pay dividends in the foreseeable future.

We have not paid any dividends since our incorporation. Even if future operations lead to significant levels of distributable profits, we currently intend that earnings, if any, will be reinvested in our business and that dividends will not be paid until we have an established revenue stream to support continuing dividends. Accordingly, shareholders cannot rely on dividend income from our ordinary shares and any returns on an investment in our ordinary shares will likely depend entirely upon any future appreciation in the price of our ordinary shares.

If we fail to maintain an effective system of internal controls, we may be unable to accurately report our results of operations or prevent fraud or fail to meet our reporting obligations, and investor confidence and the market price of our ordinary shares may be materially and adversely affected.

If we fail to maintain the adequacy of our internal control over financial reporting, we may not be able to conclude on an ongoing basis that we have effective internal control over financial reporting. If we fail to maintain effective internal control over financial reporting, we could experience material misstatements in our financial statements and fail to meet our reporting obligations, which would likely cause investors to lose confidence in our reported financial information. This could in turn limit our access to capital markets, harm our results of operations, and lead to a decline in the trading price of our ordinary shares. Additionally, ineffective internal control over financial reporting could expose us to increased risk of fraud or misuse of corporate assets and subject us to potential delisting from The Nasdaq Global Select Market, regulatory investigations and civil or criminal sanctions. Our reporting and compliance obligations may place a significant strain on our management, operational and financial resources and systems for the foreseeable future.

Risks for U.S. Holders

We have in the past qualified and in the future may qualify as a passive foreign investment company, which may result in adverse U.S. federal income tax consequence to U.S. holders.

Based on our average value of our gross assets, our cash and cash equivalents as well as the price of our shares we qualified as a passive foreign investment company (“PFIC”) for U.S. federal income tax for 2016 but not between 2017 and 2020. A corporation organized outside the United States generally will be classified as a PFIC for U.S. federal income tax purposes in any taxable year in which at least 75% of its gross income is passive income or on average at least 50% of the gross value of its assets is attributable to assets that produce passive income or are held to produce passive income. Passive income for this purpose generally includes dividends, interest, royalties, rents and gains from commodities and securities transactions. Our status in any taxable year will depend on our assets and activities in each year, and because this is a factual determination made annually after the end of each taxable year, there can be no assurance that we will continue to qualify as a PFIC in future taxable years. The market value of our assets may be determined in large part by reference to the market price of our ordinary shares, which is likely to fluctuate, and may fluctuate considerably given that market prices of biotechnology companies have been especially volatile. If we were considered a PFIC for the current taxable year or any future taxable year, a U.S. holder would be required to file annual information returns for such year, whether the U.S. holder disposed of any ordinary shares or received any distributions in respect of ordinary shares during such year. In certain circumstances a U.S. holder may be able to make certain tax elections that would lessen the adverse impact of PFIC status; however, to make such elections the U.S. holder will usually have to have been provided information about the company by us, and we do not intend to provide such information.

The U.S. federal income tax rules relating to PFICs are complex. U.S. holders are urged to consult their tax advisors with respect to the purchase, ownership and disposition of our shares, the possible implications to them of us being treated as a PFIC (including the availability of applicable election, whether making any such election would be advisable in their particular circumstances) as well as the federal, state, local and foreign tax considerations applicable to such holders in connection with the purchase, ownership, and disposition of our shares.

Any U.S. or other foreign judgments may be difficult to enforce against us in the Netherlands.

Although we now report as a U.S. domestic filer for SEC reporting purposes, we are incorporated under the laws of the Netherlands. Some of the members of our board and senior management reside outside the United States. As a result, it may not be possible for shareholders to effect service of process within the United States upon such persons or to enforce judgments against them or us in U.S. courts, including judgments predicated upon the civil liability provisions of the federal securities laws of the United States. In addition, it is not clear whether a Dutch court would impose civil liability on us or any of our Board members in an original action based solely upon the federal securities laws of the United States brought in a court of competent jurisdiction in the Netherlands.

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The United States and the Netherlands currently do not have a treaty providing for the reciprocal recognition and enforcement of judgments, other than arbitration awards, in civil and commercial matters. Consequently, a final judgment for payment given by a court in the United States, whether or not predicated solely upon U.S. securities laws, would not automatically be recognized or enforceable in the Netherlands. To obtain a judgment which is enforceable in the Netherlands, the party in whose favor a final and conclusive judgment of the U.S. court has been rendered will be required to file its claim with a court of competent jurisdiction in the Netherlands. Such party may submit to the Dutch court the final judgment rendered by the U.S. court. If and to the extent that the Dutch court finds that the jurisdiction of the U.S. court has been based on grounds which are internationally acceptable and that proper legal procedures have been observed, the Dutch court will, in principle, give binding effect to the judgment of the U.S. court, unless such judgment contravenes principles of public policy of the Netherlands. Dutch courts may deny the recognition and enforcement of punitive damages or other awards. Moreover, a Dutch court may reduce the amount of damages granted by a U.S. court and recognize damages only to the extent that they are necessary to compensate actual losses or damages. Enforcement and recognition of judgments of U.S. courts in the Netherlands are solely governed by the provisions of the Dutch Civil Procedure Code.

Therefore U.S. shareholders may not be able to enforce against us or our board members or senior management who are residents of the Netherlands or countries other than the United States any judgments obtained in U.S. courts in civil and commercial matters, including judgments under the U.S. federal securities laws.

The rights and responsibilities of our shareholders and directors are governed by Dutch law and differ in some important respects from the rights and responsibilities of shareholders under U.S. law.

Although we now report as a U.S. domestic filer for SEC purposes, our corporate affairs are governed by our articles of association and by the laws governing companies incorporated in the Netherlands. The rights of our shareholders and the responsibilities of members of our board under Dutch law are different than under the laws of some U.S. jurisdictions. In the performance of their duties, our board members are required by Dutch law to consider the interests of uniQure, its shareholders, its employees, and other stakeholders and not only those of our shareholders (as would be required under the law of most U.S. jurisdictions). As a result of these considerations our directors may take action that would be different than those that would be taken by a company organized under the law of some U.S. jurisdictions

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Item 2.Unregistered Sales of Equity Securities and Use of Proceeds

None.

Item 3.Defaults Upon Senior Securities

None.

Item 4.Mine Safety Disclosures

Not applicable.

Item 5.Other Information

None.

Item 6.Exhibits

See the Exhibit Index immediately preceding the signature page to this Quarterly Report on Form 10-Q for a list of exhibits filed or furnished with this report, which Exhibit Index is incorporated herein by reference.

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EXHIBIT INDEX

2.1*† Sale and Purchase Agreement, executed June 21, 2021, by and between uniQure N.V. and Corlieve Therapeutics SAS

3.1* Amended Articles of Association of the Company, effective as of June 16, 2021.

4.1*t2014 Share Incentive Plan, Amended and Restated, effective as of June 16, 2021.

10.1*t Employment Agreement, effective May 17, 2021, by and between uniQure biopharma B.V. and Pierre Caloz

10.2*t Equity Side Letter, effective May 17, 2021, by and between uniQure N.V. and Pierre Caloz

10.3*tAmended and Restated Employment Agreement, effective June 15, 2021, by and between uniQure biopharma B.V. and Christian Klemt

31.1*     Rule 13a-14(a)/15d-14(a) Certification of Chief Executive Officer

31.2*     Rule 13a-14(a)/15d-14(a) Certification of Chief Financial Officer

32.1±     Certification of Principal Executive Officer and Principal Financial Officer pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002

101*      The following financial information from our Quarterly Report on Form 10-Q for the period ended June 30, 2021, filed with the Securities and Exchange Commission on July 26, 2021, is formatted in Inline Extensible Business Reporting Language (“iXBRL”): (i) Consolidated Balance Sheets; (ii) Consolidated Statements of Operations and Comprehensive Loss; (iii) Consolidated Statements of Shareholders’ Equity; (iv) Consolidated Statements of Cash Flows; and (v) Notes to Consolidated Financial Statements (tagged as blocks of text)

104*      The cover page from our Quarterly Report on Form 10-Q for the period ended June 30, 2021, filed with the Securities and Exchange Commission on July 26, 2021, is formatted in Inline Extensible Business Reporting Language (“iXBRL”)

† Portions of this exhibit (indicated by asterisks) have been omitted in accordance with the rules of the Securities and Exchange Commission.

*            Filed herewith.

±            Furnished herewith.

tIndicates a management contract or compensatory plan or arrangement

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SIGNATURE

Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.

UNIQURE, N.V.

By: /s/ Matthew Kapusta

Matthew Kapusta

Chief Executive Officer

(Principal Executive Officer)

By: /s/ Christian Klemt

Christian Klemt

Chief Financial Officer

(Principal Financial Officer)

Dated July 26, 2021

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EX-2.1

Exhibit 2.1

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

SALE AND PURCHASE AGREEMENT

by and among

UNIQURE N.V.

and

EACH OF THE SHAREHOLDERS OF CORLIEVE THERAPEUTICS PARTY HERETO,

and

THE HOLDER REPRESENTATIVE

Dated as of June 21, 2021

In presence of Corlieve

Condor House

5-10 St. Paul’s Churchyard

London EC4M 8AL

Tel. +44 (0)20 3201 5000

Fax: +44 (0)20 3201 5001

www.morganlewis.com

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

TABLE OF CONTENTS

SHARE PURCHASE AGREEMENT	1
INTRODUCTION	1
ARTICLE 1 DEFINITIONS AND CONSTRUCTION	2
ARTICLE 2 SALE OF THE SHARES	18
ARTICLE 3 REPRESENTATIONS AND WARRANTIES REGARDING THE SELLERS	26
ARTICLE 4 REPRESENTATIONS AND WARRANTIES REGARDING CORLIEVE	28
ARTICLE 5 REPRESENTATIONS AND WARRANTIES OF PURCHASER	41
ARTICLE 6 COVENANTS AND AGREEMENTS	42
ARTICLE 7 POST-CLOSING COVENANTS	47
ARTICLE 8 CLOSING CONDITIONS	49
ARTICLE 9 INDEMNIFICATION	51
ARTICLE 10 TERMINATION	57
ARTICLE 11 MISCELLANEOUS	59
EXHIBIT A: THE SELLERS	65
EXHIBIT B: THE FREE SHARES HOLDERS	66
EXHIBIT C: RESALE PROSPECTUS SUPPLEMENT;	67
EXHIBIT D: FORM OF FRENCH SHORT FORM TRANSFER AGREEMENT	70

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

SHARE PURCHASE AGREEMENT

This SHARE PURCHASE AGREEMENT (this “Agreement”) dated as of June 21, 2021 (the “Agreement Date”) is made and entered into by and among uniQure N.V., a public company with limited liability (naamloze vennootschap) formed under the laws of the Netherlands and registered with the Dutch trade register under number 54385229 (“Purchaser”); all shareholders of Corlieve Therapeutics SAS, a société par actions simplifiée formed under the laws of France (“Corlieve” or the “Company”), party hereto acting severally and not jointly (non solidairement) for the purposes hereof (“Sellers” and each, individually, a “Seller”); and the Holder Representative hereunder (collectively, the “Parties”), in presence of Corlieve for purposes of Section 4.

INTRODUCTION

WHEREAS, the Sellers currently own 100,000 ordinary shares of Corlieve (excluding Free Shares) (“Ordinary Shares”) in the aggregate and 602,488 ordinary shares called “Series A” shares for identification purposes only of Corlieve (“Series A Shares”) in the aggregate, as set forth in further detail on Exhibit A attached hereto;

WHEREAS, Corlieve has allocated 52,296 free shares (actions gratuites), of which 5,464 have been cancelled effective on the Closing Date.  The holders of the remaining free shares (“Free Shares Holders” and each, individually, a “Free Share Holder”) currently own 46,832 free shares as set forth in further detail on Exhibit B attached hereto, representing all of the issued and outstanding free shares and securities giving access to the share capital of Corlieve (the “Free Shares”);¹

WHEREAS, in connection with the execution of this Agreement, Purchaser and REGENXBIO INC., a corporation organized under the laws of the State of Delaware, with offices at 9804 Medical Center Drive, Rockville, MD 20850 (“RGX”), have reached certain agreements in respect of the RGX License Agreement.

WHEREAS, certain of the Sellers have extended a shareholder loan to Corlieve (the “Shareholder Loan”), which will be repaid before the Closing Date by way of set-off against the capital contribution obligation resulting from a share capital increase in favor of certain of the Sellers, representing the issuance of new Series A Shares, at a price of €10 each (the “Loan Conversion”);

WHEREAS, the Sellers and the Free Share Holders shall hold, on the Closing Date, 100% of the issued and outstanding share capital and of the voting rights in Corlieve (the “Shares”);

WHEREAS, the Sellers desire to sell, or cause to be sold, to Purchaser, and Purchaser desires to acquire from the Sellers, on the Closing Date, all of the Shares upon the terms and conditions hereinafter set forth;

WHEREAS, Purchaser desires to acquire from the Free Share Holders all of the Free Shares upon the terms and conditions hereinafter set forth;

WHEREAS, for certain limited purposes, and subject to the terms set forth herein, the Holder Representative shall serve as a representative of the Sellers; and

WHEREAS, provisions of the SER Merger Code (SER-Fusiegedragsregels 2015) and the Act on the works councils (Wet op de ondernemingsraden) are not applicable to the transactions contemplated hereunder.

1

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

NOW, THEREFORE, in consideration of the mutual covenants and agreements set forth in this Agreement and for other good and valuable consideration, the receipt and sufficiency of which are hereby acknowledged, the parties hereto agree as follows:

ARTICLE 1

DEFINITIONS AND CONSTRUCTION

1.1Definitions. As used in this Agreement, the following terms shall have the meanings set forth or as referenced below:

“AAV9” means (a) the recombinant adeno-associated virus serotype 9 vector with the specified sequence set forth in GenBank ([*]) and (b) any recombinant adeno-associated virus derivatives of such serotype 9 vector that are covered by the claims of the Valid RGX AAV9 Patents.

“Acceptable Undertaking” means the undertaking by the Purchaser and its Affiliates to take any of the following actions or commitments: (a) to ensure that the Corlieve Intellectual Property remains available for use in France by Corlieve; (b) to cause Corlieve to (i) file in France any patent application relating to a patentable invention developed by Corlieve for the purposes of its activities, without prejudice to the ability of Corlieve to additionally register such patent(s) in any territory other than France, and (ii) ensure that Patents filed pursuant to subparagraph (i) remain available for use in France by Corlieve; (c) to maintain Corlieve’s registered office in France and substantially the same level of human resources and equipment in France to continue operating the Business for a period of not more than two years from Closing; and (d) to grant the French Ministry of Economy, Finance and Recovery (Ministère de l’Economie, des Finances et de la Relance) with information, audit and access rights customarily required for the purpose of monitoring the above undertakings, provided that such rights do not unreasonably disrupt the normal operations of Corlieve.

“Accredited Investor” shall have the meaning set forth in Section 2.11(d)..

“Affiliate” shall mean, with respect to any Person, any other Person that directly or indirectly controls, is controlled by or is under common control with such Person. For the purposes of this definition, the term “control” (including, with correlative meanings, the terms “controlled by” and “under common control with”) as used with respect to a Person shall mean (a) direct or indirect ownership of more than fifty percent (50%) of the voting securities or other voting interest of any Person (including attribution from related parties) or (b) the possession, directly or indirectly, of the power to direct or cause the direction of the management and policies of such Person, whether through ownership of voting securities, by Contract, as a general partner, as a manager, or otherwise.

“Agreement” shall have the meaning set forth in the Preamble of this Agreement.

“Agreement Date” shall have the meaning set forth in the Preamble of this Agreement.

“Alternative Product” shall mean any pharmaceutical product in all forms, presentations, formulations, methods of administration and dosage forms, that targets the expression of the Grik2 Gene Sequence for the treatment of epilepsy in humans that uses Corlieve Intellectual Property,  excluding any Corlieve Product.

“Alternative Transaction” shall have the meaning set forth in Section 6.5.

“ANSM” shall have the meaning set forth in Section 4.23(b).

“API” means active pharmaceutical ingredient.

2

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

“Applicable Law” shall mean, with respect to any Person, any Law applicable to such Person or any of such Person’s property and assets or such Person’s legal representatives, officers, directors, employees, consultants or agents in their capacity as such Person’s legal representatives, officers, directors, employees, consultants or agents, respectively.

“Benefits” shall mean: (i) all compensation or benefits provided by any Contract signed with any existing or former employee, and (ii) all medical, dental, health, welfare, life insurance agreements, programs, policies, commitments or other arrangements.

“BLA” means a Biological License Application (BLA) pursuant to 21 C.F.R. § 601.2 (or any successor regulation thereto), or a NDA filed with the FDA as described in 21 C.F.R. § 314 (or any successor regulation thereto) for purposes of obtaining Regulatory Approval for a new biologic in the United States, or any equivalent filing in a country or regulatory jurisdiction other than the United States.

“BPI Loan Agreement” shall mean that certain loan agreement (contrat d’aide en avance récupérable n°DOS0138446/00) between BPIFrance Financement and Corlieve, dated 6 January 2021. “Business” shall mean the business of Corlieve as conducted as of the Agreement Date.

“Business Day” shall mean any day other than a Saturday, a Sunday or a day on which banks in New York City, New York, Paris, France or Amsterdam, The Netherlands, are authorized or obligated by Law or executive order to close.

“Bylaws” shall mean the by-laws (statuts) of Corlieve, updated as of 27 March 2021.

“Cap” shall have the meaning set forth in Section 9.5(c).

“Capitalization Table” shall have the meaning set forth in Section 4.3(a).

“CHMP” shall mean the Committee for Medicinal Products for Human Use of the EMA and any successor committee thereto.

“Claim Notice” shall have the meaning set forth in Section 9.3(a).

“Claim Response” shall have the meaning set forth in Section 9.3(a).

“Closing” shall have the meaning set forth in Section 2.2.

“Closing Date” shall mean the date on which the Closing occurs.

“Closing Date Allocation Schedule” shall mean a schedule, which shall be prepared in good faith by the Holder Representative, that sets forth (i) the amount of Closing Date Cash Consideration payable to each Seller and (ii) the Pro Rata Share of each Seller at Closing.

“Closing Date Cash Balance” shall mean the aggregate amount as of the Closing Date of cash and cash equivalents of Corlieve, as determined in accordance with French GAAP.

“Closing Date Cash Consideration” shall mean an amount equal to:

(a)Such amount, not less than [*], as is determined by the Purchaser in its sole discretion and notified by the Purchaser to the Holder Representative by the close of business on the second Business Day prior to Closing;

(b)plus the Closing Date Cash Balance;

3

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

(c)plus the Net Working Capital (which may be positive or negative);

(d)plus the Specified Costs;

(e)less the Transaction Expenses; and

(f)less the Closing Date Indebtedness.

“Closing Date Consideration” shall mean an amount equal to:

(a)[*];

(b)plus the Closing Date Cash Balance;

(c)plus the Net Working Capital (which may be positive or negative);

(d)plus the Specified Costs;

(e)less the Transaction Expenses; and

(f)less the Closing Date Indebtedness.

“Closing Date Indebtedness” shall mean the aggregate amount of Indebtedness of Corlieve as of the Closing Date.

“Closing Statement” shall have the meaning set forth in Section 2.4(b).

“Commercially Reasonable Efforts” means, with respect to a Party, such level of efforts that would be used by a similarly situated group as such Party and its Affiliates in conducting the development and registration of pharmaceutical products to which it has rights, taking into account product labeling, anticipated labeling, safety and efficacy, market potential, medical and clinical consideration, regulatory environment, profitability, manufacturability and competitive market conditions, the nature and extent of market exclusivity (including patent coverage and regulatory exclusivity), development costs and time, financial return on such product and other relevant considerations, all of which may be evaluated for each particular market. It is understood that such efforts may change from time to time based upon changes to the above factors. As a result, the exercise of diligence by Purchaser and its Affiliates is to be determined by judging Purchaser’s commercially reasonable efforts taken as a whole. “Commercially Reasonable Efforts” shall be determined on a country-by-country (or region-by-region, where applicable) and indication-by-indication basis, without regard to the particular circumstances of a Party, including any other product opportunities of such Party and without regard to any payments owed by such Party to the other Party under this Agreement.

“Confidentiality Agreement” shall have the meaning set forth in Section 6.2.

“Contract” shall mean any binding agreement, lease, license, commitment, purchase order, arrangement, mortgage, indenture, note, bond, deed, loan, evidence of Indebtedness, security agreement or other contract.

“Control” means, with respect to any material, information, or Intellectual Property right, that a Party and/or any Affiliate(s) of such Party (a) owns such material, information, or Intellectual Property right, or (b) has a license to or a right to use such material, information, or Intellectual Property right; in each case of (a) or (b), with the ability to grant to the other Party (and, with respect to Purchaser, as an Affiliate of Corlieve) access, a right to use, or a license or sublicense (as applicable) to such material, information, or Intellectual Property right, on the terms and conditions set forth herein, without (i) violating the terms of any agreement or other arrangement with or obligation to any Third Party in

4

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

existence as of the time such Party and/or any Affiliate(s) of such Party would first be required hereunder to grant the other Party such access, right to use or (sub)license or (ii) paying any sums of money to any Third Party that assigned or licensed such material, information, or Intellectual Property right to such first Party and/or any Affiliate(s) of such Party that become payable in connection with the other Party’s exploitation thereof hereunder, unless such sums are payable under any Existing License Agreement.

“Corlieve” shall have the meaning set forth in the Preamble of this Agreement.

“Corlieve Intellectual Property” shall mean any Intellectual Property Controlled by Corlieve as of the Closing Date.

“Corlieve Partner” shall have the meaning set forth in Section 4.23(a).

“Corlieve Patents” shall mean any Patents Controlled by Corlieve as of the Closing Date.

“Corlieve Product” shall mean any pharmaceutical product using AAV9 to knock down the expression of the Grik2 Gene Sequence, which product (i) contains a DNA sequence encoding an RNA sequence that is Covered or specifically disclosed (without reference to percent sequence identity or variants thereof) in any Corlieve Patents, or (ii) contains a DNA sequence encoding an  RNA sequence that has been listed on Schedule 1.1 (or delivered on an updated Schedule 1.1 at Closing), or (iii) is developed after the Closing Date and contains a DNA sequence encoding an RNA sequence with two or less nucleotide substitutions, deletions and/or additions to the “guide” nucleotide sequence of the DNA sequences referred to in subclauses (i) and (ii), in each case (alone or with other APIs), in all forms, presentations, formulations, methods of administration and dosage forms, for all indications and diagnostic uses in humans, including all human disease.

“Court Order” shall mean any judgment, decision, award, consent decree, injunction, ruling or order of any federal, state, local or other domestic or foreign court, arbitral tribunal or Governmental Authority that is binding on any Person or its property.

“Cover”, “Covered” or “Covering” means, with respect to any given product and Patents, that the composition of matter, method of manufacture or use of such product is claimed by a Valid Claim of such Patent (i.e., in the absence of a license under, or ownership of, such Patent, the manufacture, use or sale of such would infringe such Patent as issued or, in the case of a patent application, evaluating such patent application as if it were issued as a Patent as of the date of such evaluation).

“Covered Parties,” collectively, and “Covered Party,” individually, shall have the meaning set forth in Section 9.2(c).

“CRO” shall mean a contract research organization.

“Current Assets” shall mean Corlieve’s: (a) inventories; (b) accounts receivable from third parties (including trade and service accounts receivable owed); (c) other receivables from third parties, including receivables related to Tax Credits (including for the avoidance of doubt, any claim for Tax Credit reimbursement for which the relevant filing application has been made by Corlieve prior to the date hereof); (d) prepaid insurance; (e) prepaid expenses; (f) commissions and advances to third parties; (g) subsidies; and (h) prepaid custom duties; provided, however, that in no event shall “Current Assets” hereunder include or be deemed to include any item or amount included in or described in the definition of “Closing Date Cash Balance.”

“Current Liabilities” shall mean Corlieve’s: (a) accounts payable; (b) accrued expenses including Transaction Bonus; and (c) Taxes currently payable, in each case excluding any Liabilities incurred by Corlieve with the consent of Purchaser between the Agreement Date and the Closing Date,

5

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

as the case may be, such consent to be withheld in Purchaser’s sole discretion (other than any Liabilities expressly included in the definition of “Transaction Expenses”); provided, however, that in no event shall “Current Liabilities” hereunder include or be deemed to include any item or amount included in or described in the definition of “Closing Date Indebtedness.”

“Damages” shall have the meaning set forth in Section 9.2(a).

“De Minimis Amount” shall have the meaning set forth in Section 9.5(a)).

“Default” shall mean (a) any actual breach, violation or default or (b) the existence of circumstances or the occurrence of an event that with the passage of time or the giving of notice or both will constitute a breach, violation or default or give rise to a right of termination, renegotiation or acceleration.

“Deprioritization” shall mean the manufacturing of a batch of at least 20 liters of an Alternative Product (and “Deprioritize” shall have the corresponding meaning).

“Development Notice” shall have the meaning set forth in Section 2.9(h)(ii).

“Disclosure Letter” shall have the meaning set forth in the preamble to Article 4.

“Disclosure Notification” shall have the meaning set forth in Section 6.7.

“Disputed Amounts” shall have the meaning set forth in Section 2.4(d).

“Dollars” and “$” shall each mean lawful money of the United States.

“Downstream Milestone 4b Payment” shall have the meaning set forth in Section 2.9(c).

“Downstream Milestone 5b Payment” shall have the meaning set forth in Section 2.9(d).

“Downstream Payments” shall have the meaning set forth in Section 2.9(d).

“EMA” shall mean the European Medicines Agency and any successor agency thereto.

“Employees” shall mean each of the employees of Corlieve.

“Encumbrance” shall mean with respect to any asset, any adverse claim of title, lien, pledge, option, charge, easement, servitude, title defect, security interest, deed of trust, mortgage, conditional sales agreement, encumbrance, preemptive right, right of first refusal or first offer, restriction or other right of any third party, whether voluntarily incurred or arising by operation of law, and includes any agreement to give any of the foregoing in the future other than mandatory licenses arising by operation of law.

“Environmental Claim” shall mean any claim, notice, order, or Proceeding alleging Liability for, or an obligation with respect to, any investigation, monitoring, remediation, removal, cleanup, response, corrective action, damages to natural resources, personal injury, property damage, fines, penalties or other costs resulting from, related to or arising out of (i) the presence, release or threatened release of Hazardous Material at any location or (ii) any violation or alleged violation of any Environmental Law, and shall include any claim, notice, order, or Proceeding seeking Damages, contribution, cost recovery, compensation or injunctive relief resulting from, related to or arising out of the presence, release or threatened release of Hazardous Material or alleged injury or threat of injury to health, safety or the environment.

6

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

“Environmental Law” shall mean all Applicable Law relating to pollution or protection of human health and safety or the environment.

“Escrow Account” shall have the meaning set forth in Section 2.12(a).

“Escrow Agent” shall mean the escrow agent to be selected by the Holder Representative and Purchaser.

“Escrow Agreement” shall mean that certain Escrow Agreement, dated as of the Closing Date, by and among Purchaser, the Escrow Agent and the Holder Representative. The Escrow Agreement will be in customary form, contain terms and conditions consistent with this Agreement, and be mutually reasonably acceptable to Purchaser, the Escrow Agent and the Holder Representative.

“Escrow Release Date” shall have the meaning set forth in Section 2.12(b).

“Escrowed Amount” shall mean an amount in cash from time to time being held in escrow by the Escrow Agent pursuant to the terms of the Escrow Agreement, which shall be equal to [*] the following amount: [*] minus the Non GMP Batch Payment minus the Free Share Holdback, provided that if all or part of the Non GMP Batch Payment or the Free Share Holdback are paid by the Purchaser pursuant to this Agreement prior to the Escrow Release Date, a portion thereof equal to [*] of each such payment will be made to the Escrow Account.

“Estimated Closing Date Cash Balance” shall have the meaning set forth in Section 2.4(a).

“Estimated Closing Date Cash Consideration” shall be determined by the Purchaser based on the Estimated Closing Date Consideration and the definition of Closing Date Cash Consideration.

“Estimated Closing Date Consideration” shall have the meaning set forth in Section 2.4(a)).

“Estimated Closing Date Indebtedness” shall have the meaning set forth in Section 2.4(a).

“Estimated Closing Statement” shall have the meaning set forth in Section 2.4(a).

“Estimated Net Working Capital” shall have the meaning set forth in Section 2.4(a).

“Estimated Transaction Expenses” shall have the meaning set forth in Section 2.4(a).

“Euros” and “€” shall each mean the currency introduced at the start of the third stage of the European economic and monetary union pursuant to the treaty establishing the European Community, as amended.

“Existing License Agreement” shall mean the contracts listed at Section 4.18(b) of the Disclosure Letter.

“FDA” shall mean the U.S. Food and Drug Administration and any successor agency thereto.

“Financial Statements” shall mean reports of the statutory auditors, as applicable, and notes contained therein in respect of Corlieve as at December 31, 2020.

“Foreign Investment Approval” shall have the meaning set forth in Section 8.1(a).

“Founders” shall mean [*] and the Scientific Founders.

“French GAAP” shall mean generally accepted accounting principles in France, as consistently applied over all relevant periods.

7

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

“Free Shares” shall have the meaning set forth in the Recitals.

“Free Shares Holdback” shall mean the Pro Rata Share of the Closing Date Consideration and any Milestone Payments, as applicable, corresponding to the Free Shares.

“Free Shares Holder” and “Free Shares Holders” shall have the respective meanings set forth in the Recitals.

“Free Share Transfer Date” shall mean, with respect to any given Free Share, the date on which such Free Share can be transferred in accordance with Applicable Law.

“Fundamental Representations” shall mean the representations and warranties set forth in Section 3.1 (Power and Authorization), Section 3.4 (Ownership of the Shares), Section 3.6 (No Brokers), Section 4.1 (Organization of Corlieve), Section 4.3 (Capitalization) and Section 4.22 (No Brokers).

“Governmental Authority” shall mean any: (a) nation, state, province, territory, county, municipality, district or other jurisdiction of any nature; (b) international, multinational, federal, state, local, municipal, foreign or other government, agency or authority; or (c) governmental or quasi-governmental authority of any nature (including any governmental division, department, agency, Regulatory Authority, commission, instrumentality, official, organization, unit, body or Person and any court or other judicial or arbitral tribunal).

“Governmental Order” shall mean any order, writ, judgment, injunction, decree, stipulation, determination or award of any Governmental Authority.

“Grik2 Gene Sequence” means the gene sequence encoding human glutamate ionotropic receptor kainite type subunit 2 (GluK2: previously known as ionotropic glutamate receptor 6).

“Hazardous Materials” shall mean any hazardous or toxic substance, material, chemical, pollutant, contaminant or waste which is regulated under any Environmental Law.

“Holdback Amount” shall mean a cash amount equal to [*].

“Holder Representative” shall have the meaning set forth in Section 11.4(a).

“Holder Representative’s Costs” shall have the meaning set forth in Section 11.4(e).

“Holder Representative’s Fund” shall have the meaning set forth in Section 11.4(f).

“Indebtedness” shall mean (without duplication), as to any Person, (a) all obligations for the payment of principal, accrued and unpaid interest, prepayment or redemption penalties, unpaid fees or expenses and other monetary obligations in respect of (i) indebtedness of such Person for borrowed money or (ii) indebtedness evidenced by notes debentures, bonds or other similar instruments for the payment of which such Person is liable, in each case including any breakage costs and costs and expenses related to the termination, discharge and release of all related Encumbrances, (b) any obligations to reimburse the issuer of any letter of credit, surety bond, performance bond, bank guarantee or similar obligation, in each case to the extent drawn or otherwise not contingent, (c) all capitalized lease obligations of such Person that are, or should be, classified as balance sheet liability in accordance with French GAAP, (d) all indebtedness of third parties secured by an Encumbrance (other than a Permitted Encumbrance) on property owned or acquired by such Person, (e) any obligation that would be required to be reflected as debt on the balance sheet of such Person under French GAAP, (f) any unfunded benefit liabilities, (g) all indebtedness arising out of overdrafts, acceptance credit or

8

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

similar facilities, (h) all Indebtedness of others referred to in clauses (a) through (g) above guaranteed directly or indirectly in any manner by such Person, or in effect guaranteed directly or indirectly by such Person through an agreement to pay or purchase such Indebtedness, to advance or supply funds for the payment or purchase of such Indebtedness or otherwise to assure a creditor against loss, in each case including all accrued interest and prepayment penalties, if any, and (i) the amount of repayable advances under the BPI Loan Agreement not paid prior to Closing that are in excess of  [*] of all such repayable advances; provided, however, that (1) no amount under subclauses (h) or (i) shall be counted more than one time and (2) in no event shall “Indebtedness” hereunder include or be deemed to include any item, amount, Liability or obligation (i) included in or described in the definitions of “Current Liabilities” or “Transaction Expenses” or (ii) incurred by Corlieve with the consent of Purchaser between the Agreement Date and the Closing Date, as the case may be, such consent to be withheld in Purchaser’s sole discretion.

“Indemnifying Party” shall have the meaning set forth in Section 9.3(a).

“Independent Accountants” shall have the meaning set forth in Section 2.4(d).

“Instrument” shall have the meaning set forth in Section 11.4(c).

“Intellectual Property” means any and all intellectual property rights or similar proprietary rights throughout the world, including all (i) national and multinational statutory invention registrations, patents and patent applications of any type issued or applied for in any jurisdiction, including all provisionals, non-provisionals, divisions, continuations, continuations-in-part, reissues, extensions, supplementary protection certificates, reexaminations and the equivalents of any of the foregoing in any jurisdiction, and all inventions claimed in each such registration, patent or patent application (collectively, “Patents”), (ii) trademarks, service marks, trade dress, logos, brand names, certification marks, domain names, trade names, corporate names and other indications of origin, whether or not registered, in any jurisdiction, and all registrations and applications for registration of the foregoing in any jurisdiction, and all goodwill associated with the foregoing (collectively, “Trademarks”), (iii) copyrights (whether or not registered) and registrations and applications for registration thereof in any jurisdiction, including all derivative works, moral rights, renewals, extensions, reversions or restorations associated with such copyrights, regardless of the medium of fixation or means of expression (collectively, “Copyrights”), (iv) trade secrets, information, data, specifications, processes, methods, knowledge, experience, formulae, skills, techniques, schematics, drawings, blue prints, utility models, designs, technology, software, inventions, discoveries, ideas and improvements, including manufacturing information and processes, assays, engineering and other manuals and drawings, standard operating procedures, flow diagrams, regulatory, chemical, pharmacological, toxicological, pharmaceutical, structural activity relationship information, physical and analytical, safety, quality assurance, quality control and clinical data, technical information, research records and similar data and information (collectively, “Know-How”), (v) database rights, industrial designs, industrial property rights, publicity rights and privacy rights (vi) computer software and firmware, including data files, source code, object code and software-related specifications and documentation (collectively “Software”) and (vii) the right to assert, claim or sue and collect damages for the past, present or future infringement, misappropriation or other violation of any of the foregoing.

“Knowledge” shall mean (i) in the case of the Company, the actual knowledge of the Company, after due inquiry of the Company’s President and, with respect to Section 4.18, of the Company’s President and the Scientific Founders (each designated Person, at their actual knowledge, and with respect to  [*], after reasonable inquiry of Corlieve employees with respect to subject matters falling within the responsibilities of such employees) or (ii) in the case of the Purchaser, the actual knowledge of the Purchaser, after due inquiry of  [*].

9

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

“Laws” shall mean any federal, state, local or foreign law (including common law), statute, code, ordinance, rule, regulation, directive, judgment, order, award, writ, injunction, decree or other Court Order of any Governmental Authority.

“Liabilities” shall mean any and all debts, liabilities, claims, losses and obligations, whether accrued or fixed, known or unknown, absolute or contingent, matured or unmatured, asserted or unasserted, due or to become due, determined, determinable or otherwise, and whether or not required under French GAAP, as applicable, to be accrued on the financial statements of a Person, including all costs and expenses relating thereto.

“Licensed Know-How Schedule” shall mean a schedule, dated as of the Closing Date, which shall reasonably reflect all material written documents delivered, disclosed or otherwise transferred by RGX to Corlieve pursuant to the RGX License Agreement as of the Closing Date which contain material Know-How, including an index of (A) the collaboration folder named [*], which contains (i) a folder named [*] containing documents which have been shared between the parties to the MTA (as such term is defined in the RGX License Agreement), and (ii) folders containing documents exchanged between Corlieve and RGX in the conduct of the Research Plan (as such term is defined in the RGX Agreement), and (B) the Know-How provided by RGX to Corlieve pursuant to Section 2.8 (b) of the RGX Agreement.

“Litigation Conditions” shall mean, with respect to a Third Party Claim, (a) such Third Party Claim does not seek injunctive or equitable relief or non-monetary damages from the Covered Party; (b) such Third Party Claim does not relate to or arise in connection with any criminal proceeding, action, indictment, allegation or investigation; (c) the Indemnifying Party agrees that it will be liable to a Covered Party for any indemnifiable Damages relating to the Third Party Claim; (d) the Indemnifying Party is able to reasonably demonstrate that it has sufficient financial resources to defend such Third Party Claim; (e) the amount remaining under the Cap at the time of the Third Party Claim is reasonably sufficient to satisfy any likely judgment or settlement resulting from such Third Party Claim; and (f) such Third Party Claim has not been asserted directly or indirectly by or on behalf of a Person that is a licensee or supplier of Purchaser or Corlieve.

“Loan Conversion” shall have the meaning set forth in the Recitals.

“Major EU Country” shall mean each of France, Germany, Italy and Spain.

“Majority of Sellers” shall have the meaning set forth in Section 11.4.

“Material Adverse Effect” shall mean any change, event, condition, circumstance, development, occurrence or effect that is, or would reasonably be expected to constitute, individually or in the aggregate, a material adverse effect on the business, assets, liabilities, financial condition or results of operations of Corlieve as a whole or on the ability of Corlieve or the Sellers to consummate the transactions contemplated by this Agreement, other than any change, event, condition, circumstance, development, occurrence or effect arising from or related to (i) general changes in business, economic, political, social, legal or regulatory conditions, (ii) changes in conditions generally applicable to the industry in which Corlieve operates, (iii) general changes in financial, banking or securities markets (including any disruption thereof), (iv) changes in Applicable Laws or French GAAP, (v) pandemics, outbreak of hostilities, terrorist attack (whether against a nation or otherwise) or war, or (vi) the announcement of this Agreement or the transactions contemplated hereby, in each case, that do not disproportionately adversely affect Corlieve in relation to others who participate in the same business as Corlieve.

“Material Contracts” shall have the meaning set forth in Section 4.6(a).

10

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

“Milestone #1” shall have the meaning set forth in Section 2.9(a).

“Milestone #2” shall have the meaning set forth in Section 2.9(a).

“Milestone #3” shall have the meaning set forth in Section 2.9(a).

“Milestone #4a” shall have the meaning set forth in Section 2.9(a).

“Milestone #4b” shall have the meaning set forth in Section 2.9(a).

“Milestone #5a” shall have the meaning set forth in Section 2.9(a).

“Milestone #5b” shall have the meaning set forth in Section 2.9(a).

“Milestone Payment” shall mean the payments set forth in Section 2.9(a) with respect to Milestone #1, Milestone #2, Milestone #3, Milestone #4a or Milestone #4b and Milestone #5a or Milestone #5b, subject to Section 2.7.

“Milestone Set-Off” shall have the meaning set forth in Section 2.10.

“Milestones” shall mean Milestone #1, Milestone #2, Milestone #3, Milestone #4a, Milestone #4b, Milestone #5a and Milestone #5b.

“Milestone Shares” shall mean the uniQure Ordinary Shares, if any, that are issued by the Purchaser to satisfy its obligation to make the Milestone Payments, as the case may be.

“Milestone Shares Cap” shall have the meaning set forth in Section 2.9(f).

“Most Recent Balance Sheet” shall mean the audited statutory accounts of Corlieve as at December 31, 2020.

“Nasdaq” shall mean The Nasdaq Global Select Market (or any successor thereto).

“NDA” means a new drug application as described in 21 C.F.R. § 314.50, including all amendments and supplements to the application, submitted to the FDA under Section 505(b) of the FDC Act for approval to commercialize a new drug in the United States.

“Net Working Capital” shall mean (a) the Current Assets as of the Closing Date minus (b) the Current Liabilities as of the Closing Date.

“Non-GMP Batch Delivery” shall mean the delivery by RGX to Corlieve of Corlieve Product manufactured in accordance with Non-GMP specifications to support a pivotal GLP toxicology IND-enabling study by a process representative of the GMP process to be used in GMP manufacture of such Corlieve Product, as well as supporting analytics and all CMC Data required to enable lot release and IND filing, as contemplated in Section 5.1.1 of the RGX License Agreement.

“Non GMP Batch Payment” shall mean (i) with respect to the Closing Date Consideration, an amount to be notified by the Holder Representative to the Purchaser not less  [*] prior to Closing, estimated to be  [*] (the “Initial Non GMP Batch Payment”) and (ii) with respect to any Milestone Payment if and when due, the relative increase in the Pro Rata Share of RGX following payment of the Initial Non GMP Batch Payment, if any, as set out on Schedule 2.7.

“Ordinary Course of Business” or “Ordinary Course” shall mean the ordinary and usual course of the normal day-to-day operations of Corlieve, consistent with the past practice.

11

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

“Ordinary Shares” shall have the meaning set forth in the Recitals.

“Outside Date” shall have the meaning set forth in Section 10.1(b).

“Parties” shall have the meaning set forth in the Preamble.

“Patents” shall have the meaning set forth in the definition of “Intellectual Property.”

“Paying Agent” has the meaning set forth in Section 2.3(a)(ii).

“Paying Agent Agreement” shall mean that certain Paying Agent Agreement, dated as of the Closing Date, by and among Purchaser, the Paying Agent and the Holder Representative. The Paying Agent Agreement will be in customary form, contain terms and conditions consistent with this Agreement (including that any Seller who transfers its right to receive the Milestone Payments must provide notice to Purchaser and the Paying Agent, and that Purchaser may require an opinion of counsel in connection with such transfer), and be mutually reasonably acceptable to Purchaser, the Paying Agent and the Holder Representative.

“Permit” shall mean each permit, certificate, license, consent, registration, approval or authorization of any Governmental Authority.

“Permitted Encumbrance” shall mean (a) liens for Taxes, assessments and other governmental charges, in each case, not yet due and payable or which are being contested in good faith by appropriate proceedings and for which adequate reserves have been established in accordance with French GAAP or U.S. GAAP, as applicable, (b) statutory liens arising in the Ordinary Course of Business for sums not yet due, (c) statutory and contractual landlord liens under leases pursuant to which Corlieve is a lessee and not in Default, (d) licenses to service providers (such as contract manufacturing organizations and contract research organizations) in the Ordinary Course of Business or licenses back to licensors expressly granted pursuant to the Material Contracts, (e) pledges or deposits made in the Ordinary Course of Business which do not in the aggregate materially detract from the value of the related assets or properties or materially impair the use thereof in the operation of the Business as currently conducted, and (f) deposits and pledges made in connection with, or to secure payment of, workers’ compensation, unemployment insurance or similar programs.

“Permitted Transfer” shall mean a transfer of the right to receive the Milestone Payments by any Seller (a) upon death of such Seller by will or intestacy; (b) pursuant to a Court Order; (c) by operation of law (including by consolidation or merger) or without consideration in connection with the dissolution, liquidation or termination of any corporation, limited liability company, partnership or other entity; or (d) to any Person if such transfer is of such Seller’s entire right to receive the remaining portion of the Milestone Payments which such Seller is entitled to receive; provided, that, in connection with any such transfer, (i) such transferee agrees in writing with Purchaser to be bound by the restriction on transfer contained in Section 2.9(j) as a Seller, (ii) such transfer complies with all applicable securities Laws, and (iii) such transfer is registered with the Paying Agent as provided in the Paying Agent Agreement.

“Person” shall mean an individual, a limited liability company, a joint venture, a corporation, a company, a partnership, an association, a business trust, a trust, an estate, a Governmental Authority, a division or operating group of any of the foregoing or any other entity or organization.

“Phase I/II Clinical Trial” shall mean a first in human clinical trial that is primarily intended to test the safety of the product for a specific indication in patients with the disease or condition under study, or an analogous study or trial of a medical device intended to evaluate scientifically valid evidence to be submitted in an application to a Regulatory Authority for the applicableproduct.

12

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

“Phase III Clinical Trial” means a clinical trial intended to meet the requirements for approval of an NDA or BLA for the product, or an analogous study intended to establish scientifically valid evidence to be submitted in an application to a Regulatory Authority for the product, including any Phase II Clinical Trial that meets the requirements of the foregoing.

“Post-Closing Adjustment” shall have the meaning set forth in Section 2.4(f).

“President” shall mean the Company’s president (Président).

“Proceeding” shall have the meaning set forth in Section 4.16.

“Pro Rata Share” shall mean, with respect to each Seller, as to (i) any indemnifiable Damages, (ii) the Escrowed Amount, (iii) the Milestone Shares or (iv) otherwise for purposes of this Agreement, the percentage of the Closing Date Cash Consideration (including the Free Share Holdback but excluding the Non GMP Batch Payment) to which such Seller (including any Free Share Holder) is entitled; provided that if the Non GMP Batch Payment is earned by RGX, the Pro Rata Share of each Seller shall be recalculated to reflect the relative increase in the Pro Rata Share of RGX, as illustrated on Schedule 2.7, which will be updated prior to the Closing Date.

“Purchaser” shall have the meaning set forth in the Preamble of this Agreement.

“Purchaser Indemnified Party” and “Purchaser Indemnified Parties” shall have the respective meanings set forth in Section 9.2(a).

“Reference Market Value” means, with respect to any date, the volume-weighted average price of uniQure Ordinary Shares as reported on Nasdaq for the twenty (20) consecutive Trading Day period ending two (2) Business Days prior to such date.

“Regulation S” shall mean Regulation S promulgated under the Securities Act.

“Regulatory Approval” shall mean, with respect to a country or extra-national territory, all approvals, licenses, registrations or authorizations of any Regulatory Authority necessary in order to commercially market, distribute and sell a pharmaceutical product in such country or some or all of such extra-national territory.

“Regulatory Authority” shall mean, with respect to a country or region, any national (e.g., the FDA for the United States), supra-national (e.g., the EMA for the European Union), regional, state or local regulatory agency, department, bureau, commission, council or other Governmental Authority that has jurisdiction with respect to the safety, efficacy, reliability, manufacture, investigation, sale or marketing of pharmaceuticals products or otherwise has jurisdiction with respect to the safety, efficacy, reliability, manufacture, investigation, sale or marketing of any pharmaceutical product.

“Regulatory Authorizations” shall have the meaning set forth in Section 4.23(g).

“Released Closing Date Cash Consideration” shall mean an amount of cash equal to the Estimated Closing Date Cash Consideration less (i) the Holdback Amount, (ii) the Initial Non GMP Batch Payment and (iii) the Free Share Holdback.

“Released Damages” shall have the meaning set forth in Section 7.2.

“Representative” shall mean, with respect to any Person, any officer, director, principal, member, manager, attorney, agent, advisor, employee or other representative of such Person.

“Resolution Period” shall have the meaning set forth in Section 2.4(c).

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[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

“Response Period” shall have the meaning set forth in Section 9.3(b).

“Restricted Period” shall mean the period commencing on the Closing Date and ending on the second (2^nd) anniversary of the Closing Date.

“Review Period” shall have the meaning set forth in Section 2.4(c).

“RGX License Agreement” shall mean the license and collaboration agreement between Corlieve and RGX dated 24 June 2020.

“Safety/Regulatory Reason” means that the Purchaser reasonably determines that the medical risk/benefit of such Corlieve Product is sufficiently unfavorable as to be incompatible with the welfare of patients, poses an unacceptable safety risk or is unlikely to obtain Regulatory Approval, which determination shall be supported by relevant scientific data as well as requests of CHMP or FDA, and a confirmation by Purchaser that it has determined in good faith that such issues are unlikely to be resolved within a reasonable period of time, as shall be disclosed (together with a summary of the data and requests supporting such reason) to the Holder Representative.

“Scientific Founders” shall mean  [*].

“Securities Act” means the Securities Act of 1933, as amended, and the rules and regulations promulgated thereunder.

“Seller” and “Sellers” shall have the respective meanings set forth in the Preamble to this Agreement.

“Seller Indemnified Party” and “Seller Indemnified Parties” shall have the respective meanings set forth in Section 9.2(c).

“Seller Releasing Party” shall have the meaning set forth in Section 7.2

“Series A Shares” shall have the meaning set forth in the Recitals.

“Shareholders Agreement” shall mean that certain Shareholders Agreement, dated as of 24 June 2020, by and among Corlieve and the shareholders of Corlieve, and all related contractual undertakings entered into with Free Share Holders.

“Shareholder Loan” shall have the meaning set forth in the Recitals.

“Shares” shall have the meaning set forth in the Recitals.

“Software” shall have the meaning set forth in the definition of “Intellectual Property.”

“Specified Costs” shall mean costs that Corlieve has incurred or will incur or commit prior to the Closing Date as set forth in Section 1.1 of the Disclosure Letter.

“Specified Representations” shall mean the representations and warranties set forth in Section 4.18 (Intellectual Property).

“Statement of Objections” shall have the meaning set forth in Section 2.4(c).

“Subsidiary” shall mean, with respect to any Person, (a) any corporation more than fifty percent (50%) of whose shares of any class or classes is owned by such Person directly or indirectly through one or more Subsidiaries of such Person and (b) any partnership, association, joint venture or other

14

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

entity in which such Person directly or indirectly through one or more Subsidiaries of such Person has more than a fifty percent (50%) equity interest.

“Subsidies” shall have the meaning set forth in Section 4.24.

“Survival End Date” shall have the meaning set forth in Section 9.1.

“Swiss Subsidiary” shall mean Corlieve Therapeutics AG, a Swiss corporation having its registered office at c/o Switzerland Innovation Park Basel Area AG, Gewerbestrasse 24, 4123 Allschwil, Switzerland, and registered under number CHE-486.007.268.

“Tax” (including with correlative meaning, the terms “Taxes” and “Taxable”) shall mean (a) any and all, direct or indirect, taxes and duties and similar governmental charges, levies, imposts or withholdings (including net income, gross income, gross receipts, gains, net wealth, net worth, equity, asset value, value added tax, turnover, sales, use, ad valorem, transfer, franchise, profits, license, lease, service, service use, withholding, payroll, social security contributions, employment, excise, severance, stamp, occupation, premium, property, windfall profits, escheat, customs, duties or other taxes) in the nature of a tax whenever and by whatever Governmental Authority imposed, and whether of the French or a foreign, state or local jurisdiction, together with in any such case any interest, fines, penalties, surcharges and charges incidental or relating to the imposing of any of such Taxes and any additions to tax or additional amounts with respect thereto, (b) any Liability for the payment of any items described in clause (a) above as a result of being (or ceasing to be) a member of an affiliated, consolidated, combined, unitary or aggregate group (or being included (or being required to be included)) in any Tax Return related to such group, and (c) any Liability for the payment of any amounts as a result of any express or implied obligation to indemnify any other Person, or any successor or transferee liability, by Contract or otherwise in respect of any items described in clause (a) or (b) above.

“Tax Credit” shall mean any amount which can be subtracted from the payment of Taxes to any Taxing Authority or that can be reimbursed by any Taxing Authority.

“Tax Law” shall mean all currently Applicable Law and guidelines released by any Taxing Authority relating to or regulating the assessment, determination, collection or imposition of Taxes.

“Tax Period” shall mean any period prescribed by any Taxing Authority for which a Tax Return is required to be filed or a Tax is required to be paid.

“Tax Return” individually, or “Tax Returns,” collectively, shall mean any return, declaration, report, statement, information statement and other document (including any amendment thereof or attachment thereto) required to be filed with a Governmental Authority with respect to Taxes.

“Taxing Authority” shall mean any Governmental Authority having jurisdiction over the assessment, determination, collection, or imposition of any Taxes (domestic or foreign).

“Third Party” shall mean any Person other than the Purchaser Indemnified Parties and the Seller Indemnified Parties.

“Third Party Claim” shall have the meaning set forth in Section 9.4(a).

“Threshold” shall have the meaning set forth in Section 9.5(b).

“Total Consideration” shall mean the Closing Date Consideration and the Milestone Payments.

“Trademarks” shall have the meaning set forth in the definition of “Intellectual Property.”

“Trading Day” shall mean a day on which Nasdaq is open for trading.

15

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

“Transaction Agreements” shall mean this Agreement, the Escrow Agreement and all other agreements, instruments and certificates to be executed by any party hereto at or prior to the Closing pursuant to this Agreement.

“Transaction Bonus” shall mean the bonuses that will be paid by Corlieve to its employees or consultants in connection with the transactions contemplated in this Agreement, up to a maximum aggregate amount of  [*].

“Transaction Expenses” shall mean all fees, costs (including Taxes but excluding Tax Credits), payments and expenses of Corlieve (and of the Sellers to the extent such fees, costs, payments and expenses are to be borne by Corlieve and are not paid before the Closing Date) payable to third parties, including legal counsel, that were incurred in connection with (i) the negotiation, preparation (including due diligence), drafting, review, execution, delivery or performance of this Agreement or any other document delivered or to be delivered in connection with the transactions contemplated hereby, (ii) except as otherwise set forth in this Agreement, the preparation and submission of any filing or notice required to be made or given prior to the Closing in connection with any of the transactions contemplated hereby, or (iii) the obtaining of any consent, waiver or approval required to be obtained in connection with any of the transactions contemplated hereby, including legal and accounting fees, investment banking fees, and related disbursements in connection with any of the foregoing.

“Transaction Expenses of the Sellers” shall mean all fees, costs, payments and expenses of the Holder Representative and the Paying Agent.

“Transfer Taxes” shall mean all direct and indirect stock transfer Taxes, stock registration, documentary or recording Taxes, stamp Taxes and similar Taxes (including any penalties and interest) incurred, imposed, assessed or payable pursuant to Section 7.1 in connection with the transfer of Shares pursuant to this Agreement.

“uniQure Ordinary Shares” shall mean the Ordinary Shares in the capital of uniQure N.V., nominal value €0.05 per share.

“U.S. GAAP” shall mean generally accepted accounting principles in the United States and consistently applied over all relevant periods.

“U.S. Person” shall have the meaning given to such term by Regulation S.

“Valid Claim” shall mean with respect to a particular country, a claim of any existing or future issued and unexpired Patent that has not (A) lapsed, been cancelled or abandoned or dedicated to the public (subject, in case of abandonment or dedication to the public of a Patent, to Purchaser providing notice to the Holder Representative reasonably promptly before such abandonment or dedication to the public) or (B) revoked, held unenforceable, invalid or unpatentable by a decision of a court or other government body (which decision is unappealed or unappealable within the time allowed for appeal), including through opposition, reexamination or reissue, and which has not been rendered unenforceable through disclaimer or otherwise; provided, that, on a country-by-country basis, a Patent application pending for more than five (5) years from the earliest priority date of such application shall not be considered to have any Valid Claim for purposes of this Agreement from and after such five (5) year date unless and until a Patent with respect to such application issues.

“Valid RGX AAV9 Patent” shall mean a validly issued, unexpired Patent owned or Controlled by RGX  that is included in Exhibit A of the RGX License Agreement and that Covers the composition of matter of the AAV9.

“Worker” or “Workers” shall mean any individual performing services in the capacity of an employee, legal representative, officer, director, and/or is an independent contractor.

16

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

“Worker Agreements” shall have the meaning set forth in Section 4.12(d).

1.2Other Definitional and Interpretive Matters. In this Agreement, unless the context otherwise requires:

(a)when calculating the period of time before which, within which or following which any act is to be done or step taken pursuant to this Agreement, the date that is the reference date in calculating such period shall be excluded. If the last day of such period is a non-Business Day, the period in question shall end on the next succeeding Business Day;

(b)the “Euro equivalent of” means on any day, for purposes of determining the Euro equivalent of on an amount on any non-Euro currency, the Euro amount obtained by the application to such non-Euro amount of the rate at which such currency may be exchanged into Euros at the time of determination on such day as quoted by Bloomberg on www.bloomberg.com/markets/currencies/fxc .html, or as displayed on such other information service which publishes that rate of exchange from time to time in place of Bloomberg. In the event that such rate is not displayed by Bloomberg on the webpage specified in the immediately preceding sentence, the exchange rate shall be determined by reference to such other publicly available service for displaying exchange rates as may be agreed upon by the Parties;

(c)“or” has the inclusive meaning represented by the phrase “and/or”;

(d)words expressed in the singular number shall include the plural and vice versa, and words expressed in the masculine shall include the feminine and neuter genders and vice versa;

(e)references to Articles, Sections, Exhibits, Schedules and Recitals are references to articles, sections, exhibits, schedules and recitals of this Agreement, unless another agreement is specified;

(f)references to “day” or “days” are to calendar days;

(g)references to this “Agreement” or any other agreement or document shall be construed as references to this Agreement or, as the case may be, such other agreement or document as the same may have been, or may from time to time be, amended, supplemented or otherwise modified;

(h)each reference to a Law, statute, regulation or other government rule is to it as amended from time to time and, as applicable, is to corresponding provisions of successor Laws, statutes, regulations or other government rules;

(i)“include,” “includes,” and “including” are deemed to be followed by “without limitation” whether or not they are in fact followed by such words or words of similar import;

(j)the words “hereof,” “herein,” “hereto,” “hereby,” “hereunder” and derivative or similar words refer to this Agreement as an entirety and not solely to any particular provision of this Agreement; and

(k)terms expressed in the French language or to which a French translation has been added shall be interpreted for the purposes of this Agreement under the meaning assigned to them by the French term and/or translation under French law.

17

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

ARTICLE 2

SALE OF THE SHARES

2.1Sale of the Shares. Upon the terms and subject to the conditions of this Agreement and on the basis of the representations, warranties, covenants and agreements herein contained, at the Closing, (a) Purchaser shall purchase, acquire and accept from each Seller, and (b) each Seller shall sell, transfer, assign, convey and deliver to Purchaser, all of the right, title and interest in and to their respective Shares, free and clear of all Encumbrances.

2.2Closing. Subject to the terms and conditions of this Agreement, including, without limitation, the satisfaction or waiver (where applicable) of the conditions set forth in Article 8, and unless otherwise terminated pursuant to Section 10.1, the sale and purchase of the Shares contemplated by this Agreement shall take place at a closing (the “Closing”) to take place at the offices of Morgan, Lewis & Bockius UK LLP, 68 Rue du Faubourg Saint-Honoré, 75008 Paris, France, no later than five (5) Business Days following the date on which all of the conditions set forth in Article 8 have been satisfied or waived (other than those that by their terms are to be satisfied or waived at the Closing), or at such other place or on such other date as Purchaser and the Holder Representative may mutually agree to in writing.

2.3Payment of Closing Date Consideration.

(a)Closing Date Consideration. The Closing Date Consideration payable on the Closing Date by Purchaser in consideration for all of the outstanding Shares shall be paid as follows:

(i)the Released Closing Date Cash Consideration, payable to a paying agent appointed by the Holder Representative (the “Paying Agent”); and

(ii)the issuance and deposit of the Escrowed Amount into the Escrow Account at the Closing in accordance with the terms of the Escrow Agreement.

(b)Payment of Closing Date Cash Consideration. On the Closing Date, the Holder Representative shall direct the Paying Agent to make a cash payment to (i) each Seller in accordance with the Closing Date Allocation Schedule by wire transfer in immediately available funds, in an amount equal to the portion of the Released Closing Date Cash Consideration that such Seller is entitled to hereunder in respect of the Shares held by such Seller immediately prior to Closing (after deduction of the Transaction Expenses of the Sellers), and thereafter such Seller shall be entitled to receive any other portion of the Closing Date Consideration (after deduction of the Transaction Expenses of the Sellers) in respect of the Shares held by such Seller immediately prior to Closing to which it is entitled pursuant to the Transaction Agreements and (ii) the relevant payee with respect to the Transaction Expenses of the Sellers which the Sellers expressly acknowledge and agree; provided, that Purchaser shall not be responsible for the payment to any Seller of any further amounts with respect to the Closing Date Consideration, other than the Post-Closing Adjustment, if applicable.

2.4Closing Date Cash Consideration Adjustment.

(a)At least [*] before the Closing, (A) the Sellers, within the limit of their respective powers within the Company as officer and/or shareholder, shall cause Corlieve to prepare and the Holder Representative shall deliver to Purchaser a complete and accurate Closing Date Allocation Schedule and (B) a statement (the “Estimated Closing Statement”) setting forth its good faith estimate calculated in accordance with French GAAP of the Closing Date Consideration (the “Estimated Closing Date Consideration”), Closing Date Cash Balance (the “Estimated Closing Date Cash Balance”), Net Working Capital (the “Estimated Net Working Capital”), Closing Date Indebtedness (the “Estimated Closing Date Indebtedness”) and Transaction Expenses (the “Estimated Transaction Expenses”)

18

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

(without giving effect to the transactions contemplated herein), and a certificate of Purchaser and the Holder Representative that the Estimated Closing Statement was prepared in accordance with French GAAP consistently applied using the same accounting methods, practices, principles, policies and procedures, with consistent classifications, judgments and valuation and estimation methodologies that were used in the preparation of the financial statements of Corlieve for the most recent fiscal year end as if such Estimated Closing Statement was being prepared and audited as of a fiscal year end.

(b)Within [*] after the Closing Date, Purchaser shall prepare and deliver to the Holder Representative a statement (the “Closing Statement”) setting forth its calculation of the Closing Date Cash Consideration, Closing Date Cash Balance, Net Working Capital, Closing Date Indebtedness and Transaction Expenses (without giving effect to the transactions contemplated herein) and a certificate of Corlieve and Purchaser that the Closing Statement was prepared in accordance with French GAAP applied using the same accounting methods, practices, principles, policies and procedures, with consistent classifications, judgments and valuation and estimation methodologies that were used in the preparation of the Estimated Closing Statement.

(c)After receipt of the Closing Statement, the Sellers shall have [*] (the “Review Period”) to review the Closing Statement. During the Review Period, the Holder Representative’s accountants shall have reasonable access (during regular business hours, upon reasonable advance notice, under reasonable circumstances, subject to restrictions under applicable Law and without undue disruption to the normal business activities of Corlieve) to the books and records of Corlieve and work papers prepared by Corlieve and/or Corlieve’s accountants to the extent that they relate to the Closing Statement and to such historical financial information (to the extent in Corlieve’s possession) relating to the Closing Statement as the Holder Representative may reasonably request for the purpose of reviewing the Closing Statement and to prepare a Statement of Objections, if any. On or prior to the last day of the Review Period, the Holder Representative may object to the Closing Statement by delivering to Purchaser a written statement setting forth the Holder Representative’s objections in reasonable detail, indicating each disputed item or amount and the basis for the Holder Representative’s disagreement therewith (the “Statement of Objections”). If the Holder Representative fails to deliver the Statement of Objections before the expiration of the Review Period, the Closing Statement and the Post-Closing Adjustment, as the case may be, reflected in the Closing Statement shall be deemed to have been accepted by the Holder Representative. If the Holder Representative delivers the Statement of Objections before the expiration of the Review Period, Purchaser and the Holder Representative shall negotiate in good faith to resolve such objections within [*] after the delivery of the Statement of Objections (the “Resolution Period”), and, if the same are so resolved within the Resolution Period, the Post-Closing Adjustment and the Closing Statement with such changes as may have been agreed in writing by Purchaser and the Holder Representative shall be final and binding.

(d)If the Holder Representative and Purchaser fail to reach an agreement with respect to all of the matters set forth in the Statement of Objections before expiration of the Resolution Period, then any amounts remaining in dispute (“Disputed Amounts”) shall be submitted for resolution to EY or, if EY is unable to serve, Purchaser and the Holder Representative shall appoint by mutual agreement an impartial public accounting firm of nationally recognized standing in France other than the Holder Representative’s or any Seller’s accountants or Purchaser’s accountants (the “Independent Accountants”) who, acting as experts and not arbitrators, shall resolve the Disputed Amounts only and make any adjustments to the Post-Closing Adjustment and the Closing Statement, as the case may be. All adjustments shall be made without regard to materiality. The Independent Accountants shall only decide the specific items under dispute by the parties and their decision for each Disputed Amount must be within the range of values assigned to each such item in the Closing Statement and the Statement of Objections, respectively.

(e)The Holder Representative shall pay (which payment shall be from the Holder Representative’s Fund if there is one) a portion of the fees and expenses of the Independent Accountants equal to [*] multiplied by a fraction, the numerator of which is the amount of Disputed Amounts

19

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

submitted to the Independent Accountants that are resolved in favor of Purchaser (that being the difference between the Independent Accountants’ determination and the Holder Representative’s determination) and the denominator of which is the total amount of Disputed Amounts submitted to the Independent Accountants (that being the sum total by which Purchaser’s determination and the Holder Representative’s determination differ from the determination of the Independent Accountants). Purchaser shall pay that portion of the fees and expenses of the Independent Accountants that the Holder Representative is not required to pay hereunder. The Independent Accountants shall make a determination as soon as practicable within [*] (or such other time as the Parties shall agree in writing) after their engagement, and their resolution of the Disputed Amounts and their adjustments to the Closing Statement and/or the Post-Closing Adjustment shall be conclusive and binding upon the Parties.

(f)The post-closing adjustment to the Closing Date Cash Consideration shall be an amount equal to the Estimated Closing Date Cash Consideration as set forth in the Estimated Closing Statement minus the Closing Date Cash Consideration as set forth on the Closing Statement as finally determined under subsection (c) or (d) above (the “Post-Closing Adjustment”). If the Post-Closing Adjustment is a negative number, Purchaser shall pay to the Paying Agent (i) the Holdback Amount and (ii) an amount in cash equal to the absolute value of the Post-Closing Adjustment (and the Paying Agent shall pay such amounts to the Sellers based upon their Pro Rata Share). If the Post-Closing Adjustment is a positive number, Purchaser shall retain that portion of the Holdback Amount equal to the absolute value of the Post-Closing Adjustment and, in the event the Post-Closing Adjustment due by the Sellers to Purchaser exceeds the Holdback Amount, such amount by which the Post-Closing Adjustment due by the Sellers to Purchaser exceeds the Holdback Amount shall be paid in cash by the Sellers (based upon their Pro Rata Share) to Purchaser; provided, that Purchaser shall have the right to set-off any such amount due by the Sellers to Purchaser in excess of the Holdback Amount from any Milestone Payment that may become due by Purchaser under this Agreement and reduce the amount of any such Milestone Payment due to the Sellers accordingly. If the Post-Closing Adjustment is a positive number that is less than the Holdback Amount, Purchaser shall pay to the Paying Agent an amount in cash equal to the difference between the Holdback Amount and the Post-Closing Adjustment (and the Paying Agent shall pay such amounts to the Sellers based upon their Pro Rata Share). Any payments made pursuant to this Section 2.4 shall be treated as an adjustment to the Closing Date Cash Consideration by the Parties for Tax purposes, unless otherwise required by Applicable Law.

2.5Payment of Closing Date Transaction Expenses. On the Closing Date, upon the terms and conditions of this Agreement, Purchaser shall pay, in cash by wire transfer of immediately available funds, the Estimated Transaction Expenses, in the amount and to the extent set forth in the Estimated Closing Statement delivered hereunder, and pursuant to the wiring instructions provided by the Holder Representative at least [*] prior to the Closing.

2.6Treatment of Free Shares. With respect to any given Free Share, within five (5) Business Days following the applicable Free Share Transfer Date, each Free Share Holder shall deliver a share transfer certificate (ordre de mouvement) duly signed by the Free Share Holder, and within [*] of receipt thereof, Purchaser shall pay, in cash by wire transfer of immediately available funds, the portion of the Free Share Holdback corresponding to such Free Share and pursuant to the wiring instructions provided by the applicable Free Share Holder, provided that, if any such payment is to be made prior to the Escrow Release Date,  a portion thereof equal to [*] of the applicable Free Share Holdback shall be paid to the Escrow Account.  Each Free Share Holder shall hold all Free Shares in trust for Purchaser until such transfer is consummated, and hereby agrees, prior to such transfer, to vote all such Free Shares in accordance with the recommendations of the Purchaser. In no event shall any Free Share Holder transfer the beneficial or legal interest in any Free Share to any person other than the Purchaser.

2.7Non GMP Batch Payment. Within [*] following the Non-GMP Batch Delivery, if any, Purchaser shall pay the Non-GMP Batch Payment to RGX, provided that, if any such payment is to be made prior to the Escrow Release Date,  a portion thereof equal to [*] of the Non-GMP Batch Payment

20

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

shall be paid to the Escrow Account; and the relative Pro Rata Share of each Seller shall be automatically amended as set forth on Schedule 2.7.

2.8Withholding Tax. Subject to prior consultation with the Holder Representative, Purchaser shall be entitled to deduct and withhold, and to cause the Escrow Agent to deduct and withhold, from any amounts payable in respect of the Shares, such amount, if any, as Purchaser or the Escrow Agent may be required to deduct and withhold with respect to the making of such payment under domestic or foreign Tax Law, and shall pay over (or cause to be paid over) such amounts to the proper Taxing Authority. To the extent that amounts are so deducted and withheld in accordance with the preceding sentence and paid to the proper Taxing Authority, such amounts shall be treated for all purposes of this Agreement as having been paid to, or for the benefit of, the former holder of the Shares in respect of which such deduction or withholding was made.  Based on its review of current Applicable Laws, the Purchaser has not identified any withholding taxes applicable to the Closing Date Consideration or the Total Consideration.

2.9Milestone Payments.

(a)As additional consideration for the Shares and upon the first achievement by Purchaser, any of its Affiliates or any of the respective licensees, sublicensees, assignees or transferees of Purchaser or any of its Affiliates (or any Affiliates of such licensees, sublicensees, assignees or transferees) of any of the events set forth in the table below under “Milestone Trigger Event”, Purchaser shall promptly (and in any event no later than [*] thereafter) notify the Holder Representative of such achievement and, within [*] of such notice, pay or deposit with the Paying Agent, subject to Section 2.10, either in cash or a combination of cash and issuance of uniQure Ordinary Shares (such uniQure Ordinary Shares being referred to as the “Milestone Shares”), the amount set forth in the table below under “Milestone Payment” opposite such “Milestone Trigger Event” (each, a “Milestone Payment”), and net of any required withholding of Taxes under Applicable Law, which shall be promptly paid over to the applicable Taxing Authority by Purchaser.  Promptly following the payment or deposit of any Milestone Payment, the Paying Agent, as instructed by Holders Representative, shall, subject to ‎Section 2.9(k), pay to each Seller, by wire transfer of immediately available funds, such Seller’s Pro Rata Share of such Milestone Payment.  It is expressly understood and agreed that Purchaser, the Company and their respective Affiliates shall have no Liability to any Seller with respect to any portion of any Milestone Payment that has been paid or deposited by or on behalf of Purchaser to the Paying Agent. For clarity, the Milestones shall by payable only once for the first Corlieve Product to achieve such Milestone Trigger Event.

[*]

(b)For the avoidance of doubt, (i) the maximum aggregate amount Purchaser and any of its Affiliates shall be obligated to pay pursuant to Section 2.9(a) with respect to Milestones shall be [*] and (ii) no more than one Milestone Payment shall be payable with respect to any Milestone (other than, for the avoidance of doubt, any additional payments in respect of Milestone #4b and Milestone #5b to the extent they become payable as set forth in Section 2.9(c) and Section 2.9(c) below).

(c)In the event that Milestone #4b is triggered pursuant to Section 2.9(a) with respect to any Corlieve Product, then upon expiry of the Valid RGX AAV9 Patent Covering such Corlieve Product in the United States, the Sellers shall be entitled to receive a payment in an amount equal to [*].

(d)In the event that Milestone #5b is triggered pursuant to Section 2.9(a), then upon expiry of the Valid RGX AAV9 Patent Covering such Corlieve Product in all Major EU Countries,  the Sellers shall be entitled to receive a payment in an amount equal to [*].

(e)Valuation of the Milestone Shares. If any Milestone Payment becomes due, if Purchaser elects to make any portion of a Milestone Payment by issuing uniQure Ordinary Shares, each

21

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

Milestone Share issued by Purchaser will be deemed to have a value equal to the Euro equivalent of the Reference Market Value with respect to the date on which the relevant Milestone payment is made.

(f)Milestone Shares Cap. Notwithstanding any other provision in this Agreement, the total number of Milestone Shares that may be issued to each Seller in respect of each of the Milestones shall be limited to a number of Milestone Shares having an aggregate value equal to [*] of such Seller’s Pro Rata Share of such aggregate Milestone Payment (the “Milestone Shares Cap”).

(g)Milestone Payments Not Certain. Each of Purchaser, Corlieve and each of the Sellers hereby acknowledges that the achievement of the Milestones is uncertain, and it is therefore not assured that Purchaser’s obligation to pay any Milestone Payments will ever arise, despite Purchaser’s use of the efforts required pursuant to Section 2.9(h).

(h)Diligence.

(i)Following the Closing, Purchaser and its Affiliates shall have the right, in their sole and absolute discretion, to direct and control the research, development and commercialization of the Corlieve Product in all respects, including the determination to test, develop, pursue, market, make any regulatory filings with respect to, or make any strategic product portfolio decisions affecting, any such Corlieve Product; provided that Purchaser and its Affiliates shall use Commercially Reasonable Efforts with respect to the Corlieve Product to develop and seek Regulatory Approval in the United States and at least one Major EU Country for the Corlieve Product.

(ii)If the Purchaser, or its Affiliates, or any of the respective licensees, sublicensees, assignees or transferees of Purchaser or any of its Affiliates, as the case may be, makes a definitive determination to cease research, development or commercial activities for the Corlieve Product due to a Safety/Regulatory Reason, then (x) Purchaser or its Affiliates shall promptly notify the Holders Representative of such determination together with all supporting data and regulatory information in writing (a “Development Notice”) and (y) no additional Milestone Payments or Downstream Payments shall be payable with respect to a Corlieve Product or Alternative Product pursuant to this Agreement.

(iii)Provided that no such Development Notice has been delivered pursuant to Section 2.9(h)(ii) herein, and provided further that any Milestone Payments payable pursuant to this Section 2.9(h)(iii) shall be payable once for the first Corlieve Product or Alternative Product (as the case may be) to reach the applicable Milestone, then:

a)To the extent that Purchaser, or its Affiliates, or any of the respective licensees, sublicensees, assignees or transferees of Purchaser or any of its Affiliates, makes the decision to Deprioritize or cease to develop the Corlieve Product within two (2) years of the Closing Date, then the Milestone Payments in Section 2.9(a) will be payable upon achievement of the corresponding Milestone by the Corlieve Product or the Alternative Product whichever comes first, provided that with respect to the Alternative Product, no Milestone Payments in Section 2.9(a) shall be payable by the Purchaser or its Affiliates after ten (10) years following the Deprioritization; or

b)To the extent that Purchaser or its Affiliates or any of the respective licensees, sublicensees, assignees or transferees of Purchaser or any of its Affiliates, makes a Deprioritization decision more than two (2) years after the Closing Date, then the Milestone Payments in Section 2.9(a) will be payable upon achievement of the corresponding Milestone by the Corlieve Product or the Alternative Product whichever comes first, provided that with respect to such Alternative Product, no Milestone Payments in Section 2.9(a) shall be payable by the Purchaser or its Affiliates after [*] following the Deprioritization;

22

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

(iv)For purposes of this Section 2.9(h), Purchaser shall notify the Holder Representative of its rationale for any Safety/Regulatory Reason determination or Deprioritization decision, with supporting information, and discuss it in good faith with the Holder Representative.

(v)Further, no later than December 15^th of each year until the fifth (5^th)  anniversary of the Closing Date, Purchaser shall provide an annual report on the development of the Corlieve Product and Alternative Products by Purchaser, its Affiliates and any of the respective licensees, sublicensees, assignees or transferees of Purchaser or any of its Affiliates, over the preceding twelve months. Purchaser shall respond to all reasonable questions that the Holder Representative may have on such report and related activities.

(i)Nontransferability of Rights to Milestone Payments. The right of the Sellers to receive the Milestone Payments pursuant to this Section 2.9. may not be sold, assigned, transferred, pledged, encumbered or in any other manner transferred or disposed of, in whole or in part, other than through a Permitted Transfer. For the avoidance of doubt, if Purchaser elects to make any portion of a Milestone Payment by issuing Milestone Shares, such Milestone Shares will not be subject to the restriction provided in the previous sentence, but will be subject to any applicable restrictions on transfer provided in any applicable securities Laws.

(j)Milestone Payments an Integral Part of Consideration. The Parties acknowledge that the right of the Sellers to receive the Milestone Payments represents an integral part of the consideration the Sellers are receiving in exchange for their Shares and is a significant factor in the willingness of the Sellers to enter into this Agreement.

(k)Tax Treatment. Any payments made pursuant to this Section 2.9 shall be treated as an adjustment to the Total Consideration for all Tax purposes, unless otherwise required by Applicable Law.

2.10Set-Off. Notwithstanding any provision of this Agreement to the contrary, the parties hereby acknowledge and agree that, in addition to any other right hereunder, Purchaser shall have the right, but not the obligation, to set off against any Milestone Payment (or Downstream Payment that is owed and has not yet been paid the following amounts, to the extent not disputed in good faith by the Holder Representative [*] (each, a “Milestone Set-Off”).  To the extent that any amounts of any Milestone Payment are set-off in accordance with this Section 2.10, such amounts shall be treated for all purposes of this Agreement as having been paid to the Sellers. For the avoidance of doubt, once actually paid to the Sellers, the Milestone Payments shall not be recoverable by Purchaser pursuant to the set-off rights contained in this Section 2.10.

2.11Form of Consideration Payable by Purchaser.

(a)uniQure Ordinary Shares Issued as part of a Milestone Payment. Subject to the provisions of Section 2.9 and Section 2.11(c), upon its election, Purchaser may satisfy part of its obligation to make any portion of a Milestone Payment by the issuance to each Seller of such Seller’s Pro Rata Share of the Milestone Shares being issued by Purchaser, with any fraction of a uniQure Ordinary Share being treated as provided in Section 2.11(d) below, subject to the Milestone Shares Cap.

(b)Covenants. Subject to Section 2.12, prior to the issuance of any uniQure Ordinary Shares pursuant to this Agreement, Purchaser shall cause the following to occur:

(i)any and all uniQure Ordinary Shares issued pursuant to this Agreement shall be saleable, subject to any restrictions that would be imposed under applicable Law as a result of a Seller’s individual circumstance (i.e., the restrictions under Regulation S and any volume limitations imposed pursuant to Rules 144 or 145 under the Securities Act), without requiring further registration

23

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

under the Securities Act, but subject to any holding period or similar restrictions arising under Rule 144;

(ii)any and all uniQure Ordinary Shares issued pursuant to this Agreement shall be listed on each securities exchange on which similar securities, including as to class and series, issued by Purchaser are then listed and, if not so listed, such shares shall be listed on Nasdaq, subject only to notice of issuance;

(iii)any and all such uniQure Ordinary Shares issued pursuant to this Agreement shall be duly authorized and reserved for issuance, and upon issuance thereof in accordance with this Agreement, be validly issued, fully paid and nonassessable; and

(iv)any and all Milestone Shares shall be subject to the right contained in Section 7.4 to have Purchaser register such Milestone Shares for resale.

(c)No Fractional Shares. Notwithstanding anything to the contrary herein, no fractional uniQure Ordinary Shares shall be issued pursuant to this Agreement, but an amount in cash equal to the aggregate Reference Market Value of all such fractional shares shall instead be deposited with the Holder Representative by Purchaser and shall be an addition to the Holder Representative’s Fund if there is one.

(d)Status of Seller. In no event shall Purchaser issue Milestone Shares to any Seller who is a U.S. Person unless such Seller is (i) an “accredited investor” as defined in Regulation D promulgated under the Securities Act or (ii) a “qualified institutional buyer” as defined in Rule 144A promulgated under the Securities Act ((i) and (ii), an “Accredited Investor”).

2.12Escrow.

(a)Escrowed Amount. At the Closing, Purchaser shall deposit the Escrowed Amount into an escrow account (the “Escrow Account”) to be held by the Escrow Agent pursuant to the terms of the Escrow Agreement. The Escrowed Amount shall serve as security for the indemnity obligations under Sections 9.2(a) and 9.2(b).

(b)Release of Escrow. The Escrow Agreement shall provide that on the date that is twelve (12) months after the Closing Date (the “Escrow Release Date”), the Escrow Agent shall release the entire Escrowed Amount then remaining in the Escrow Account in accordance with the Closing Date Allocation Schedule, provided, that if prior to the Escrow Release Date, a Purchaser Indemnified Party has delivered a Claim Notice pertaining to a matter described in Sections 9.2(a) or 9.2(b) and, despite the Purchaser’s and the Holder Representative’s good faith discussion,  such Claim Notice remains unresolved as of the Escrow Release Date, then Purchaser shall be entitled to instruct the Escrow Agent to retain such portion of the remaining Escrowed Amount as, in Purchaser’s reasonable judgment, may be necessary to satisfy any unresolved or unsatisfied claims for indemnifiable Damages specified in such Claim Notice.

(c)Escrow Agent Fees and Expenses. Any fees and expenses of the Escrow Agent shall be paid one half by Purchaser and one half by the Holder Representative (such payment to be made from the Holder Representative’s Fund if there is one). The Sellers shall be entitled to receive, upon the release of the Escrowed Amount, any and all interest on such Escrowed Amount (provided, however, that if the Escrow Agent charges “negative” interest on the Escrowed Amount, such expense shall be shared one half by Purchaser and one half by the Sellers) or dividends paid by Purchaser on uniQure Ordinary Shares during the period in which the Escrowed Amount are retained in the Escrow Account.

2.13Closing Deliverables. At the Closing:

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[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

(a)Deliveries to Purchaser. Purchaser shall have received the following:

(i)a certified copy of the power and authority of the individual(s) acting on behalf of any of the Sellers, or any of the Free Shares Holders, of the Holder Representative for the purposes of the Closing;

(ii)the share transfer forms (ordres de mouvement) related to the Shares, duly completed and signed by each Seller in favor of Purchaser;

(iii)three (3) originals of the French Short Form Transfer Agreement, duly executed by the Sellers in the form of the draft attached hereto as Exhibit D;

(iv)the up-to-date originals of (i) the share transfer register (registre des mouvements de titres) of Corlieve, (ii) the shareholders’ accounts (comptes d’actionnaires) of Corlieve, (iii) the registries of the minutes of the meetings of the Comité de Supervision (supervisory board) and of the decisions of the President, (iv) the registry(ies) of the minutes of the general shareholders’ meetings of Corlieve (registre(s) des procès-verbaux des Assemblées Générales) and the attendance sheets to such shareholders’ meetings (feuilles de présence aux Assemblées Générales);

(v)a copy of the Escrow Agreement, signed by the Holder Representative and the Escrow Agent;

(vi)the resignations, effective as of the Closing, of all of the members of the management (including the President of Corlieve) and the Comité de Supervision (supervisory board) of Corlieve;

(vii)a certificate duly executed by the Holder Representative, dated the Closing Date, certifying that the conditions with respect to Purchaser’s obligations under this Agreement set forth in Section 8.2(a) and Section 8.2(b) have been satisfied;

(viii)an instrument evidencing the termination of the Shareholders Agreement, signed by each party thereto;

(ix)a copy of the Paying Agent Agreement, signed by the Holder Representative and the Paying Agent;

(x)a copy of the Licensed Know-How Schedule, dated as of the Closing Date;

(xi)Execution and delivery of such other documents and agreements as have been mutually agreed by the Parties as of the date hereof.

(xii)Execution and delivery of new employment agreements (including in respect of the amended vesting terms of outstanding unvested Free Share awards, with a vesting at six (6) months following the Closing Date) between the Purchaser or its Affiliates and each of [*] on the terms mutually agreed as of the date hereof ; and

(xiii)such other certificates or other documents reasonably requested and necessary to effectuate the transactions contemplated hereby.

(b)Deliveries to the Holder Representative. The Holder Representative shall have received the following:

(i)a certified copy of the power and authority of the individual(s) acting on behalf of Purchaser for the purposes of the Closing;

25

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

(ii)evidence of the deposit of the Closing Date Cash Consideration by wire transfer in immediately available funds to the Paying Agent;

(iii)evidence of the deposit of the Escrowed Amount in the Escrow Account;

(iv)a certificate duly executed by Purchaser, dated the Closing Date, certifying that the conditions with respect to the Sellers’ obligations under this Agreement set forth in Section 8.3(a) and Section 8.3(b) have been satisfied

(v)a copy of the Escrow Agreement, signed by Purchaser;

(vi)three (3) originals of the French Short Form Transfer Agreement, duly executed by Purchaser in the form of the draft attached hereto as Exhibit D;

(vii)evidence of the obtaining of the Foreign Investment Approval;

(viii)a copy of the Paying Agent Agreement, signed by Purchaser; and

(ix)such other certificates or other documents reasonably requested and necessary to effectuate the transactions contemplated hereby.

2.14Payments to Paying Agent. The Sellers and the Holder Representative agree that any payment made by or on behalf of Purchaser (including via release of the Holdback Amount, the Non GMP Batch Payment, the Free Share Holdback, release from the Escrow Account or with respect to payment of Milestone Payments) to the Paying Agent in accordance with this Agreement for distribution to a given Seller shall be deemed to be a payment made by Purchaser directly to such Seller and shall be deemed to satisfy the corresponding obligation of Purchaser to such Seller to the extent of such amount paid to the Paying Agent. Notwithstanding anything to the contrary contained herein, any amounts paid to the Paying Agent for distribution to the Sellers shall be deemed to be paid to the Sellers, and upon such payment, neither Purchaser, Corlieve nor any of their respective Affiliates shall have any further Liability for any payment owed to any Seller if so paid to the Paying Agent.

2.15Further Assurances. If at any time after the Closing Date, Purchaser shall determine, in its reasonable discretion, that any deeds, bills of sale, instruments of conveyance, assignments, assurances or any other actions or things are necessary or desirable to vest, perfect or confirm of record or otherwise in Purchaser its right, title or interest in, the Shares and to or under any of the rights, properties or assets of Corlieve indirectly acquired by Purchaser as a result of, or in connection with, the transaction contemplated by this Agreement or otherwise to carry out this Agreement, then the officers and directors of Purchaser shall be authorized to execute and deliver, in the name and on behalf of Corlieve, all such deeds, bills of sale, instruments of conveyance, assignments and assurances and to take and do, in the name and on behalf of Corlieve, all such other actions and things as may be necessary or desirable to vest, perfect or confirm any and all right, title or interest in, to and under such rights, properties or assets in Purchaser or otherwise to carry out this Agreement, so long as such action is consistent with the terms of this Agreement.

ARTICLE 3

REPRESENTATIONS AND WARRANTIES REGARDING THE SELLERS

Each Seller, severally and not jointly (sans solidarité entre eux), hereby represents and warrants to Purchaser that the statements contained in this Article 3 are true and correct as of the Agreement Date and as of the Closing Date:

3.1Power and Authorization. Such Seller has full capacity, legal right, power, and authority (including pursuant to his/her respective matrimonial regime) to enter into and perform its

26

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

obligations and consummate the transactions contemplated under this Agreement. Such Seller that is an entity is validly existing and is in good standing under the Applicable Laws of the jurisdiction of its organization and has all requisite power and authority to own, lease and operate properties and carry on its business as now conducted. The execution, delivery, and performance by such Seller of this Agreement and the other Transaction Agreements to which such Seller will be a party, and the consummation by such Seller of the transactions contemplated hereby and thereby, have been duly authorized with respect to such Seller by all necessary action. This Agreement has been, and each other Transaction Agreement to which such Seller will be a party will be, duly and validly executed and delivered by such Seller and constitutes, or will constitute, the legal, valid, and binding obligation of such Seller, enforceable against such Seller in accordance with its terms, except as enforcement may be limited by equitable principles limiting the right to obtain specific performance or other equitable remedies, or by applicable bankruptcy or insolvency laws and related decisions affecting creditors’ rights generally.

3.2No Conflicts. The execution, delivery, and performance of this Agreement and the other Transaction Agreements to which such Seller will be a party, and the consummation of the transactions contemplated hereby and thereby, do not and will not (with or without the passage of time or the giving of notice):

(a)violate or conflict with any provision of the organizational documents of such Seller;

(b)violate any Applicable Law or Governmental Order applicable to such Seller or by which any of the properties or assets of such Seller are bound;

(c)violate or conflict with, result in a breach of, or constitute a Default or otherwise cause any loss of benefit under any Contract or Permit to which such Seller is a party, or give to others any rights (including rights of termination, foreclosure, cancellation or acceleration), in or with respect to such Seller or the Shares or Free Shares as applicable, held by such Seller; or

(d)result in, require or permit the creation or imposition of any Encumbrances of any nature upon or with respect to the Shares held by such Seller.

3.3Absence of Litigation. There are no judicial, administrative or other governmental actions, proceedings or investigations pending or, to the knowledge of such Seller, threatened involving such Seller that question any of the transactions contemplated by, or the validity of, this Agreement which, if adversely determined, would materially impair the ability of such Seller to enter into or perform its obligations under this Agreement or materially delay, affect or prohibit the consummation of the transactions contemplated by this Agreement. Such Seller has not received any request from any Governmental Authority for information with respect to the transactions contemplated hereby.

3.4Ownership of the Shares. Such Seller owns the Shares set forth opposite such Seller’s name in Exhibit A, beneficially and of record, free and clear of any Encumbrances. There are no shareholder or other agreements  that will not be terminated at the Closing Date affecting the right of such Seller to convey the Shares to Purchaser or any other right of such Seller with respect to the Shares that will not be terminated prior to Closing, and at the Closing such Seller will have the absolute right, authority, power, and capacity to sell, assign, and transfer the Shares owned by it to Purchaser free and clear of any Encumbrance (except for restrictions imposed generally by Applicable Law). Upon the exchange of the documents mentioned in Section 2.15, Purchaser will acquire good, valid, and marketable title to the Shares, free and clear of any Encumbrances. Such Seller does not own any shares in the capital of Corlieve, nor does such Seller own or claim any other interest in any options, warrants or other rights to acquire any shares in the capital of Corlieve.

3.5Approvals, etc. Except for the approval of the French Ministry of Economy, Finance and Recovery (Ministère de l’Economie, des Finances et de la Relance) and the supervisory board’s

27

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

meeting of Corlieve pursuant to Article 14.11 of the Bylaws, all consents, approvals, authorizations and orders (corporate, governmental or otherwise) necessary for the due authorization, execution and delivery by such Seller of this Agreement and the other Transaction Agreements to which such Seller will be a party, the performance of such Seller of any of its obligations hereunder and thereunder and the consummation of the transactions contemplated hereby and thereby will have been obtained as of the Closing.

3.6No Brokers. Such Seller and its officers, directors or employees have not entered into any Contract with any broker, finder or similar agent or any Person which will result in an obligation of Purchaser, Corlieve, or any of their respective Affiliates to pay any finder’s fee, brokerage fees or commission or similar payment in connection with the transactions contemplated hereby.

3.7Compliance with Securities Laws.

(a)Such Seller (i) is either not a U.S. Person or qualifies as an Accredited Investor, (ii) acknowledges that, subject to Section 7.4, the Milestone Shares are not registered under the Securities Act, and that the Milestone Shares may not be transferred or sold except pursuant to the registration provisions of the Securities Act or pursuant to an applicable exemption therefrom and, accordingly, that it may not be able to liquidate the Milestone Shares, and (iii) is able to bear the economic risk of holding the Milestone Shares (including total loss of its investment).

(b)If such Seller is not a U.S. Person, such Seller is not acquiring the Milestone Shares for the account or benefit of any U.S. Person.

(c)If such Seller is an Accredited Investor, such Seller is acquiring the Milestone Shares solely for its own account for investment purposes and not with a view to, or for offer or sale in connection with, any distribution thereof in violation of the Securities Act.

3.8Ownership of uniQure Ordinary Shares. Such Seller does not, directly or indirectly, own any uniQure Ordinary Shares.

ARTICLE 4

REPRESENTATIONS AND WARRANTIES REGARDING CORLIEVE

Except as set forth herein or in the disclosure letter delivered by the Holder Representative to Purchaser in connection with the execution of this Agreement as may be updated pursuant to Section 6.10 (the “Disclosure Letter”), the Company hereby represents and warrants to Purchaser that the statements contained in this Article 4 are true and correct as of the Agreement Date and as of the Closing Date (except for the representations and warranties that are made as of a specific date, which shall be true and correct as of such date). The Disclosure Letter will be arranged in sections corresponding to the numbered and lettered paragraphs contained in this Article 4. The Sellers shall not be liable under Article 9 for a breach of a specific representation or warranty for any fact or item fairly disclosed in the Disclosure Letter against such specific representation and warranty; provided, that any fact or item disclosed on any Section of the Disclosure Letter shall be deemed disclosed on all other Sections of the Disclosure Letter to which an appropriate cross-reference is made or in all other Sections of the Disclosure Letter where it is reasonably apparent on its face that such disclosure fairly applies to such other Sections of the Disclosure Letter (irrespective of the language in which they are disclosed).

4.1Organization of Corlieve. Corlieve is duly incorporated and organized, validly existing and in good standing under Applicable Laws of France with full power and authority to conduct its business as it is presently being conducted, and to own, lease or operate, as applicable, its assets and properties. A true, correct and complete copy of the Bylaws, and all amendments thereto, have

28

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

heretofore been made available to Purchaser and are accurate and complete as of the Agreement Date. Corlieve is not in violation of the Bylaws.

4.2Subsidiaries. All the outstanding shares of capital stock of, or other equity or voting interests in the Swiss Subsidiary have been validly issued and are fully paid and nonassessable and are owned by Corlieve free and clear of all liens and are duly authorized, validly issued, full paid and nonassessable.  Other than the Swiss Subsidiary, neither the Company nor the Swiss Subsidiary owns or has owned any capital stock of, or other equity or voting interests of any nature in, or any interest convertible, exchangeable or exercisable for, capital stock of, or other equity or voting interests of any nature in, any other Person. With the exception of the Swiss Subsidiary, Corlieve does not have, and has never had, any Subsidiary and does not otherwise own or control, directly or indirectly, or hold any rights to acquire, any capital stock or any other securities, interests or investments (other than investments that constitute cash or cash equivalents) in any other corporation, partnership, trust, joint venture, association or other Person.

4.3Capitalization.

(a)Section 4.3(a) of the Disclosure Letter sets forth the name of each shareholder of Corlieve and the number of Shares held by each such shareholder as at the date hereof and the Closing Date. At the Closing, the Shares held by the Sellers will represent 100% of the issued, outstanding and authorized capital of Corlieve. All of the Shares have been and will be duly and validly issued. None of the Shares were or will be issued in violation of preemptive or similar rights or in violation of any applicable securities laws. Section 4.3(a) of the Disclosure Letter also sets forth a capitalization table of Corlieve as of the Agreement Date (the “Capitalization Table”).

(b)Section 4.3(b) of the Disclosure Letter sets forth the name of each Free Share Holder and the number of Free Shares held by each Free Share Holder. Except as set forth on Section 4.3(b) of the Disclosure Letter, there is no existing option, warrant, call, right (including preemptive rights, subscription rights, rights of first refusal and commitments) or Contract of any character to which Corlieve is a party requiring, and there are no securities of Corlieve outstanding which upon conversion or exchange would require, the issuance of any shares in the capital of Corlieve or other securities convertible into, exchangeable for or evidencing the right to subscribe for or purchase any shares in the capital of Corlieve or that provides for any share appreciation or similar right. Except as set forth in Section 4.3(b) of the Disclosure Letter, Corlieve is not a party to any voting trust or other Contract or arrangement with respect to the voting, redemption, sale, transfer or other disposition of any shares in the capital, or other equity securities of Corlieve.

4.4Title to Properties and Assets.

(a)Corlieve has good and marketable title to or, in the case of leased properties or properties held under license, a good and valid leasehold or license interest in all of its properties and assets free and clear of any Encumbrances other than Permitted Encumbrances.

(b)All of the tangible assets of Corlieve are in reasonably serviceable operating condition and repair (giving due account to the age and length of use of same, ordinary wear and tear excepted) and are adequate for the conduct of the Business in substantially the same manner as it has heretofore been conducted.

(c)Corlieve does not own or lease, and has never owned or leased, any real property.

4.5Absence of Certain Activities or Changes. Except as set forth in Sections 4.5(ii) and 4.5(viii) of the Disclosure Letter or for the Specified Costs, since the date of the Most Recent Balance Sheet, (a) Corlieve has conducted its operations in the Ordinary Course of Business, (b) there has been no Material Adverse Effect and (c) Corlieve has not:

29

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

(i)made any capital expenditures in excess of [*];

(ii)amended or changed the Bylaws;

(iii)changed its accounting methods, principles or practices;

(iv)sold, assigned, transferred, encumbered, abandoned, licensed or permitted to lapse or expire any Corlieve Intellectual Property or other intangible assets other than in the Ordinary Course of Business;

(v)sold or disposed of any material assets or rights;

(vi)been involved in any transaction or event which has given rise to a Tax Liability other than in respect of matters arising in the Ordinary Course of Business.

(vii)made, changed or rescinded any of its Tax elections, filed any Tax Return out of the ordinary course or in a manner that is not consistent with past practice and applicable law, filed any amended Tax Returns, settled or compromised any claim or assessment, entered into any closing agreement, settled or compromised any claim, action, suit, litigation, proceeding, arbitration, investigation, audit controversy relating to a material amount of Taxes, except as required by applicable law or French GAAP, made any change to any of its methods of reporting income or deductions for federal income Tax purposes from those employed in the preparation of Corlieve’s most recent income Tax Return; surrendered any claim for a refund of Taxes; or

(viii)except for (x) the Transaction Bonus, (y) non significant changes in the Ordinary Course of Business, changed the employment terms of, paid any bonus to, increased any salary or wages for, or entered into any employment, consulting or other service Contract with, any Person, or instituted, adopted, amended or terminated any Benefit (all except as required by Applicable Law or in accordance with existing contracts).

4.6Material Contracts.

(a)Section 4.6(a) of the Disclosure Letter sets forth all of the following outstanding Contracts to which Corlieve is a party or by which it is bound (collectively, the “Material Contracts”):

(i)Contracts that contain covenants requiring Corlieve not to (or that otherwise restrict or limit Corlieve’s ability to) compete in any line of business or geographical area (including any covenant not to compete with respect to the research, development, manufacture, marketing, distribution or sale of any product or product line) or transact business or deal in any other manner with any other Person;

(ii)Contracts that involve real property;

(iii)Contracts that involve a joint venture, strategic alliance, partnership or limited liability company relationship;

(iv)Contracts that govern or relate to Indebtedness for borrowed money (other than accounts payable), or guarantees for money borrowed by others;

(v)supply agreements involving aggregate payments in excess of [*] per annum;

(vi)Contracts that relate to the acquisition or disposition of any assets in excess of [*] outside of the Ordinary Course of Business;

30

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

(vii)Contracts that require payments or the receipt of payments by Corlieve in excess of [*] per annum outside of the Ordinary Course of Business;

(viii)powers of attorney pursuant to which Corlieve has granted authority to act on its behalf;

(ix)Contracts that involve the payment of royalties or other amounts calculated upon the revenues or income of Corlieve or income or revenues related to any product or Intellectual Property of Corlieve in excess of [*] per annum;

(x)Contracts between Corlieve and any Person that relate to the acquisition, transfer, assignment, sale, use, development, sharing, license or commercialization (including covenants not to sue) of Intellectual Property or any Corlieve Product (other than (A) agreements between Corlieve and its Workers in Corlieve’s standard forms thereof, (B) non-exclusive licenses to third-party Software with license fees less than [*] per year and (C) customary confidentiality agreements, material transfer agreements, or service agreements with third parties that provide only a license to evaluate the Intellectual Property subject to such agreement or provide the services contemplated in such agreement or transfer Intellectual Property to Corlieve in the Ordinary Course of Business);

(xi)Contracts in which Corlieve has granted any exclusive rights, rights of first refusal or rights of first negotiation, including with respect to any Intellectual Property of Corlieve, to any Person;

(xii)employment Contracts and Contracts with any consultant requiring an annual payment of cash compensation in excess of [*];

(xiii)Contracts with any current or former officer, director, Affiliate or shareholder of Corlieve;

(xiv)Contracts to which any Governmental Authority is a party (other than clinical trial agreements).

(b)True, correct and complete copies of all Material Contracts, and all amendments thereto, have been provided (or made available) to Purchaser. Each Material Contract is in full force and effect and is valid, binding and enforceable against Corlieve and, to the Knowledge of Corlieve, each other party thereto in accordance with its terms except as enforcement may be limited by applicable bankruptcy, insolvency, reorganization, moratorium and other Laws affecting enforcement of creditors’ rights generally and except insofar as the availability of equitable remedies may be limited by Applicable Law. Corlieve is not in Default under any Material Contract and, to the Knowledge of Corlieve, no other party is in Default under such Material Contracts. No written notice of any claim of Default under a Material Contract has been given to Corlieve. Except as set forth in Section 4.6(b) of the Disclosure Letter, immediately following the Closing, Corlieve will continue to be permitted to exercise all of its rights under each Material Contract pursuant to the terms thereof without the payment of any additional amounts of consideration other than ongoing fees, royalties or payments that Corlieve would otherwise be required to pay in accordance with the terms of such Material Contract had the transactions contemplated by each Transaction Agreement not occurred.

(c)Except as set forth in Section 4.6(c) of the Disclosure Letter or as required by Applicable Law, no Person (including, in particular, any current or former employee, legal representative, officer and/or consultant of Corlieve) has any option or any other right to participate in or receive any payment as a result of the development, commercialization and/or marketing of any Corlieve Products under any Contract (including employment contracts) to which Corlieve is party or by which it is bound.

31

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

4.7Compliance with Other Instruments. The execution, delivery and performance of and compliance with each Transaction Agreement and the consummation of the transactions contemplated hereby and thereby will not (i) assuming delivery of the document referenced in Section 2.15(a)(v), result in a violation or breach of, or be in conflict with any provision of the Bylaws, (ii) assuming the consents and approvals referred to in Section 4.15 are duly obtained, result in a violation of, or be in conflict with or constitute, with or without the passage of time or the giving of notice or both, a Default under, any Applicable Laws, Court Orders or Permits applicable to Corlieve, (iii) assuming the consents and approvals referred to in Section 4.15 are duly obtained, violate, conflict with, result in a Default under, or result in the creation of any Encumbrance (other than a Permitted Encumbrance) upon any of the material properties or material assets of Corlieve, or (iv) result in a Default under a Material Contract.

4.8Financial Statements.

(a)The Financial Statements have been prepared in accordance with French GAAP applied on a consistent basis during the periods referred to in the Financial Statements and fairly present, in all material respects, the financial condition of Corlieve as at the respective dates referred to in the Financial Statements, and the results of operations and cash flows of Corlieve for the respective periods referred to in the Financial Statements. The Financial Statements have been prepared in accordance with the books and records of Corlieve, which books and records are accurate and complete in all material respects. There have been no instances of fraud by Corlieve or its officers, whether or not material, that occurred during any period covered by the Financial Statements.

4.9Liabilities. Except as set forth in Section 4.9 of the Disclosure Letter, Corlieve has no Liabilities of any nature which would be required to be disclosed on a balance sheet or in any notes thereto prepared in accordance with French GAAP applied consistently with the Most Recent Balance Sheet that were not so disclosed, except for Liabilities (i) incurred or accrued in Corlieve’s Ordinary Course of Business since the date of its Most Recent Balance Sheet, (ii) reasonably incurred in connection with the execution of this Agreement or the other Transaction Agreements (iii) included within the Specified Costs, or (iv) disclosed in the Disclosure Letter.

4.10Taxes. Corlieve has duly, timely and correctly filed all Tax Returns it is required to have filed on or prior to the date of the Agreement and maintained all records and supplied all other information in relation to Tax which they were required to make, give, maintain or supply and all such Tax Returns, notices, records and information were accurate, complete and correct in all material respects. Corlieve has delivered to Purchaser true, complete and correct copies of all Tax Returns of Corlieve relating to Taxes for all Tax Periods for which the applicable statute of limitations has not yet expired.

(a)Corlieve has timely paid all Taxes required to have been paid (whether or not shown as due on any Tax Return) and all Taxes due in connection with its operations have been timely paid in all material respects. Taxes that have accrued and/or are due but have not been paid have been fully provided for in the Most Recent Balance Sheet.

(b)Each transaction between any of the Sellers on the one hand and Corlieve on the other hand and between Corlieve and its Affiliates has been carried out on an arm's length basis.

(c)There neither is nor has been any dispute, including litigation, between Corlieve and any Tax Authority, nor have Corlieve been the subject of any extraordinary investigation by any Tax Authority and there are no facts which are likely to give rise to any such dispute or investigation.

(d)For all periods for which the applicable statute of limitations has not expired, with respect to Corlieve:

32

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

(i)no written claim has been made by any Taxing Authority in any jurisdiction where Corlieve does not file Tax Returns that it is or may be subject to Tax by that jurisdiction;

(ii)no extensions or waivers of (A) statutes of limitations with respect to the Tax Returns of Corlieve or (B) the period of collection of any Taxes of Corlieve, in each case have been given by or requested from Corlieve; and

(iii)no written claim for assessment or collection of Taxes has been asserted against Corlieve which remains unpaid or unresolved, and there is no presently pending audit examination, refund claim, litigation, Proceeding, proposed adjustment or matter in controversy with respect to any Taxes of or with respect to Corlieve.

(e)Corlieve has always been resident for Tax purposes in the jurisdiction in which it is incorporated and has never been resident in any other jurisdiction or, except for the Swiss Subsidiary, traded through a branch or permanent establishment located outside such jurisdiction.

(f)All deficiencies asserted or assessments made against Corlieve as a result of any examinations by any Taxing Authority have been fully paid.

(g)There are no Encumbrances for Taxes (other than Permitted Encumbrances) upon the assets of Corlieve.

(h)Corlieve is not party to or bound by any Tax indemnity, Tax sharing, Tax allocation or similar agreement, other than an agreement entered into in the Ordinary Course of Business, the principal purpose of which is not the sharing of Taxes (such as a lease).

(i)Corlieve is not party to or bound by any ruling, closing agreement, offer in compromise or other agreement with any Taxing Authority.

(j)Corlieve has never (1) made an election to be treated as a United States domestic corporation for United States tax purposes, (2) made an election to be classified for United States federal income tax purposes as an entity that is not an association Taxable as a corporation.

4.11Environmental Matters. Corlieve has been and is in material compliance with applicable Environmental Laws. There have been no disposals, releases or, to Corlieve’s Knowledge,  threatened releases of Hazardous Materials by Corlieve. There has never been an Environmental Claim pending or, to the Knowledge of Corlieve, threatened against Corlieve. Corlieve is not subject to any Court Order, letter or memorandum by or with any Governmental Authority imposing any Liability under any Environmental Law.

4.12Employee Benefits.

(a)Section 4.12(a) of the Disclosure Letter sets forth a true, complete and accurate list of each Benefit other than those required by Applicable Laws. True, correct and complete copies of each benefit plan have been provided or made available to Purchaser as of the Agreement Date, including true, correct and complete copies of, where applicable, (i) all material documents embodying and relating to each Benefit, including any plan document (or, in the case of an unwritten Benefit, a written description thereof) and all amendments thereto, the most recent summary plan description (and any summaries of material modifications with respect thereto), and (ii) all material written Contracts, instruments or agreements relating to each Benefit.

(b)Each Benefit complies in form and has been established, maintained and administered in accordance with its terms, and in compliance in all material respects with the requirements of Applicable Law. All payments with respect to each Benefit, including without limitation all contributions (including all employer contributions and employee salary reduction contributions),

33

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

insurance premiums or intercompany charges, required to have been made under the terms of any document providing for such Benefit or other agreement or by Applicable Law, have been made or paid by the due date thereof in accordance with the provisions of each document providing for the Benefit, Applicable Law, and all contributions or payments for any period ending on or before the Closing Date which are not yet due will have been paid or accrued prior to the Closing Date in accordance with the provisions of each document providing for the Benefit, Applicable Law.

(c)With respect to each Benefit of Corlieve that is funded mostly or partially through an insurance policy, Corlieve has no liability in the nature of retroactive rate adjustment, loss sharing arrangement or other actual or contingent liability arising wholly or partially out of events occurring on or before the Closing Date or is reasonably expected to have such liability with respect to periods through the Closing.

(d)Section 4.12(d) of the Disclosure Letter sets forth a list of all (i) employment agreements with officers of Corlieve, (ii) agreements with consultants who are individuals obligating Corlieve to make, pursuant to which Corlieve has made, or pursuant to which it is reasonably likely that Corlieve will in the future make, annual cash payments in an amount of [*] or more, (iii) Contracts, programs and policies of Corlieve under which Corlieve may be obligated to provide a Worker severance or any other compensation or benefits as a result of the transaction contemplated by this Agreement or upon termination of employment or any other relationship with Corlieve, (iv) plans, programs, agreements and other arrangements of Corlieve with or relating to its Workers that contain change in control benefit provisions and (v) all written agreements between Corlieve and any Worker of Corlieve (collectively, the “Worker Agreements”). Corlieve has provided or made available to Purchaser true, correct and complete copies of all such Worker Agreements.

(e)Except for any Free Shares and Transaction Bonus, the execution and delivery of this Agreement by the Sellers and the consummation of the transactions contemplated hereby (either alone will not (i) result in any payment becoming due, or increase the amount of any compensation or benefits due, to any current or former Worker of Corlieve under any Worker Agreement or otherwise or with respect to any benefit plan of Corlieve; (ii) increase any benefits or payments otherwise payable under any Worker Agreement or any Benefit of Corlieve; (iii) result in the acceleration of the time of payment or vesting of any such compensation or benefits; or (iv) result in the forgiveness in whole or in part of any outstanding loans made by Corlieve to any Person.

(f)None of the Benefits or Worker Agreements provide retiree health or welfare insurance benefits to any current or former employee of Corlieve except as may be required by Applicable Laws.

(g)None of Corlieve or any of its existing or former employees, current legal representatives, officers, directors, shareholders or other Workers has made any promises or commitments, whether legally binding or not, to create any additional Worker Agreement or Benefits, agreement or arrangement, or to modify or change in any material way any existing Worker Agreement or Benefit.

(h)Corlieve has no unfunded liabilities pursuant to any Contract or other arrangement implementing a Benefit.

4.13Compliance with Law.

(a)The operation of Corlieve’s business has been and is being conducted in all material respects in compliance with all Applicable Laws and all Court Orders applicable to Corlieve or its business. Corlieve has not received any written notice from a Governmental Authority to the effect that, or otherwise been advised that, it is not in compliance with any such Applicable Laws or Court Orders, and, to Corlieve’s Knowledge, no circumstance exists that could reasonably be expected to result in material violations of any Applicable Law or Court Order.

34

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

(b)Corlieve is not required by Applicable Law to notify or consult with its employees in respect of the sale of the Shares.

4.14Permits. Corlieve has all Permits required for the operation of the Business as presently conducted, all of which are valid and in full force and effect. Section 4.14 of the Disclosure Letter sets forth a complete list of all such Permits issued to Corlieve, and true, correct and complete copies of such Permits have been made available to Purchaser. Corlieve is not in Default, nor has it received any written notice of any claim of Default, with respect to any such Permit. No suspension, termination, revocation, cancellation or restriction of any such Permits is pending or, to the Knowledge of Corlieve, threatened. No Governmental Authority is challenging or has threatened in writing to challenge the right of Corlieve to design, research, develop, pre-clinically or clinically test, manufacture, license, commercialize, offer or sell any of its products or services.

4.15Consents and Approvals. Except for the approval of the French Ministry of Economy, Finance and Recovery (Ministère de l’Economie, des Finances et de la Relance), no consent, approval, order or authorization of, or declaration to, or filing or registration with, any Governmental Authority, or any other Person, is required to be made, obtained or given by Corlieve in connection with the execution, delivery and performance of this Agreement and the consummation of the transactions contemplated hereby.

4.16Litigation. There is no action, suit, proceeding, claim, arbitration, audit of Governmental Authority, criminal prosecution, unfair labor practice charge or complaint, examination or investigation from any third party (“Proceeding”) pending (or, to the Knowledge of Corlieve, threatened) against Corlieve, or relating to its activities, business, properties or assets, or, to Corlieve’s Knowledge, against any officer, director or employee of Corlieve in connection with such officer’s, director’s or employee’s relationship with, or actions taken on behalf of, Corlieve. None of Corlieve or its assets or properties is a party to or subject to the provisions of any Court Order, and there is no Proceeding by Corlieve currently pending or which Corlieve intends to initiate.

4.17Labor Matters.

(a)Corlieve has no labor unions, works councils or other organizations representing any employees of Corlieve. No Person (including consultants, independent workers, temporary workers, agents, trainees, apprentices and fixed-term/seasonal employees), other than the employees of Corlieve, has the right to claim the status of employee or permanent employee of Corlieve.

(b)Corlieve is and has been in compliance in all material respects with all Applicable Laws regarding employment, termination of employment, employment practices, terms and conditions of employment, wages and hours, duration of work, overtime, applicable collective bargaining, employment discrimination, leaves of absence, immigration, civil rights, safety and health, workers’ compensation, pay equity, classification of employees, the collection and payment of withholding and/or social security Taxes and other employment related Taxes. In addition, each employee of Corlieve is in compliance with all applicable visa and work permit requirements. Except as set forth in Section 4.17 of the Disclosure Letter, no visa or work permit held by an employee of Corlieve will expire during the six (6)-month period beginning at the Closing Date.

(c)No claims, disputes, grievances, controversies or labor disputes are pending, or to Corlieve’s Knowledge, threatened or anticipated involving any Worker or group of Workers of Corlieve. There have been no written claims, charges, investigations, administrative proceedings or written complaints of harassment, discrimination (including discrimination based upon sex, age, marital status, race, national origin, sexual orientation, disability or veteran status), retaliatory action pertaining to any Worker against Corlieve or any employee, legal representative, officer or director of Corlieve at any time during the past four (4) years, and to the Knowledge of Corlieve, no facts exist that could reasonably be expected to give rise to such claims or actions.

35

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

(d)Section 4.17(e) of the Disclosure Letter sets forth an accurate and complete list of all (i) employees of Corlieve, including each employee’s name, title or position, present annual compensation (including bonuses, commissions and deferred compensation), accrued and unused paid vacation and other paid leave, years of service, interests in any incentive compensation plan, vested and unvested equity interests, and estimated entitlements to receive supplementary retirement benefits or allowances (whether pursuant to a contractual obligation or otherwise), and (ii) individuals who are currently performing services for Corlieve who are classified as independent contractors, including the respective compensation of each consultant or independent contractor.

(e)To the Knowledge of Corlieve, no existing or former employee of Corlieve is or has been in any material respect in violation of any term of any employment Contract, non-disclosure agreement, non-competition agreement, or any restrictive covenant to a former employer relating to the right of any such employee to be employed by Corlieve because of the nature of the business conducted or presently proposed to be conducted by Corlieve or to the use of trade secrets or proprietary information of others.

4.18Intellectual Property.

(a)Section 4.18(a) of the Disclosure Letter sets forth, with respect to all Corlieve Intellectual Property, a complete and accurate list of all (1) Patents and other indicia of ownership or license of any invention issued or filed with any Governmental Authority, (2) trade names, common law trademarks, common law service marks, registered trademarks, registered service marks, and applications for trademark registration or service mark registration, (3) registered Copyrights and (4) domain name registrations and websites in each case owned, licensed, used or held for use by Corlieve in the conduct of its business, specifying as to each such item, as applicable (i) the owner(s) and licensor or licensee, of the item, (ii) the jurisdictions in which the item is issued or registered or in which any application for issuance or registration has been filed, (iii) the respective issuance, registration, and application number of the item, and (iv) the date of application and issuance or registration of the item.

(b)Section 4.18(b) of the Disclosure Letter sets forth, with respect to Corlieve, a complete and accurate list of all written licenses, sublicenses, consents and other agreements (whether written or otherwise) (i) pertaining to any Intellectual Property held for use or used by Corlieve in the conduct of its business (the “Head Licenses”), or (ii) by which Corlieve licenses or otherwise authorizes a third party to use or covenants not to sue or grants an immunity from suit any Corlieve Intellectual Property (other than (1) agreements between Corlieve and its employees in Corlieve’s standard forms thereof, (2) non-exclusive licenses to third-party Software with license fees less than [*] per year and (3) customary confidentiality agreements, material transfer agreements or service agreements with third parties that provide only a license to evaluate the Intellectual Property subject to such agreement or provide the services contemplated in such agreement). Neither Corlieve nor, to Corlieve’s Knowledge, any Third Party is in Default under any such license or other agreement in any material respect, and each such license or other agreement is now and immediately following the Closing shall be in full force and effect. The execution, delivery and performance of this Agreement and the consummation of the transactions contemplated hereby will not result in the loss, forfeiture, termination, license, or impairment of, or give rise to a right to limit, terminate, or consent to the continued use of any Corlieve Intellectual Property. To Corlieve’s Knowledge the Corlieve Intellectual Property includes all Intellectual Property necessary for the conduct of the business of Corlieve as currently conducted and contemplated to be conducted as of the Closing Date.

(c)To Corlieve’s Knowledge, the making, using or selling of the Corlieve Products and Corlieve’s operation of the business do not and have not infringe(d), misappropriate(d) or otherwise violate(d) the Intellectual Property rights of any Person, or constitute unfair competition or trade practices under the Laws of any jurisdiction. To Corlieve’s Knowledge, no Person is infringing, misappropriating or otherwise violating,  or has infringed, misappropriated or otherwise violated, the Corlieve Intellectual Property, and Corlieve has not filed or threatened in writing any claims alleging

36

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

that a Third Party has infringed, misappropriated or otherwise violated any Corlieve Intellectual Property. No Third Party has filed or threatened to file any claims alleging that Corlieve has infringed, misappropriated or otherwise violated any Person’s Intellectual Property rights. Except in the Material Contracts, Corlieve has not given any indemnification, release or covenant to any Third Party against infringement, misappropriation or other violation of Intellectual Property related to  Corlieve Products or Corlieve Intellectual Property. Except as set forth in Section 4.18(c) of the Disclosure Letter, Corlieve has not requested (whether or not received) any written opinion of patent or other intellectual property counsel and Corlieve has not been provided with any written of opinion of patent or other intellectual property counsel, that concerns infringement, validity or enforceability of any Person’s Intellectual Property  rights.

(d)All of the items listed in Section 4.18(a) of the Disclosure Letter are, except as otherwise indicated therein, owned solely and exclusively by Corlieve, or licensed solely and exclusively to Corlieve through sole and exclusive ownership of the respective licensor, or co-owned with Corlieve in accordance with the terms of the applicable Material Contracts, and are free and clear of all Encumbrances (other than co-ownership listed in Section 4.18(a) of the Disclosure Letter, the terms of the applicable Material Contracts, or Permitted Encumbrances).   The issued Patents listed in Section 4.18(a) of the Disclosure Letter are not the subject of any cancellation, opposition, nullity, reexamination proceeding or any other proceeding challenging their scope, validity, enforceability, ownership, or use.  No opposition, extension of time to oppose, interference, rejection, or refusal (such rejection or other refusal only in the normal course of Patent prosecution prior to grant) to register has been filed in connection with any patent or patent application listed in Section 4.18(a) of the Disclosure Letter.  The issued Patents listed in Section 4.18(a) of the Disclosure Letter are valid and enforceable, and the ownership of the entire right, title and interest therein of each owned or licensed item listed in Section 4.18(a) of the Disclosure Letter is recorded or registered with the applicable Governmental Authority solely in the name of Corlieve or its licensor(s). All fees, Taxes, annuities and other payments associated with filing, prosecuting, issuing, recording, registering or maintaining any such Intellectual Property have been paid in full in a timely manner to the proper Governmental Authority. None of the Intellectual Property owned, co-owned, or licensed by Corlieve is subject of any order, decree or injunction of any Governmental Authority and Corlieve has not been subject to any order, decree or injunction of any Governmental Authority in respect of any other Person’s Intellectual Property. None of the Intellectual Property used by Corlieve is the subject of any order, decree or injunction of any Governmental Authority and Corlieve has not been subject to any order, decree or injunction of any Governmental Authority in respect of any other Person’s Intellectual Property.  The items listed in Section 4.18(a) of the Disclosure Letter as co-owned by Corlieve and another party, are either (i) exclusively licensed to Corlieve, or (ii) are subject to Corlieve’s exclusive option to be exclusively licensed to Corlieve under the terms of the applicable Material Contracts.

(e)None of the trade secrets or material confidential or proprietary information of Corlieve has been disclosed to any Person unless such disclosure was necessary and made pursuant to an appropriate confidentiality agreement and, to Corlieve’s Knowledge, there has not been any breach by any such Person of any such agreement. All Workers either (i) work in jurisdictions where their inventions are automatically assigned to Corlieve by operation of law, or (ii) have entered into a written agreement automatically assigning to Corlieve all rights to such contributions, which law or agreement includes a present-tense assignment of future inventions, discoveries, and data, copies of which have been provided to Purchaser prior to the Agreement Date. All Workers have entered into a written confidentiality agreement with Corlieve, copies of which have been provided to Purchaser prior to the Agreement Date.

(f)Except for any fees payable (i) to a Governmental Authority to issue, register or maintain any of the Intellectual Property listed in Section 4.18(a) of the Disclosure Letter, (ii) as required by Applicable Law and (iii) the payments due under the Head Licenses, no payment of any kind is required to be made by Corlieve to any Person (including directors, officers, employees, consultants, contractors and agents of Corlieve and inventors of owned or licensed Corlieve Intellectual

37

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

Property) for the ownership, license or use of any Corlieve Intellectual Property. Except for mandatory licenses listed in Section 4.18(f) of the Disclosure Letter, no Governmental Authority or other Third Party has any rights in the Corlieve Intellectual Property.

(g)All data and personal information used or maintained by Corlieve has been collected, maintained, used and transferred in accordance with Corlieve’s applicable data protection and privacy principles and policies and in all material respects in accordance with Applicable Law. No Person has claimed any compensation from Corlieve for the loss of or unauthorized disclosure or transfer of personal data or information, and, to Corlieve’s Knowledge, no facts or circumstances exist that might give rise to such a claim.

(h)RGX had and continues to have the right to grant all rights and licenses it purported to grant to Corlieve in the RGX License Agreement.

(i)The French National Institute of Health and Medical Research (INSERM), Universite de Bordeaux, Aix-Marseille Université and Centre National de Recherche Scientifique (CNRS), are the sole owners of the patent family claiming priority to patent filing [*], any foreign patent application corresponding thereto, and any divisional, additions, continuations, continuations in part, or re-examination application, as well as national or foreign equivalents, and each patent that issues or reissues from any of these patent applications.

(j)Except as set forth in Section 4.18(j) of the Disclosure Letter, the inventions disclosed and claimed in the Corlieve Intellectual Property, (i) were not conceived, discovered, developed or otherwise made in connection with any research activities funded, in whole or in part, by any Governmental Authority, and (ii) no entity other than Corlieve and its licensors has any ownership, license or other interest in such inventions.

(k)To the Company’s Knowledge, the Licensed Know-How Schedule shall be true and accurate in all material respects as of the Closing Date.

4.19Transactions with Certain Persons. Except for the Material Contracts or employment agreements of Corlieve, no officer, director, Seller, Free Share Holder or Affiliate of Corlieve or, to the Knowledge of Corlieve, any individual in such officer’s, director’s, Seller’s or Free Share Holder’s immediate family has or has had, either directly or indirectly, a material interest in: (i) any Person or entity which purchases from or sells, licenses or furnishes to Corlieve any material goods, property, technology, intellectual or other property rights, (ii) any Material Contract to which Corlieve is a party or by which it is bound or to which any of its properties or assets is subject, or (iii) any property used by Corlieve with a book value or market value in excess of [*]. To the Knowledge of Corlieve, no officer, director, Seller or Free Share Holder of Corlieve has a claim or cause of action against Corlieve or Purchaser.

4.20Insurance. Section 4.20 of the Disclosure Letter sets forth a complete and correct list of all insurance policies of Corlieve of any kind currently in force, including for each such policy the type of coverage, the name of the insureds, the insurer, the expiration date, and the amounts of coverage. True, correct and complete copies of such insurance policies have been provided or made available to Purchaser. All such insurance policies are in full force and effect and all premiums due thereunder have been paid. The insurance policies maintained by Corlieve insure Corlieve against such losses and risks (including risks related to clinical trials and product liability) and in such amounts as are prudent and customary in the business in which it is engaged and are sufficient for compliance in all material respects with all requirements of Applicable Law and of all Material Contracts. There is no claim pending under any such policies. Corlieve is not in Default under any material provision of any such insurance policy, and Corlieve has not taken any action or failed to take any action which, with notice or lapse of time, would constitute a Default under any material provision of any such insurance policy. Corlieve has not

38

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

received notice of an increase in premiums with respect to, or cancellation, nonrenewal or termination of, any such insurance policy.

4.21Certain Business Practices. To the Knowledge of Corlieve, none of the directors, officers, agents or employees of Corlieve or any of their Affiliates has, in each case in connection with Corlieve’s business, (i) used any funds for unlawful contributions, gifts, entertainment or other unlawful expenses, including unlawful expenses related to political activity, (ii) made any unlawful payment to foreign or domestic government officials or employees or to foreign or domestic political parties or campaigns, made any bribes or kickback payments or violated any provision of any Applicable Law, or (iii) made any payment to any customer or supplier of Corlieve, or given any other consideration to any such customer or supplier in respect of Corlieve’s business that violates any Applicable Law.

4.22No Brokers. Neither Corlieve nor any of its officers, directors, employees or Affiliates has entered into any Contract with any broker, finder or similar agent or any Person which will result in an obligation of Purchaser, Corlieve, or any of their respective Affiliates to pay any finder’s fee, brokerage fees or commission or similar payment in connection with the transactions contemplated hereby.

4.23Regulatory Matters.

(a)Corlieve has no Knowledge (and has not been notified by a Corlieve Partner) of any pending investigation, review, enforcement action or adverse regulatory action by any Regulatory Authority against Corlieve or any Person that manufactures or develops Corlieve Products pursuant to a development, contract research, manufacturing, supply or other collaboration arrangement with Corlieve (each, a “Corlieve Partner”).

(b)All Corlieve Products are being and have been developed, manufactured, used, processed, packaged, labeled, stored and tested by or on behalf of Corlieve in compliance in all material respects with all applicable requirements under all Applicable Laws, including, as applicable, those requirements relating to the conduct of preclinical and clinical studies under the French National Agency for the Safety of Medicine and Health Products (“ANSM”).

(c)Neither Corlieve nor, to the Knowledge of Corlieve, any of its respective agents or subcontractors or the Corlieve Partners has been convicted of any crime or engaged in any conduct which could result in debarment or disqualification by any Regulatory Authority, and there are no Proceedings pending or threatened in writing that would reasonably be expected to result in criminal liability or debarment or disqualification by any Regulatory Authority.

(d)Corlieve, and to Corlieve’s Knowledge the Corlieve Partners, have not imported, exported, marketed, sold, offered for sale, or distributed any investigational drug substance or drug product except in compliance with all Applicable Laws. Corlieve has made available to Purchaser true, correct, complete and unredacted copies of all material data and information with respect to the Corlieve Products and any material correspondence to and from any Regulatory Authority, including each clinical trial application submitted to the ANSM, adverse event files and manufacturing records. All applications, notifications, submissions, information, claims, reports and statistics and other data that have been utilized, or prepared with the intention to be utilized, as the basis for or submitted in connection with any regulatory or marketing approvals or Permits from ANSM or any other Regulatory Authority relating to the Corlieve Products were true, complete and correct in all material respects as of the date of preparation and submission, as applicable, and any necessary or required updates, changes, corrections or modifications to such applications, submissions, information and data have been submitted to ANSM or other Regulatory Authority.

(e)Corlieve and, to Corlieve’s Knowledge, the Corlieve Partners have not received from any Regulatory Authority any (i) inspection reports, (ii) notices of adverse findings, warnings, untitled

39

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

letters, minutes of meetings, or (iii) other correspondence from any Regulatory Authority concerning the Corlieve Products in which any Regulatory Authority asserted that the operations of Corlieve may not be in compliance with Applicable Law or that the Corlieve Products may not be approvable.

(f)Corlieve has not received written notice from any Corlieve Partner of any interruption of supply or manufacturing capacity, shortage of raw materials, components or other manufacturing problems that would have a material effect on the subsequent development (as such development is contemplated as of the Closing Date) of the Corlieve Products, nor do any conditions currently exist that reasonably could be expected to lead to such manufacturing problems.

(g)Section 4.23(g) of the Disclosure Letter sets forth a true and complete list of all authorizations, approvals, applications, clearances, consents, qualifications and other rights from any Regulatory Authority relating to the ability of Corlieve to research, develop and manufacture the Corlieve Products (“Regulatory Authorizations”) and there are no other Regulatory Authorizations required for the Corlieve Products in connection with the conduct of the Business as presently conducted. All such Regulatory Authorizations are (i) validly registered and on file with applicable Regulatory Authorities and (ii) in compliance with all formal filing and maintenance requirements.

(h)The studies, tests and preclinical studies conducted by Corlieve and, to the Knowledge of Corlieve, the studies, tests and preclinical tests conducted on behalf of the Corlieve, were and, if still pending, are being conducted in all material respects in accordance with approved experimental protocols, procedures and controls pursuant to accepted professional scientific standards and all Applicable Laws, including, without limitation, the applicable regulations in France. Corlieve is not aware of any studies, tests or trials the results of which Corlieve believes reasonably call into question the study, test, or trial results when viewed in the context of the clinical state of development. Corlieve has not received any notices or other written correspondence from any Regulatory Authority requiring the termination, suspension or material modification of any studies, tests or preclinical or clinical trials conducted by or on behalf of Corlieve.

4.24Subsidies. Section 4.24 of the Disclosure Letter sets forth a list of (i) all subsidies and/or grants granted to Corlieve by any Governmental Authority or any other Person and which are outstanding, (ii) all repayable or reimbursable subsidies and/or grants, whether outstanding or not, granted to Corlieve, including, without limitation, in the form of repayable advances, and (iii) all other subsidies and grants received by Corlieve since its incorporation (collectively the “Subsidies”). Corlieve is not in breach of any of the terms and conditions governing the Subsidies and Corlieve and, other than the transactions contemplated hereunder, the Sellers have not undertaken to do anything that would result in the granting Governmental Authority or Person being entitled to claim for the repayment or reimbursement, in whole or in part, of any such Subsidies.

4.25Books and Records. Corlieve has made and kept (and made available to Purchaser) correct and complete, in all material respects, minute books and records and accounts, which, in reasonable detail, accurately and fairly summarize the material corporate activities of Corlieve. The minute books of Corlieve made available to Purchaser accurately and adequately reflect all action previously taken by the shareholders of Corlieve, the President of Corlieve, and the Comité de Supervision (supervisory board) of Corlieve. All registration and publication formalities required to be carried out pursuant to Applicable Laws have duly and timely been carried out by Corlieve.

40

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

4.26Bank Accounts. Section 4.26 of the Disclosure Letter contains a true, correct and complete list of all bank accounts maintained by Corlieve, including each account number and the name and address of each bank and the name of each Person who has signature power with respect to each such account or power of attorney to act on behalf of Corlieve.

ARTICLE 5

REPRESENTATIONS AND WARRANTIES OF PURCHASER

Purchaser represents and warrants to the Sellers that the following statements contained in this Article 5 are true and correct as of the Agreement Date and as of the Closing Date.

5.1Organization and Standing. Purchaser is a public company with limited liability under the laws of the Netherlands (naamloze vennootschap), validly existing and in good standing under the Laws of the Netherlands.  Purchaser has all requisite corporate power and authority to execute and deliver this Agreement and the other Transaction Agreements, to consummate the transactions contemplated hereby and thereby and to perform its obligations hereunder and thereunder.

5.2Authorization. All proceedings required to be taken on the part of Purchaser to authorize Purchaser to enter into and carry out this Agreement and the other Transaction Agreements have been duly and properly taken. This Agreement has been, and each other Transaction Agreement will be, duly executed and delivered by Purchaser  and is, or will be, the valid and binding obligation of Purchaser enforceable against  Purchaser in accordance with its terms, except as enforcement may be limited by equitable principles limiting the right to obtain specific performance or other equitable remedies, or by applicable bankruptcy or insolvency Laws and related decisions affecting creditors’ rights generally.

5.3Compliance. The execution and delivery of this Agreement and the other Transaction Agreements, and the consummation of the transactions contemplated hereby and thereby, will not:

(a)result in the breach of any of the terms or conditions of, or constitute a Default under or violate, as the case may be, the articles of association of Purchaser; or

(b)violate any Law or Governmental Order applicable to Purchaser, or by which any of the properties or assets of Purchaser is bound, solely to the extent that such breach or violation would have a material adverse impact on Purchaser’s ability to consummate the transactions contemplated hereby.

5.4Milestone Shares.

(a)The Milestone Shares, if and when issued as contemplated herein, will be duly authorized, validly issued, fully paid, nonassessable, and free of preemptive rights and Encumbrances, except for restrictions on transfer imposed under applicable securities Laws. The issuance of such Milestone Shares does not contravene any Law or the rules and regulations of The NASDAQ Stock Market. Assuming the accuracy of the representations and warranties in Article 3, compliance by each Seller with the covenants in Article 7 and the compliance by each Seller with his or her obligations set forth in Section 2.13, the offer, issuance and sale of the Milestone Shares will be exempt from registration pursuant to Regulation S promulgated under the Securities Act and other applicable state securities Laws.

5.5Absence of Litigation. There are no judicial, administrative or other governmental actions, proceedings or investigations pending or, to the Knowledge of Purchaser, threatened against Purchaser that question any of the transactions contemplated by, or the validity of, this Agreement which, if adversely determined, would materially impair the ability of Purchaser to enter into or perform

41

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

its obligations under this Agreement or materially delay, affect or prohibit the consummation of the transactions contemplated by this Agreement. Purchaser has not received any request from any governmental agency or instrumentality for information with respect to the transactions contemplated hereby.

5.6Approvals, etc. Except for the approval of the French Ministry of Economy, Finance and Recovery (Ministère de l’Economie, des Finances et de la Relance), all consents, approvals, authorizations and orders (corporate, governmental or otherwise) necessary for the due authorization, execution and delivery by Purchaser of this Agreement and the other Transaction Agreements, the performance of Purchaser of any of its obligations hereunder and thereunder and the consummation of the transactions contemplated hereby and thereby will have been obtained as of the Closing.

5.7Funding. Purchaser has and will have sufficient available funds on hand at Closing to pay the Closing Date Cash Consideration.

5.8No Brokers. Neither Purchaser nor any of its officers, directors or employees has entered into any Contract with any broker, finder or similar agent or any Person which will result in an obligation of the Sellers to pay any finder’s fee, brokerage fees or commission or similar payment in connection with the transactions contemplated hereby.

5.9SEC Reports. Purchaser has timely filed all reports, registration statements, proxy statements and other materials, together with any amendments required to be made with respect thereto, that it was required to file with the SEC since December 31, 2019, and all such reports, registration statements, proxy statements, other materials and amendments have complied in all material respects with all legal requirements relating thereto (the “Public Documentation”). None of the Public Documentation, as of its respective date, contained  any untrue statement of a material fact or, when taken together, omitted to state a material fact necessary to make the statements therein in the light of the circumstances under which such statements were made, not misleading.

ARTICLE 6

COVENANTS AND AGREEMENTS

6.1Public Disclosures.

(a)Without the prior written consent of Purchaser, the Sellers shall not, and shall not authorize or permit Corlieve or any of their Representatives or Affiliates to, use Purchaser’s name or refer to Purchaser in a manner in which Purchaser’s identity is readily apparent in connection with Purchaser’s relationship with Corlieve, including in any media interview, advertisement, news release, press release or professional or trade publication, or in any print media, whether or not in response to an inquiry, unless otherwise required by Applicable Law. Notwithstanding anything herein to the contrary, (i) Purchaser and the Holder Representative shall mutually agree on the content of an initial press release announcing the entry into this Agreement and a subsequent press release on the Closing, and (ii) the Parties shall be entitled to use the content of such press releases and any other mutually approved press release in subsequent communication and public disclosure.

(b)Without limiting any other provision of this Agreement, except as otherwise contemplated by this Agreement, no Party shall make (or cause or permit any controlled Affiliate to make), directly or indirectly, any public announcement with respect to this Agreement and the transactions contemplated hereby without the consent of Purchaser and the Holder Representative, except as may be required by Applicable Law or the regulations of The NASDAQ Stock Market, in which case the party making such public announcement shall inform Purchaser or the Holder Representative, as applicable, promptly by written notice.

42

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

6.2Confidentiality. Purchaser and the Sellers each acknowledge that the information provided to it in connection with this Agreement and the transactions contemplated hereby is subject to the terms of the confidentiality agreement between Purchaser and Corlieve dated January 20, 2021 (the “Confidentiality Agreement”). Effective upon, and only upon, the Closing, Purchaser’s obligations under the Confidentiality Agreement shall terminate.

6.3Access to Information. Prior to the Closing, the Sellers, within the limit of their respective powers within the Company as officer and/or shareholder, shall cause Corlieve to provide Purchaser, from time to time, with reasonable access to the offices, properties, appropriate officers, books and records of Corlieve (during regular business hours, upon reasonable advance notice, under reasonable circumstances, subject to restrictions under applicable Law and without undue disruption to the normal business activities of Corlieve). Purchaser and its Representatives shall cooperate with Corlieve and its Representatives and they shall use their reasonable efforts to minimize any disruption to the Business. Notwithstanding anything herein to the contrary, no such investigation or examination shall be permitted to the extent that it would require Corlieve or any of the Sellers to disclose information subject to attorney-client privilege.

6.4Conduct of the Business Pending the Closing

(a)Conduct in the Ordinary Course of Business. Prior to the Closing, except: (i) as required by applicable Law; (ii) as expressly contemplated by this Agreement; or (iii) with the prior written consent of Purchaser (which consent shall not be unreasonably withheld, delayed or conditioned), the Sellers, within the limit of their respective powers within the Company as officer and/or shareholder, shall cause Corlieve to conduct the Business in the Ordinary Course of Business and, to the extent consistent therewith, use reasonable efforts to preserve business relationships with suppliers and others with whom Corlieve deals with in connection with the conduct of the Business.

(b)Restricted Conduct. Prior to the Closing, except (i) as required by applicable Law, (ii) as expressly contemplated by this Agreement, (iii) proposed agreements set out on Schedule 6.4(a), or (iv) with the prior written consent of Purchaser (which consent shall not be unreasonably withheld, delayed or conditioned and shall be deemed granted in the absence of a response by Purchaser within [*] of Corlieve’s request), the Sellers, within the limit of their respective powers within the Company as officer and/or shareholder,  shall cause Corlieve not to:

(i)transfer, issue, sell or dispose of any shares in the capital, or other securities of, Corlieve or grant options, warrants, calls or other rights to purchase or otherwise acquire any shares in the capital, or other securities, of Corlieve;

(ii)declare or pay any non-cash dividends on or make other non-cash distributions in respect of any of its share capital or other equity interests;

(iii)effect any split, combination, capitalization or reclassification of any capital stock or any like change in the capitalization of Corlieve;

(iv)(A) increase the annual level of compensation payable or to become payable by Corlieve to any of their respective legal representatives, members of the Comité de Supervision (supervisory board) or employees (other than any increase required by Applicable Laws or the terms of existing Contracts); (B) grant any bonus, benefit or other direct or indirect compensation to any legal representative, members of the Comité de Supervision (supervisory board) or employee (other than Transaction Bonus) of Corlieve other than as contemplated by existing Contracts or materially amend any term of their employment Contract with Corlieve; or (C) hire any new employee;

43

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

(v)incur, create, assume, guarantee or become liable for any Indebtedness other than (A) in the Ordinary Course of Business, (B) any draw under the Shareholder Loan, or (C) any loan made to Corlieve by Purchaser;

(vi)subject any of the properties or assets (whether tangible or intangible) of Corlieve to any Encumbrance, except for Permitted Encumbrances;

(vii)enter into, adopt, extend, renew or amend any collective bargaining agreement or other Contract with any labor organization, union or association;

(viii)acquire or commit to acquire by merger or consolidation with, or merge or consolidate with, or purchase substantially all of the assets of, any corporation, partnership, association, joint venture or other business organization or division thereof, or acquire any material properties or assets (except for the acquisition of supplies in the Ordinary Course of Business);

(ix)sell, assign, license, sublicense, transfer, convey, lease, abandon, permit to lapse or otherwise dispose of any of the properties or assets material to the Business, including Corlieve Intellectual Property (except for the purpose of disposing of obsolete or worthless assets or in the Ordinary Course of Business);

(x)cancel or compromise any material debt or claim owing to Corlieve;

(xi)enter into any Contract that restricts or limits the conduct or operations of the Business in any material respect;

(xii)adopt or enter into any plan of complete or partial liquidation, dissolution, restructuring or other reorganization, or file a petition in bankruptcy under any provisions of any bankruptcy or similar Law or consent to the filing of any bankruptcy petition against it under any bankruptcy or similar Law;

(xiii)make or rescind any material election relating to Taxes, settle or compromise any claim, action, suit, litigation, proceeding, arbitration, investigation, audit controversy relating to Taxes, or except as required by Applicable Law or French GAAP, make any change to any of its methods of reporting income or deductions for income Tax purposes from those employed in the preparation of Corlieve’s most recent Tax Return; enter into any closing agreement, settle or compromise any material claim or assessment, amend any Tax Return, file any Tax Return out of the ordinary course or in a manner that is not consistent with past practice and applicable law; or surrender any claim for a refund of material Taxes;

(xiv)fail to pay or discharge when due and payable any Liabilities;

(xv)change the accounting methods, principles or practices used by Corlieve to keep its books and records, except as required by French GAAP;

(xvi)extend, modify, terminate, cancel or renew, or waive a material right under, any Material Contract, except in the Ordinary Course of Business or pursuant to the terms of such Material Contract as of the date hereof;

(xvii)enter into any Contract which would qualify as a Material Contract;

(xviii)initiate, settle or compromise any litigation or other disputes (whether or not commenced prior to the date of this Agreement)

(xix)enter into or be a party to any transaction with any Seller or Free Share Holder other than in accordance with existing Contract or in the Ordinary Course of Business;

44

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

(xx)take any action inconsistent with this Agreement of the consummation of the transactions contemplated by this Agreement; or

(xxi)agree, whether in writing or otherwise, to do any of the foregoing.

6.5Exclusivity. From the Agreement Date until the earlier of the termination of this Agreement and the Closing, the Sellers will not, will cause Corlieve not to, and will use their reasonable efforts to cause their Representatives not to, directly or indirectly, facilitate or attempt to obtain or arrange for (i) any equity capital financing that would have the effect of any Person (other than the Purchaser or its Affiliates) acquiring equity interests (or rights convertible into equity interests) of Corlieve (other than as expressly contemplated herein), (ii) the merger, acquisition, consolidation, or recapitalization of Corlieve or (iii) a sale of the securities of Corlieve or a material portion of the assets of Corlieve, in each case to any third party (excluding Purchaser and its Affiliates) (each such transaction in the above subclauses (i), (ii) and (iii), an “Alternative Transaction”) or discuss or provide any other Person (other than Purchaser and its Affiliates and their respective Representatives) any information in connection with any such Alternative Transaction. In the event that Corlieve, its Representatives or any of its equityholders, including a Seller, receives, at any time between the date hereof and the Closing Date, a written notice or an inquiry from any Person (other than Purchaser and its Affiliates and their respective Representatives) regarding an Alternative Transaction, the Holder Representative will promptly notify Purchaser and provide such information regarding such Alternative Transaction as Purchaser reasonably requests.

6.6Consents and Approvals.

(a)Each of the parties agrees to use its reasonable efforts to take, or cause to be taken, all actions to file, or cause to be filed, all documents and to do, or cause to be done, all things necessary, proper or advisable to consummate the transactions contemplated by this Agreement, including preparing and filing as promptly as practicable all documentation to effect or obtain all necessary filings, consents, waivers, approvals, authorizations, permits, licenses or orders from all Governmental Authorities, including the French Ministry of Economy, Finance and Recovery (Ministère de l’Economie, des Finances et de la Relance) as provided in Section 8.1(a), or any other third party.

(b)With respect to the condition precedent set forth in Section 8.1(a), Purchaser hereby undertakes:

(i)to make all necessary notifications and filings with the relevant Governmental Authorities with respect to the transactions contemplated herein in order to obtain the Foreign Investment Approval as soon as reasonably possible and in compliance with Applicable Laws. Purchaser shall further respond as soon as reasonably possible to any requests received from any relevant Governmental Authority for additional documents or information;

(ii)to take all actions reasonably necessary under Applicable Law to obtain the Foreign Investment Approval. Notwithstanding the foregoing, if the competent Governmental Authority requests any material condition to the granting of the Foreign Investment Approval (other than, without limitation, any Acceptable Undertaking), such as Purchaser’s undertaking to divest, dispose of, or hold separate any of the businesses or assets of Corlieve or Purchaser and/or any of its Affiliates, then Purchaser  shall not be required to comply with any condition, obligation or other requirement imposed or contained in any decision by such relevant Governmental Authority;

(iii)to keep the Holder Representative regularly informed of the processing of the above filings and notifications in particular, if it becomes aware of any fact or event which could reasonably result in the Foreign Investment Approval being delayed or denied;

45

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

(iv)to provide the Holder Representative, as soon as reasonably practicable, with copies of all material relevant correspondence, documents or other communications received from or sent to the relevant Governmental Authority.

(v)to give notice to the Holder Representative of the obtaining of the Foreign Investment Approval within [*] of becoming aware of the same.

(c)For the purposes of the covenant contained in Section 6.6(b), the Sellers hereby undertake to provide, through the Holder Representative, and to cause Corlieve to provide, all assistance reasonably required by Purchaser in respect of the filings and notifications relating to the Foreign Investment Approval and any requests for information relating to Corlieve and/or the Sellers from the competent Governmental Authority.

(d)Further, and without limiting any other provision of this Section 6.6, each of the parties shall cooperate in all respects with each other in connection with any filing or submission and in connection with any investigation or other inquiry and shall promptly: (i) furnish on a confidential basis to the other such necessary information and reasonable assistance as the other parties may request in connection with the foregoing; (ii) inform the other of any communication from any Governmental Authority regarding transactions contemplated by this Agreement; and (iii) provide counsel for the other parties with copies of all filings made by such party, and all correspondence between such party (and its advisors) with any Governmental Authority and any other information supplied by such party and such party’s Affiliates to a Governmental Authority or received from a Governmental Authority in connection with the transactions contemplated by this Agreement; provided, however, that materials may be redacted as necessary to comply with contractual arrangements and with Applicable Law. Each party hereto shall, subject to Applicable Law, permit counsel for the other parties to review in advance, and consider in good faith the views of the other parties in connection with, any proposed written communication to any Governmental Authority in connection with the transactions contemplated by this Agreement. The parties agree not to participate, or to permit their Affiliates to participate, in any substantive meeting or discussion, either in person or by telephone, with any Governmental Authority in connection with the transactions contemplated hereby unless it consults with the other parties in advance and, to the extent not prohibited by such Governmental Authority, gives the other parties the opportunity to attend and participate. The parties further agree to execute or deliver any additional instruments necessary to consummate the transactions contemplated by, and to fully carry out the purposes of, this Agreement.

6.7Notification of Certain Matters. During the period from the Agreement Date until the earlier of the Closing and the termination of this Agreement, each party hereto shall give prompt notice to the other parties hereto of (a) the occurrence of any event that would cause any representation or warranty of such party in this Agreement to be materially untrue or inaccurate at or prior to the Closing Date, (b) any material failure of such party to comply with or satisfy its covenants, conditions, agreements and other obligations in this Agreement, (c) in the case of Corlieve, the occurrence of an event that has had or would reasonably be expected to have, individually or in the aggregate, a Material Adverse Effect, or (d) in the case of Purchaser, the occurrence of an event that would reasonably be expected to have a material adverse effect on the ability of Purchaser to consummate the transactions contemplated by this Agreement. The Holder Representative shall have the right (but not the obligation) to provide updates to the Disclosure Letter (each a “Disclosure Notification”) with respect to events, facts or circumstances (i) arising after the Agreement Date or (ii) that came to the Knowledge of Corlieve after the Agreement Date only for those representations and warranties that are qualified by any reference to the “Knowledge of the Sellers”. Except for the delivery of an updated Capitalization Table that reflects any changes to the Capitalization Table arising between the Agreement Date and the Closing Date, no Disclosure Notification shall be deemed incorporated into or to supplement and amend the Disclosure Letter for purposes of determining whether the condition with respect to Purchaser’s obligations under the Agreement set forth in Section 8.2(a) has been satisfied. Following the Closing, no claim for indemnification pursuant to Article 9 may be made by a Purchaser Indemnified Party with

46

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

respect to any matter included in a Disclosure Notification, any such claim being hereby irrevocably waived and released with respect to such matter.

6.8Waiver of Rights Under Shareholders Agreement. The Sellers hereby agree to waive as from the Closing Date any rights under the Shareholders Agreement that may be exercisable in connection with the transactions contemplated by this Agreement, including any preemptive rights. The Sellers hereby agree that any exercise of any such preemptive rights shall be null and void and that Purchaser may seek to enjoin the exercise of such preemptive rights pursuant to the provisions of this Agreement, including Section 11.12.

6.9No Directed Selling Efforts. Neither Purchaser nor any of its Affiliates nor any person acting on its or their behalf has engaged or will engage in any “directed selling efforts” (as defined in Regulation S) in connection with the offering of the Milestone Shares, and Purchaser has complied and will comply with the offering restrictions requirement of Regulation S.

6.10Update to the Disclosure Letter. No later than [*] before the Closing Date, Corlieve shall provide to the Seller an updated version of the Disclosure Letter, including possible facts and circumstances occurred after the date hereof, or with respect to representations and warranties qualified by Corlieve’s Knowledge, that came to the Knowledge of Corlieve after the Signing Date.

6.11Free Shares. In the event that Closing has not occurred by July 22, 2021, [*]  and Corlieve shall enter into agreements, prior to July 24, 2022 amending the vesting terms of certain Free Share awards on the terms mutually agreed among the Purchaser, [*] and Corlieve as of the date hereof.

ARTICLE 7

POST-CLOSING COVENANTS

7.1Transfer Taxes. Transfer Taxes payable in connection with the consummation of the transactions contemplated hereby, if any, shall be borne and paid by the Purchaser.

7.2Release by Sellers. Effective as of the Closing, each Seller, on behalf of itself and its Subsidiaries and Affiliates and each of their respective directors, officers, shareholders, managers, members, partners, principals, employees, agents, Representatives, heirs, predecessors, successors and assigns (each, a “Seller Releasing Party”), hereby (i) voluntarily and knowingly releases, remises, acquits and forever discharges Purchaser, Corlieve, any Person who controls (as such term is defined in Section 15 of the Securities Act) any of the foregoing, and their respective directors, officers, shareholders, managers, members, partners, principals, employees, Affiliates, agents, Representatives, heirs, predecessors, successors and assigns from any and all Damages, whether known or unknown, certain or speculative, asserted or unasserted, that any Seller Releasing Party may have had prior to, or has as of, the Closing or that arise in the future based on events occurring prior to or as of the Closing, including with respect to any rights contained in the Shareholders Agreement and including with respect to any liquidation right or preference any Seller may be entitled to under the Bylaws (the “Released Damages”); (ii) expressly waives any defense that the release provided under this Section 7.2 does not extend to Damages such Seller Releasing Party did not know or suspect to exist on the Closing Date; (iii) represents and warrants that none of the Released Damages of such Seller Releasing Party has been sold, assigned or transferred to any other Person; and (iv) agrees and covenants not to commence or cause to be commenced any Proceeding seeking Damages or remedies of any kind based on, related to or arising from the Released Damages; provided, however, that the Released Damages do not include any Damages by any of the Seller Releasing Parties related to (a) the payment of the Total Consideration subject to the terms and conditions of this Agreement, (b) with respect to RGX, any of its rights contained in the RGX License Agreement or (c) subject to the terms and conditions of this Agreement, the enforcement of, or the exercise of any rights and remedies, if any, under any provisions of this

47

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

Agreement, any exhibit to this Agreement or any document delivered in connection with this Agreement.

7.3Compliance with the Securities Act.

(a)Each Seller agrees to resell the Milestone Shares received pursuant to the transactions contemplated by this Agreement only (i) in accordance with the provisions of Regulation S, (ii) pursuant to registration under the Securities Act or (iii) pursuant to an available exemption from registration under the Securities Act.

(b)Each Seller agrees not to engage in hedging transactions with regard to the Milestone Shares unless such hedging transactions are made in compliance with the Securities Act.

7.4Registration Rights with Respect to Milestone Shares. If a Milestone Payment becomes due and Purchaser elects to make any portion of a Milestone Payment by issuing uniQure Ordinary Shares, Purchaser shall register such Milestone Shares for resale by the Sellers in accordance with the provisions of Exhibit C.

7.5Transfer of Milestone Shares. Purchaser shall refuse to register any transfer of Milestone Shares not made (a) in accordance with the provisions of Regulation S, (b) pursuant to registration under the Securities Act, or (c) pursuant to an available exemption from registration under the Securities Act.

7.6Closing Statement.  The Sellers shall ensure that all information reasonably requested by the Purchaser and necessary for the accurate and complete preparation of the Closing Statement described in Section 2.4(b) is provided or made available to the Purchaser no later than [*] following Closing.

7.7Restrictive Covenants.

(a)Non-Competition. Each of the Founders hereby irrevocably agrees that, during the Restricted Period, he or she shall not, directly or indirectly, as a proprietor, partner, stockholder, director, executive, employee, consultant, independent contractor, joint venturer, member, investor, lender or otherwise:

(i)in the case of [*], carry out in North America, Europe, Japan or China any research or development activities intended to treat epilepsy by directly inhibiting the expression of the kainate receptor subunits named GluK2 and/or GluK5 (the “Restricted Field”);

(ii)hold any shares, or have any other financial interest, in any commercial enterprise (other than up to [*] of the share capital of publicly listed companies) engaged in the Restricted Field;

(iii)employ or solicit any manager (dirigeant social), employee, agent or contractor (with respect to the latter two categories, in a manner that would have a detrimental impact on their relationship as agent or contractor of the other Party) of Corlieve or the Purchaser or any of its affiliates or of the Company, without the prior written consent of the Purchaser; or

(iv)participate in any research or development activities in the Restricted Field, other than pursuant to an agreement with Corlieve or the Purchaser.

(b)Exclusions. Notwithstanding the foregoing, outside the Restricted Field, the Scientific Founders, acting as public agents, shall be entitled to conduct any research activities, including:

48

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

(i)any research and development activities to develop any approach to treat epilepsy which is not based on the Grik2 Gene Sequence; and

(ii)any research and development activities on the Grik2 Gene Sequence which are not aimed at treating epilepsy.

(c)Non-Solicitation. Each of the Sellers (other than RGX), agrees that, during the Restricted Period, such Seller shall not (except on behalf of Purchaser, Corlieve or any of their Affiliates) knowingly, directly or indirectly, on his or her own behalf or on behalf of any other Person, solicit, employ or interfere with (or attempt to do any of the foregoing) any individual who either  is employed by Purchaser, Corlieve or any of their Subsidiaries at the time of such solicitation, employment, interference or attempt thereof in a manner that conflict with his employment by Purchaser, Corlieve or any of their Subsidiaries.

(d)Blue Pencil. If for any reason any court of competent jurisdiction shall find any provisions of this Section 7.7 (including the defined terms used herein) unreasonable in duration or geographic scope or otherwise, it is the intention of the Parties that such restrictions and prohibitions shall be modified by the court to be effective to the fullest extent allowed under applicable law in such jurisdiction. Each Seller acknowledges that the territorial and time limitations set forth in this Section 7.6 (including the defined terms used herein) are reasonable and properly required for the adequate protection of the business of Purchaser and its Affiliates, and each Seller hereby waives, to the extent permitted by law, any and all right to contest the validity of any provision of this Section 7.6 (including the defined terms used herein) on the ground of breadth of its geographic or service coverage or length of term or otherwise.

(e)Future Employers; Third Party Beneficiaries. Each of the Sellers (other than RGX) shall inform any future employer of the non-competition and non-solicitation restrictions to which he or she is subject and provide such employer with a copy thereof, prior to the commencement of that employment. Each Affiliate of Purchaser is an intended third party beneficiary of the non-competition and non-solicitation restrictions set forth in this Section 7.7 (including the defined terms used herein) and may enforce the terms of this section as if it was a party hereto.

7.8Within three (3) months after the Closing Date, the Purchaser may ask the assistance of  [*] and the Scientific Founders to comprehensively identify the Know-How and to determine whether items of Know How provided by RGX as specifically identified in the Purchaser’s request are Licensed Background Know-How or Licensed Foreground Know-How pursuant to the RGX License.  [*] and the Scientific Founders shall use reasonable efforts to respond to such request.

ARTICLE 8

CLOSING CONDITIONS

8.1Conditions Precedent to the Obligations of Each Party. The respective obligations of each party hereto to consummate the transactions contemplated by this Agreement shall be subject to the satisfaction at or prior to the Closing Date of the following conditions, any one of which may be waived on behalf of Purchaser by Purchaser or on behalf of the Sellers by the Holder Representative:

(a)Foreign Investment Clearance. The foreign investment notification has been made with the French Ministry of Economy, Finance and Recovery (Ministère de l’Economie, des Finances et de la Relance) pursuant to articles L.151-3 and R.151-1 and seq. of the French Code Monétaire et Financier, and in relation to said notification, (i) the French Ministry of Economy, Finance and Recovery (Ministère de l’Economie, des Finances et de la Relance) has provided notification that the transactions contemplated hereunder do not fall within its approval requirement pursuant to the above-mentioned legislation, (ii) the underlying approval has been granted or (iii) all appropriate waiting

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[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

periods (including any extensions thereof) have expired, lapsed or been waived (the “Foreign Investment Approval”).

(b)No Order. Without prejudice to the Foreign Investment Approval, no Governmental Authority of any competent jurisdiction shall have enacted, issued, promulgated, enforced or entered any statute, rule, regulation, executive order, decree, injunction or other Court Order which (i) is in effect and (ii) has the effect of otherwise prohibiting or preventing the consummation of the transactions contemplated hereby.

8.2Conditions Precedent to the Obligations of Purchaser. The obligation of Purchaser to effect the transactions contemplated by this Agreement shall be subject to the satisfaction at or prior to the Closing Date of the following conditions, any one of which may be waived in writing by Purchaser in its sole discretion:

(a)Representations and Warranties. The representations and warranties contained in Section 4.3, as such representations and warranties may be updated by the delivery of an updated Capitalization Table pursuant to Section 6.7, shall be true and correct in all respects except for de minimis inaccuracies as of the Agreement Date and the Closing Date. Each of the other representations and warranties contained in Article 3 and Article 4 of this Agreement shall be true and correct in all material respects as of the Agreement Date and the Closing Date (except that those representations and warranties which address matters only as of a particular date or range of dates shall be limited to the date or range of dates so specified). For purposes of the condition contained in this Section 8.2(a), the references contained in any representation or warranty contained in Article 3 and Article 4 of this Agreement to “Material Adverse Effect,” “material,” “in all material respects” or other materiality qualifications (or correlative terms), including as expressed in accounting concepts, shall be disregarded.

(b)Covenants. The Sellers shall have performed or complied in all material respects with all agreements and covenants required by this Agreement and the other Transaction Agreements to be performed or complied with at or prior to the Closing Date.

(c)Material Adverse Effect. During the period from the Agreement Date until Closing, no Material Adverse Effect shall have occurred, and no event shall have occurred that, individually or in the aggregate, with or without notice or the lapse of time, would reasonably be expected to result in a Material Adverse Effect.

(d)Equityholders. The Sellers having signed the Agreement as at the Agreement Date shall hold Shares representing 100% of the issued and outstanding Ordinary Shares of Corlieve and all said Sellers shall be effectively represented at Closing and transfer their Shares in accordance with this Agreement.

(e)Exercise of the options under the Collaboration Agreements.

(f)Delivery by the Holder Representative of an updated Schedule 2.7.

8.3Conditions Precedent to the Obligations of the Sellers. The obligations of the Sellers to consummate the transactions contemplated by this Agreement are subject to the fulfillment prior to or at the Closing Date of each of the following conditions, any one or more of which may be waived in writing by the Holder Representative in its sole discretion:

(a)Representations and Warranties. Each of the representations and warranties of Purchaser contained in Article 5 of this Agreement (i) that is qualified by any reference to “material,” “in all material respects” or other materiality qualifications (or correlative terms) shall be true and correct in all respects as of the Agreement Date and the Closing Date and (ii) that is not so qualified is

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[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

true and correct in all material respects as of the Agreement Date and the Closing Date (except that those representations and warranties which address matters only as of a particular date or range of dates shall be limited to the date or range of dates so specified).

(b)Covenants. Purchaser shall have performed or complied in all material respects with all agreements and covenants required by this Agreement and the other Transaction Agreements to be performed or complied with by it on or prior to the Closing Date.

(c)Material Adverse Effect. During the period from the Agreement Date until Closing, no event has occurred that would reasonably be expected to have a material adverse effect on the ability of Purchaser to consummate the transactions contemplated by this Agreement.

8.4Shareholder Meeting. One or more shareholders’ meetings of Corlieve shall be held immediately prior to the Closing Date (subject to the condition precedent of Closing) or immediately after the Closing Date in order to (i) issue the Shares corresponding to the Loan Conversion, (ii) acknowledge the resignation of the members of the Comité de Supervision (supervisory board) and of the President and (ii) appoint a new President and, at Purchaser’s option, new members of the Comité de Supervision (supervisory board).

ARTICLE 9

INDEMNIFICATION

9.1Survival.

(a)All covenants and agreements contained in this Agreement that by their nature are required to be performed by the Sellers by or prior to Closing shall expire and will no longer be required to be performed as of the Closing Date; provided, that Purchaser may make a claim with respect to such covenants for a period of twelve (12) months following the Closing Date.

(b)All covenants contained in this Agreement that by their nature are required to be performed after Closing by any Party hereto shall survive according to their respective terms.

(c)The representations and warranties contained in this Agreement (other than the Fundamental Representations and the Specified Representations) shall survive the Closing and continue in full force and effect for the twelve (12) month period immediately following the Closing Date.

(d)The Fundamental Representations shall survive the Closing and continue in full force and effect indefinitely.

(e)The Specified Representations shall survive the Closing and continue in full force and effect for a period of three (3) years thereafter.

(f)Any claim based on fraud or intentional misrepresentation shall survive the Closing and continue in full force and effect indefinitely.

(g)Immediately following the last day of each such survival period (the “Survival End Date”), such representations and warranties shall expire automatically. If written notice of a claim has been given in accordance with Section 9.3 prior to the expiration of the applicable representations, warranties, covenants or agreements, then the applicable representations, warranties, covenants or agreements shall survive as to such claim, until such claim has been finally resolved. Following the expiration of a representation, warranty, covenant or agreement, no claim, action, suit or Proceeding may be initiated by any Purchaser Indemnified Party or Seller Indemnified Party with respect thereto, regardless of any statute of limitations period that would otherwise apply. For the avoidance of doubt, if the Closing does not occur as a result of any condition precedent not being satisfied or waived, none

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and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

of the Sellers or Purchaser shall have any Liability whatsoever, including for breach of covenant or otherwise, except with respect to its breach of the obligations surviving termination of this Agreement as set forth in Section 10.3 or for its fraud or willful breach of this Agreement.

9.2Indemnification.

(a)Following and subject to the occurrence of the Closing, subject to the limitations described in Section 9.1 and Section 9.5, each Seller shall, severally but not jointly (sans solidarité), indemnify, defend, and hold harmless each of Purchaser and its Affiliates, their respective directors, officers and employees, and their respective heirs, successors and assigns (each, a “Purchaser Indemnified Party” and, collectively, the “Purchaser Indemnified Parties”), subject to the provisions of this Article 9, from and against any and all losses, costs, reasonable expenses, claims, damages, actions, suits, proceedings, hearings, investigations, charges, complaints, demands, injunctions, judgments, orders, decrees, rulings, directions, fines, deficiencies, amounts paid in settlement, Liabilities, Taxes, liens, and fees and court costs, including interest, penalties, and reasonable attorneys’, consultants’ and other professional fees and disbursements and reasonable expenses of investigation and enforcement of rights under this Agreement (collectively, “Damages”) incurred by such Purchaser Indemnified Party that arise out of or result from:

(i)any breach of any representation or warranty contained in Article 4 of this Agreement;

(ii)any breach of any covenant or obligation in this Agreement that the Sellers are to cause Corlieve to perform at or prior to the Closing;

(iii)any breach of any covenant or obligation in this Agreement to be performed by the Holder Representative;

(iv)any inaccuracies in the Closing Date Allocation Schedule; and

(v)any error by the Paying Agent in executing its responsibilities under the Paying Agent Agreement.

With respect to Damages referred to in Section 9.2(a), each Seller shall only be liable for the portion of such Damages corresponding to its Pro Rata Share.

(b)Following and subject to the occurrence of the Closing, subject to the limitations described in Section 9.1 and Section 9.5, each Seller, severally and not jointly (sans solidarité), shall indemnify, defend, and hold harmless the Purchaser Indemnified Parties from and against any and all Damages incurred by such Purchaser Indemnified Party that arise out of or relate to:

(i)any breach of any representation or warranty of such Seller contained in Article 3 of this Agreement;

(ii)any breach of any covenant or obligation in this Agreement to be performed by such Seller.

With respect to Damages referred to in Section 9.2(b), only the relevant breaching Seller shall be liable for the full amount of the relevant Damages and the other Sellers shall not be liable.

(c)Following and subject to the occurrence of the Closing, subject to the limitations described in Section 9.1 and Section 9.5, Purchaser shall indemnify, defend, and hold harmless the Sellers and each of their respective heirs and Affiliates (each, an “Seller Indemnified Party” and, collectively, the “Seller Indemnified Parties,” and, collectively with the Purchaser Indemnified Parties,

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[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

the “Covered Parties,” and each, a “Covered Party”) from and against any and all Damages incurred by such Seller Indemnified Party that arise out of or relate to

(i)any breach of any representation or warranty made by Purchaser in Article 5 of this Agreement or in any other Transaction Agreement; or

(ii)any breach of any covenant or obligation in this Agreement to be performed by Purchaser.

(d)For the avoidance of doubt, neither Party shall be liable under this Agreement and any Transaction Agreement in respect of any loss of profit, loss of revenue, loss of contract, loss of goodwill, loss of claim, indirect Damages, punitive Damages or consequential Damages, except to the extent such Damages are payable to a Third Party.

9.3Notice of Claims.

(a)Any Covered Party seeking indemnification hereunder shall, no later than [*] upon becoming aware of a potential claim for indemnification and in all cases prior to the relevant Survival End Date, give to Purchaser, in the case of a Seller Indemnified Party, or to the Holder Representative, in the case of a Purchaser Indemnified Party, (the “Indemnifying Party”) a bona fide notice describing in reasonable detail the facts giving rise to any claims for indemnification hereunder, together with an good faith estimate of the claimed amount and all supporting documents (a “Claim Notice”); provided, that failure to give such notice in accordance with the requirements of this Section 9.3(a) shall not affect such Covered Party’s right to indemnification hereunder except to the extent the Indemnifying Party shall have been materially prejudiced by such failure, in which case Sellers shall not be liable under this Article 9 for the portion of the relevant Damage resulting from such failure.

(b)Each Indemnifying Party to whom a Claim Notice is given shall respond to any Covered Party that has given a Claim Notice (a “Claim Response”) within [*] (the “Response Period”) after the date that the Claim Notice is given. Any Claim Response must either (i) agree to the amount or method of determination set forth in the Claim Notice and to pay such amount to (or on behalf of) such Covered Party by wire transfer of immediately available funds (or release of all or a portion of the Escrowed Amount to a Purchaser Indemnified Party) or (ii) to provide such Covered Party with notice that they disagree with the amount or method of determination set forth in the Claim Notice. If any Indemnifying Party fails to give a Claim Response within the Response Period, such Indemnifying Party shall be deemed not to dispute the claim described in the related Claims Notice. If any Indemnifying Party elects not to dispute a claim described in a Claims Notice, whether by failing to give a timely Claim Response or otherwise, then the amount of such claim shall be conclusively deemed to be an obligation of such Indemnifying Party.

(c)If an Indemnifying Party delivers a Claim Response disputing the claim of indemnification, the Covered Party that has given the Claim Notice and the Indemnifying Party shall attempt in good faith for a period of [*] after receipt of the Claim Response to resolve such objection. If the Indemnifying Party and the Covered Party shall so agree, a memorandum setting forth such agreement shall be prepared and signed by both parties, and shall be binding and conclusive upon the Indemnifying Party and the Covered Party. If no such agreement can be reached during the 30-day period for good faith negotiation, then upon the expiration of such 30-day period, either the Indemnifying Party or the Covered Party may bring suit in accordance with Section 11.10.

(d)Subject to the terms of the Escrow Agreement, if, pursuant to the procedures described in this Section 9.3, an Indemnifying Party is determined to be obligated to indemnify a Covered Party hereunder, the Indemnifying Party shall pay (or cause to be paid) to such Covered Party the amount to which such Covered Party shall be entitled within [*] after such amount has been finally determined in accordance with this Section 9.3. Subject to the provisions of this Article 9, the Purchaser Indemnified

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[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

Parties shall recover the amount of any Damages first from the Escrow Account, in which case Purchaser and the Holder Representative shall jointly instruct the Escrow Agent to distribute from the Escrow Account an amount of cash equal to the amount of any such Damages .

9.4Third Party Claims.

(a)If a Covered Party receives written notice of a claim from a Third Party that such Covered Party believes may result in a claim for indemnification under this Article 9 (a “Third Party Claim”), such Covered Party shall deliver a Claim Notice to the Indemnifying Party in accordance with the provisions of Section 9.3.

(b)So long as the Litigation Conditions are satisfied, the Indemnifying Party shall have the right to assume and control the defense of the Third Party Claim, at its own expense with counsel selected by it and reasonably acceptable to the Covered Party, by delivering written notice of its assumption of such defense to the Covered Party within [*] of its receipt of a Claim Notice of such Third Party Claim; provided, however, that the Covered Party shall have the right to retain its own counsel and assume and control the defense of such Third Party Claim, with the reasonable fees and expenses to be paid by the Indemnifying Party, if (i) the Covered Party receives advice from outside counsel that (A) representation of the Covered Party by the counsel retained by the Indemnifying Party would be inappropriate due to actual or reasonably anticipated potential conflicts of interests between such Covered Party and the Indemnifying Party or (B) there may be legal defenses or counterclaims available to the Covered Party that are inconsistent with, different from or in addition to those available to the Indemnifying Party, or (ii) at any time the Litigation Conditions are not satisfied with respect to such Third Party Claim. If the Indemnifying Party assumes and controls the defense of such Third Party Claim, the Indemnifying Party shall keep the Covered Party reasonably apprised of the status of the Third Party Claim (including by providing copies of all pleadings, notices and communications with respect to the Third Party Claim to the extent that receipt of such documents does not affect attorney-client privilege) and the Covered Party shall be entitled to participate in, but not determine or conduct, the defense of, and/or any settlement negotiations with respect to, such Third Party Claim at its sole cost and expense.

(c)If the Litigation Conditions are not satisfied, or if the Indemnifying Party does not assume the defense of the Third Party Claim as described in Section 9.4(b), the Covered Party shall assume and control the defense of such Third Party Claim at the expense of the Indemnifying Party and shall not settle or compromise the Third Party Claim without the prior written consent of the Indemnifying Party, such consent not to be unreasonably withheld, conditioned or delayed. If the Covered Party assumes and controls the defense of such Third Party Claim, the Covered Party shall keep the Indemnifying Party reasonably apprised of the status of the Third Party Claim (including by providing copies of all pleadings, notices and communications with respect to the Third Party Claim to the extent that receipt of such documents does not affect attorney-client privilege) and the Indemnifying Party shall be entitled to participate in, but not determine or conduct, the defense of, and/or any settlement negotiations with respect to, such Third Party Claim at its sole cost and expense.

(d)If the Indemnifying Party has assumed and controls the defense of the Third Party Claim in accordance with Section 9.4(b), then (i) the Covered Party shall not settle or compromise the Third Party Claim without the prior written consent of the Indemnifying Party, such consent not to be unreasonably withheld, conditioned or delayed and (ii) the Indemnifying Party shall not (A) settle or compromise the Third Party Claim or consent to the entry of any judgment which does not include an unconditional written release by the claimant or plaintiff of the Covered Party from all Liability in respect of such Third Party Claim, or (B) settle or compromise the Third Party Claim if the settlement imposes equitable remedies or material obligations on the Covered Party other than financial obligations for which such Covered Party will be indemnified hereunder, in each case, without the prior written consent of the Covered Party. In each case, the party that is not controlling the defense of the Third Party Claim shall reasonably cooperate with the party that is controlling the defense of such Third Party

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and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

Claim, at the non-controlling party’s expense, and shall make available to the controlling party all pertinent information under the control of the non-controlling party.

9.5Limitation on Indemnity. Notwithstanding anything to the contrary contained in this Agreement:

(a)No amount payable by either Purchaser or the Sellers pursuant to Section 9.2(a)(i), Section 9.2(b)(i) or Section 9.2(c)(i) shall become due until and unless the Damages in respect of any individual claim or series of related claims exceeds [*] (the “De Minimis Amount”); provided, however, that any series of related claims shall be considered as one single claim for the purposes of the De Minimis Amount; and provided, further, that the De Minimis Amount shall not apply to breaches of Fundamental Representations or with respect to fraud or intentional misrepresentation.

(b)No amount payable by either Purchaser or the Sellers pursuant to Section  9.2(a)(i), Section 9.2(b)(i) or Section 9.2(c)(i) shall become due until and unless the aggregate amount of all Damages shall exceed [*] (the “Threshold”), whereupon Purchaser or the Sellers, as applicable, shall be liable for the relevant Damages from the first Euro, provided however, that the Threshold shall not apply to breaches of Fundamental Representations or with respect to fraud or intentional misrepresentation. In calculating whether the Threshold has been exceeded, only individual claims or series of related claims in excess of the De Minimis Amount shall be considered.

(c)Subject to the remainder of this subparagraph (c), the maximum aggregate amount of Damages for which indemnity may be recovered by the Purchaser Indemnified Parties from the Sellers pursuant to Section 9.2(a)(i) and Section 9.2(b)(i) shall be an amount equal to the Escrowed Amount (the “Cap”). The Cap shall not apply to any claim by any Purchaser Indemnified Party against the Sellers pursuant to Section 9.2(a)(i) through 9.2(a)(v) or Section 9.2(b)(i) or Section 9.2(b)(ii), for breaches of Fundamental Representations, or with respect to fraud or intentional misrepresentation. Notwithstanding the foregoing, no Seller shall be liable for Damages pursuant to Section 9.2(a)(i) through 9.2(a)(v) or Section 9.2(b)(i) or Section 9.2(b)(ii) or for breaches of Fundamental Representations in an amount in excess of the Total Consideration which such Seller has actually received hereunder (subject to the right of Purchaser under Section 2.10 to set-off from part of the future Milestone Payments due to such Seller any Damages referred to in said Section  2.10 that a Purchaser Indemnified Party would be entitled to from such Seller but for this limitation). Further, notwithstanding the foregoing, the Cap shall not apply to any claim by any Purchaser Indemnified Party against the Sellers for breaches of Specified Representations; provided, however, that the only recourse of the Purchaser in respect of the amount of any such Damages in excess of the Cap shall be the right of Purchaser under Section  2.10 to set-off from part of the future Milestone Payments due to such Seller any Damages referred to in said Section  2.10 that a Purchaser Indemnified Party would be entitled to from such Seller in respect of such breach, up to a maximum amount equal to [*] of the total Milestone Payments potentially payable hereunder.

(d)The maximum aggregate amount of Damages for which indemnity may be recovered by the Seller Indemnified Parties from Purchaser pursuant to Section 9.2(c)(i) shall be an amount equal to the Escrowed Amount; provided, however, that this Section 9.5(d) shall not limit in any way Purchaser’s obligation to pay the Sellers the Closing Date Consideration, the Milestone Payments. Purchaser shall not be liable for Damages pursuant to Section 9.2(c)(ii) in an amount in excess of the Total Consideration Purchaser has actually paid hereunder.

(e)The right to indemnification or other remedy based on the representations, warranties, covenants, obligations and agreements herein will not be affected or deemed waived by reason of any investigation or audit made by or on behalf of any party (including by any of its Representatives) or by reason of the fact that such party or any of its Representatives knew or should have known that any such representation or warranty is, was or might be inaccurate, except when set forth in the Disclosure Letter.

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[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

(f)To the extent permitted by Applicable Law, any payment made by an Indemnifying Party to a Covered Party pursuant to this Article 9 shall be treated on the Parties’ Tax Returns and otherwise as an adjustment to the Total Consideration for all Tax purposes.

(g)For purposes of calculating the amount of Damages incurred out of or relating to any breach of a representation or warranty contained in Article 3, Article 4 or any Transaction Agreement delivered by any Seller or the Holder Representative, the references to “Material Adverse Effect,” “material,” “in all material respects” or other materiality qualifications (or correlative terms), including as expressed in accounting concepts, shall be disregarded.

(h)No Seller shall have, exercise or assert (or attempt to exercise or assert), any right of contribution, right of indemnity or other right or remedy against Purchaser or Corlieve, or any of their respective directors, officers, employees, Affiliates, agents, attorneys, Representatives, assigns or successors, for any indemnification claims asserted by any Purchaser Indemnified Parties in connection with any indemnification obligation or any other Liability to which such Seller may become subject under or in connection with this Agreement, it being acknowledged that the representations, warranties, covenants and agreements of the Sellers are solely for the benefit of the Purchaser Indemnified Parties.

9.6Sole Remedies. Subject to Section 9.5, if the Sellers shall be obligated to indemnify the Purchaser Indemnified Parties with respect to a claim for Damages pursuant to Section 9.2(a)(i), recovery and payment therefor shall be made first from the Escrowed Amount and then, to the extent that the then-remaining Escrowed Amount is insufficient to fully indemnify the Purchaser Indemnified Parties for such Damages, the Purchaser Indemnified Parties shall have the right to  set-off such Damages from part of the future Milestone Payments pursuant to Section 2.10. For the avoidance of doubt, the Purchaser shall have no right to seek recovery and payment of Damages directly against the Sellers, except in respect of Damages described in Section 9.2(b) or for breaches of Fundamental Representations by each relevant Seller.

9.7Treatment of Insurance. With respect to each claim for indemnification, any Damages that may be recovered by the Covered Party with respect to such claim shall be net of any insurance proceeds actually received with respect thereto (net of the out-of-pocket costs reasonably incurred in pursuing or obtaining such insurance proceeds, deductibles and any increased premium amounts directly attributable to such claim). To the extent that insurance proceeds are actually collected after an indemnification claim has been settled, the Covered Party shall restore the Indemnifying Party to the same economic position as would have existed had such insurance proceeds been collected prior to the settlement of such indemnification claim (net of the out-of-pocket costs reasonably incurred in pursuing or obtaining such insurance proceeds, deductibles and any increased premium amounts directly attributable to such claim).

9.8Tax reassessment. The Sellers shall not be liable under Article 9 in respect of any breach of the Sellers’ representations contained in Section 4.10 until such breach has resulted in (i) an enforceable decision (“titre exécutoire”) of the competent Tax Authority, or a competent court delivered against Corlieve, or (ii) a settlement agreement entered into between Corlieve and the Tax Authority, provided, however, that this Section 9.8 shall not have the effect of preventing the Purchaser from validly making a claim in respect of any Sellers’ representations contained in Section 4.10 within (where applicable) the respective time limits specified in Section 9.1, even though no such enforceable decision or settlement agreement is provided for yet.

9.9Tax Benefit. To the extent a Damage gives rise to a Tax benefit or saving in the form of an actual and effective reduction in the corporate tax paid by Corlieve or Purchaser with respect to the tax year during which such Damage is recorded in the accounts of Corlieve (including, without limitation, a Tax reduction or loss, basis adjustment and/or shifting income, deductions, gains, loss and/or credits but to the exclusion of any increase in the amount of available tax losses carried forward or carried back), the amount of the Damage shall be reduced by the amount of such actual and effective

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[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

reduction. In determining the amount of such Tax benefit or saving during the fiscal year in which the loss is recorded in the accounts of Corlieve, the impact of any allowance or cancellation of reserves by Corlieve shall be neutralized.

9.10Tax exclusions. The consequences from any Tax reassessment whose effect would merely be a shift of Tax from a fiscal year to the following fiscal year shall not be taken into account except for any interest or penalties resulting therefrom. The consequences from any increased liability to value added Tax will not be taken into account, except for any interest or penalties resulting therefrom, where such value added Tax can be (from a Tax and commercial perspective) deducted or recovered from third parties.

9.11Reserves. The amount of the indemnification/price reduction obligation of the Sellers with respect to any Damage shall be reduced by the amount of the reserves included in the Financial Statements to the extent such reserves are directly and specifically related to such Damage.

9.12Changes in Laws. The Sellers shall not be held liable for Damages directly and exclusively arising (i) from changes in Laws applicable to the operations of Corlieve not in force on or prior to the Closing Date, (ii) post-Closing reorganizations, or (iii) changes in accounting policies or procedures of Corlieve, Purchaser or its Affiliates applied after the Closing Date.

9.13Duty to Mitigate. Each Covered Party shall be responsible for taking or causing to be taken all commercially reasonable steps to mitigate its Damages upon and after becoming aware of any event or condition that could reasonably be expected to give rise to any Damages that are indemnifiable under this Article 9.

9.14Remedies. The remedies in this Article 9 shall be the sole and exclusive remedies of the Parties with respect to any breach of the respective representations, warranties, covenants, obligations and agreements pursuant to this Agreement or otherwise arising out of this Agreement, regardless of the theory or cause of action pled, except for the remedies of specific performance, injunction and other similar equitable relief; provided, however, that no party shall be deemed to have waived any rights, claims, causes of action or remedies, and the limitations set forth in Section 9.5 shall not limit any recovery related thereto, if and to the extent fraud or intentional misrepresentation is proven on the part of a party by another party or such rights, claims, causes of action or remedies may not be waived under Applicable Law; provided, further, that in the event fraud or intentional misrepresentation is proven, each Seller shall be liable for Damages under Section 9.2(b) in excess of the Cap only to the extent such Damages arise out of such fraud or intentional misrepresentation committed by such Seller.

9.15Duplication. Any liability for indemnification hereunder shall be calculated without duplication by reason of the same set of facts giving rise to such liability constituting a breach of more than one representation, warranty, covenant, obligation or agreement.

ARTICLE 10

TERMINATION

10.1Termination. This Agreement may be terminated at any time prior to the Closing Date:

(a)by mutual written consent of Purchaser and the Holder Representative;

(b)by either Purchaser or the Holder Representative upon written notice if the Closing shall not have occurred on or before October 30, 2021 (the “Outside Date”); provided, however, that the right to terminate this Agreement under this Section 10.1(b) shall not be available to any party

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and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

whose breach of any covenant or agreement hereunder caused, or resulted in, the failure of the Closing to occur on or before the Outside Date;

(c)by Purchaser, if (i) there is a breach of any representation, warranty, covenant or obligation of the Sellers such that the conditions set forth in Section 8.1(a) or Section 8.1(b) would not be satisfied; (ii) Purchaser shall have delivered to the Holder Representative a written notice of such breach; and (iii) at least [*] shall have elapsed since the delivery of such notice without such breach being cured; provided, however, that Purchaser shall have no right to terminate this Agreement pursuant to this Section 10.1(c) if Purchaser is in material breach of its representations and warranties under this Agreement or has failed in any material respect to perform its obligations under this Agreement;

(d)by the Holder Representative, if: (i) there is a breach of any representation, warranty, covenant or obligation of Purchaser such that the conditions set forth in Section 8.3(a) or Section 8.3(b) would not be satisfied; (ii) Holder Representative shall have delivered to Purchaser a written notice of such breach; and (iii) at least [*] shall have elapsed since the delivery of such notice without such breach being cured; provided, however, that the Holder Representative shall have no right to terminate this Agreement pursuant to this Section 10.1(d) if the Sellers are in material breach of their representations and warranties under this Agreement or have failed in any material respect to perform their obligations under this Agreement;

(e)by either Purchaser or the Holder Representative, if (i) a Governmental Authority of any competent jurisdiction shall have enacted, issued, promulgated, enforced or entered any Law or Governmental Order which (1) is in effect, (2) has the effect of permanently restraining, enjoining or otherwise prohibiting the Closing and (3) is final and non-appealable; or

(f)by Purchaser, in its sole discretion pursuant to Section 6.6(a), if the competent Governmental Authority requests any material condition to the granting of the Foreign Investment Approval, such as Purchaser’s undertaking to divest, dispose of, or hold separate any of the businesses or assets of Corlieve or Purchaser and/or any of its Affiliates, other than an Acceptable Undertaking; provided, however that, prior to so terminating this Agreement, the Purchaser shall first consult with the Holder Representative regarding such conditions for a period of at least [*].

10.2Notice of Termination. If Purchaser wishes to terminate this Agreement pursuant to Sections 10.1(b), 10.1(c), 10.1(e) or 10.1(f), Purchaser shall deliver to the Holder Representative a written notice stating that Purchaser is terminating this Agreement and setting forth a brief description of the basis on which Purchaser is terminating this Agreement. If the Holder Representative wishes to terminate this Agreement pursuant to Sections 10.1(b), 10.1(d) or 10.1(e),  the Holder Representative shall deliver to Purchaser a written notice stating that the Holder Representative is terminating this Agreement and setting forth a brief description of the basis on which the Holder Representative is terminating this Agreement. Except for any termination pursuant to Sections 10.1(c) or 10.1(d), which shall be effective in accordance with the terms thereof, any termination of this Agreement under Section 10.1 shall be effective immediately upon the delivery of a valid written notice of the terminating Party to the other Party as set forth in this Section 10.2.

10.3Effect of Termination. In the event of the termination of this Agreement as provided under Section 10.1 and Section 10.2, this Agreement shall be of no further force or effect and the parties hereto shall be relieved of their duties and obligations arising under this Agreement after the date of such termination and such termination shall be without Liability to Purchaser, Corlieve or the Sellers; provided, that Section 6.1, Section 10.4, Section 11.3, Section 11.10, Section 11.11 and this Section 10.3 shall survive the termination of this Agreement; and provided, further, that nothing herein shall relieve any party from Liability for (i) fraud or (ii) any willful breach of this Agreement.

10.4Expenses. Except as otherwise set forth herein, all fees and expenses incurred in connection with this Agreement and the transactions contemplated hereby, including fees and expenses

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and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

of financial advisors, legal counsel and other advisors, shall be paid by the party incurring such expenses whether or not the transactions contemplated hereby are consummated.

ARTICLE 11

MISCELLANEOUS

11.1Binding Effect; Assignment. No Party may, without the consent of the other Parties, assign or transfer any of its rights and obligations hereunder; provided that no such consent is required for an assignment or transfer by Purchaser, in whole or in part, to (i) a Subsidiary of Purchaser (and a Subsidiary of Purchaser may assign this Agreement to another Subsidiary of Purchaser or to Purchaser) or (ii) a successor-in-interest of Purchaser by reason of merger or consolidation or sale of all or substantially all of the assets of Purchaser relating to the subject matter of this Agreement; provided, that any shares that may be used to make a Milestone Payment must be either uniQure Ordinary Shares or shares of a successor-in-interest of Purchaser whose stock is publicly traded. Subject to the foregoing, this Agreement shall inure to the benefit of and be binding on the Parties’ successors and permitted assigns. Any assignment or transfer in violation of the foregoing shall be null and void ab initio, the assignee or transferee in any such assignment or transfer shall acquire no rights whatsoever, and the non-assigning, non-transferring Party shall not recognize, nor shall it be required to recognize, such assignment or transfer.

11.2Notices.

(a)All notices, deliveries and other communications pursuant to this Agreement will be in writing and will be deemed given if delivered personally, by facsimile, by email or delivered by globally recognized express delivery service to the parties at the addresses or facsimile numbers set forth below or to such other address or facsimile number as the party to whom notice is to be given may have furnished to the other Parties in writing in accordance herewith. Any such notice, delivery or communication will be deemed to have been delivered and received (i) in the case of personal delivery, on the date of such delivery, (ii) in the case of facsimile, on the Business Day that the party giving notice receives electronic confirmation of sending from the sending telecopy machine or, if the facsimile is not sent on a Business Day, the next Business Day after sending, (iii) in the case of email, on the day that the party giving notice receives electronic confirmation of sending from their email provider and (iv) in the case of a globally recognized express delivery service, on the Business Day that receipt by the addressee is confirmed pursuant to the service’s systems.

If to Purchaser:

uniQure N.V.

Attention: [*]

Paasheuvelweg 25A

1105 BP Amsterdam

Netherlands

Email: [*]

with copies (which shall not constitute notice) to:

uniQure, Inc.

Attention: [*]

113 Hartwell Avenue

Lexington, MA 02421

Email: [*]

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[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

Morgan Lewis UK LLP

Condor House, 5-10 St. Paul's Churchyard

London EC4M 8AL

United Kingdom

Attention: Timothy Corbett

Email: timothy.corbett@morganlewis.com

If to the Holder Representative, to:

Kurma Partners

24 Rue Royale

75008 Paris

France

Attn: [*]

Email: [*]

with copies (which shall not constitute notice) to:

McDermott Will & Emery AARPI

23 rue de l'Université

75007 Paris

France

Attention: Emmanuelle Trombe

Fax +33 1 81 69 15 15

Email: etrombe@mwe.com

If to a Seller, to the address set forth on the signature page of such Seller.

11.3Entire Agreement; Amendments and Waivers. This Agreement, the Confidentiality Agreement and the other documents and instruments referred to herein and all exhibits and schedules hereto, constitutes the entire agreement among the Parties pertaining to the subject matter hereof and supersedes all prior agreements, understandings, negotiations and discussions, whether oral or written, of the Parties. Any provision of this Agreement may be amended or waived if, but only if such amendment or waiver is in writing and is signed by Purchaser and the Holder Representative (it being acknowledged and agreed that the Holder Representative may amend this Agreement and waive matters on behalf of the Sellers, all as contemplated by Section 11.4 hereof). No waiver of any of the provisions of this Agreement shall be deemed or shall constitute a waiver of any other provision hereof (whether or not similar), nor shall such waiver constitute a continuing waiver unless otherwise expressly provided. No failure on the part of any party to exercise, and no delay in exercising, any right, power or remedy hereunder shall operate as a waiver thereof, nor shall any single or partial exercise of such right, power or remedy by such party preclude any other or further exercise thereof or the exercise of any other right, power or remedy.

11.4Appointment of the Holder Representative.

(a)Appointment. As used in this Agreement, the term “Holder Representative” shall mean that Person designated by the Sellers who together were entitled to receive a majority of the Closing Date Cash Consideration (including RGX) (a “Majority of Sellers”), who shall initially be Kurma Partners until Closing and as from Closing, a service provider as appointed pursuant to Section 11.4(b). By execution hereof, the Holder Representative hereby accepts its appointment as the initial Holder Representative. Effective upon the execution of this Agreement, without any further action by any other Person, the Holder Representative shall be appointed and constituted in respect of each Seller, as his, her or its agent, to act in his, her or its name, place and stead, as such Seller’s attorney-in-fact, as more fully set forth in Section 11.4(c).

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Securities Exchange Act of 1934, as amended.

(b)Election and Replacement. From and after the execution of this Agreement until the date when all obligations under this Agreement have been discharged (including all indemnification obligations under Article 9), a Majority of Sellers may, from time to time upon written notice to the Holder Representative and Purchaser, (i) remove any Holder Representative (including any Holder Representative appointed by Purchaser as provided below) or (ii) appoint a new Holder Representative to fill any vacancy created by the death, incapacitation, resignation or removal of any Holder Representative. If a Majority of Sellers is required to but has not appointed a successor Holder Representative to fill any vacancy within [*] after Purchaser has sent a written notice to a Majority of Sellers requesting that they appoint a successor Holder Representative, Purchaser shall have the right to appoint a Holder Representative to fill any such vacancy; provided, however, that a Majority of Sellers shall thereafter retain the right to remove the Holder Representative or appoint a new Holder Representative pursuant to this Section 11.4(b). A copy of any appointment by a Majority of Sellers of any successor Holder Representative shall be provided to Purchaser promptly after it shall have been effected. Each successor Holder Representative shall have all of the power, authority, rights and privileges conferred by this Agreement upon the original Holder Representative, and the term “Holder Representative” as used herein shall be deemed to include any successor Holder Representative.

(c)Authority. The Holder Representative shall be authorized, on behalf of each and every Seller, (i) to execute, as Holder Representative, this Agreement and any agreement or instrument entered into or delivered in connection with the transactions contemplated hereby (including, for the avoidance of doubt, the Disclosure Letter for and on behalf of the Sellers); (ii) to discuss, negotiate, agree to, enter into, consent to, resolve and fully and finally settle on behalf of the Sellers when it relates to all Sellers (but not individual Seller) any claims for indemnification by any Purchaser Indemnified Party under Article 9 hereof, including the authorization to comply with Court Orders with respect to any such claim for indemnification and the delivery to Purchaser of cash from the Escrow Account in satisfaction of claims asserted by Purchaser pursuant to Article 9 hereof; (iii) to object to such claims pursuant to Section 9.3; (iv) to act as, or retain a, Paying Agent for the Closing Date Cash Consideration and any post-closing distribution of the Holdback Amount, the Non GMP Batch Payment or the Milestone Payments; (v) to receive all documents, certificates and notices and make all determinations on behalf of the Sellers required under this Agreement.  A decision, act, consent or instruction of the Holder Representative in accordance with this Agreement shall constitute a decision of the Sellers and shall be final, binding and conclusive upon the Sellers. Purchaser shall have the right to rely in good faith upon an Instrument received from Holder Representative and to act in accordance with the Instrument without independent investigation. Any notice or communication given or received by, and any decision, action, failure to act within a designated period of time, agreement, consent, settlement, resolution or instruction of, the Holder Representative that is within the scope of the Holder Representative’s authority under Section 11.4 shall constitute a notice or communication to or by, or a decision, action, failure to act within a designated period of time, agreement, consent, settlement, resolution or instruction of all the Sellers and shall be final, binding and conclusive upon each such Seller.

(d)No Liability of Holder Representative or Purchaser. Neither the Holder Representative, nor any of the directors, officers, agents or employees of the Holder Representative, if applicable, shall be liable to any Seller or any other Person for any error of judgment, or any action taken, suffered or omitted to be taken, under this Agreement, except in the case of the Holder Representative’s fraud, gross negligence or willful misconduct. The Holder Representative may consult with legal counsel, independent public accountants and other experts selected by the Holder Representative and shall not be liable to any Seller for any action taken or omitted to be taken in good faith in accordance with the advice of such counsel, accountants or experts. As to any matters not expressly provided for in this Agreement, the Holder Representative shall not be required to exercise any discretion or take any action. Neither Purchaser, nor Corlieve nor any of their Affiliates shall have any Liability to any of the Sellers or otherwise arising out of the acts or omissions of the Holder Representative or any disputes among the Sellers or between the Sellers and the Holder Representative. Purchaser may rely entirely on its

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and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

dealings with, and notices to and from, the Holder Representative to satisfy any obligations it may have under this Agreement or otherwise to the Sellers.

(e)Indemnity; Costs and Expenses. Each Seller shall, severally and not jointly (sans solidarité), in proportion to the portion of the Closing Date Cash Consideration then previously received by such Sellers (or if no portion of the Closing Date Cash Consideration has been paid, in proportion to the portion of the Closing Date Cash Consideration to which such Seller is entitled), indemnify, hold harmless and defend the Holder Representative (and its directors, officers, employees, shareholders, agent and representatives) against any Damages incurred without fraud, gross negligence or willful misconduct by the Holder Representative and arising out of or in connection with the acceptance, performance or administration of the Holder Representative’s duties under this Agreement. Any Damages (including costs of defending claims prior to the final adjudication or settlement of such claims) incurred by the Holder Representative in connection with the acceptance, performance and administration of its duties as the Holder Representative pursuant to this Agreement (including the hiring of legal counsel, accountants or auditors and other advisors pursuant to the terms of this Agreement, but excluding any of the foregoing arising out of the Holder Representative’s fraud, gross negligence or willful misconduct) and all fees payable hereunder to the Holder Representative by the Sellers (“Holder Representative’s Costs”) shall be paid as follows: (i) first by recourse to the Holder Representative’s Fund, if any; and (ii) if such amounts are insufficient to pay such Holder Representative’s Costs, then by recourse directly to the Sellers (in proportion to the portion of the Closing Date Cash Consideration then previously received by each such Seller (or if no portion of the Closing Date Cash Consideration has been paid, in proportion to the portion of the Closing Date Cash Consideration to which such Seller is entitled).

(f)Holder Representative’s Fund. At the Closing, the Paying Agent may deposit with the Holder Representative an amount to be determined in the reasonable discretion of the Sellers, which shall be held by the Holder Representative in trust solely for the purpose of paying the expenses, if any, incurred by the Holder Representative in connection with the transactions contemplated by this Agreement (such fund created by the provision of such amount, the “Holder Representative’s Fund”). Upon request of all Sellers, the Holder Representative shall distribute the remaining Holder Representative’s Fund (if any) to the Sellers, in accordance with the Closing Date Allocation Schedule. Purchaser shall have no obligation whatsoever with respect to the Holder Representative’s Fund, if any, and Purchaser shall not have Liability for the manner in which the Holder Representative administers the Holder Representative’s Fund, or for causing or ensuring that all or any portion of the Holder Representative’s Fund is ultimately paid or distributed to the Sellers.

11.5Counterparts; Electronic Delivery of Signatures. This Agreement may be executed (including by facsimile or by email of a .pdf attachment) in one or more counterparts, each of which shall be deemed an original, but all of which together shall constitute one and the same instrument, it being understood that all Parties need not sign the same counterpart. In the event that any signature is delivered by facsimile or by email of a .pdf attachment, such signature shall create a valid and binding obligation of the party executing (or on whose behalf such signature is executed) with the same force and effect as if such facsimile or .pdf signature page were an original thereof.

11.6Severability. If any term or other provision of this Agreement is deemed or held to be invalid, illegal, or unenforceable in any respect, all other terms or provisions of this Agreement shall nevertheless remain in full force and effect so long as the economic or legal substance of the Agreement is not affected in any manner materially adverse to any party. Upon such determination that any term or other provision is invalid, illegal, or unenforceable in any respect, the parties hereto shall negotiate in good faith to modify this Agreement so as to effect the original intent of the parties as closely as possible in an acceptable manner. If it is ever held that any restriction hereunder is too broad to permit enforcement of such restriction to its fullest extent, each party agrees that a court of competent jurisdiction may enforce such restriction to the maximum extent permitted by Law, and each party

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and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

hereby consents and agrees that such scope may be judicially modified accordingly in any proceeding brought to enforce such restriction.

11.7Third Party Beneficiaries. Other than as provided in Section 7.7 and Article 9, nothing expressed or referred to in this Agreement will be construed to give any Person other than the Parties to this Agreement (and their successors and permitted assigns) any legal or equitable right, remedy, or claim under or with respect to this Agreement or any provision of this Agreement.

11.8No Strict Construction. The Parties have participated jointly in the negotiation and drafting of this Agreement. In the event an ambiguity or question of intent or interpretation arises, this Agreement shall be construed as if drafted jointly by the Parties, and no presumption or burden of proof shall arise favoring or disfavoring any party by virtue of the authorship of any of the provisions of this Agreement.

11.9GOVERNING LAW. THIS AGREEMENT SHALL BE GOVERNED BY AND CONSTRUED AND INTERPRETED IN ACCORDANCE WITH THE INTERNAL LAWS OF THE STATE OF NEW YORK, IRRESPECTIVE OF THE CHOICE OF LAWS PRINCIPLES OF THE STATE OF NEW YORK, AS TO ALL OTHER MATTERS, INCLUDING MATTERS OF VALIDITY, CONSTRUCTION, ENFORCEABILITY, PERFORMANCE AND REMEDIES.

11.10Jurisdiction; Venue; Service of Process. Except as otherwise provided in Section 11.12, all disputes arising out of or in connection with this Agreement shall be finally settled under the Rules of Arbitration of the International Chamber of Commerce by three arbitrators. The claimant shall nominate an arbitrator in its request for arbitration. The respondent shall nominate an arbitrator in its answer. The two arbitrators shall nominate a third arbitrator [*] after the nomination of the second arbitrator. The third arbitrator shall act as president of the tribunal. If any of the three arbitrators is not nominated within the time limits prescribed above, then the International Court of Arbitration of the International Chamber of Commerce shall appoint that arbitrator. The place of arbitration shall be Geneva Switzerland. The language of the arbitration shall be English. The arbitration award shall be final and binding on the Parties. The Parties undertake to carry out any award without delay and waive their right to any form of recourse based on grounds other than those contained in the United Nations Convention on the Recognition and Enforcement of Foreign Arbitral Awards of 1958 insofar as such waiver can validly be made. Judgment upon the award may be entered by any court having jurisdiction thereof or having jurisdiction over the relevant Party or its assets.

11.11WAIVER OF JURY TRIAL. EACH PARTY HERETO ACKNOWLEDGES AND AGREES THAT ANY CONTROVERSY WHICH MAY ARISE UNDER THIS AGREEMENT IS LIKELY TO INVOLVE COMPLICATED AND DIFFICULT ISSUES AND, THEREFORE, EACH SUCH PARTY HEREBY IRREVOCABLY AND UNCONDITIONALLY WAIVES TO THE FULLEST EXTENT PERMITTED BY APPLICABLE LAW, ANY RIGHT SUCH PARTY MAY HAVE TO A TRIAL BY JURY IN RESPECT OF ANY PROCEEDING DIRECTLY OR INDIRECTLY ARISING OUT OF, UNDER OR IN CONNECTION WITH OR RELATING TO THIS AGREEMENT OR THE TRANSACTIONS CONTEMPLATED BY THIS AGREEMENT. EACH PARTY HERETO CERTIFIES AND ACKNOWLEDGES THAT (A) NO REPRESENTATIVE OF ANY OTHER PARTY HERETO HAS REPRESENTED, EXPRESSLY OR OTHERWISE, THAT SUCH OTHER PARTY WOULD NOT, IN THE EVENT OF SUCH PROCEEDING, SEEK TO ENFORCE THE FOREGOING WAIVER, (B) EACH SUCH PARTY UNDERSTANDS AND HAS CONSIDERED THE IMPLICATIONS OF THIS WAIVER, (C) EACH SUCH PARTY MAKES THIS WAIVER VOLUNTARILY, AND (D) EACH SUCH PARTY HAS BEEN INDUCED TO ENTER INTO THIS AGREEMENT BY, AMONG OTHER THINGS, THE MUTUAL WAIVERS AND CERTIFICATIONS IN THIS SECTION 11.11.

11.12Injunctive Relief; Specific Performance. The Parties agree that irreparable damage would occur in the event that any of the provisions of this Agreement, including the restrictive covenants

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and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

contained in Section 7.7, were not performed in accordance with their specific terms or were otherwise breached, and that money damages or legal remedies would not be an adequate remedy for any such damages. Therefore, it is accordingly agreed that the Parties shall be entitled to an injunction or injunctions to prevent or restrain any breach or threatened breach of this Agreement, including Section 7.7, by any other Party and to enforce specifically the terms and provisions of this Agreement, including Section 7.7, to prevent breaches or threatened breaches of, or to enforce compliance with, the covenants and obligations of any other Party in any court of competent jurisdiction, in addition to any and all other rights and remedies at law or in equity. Each of the Parties hereto hereby waives (i) the defense that monetary damages would be an adequate remedy in any action for specific performance or any other form of equitable relief and (ii) any requirement under any Law to post a bond or any other security as a prerequisite to obtaining equitable relief.

11.13Headings. The descriptive headings used in this Agreement have been inserted for convenience of reference only, and are not intended to describe, interpret, define or limit the scope, extent or intent of this Agreement or any provision hereof.

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[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

EXHIBIT A: THE SELLERS [*]

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[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

EXHIBIT B: THE FREE SHARES HOLDERS [*]

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and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

EXHIBIT C: RESALE PROSPECTUS SUPPLEMENT

1. Registrable Securities. For purposes of this Agreement, the term “Registrable Securities” shall mean all of the Milestone Shares actually issued to Sellers under this Agreement, other than Milestone Shares issued to a Seller that has not provided the information required by Paragraph 7 of this Exhibit C at least one Business Day prior to the filing of the applicable Resale Registration Statement.

2. Resale Registration Statement. Purchaser agrees that it will file a prospectus supplement to Purchaser’s registration statement on Form S-3ASR filed on March 1, 2021 (File No. 333-253749) (or another registration statement on Form S-3, or such other form under the Securities Act then available to Purchaser), providing for the resale pursuant to Rule 415 from time to time, and on a continuing basis, by each holder thereof (each a “Holder”), of the Registrable Securities (such registration statement, including the prospectus, any pre-effective or post-effective amendments and supplements thereto, all exhibits thereto and all material incorporated by reference or deemed to be incorporated by reference, if any, therein being hereinafter referred to as a “Resale Registration Statement” and such prospectus supplement, including the base prospectus included in a Resale Registration Statement, a “Resale Prospectus Supplement”). Purchaser agrees to file a Resale Prospectus Supplement in respect of any Milestone Shares within [*] after the issuance thereof; provided, however, that Purchaser will be permitted to postpone or suspend (upon written notice to the Holders) the filing or use of a Resale Prospectus Supplement or a Resale Registration Statement if the disclosure requirements of the Securities Act in connection with such Resale Registration Statement would require Purchaser to include material non-public information (including information to supplement, update or correct existing disclosures) that has not theretofore been included or incorporated by reference in such Resale Registration Statement or otherwise in the public domain and Purchaser’s Board of Directors has determined in its reasonable judgment that Purchaser has a bona fide business reason not to disclose such material non-public information; provided, that the aggregate number of days Purchaser shall be permitted to so postpone or suspend the use of each such Resale Prospectus Supplement or effectiveness of each such Resale Registration Statement shall not exceed [*] or an aggregate of [*] days in any period of [*].

3. Withdrawal. Any Holder on behalf of whom any Registrable Securities have been included in a Resale Prospectus Supplement shall have the right to withdraw any or all of the Registrable Securities to be registered thereby by giving written notice to such effect to Purchaser prior to the filing of such Resale Prospectus Supplement.

4. Registration Procedures. In connection with a Resale Registration Statement, Purchaser shall:

(a) use reasonable best efforts to cause the registration statement to remain effective until the termination of the Purchaser’s registration obligations pursuant to Paragraph 6 of this Exhibit C, and shall use reasonable best efforts to obtain as promptly as is practicable the withdrawal of any order suspending the registration or qualification (or the effectiveness thereof) or suspending or preventing the use of any related prospectus in any jurisdiction with respect thereto;

(b) notify as promptly each seller of Registrable Securities of each of (A) the filing of the Resale Prospectus Supplement and any amendment or supplements thereto, (B) the receipt of any comments from the SEC or any state securities law authorities or any other Governmental Authorities with respect to any such registration statement or Resale Prospectus Supplement or any amendments or supplements thereto, and (C) any oral or written stop order with respect to such registration, any suspension of the registration or qualification of the sale of such Registrable Securities in any jurisdiction, or any initiation or threatening of any proceedings with respect to any of the foregoing; provided that Purchaser shall not be required to notify sellers of Registrable Securities of any events

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and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

described in (A) to (C) of this Paragraph 4 related to any Shelf Registration Statement that do not affect the availability of the applicable Resale Prospectus Supplement;

(c)if requested, furnish to each seller of Registrable Securities, the underwriters, and the sales or placement agent, if any, and counsel for each of the foregoing, a conformed copy of such registration statement and each amendment and supplement thereto (in each case, including all exhibits thereto and documents incorporated by reference therein) and such additional number of copies of such registration statement, each amendment, and supplement thereto (in such case without such exhibits and documents), the prospectus (including each preliminary prospectus) included in such registration statement, and the Resale Prospectus Supplements and all exhibits thereto and documents incorporated by reference therein, and such other documents as such seller, underwriter, agent, or counsel may reasonably request in order to facilitate the disposition of the Registrable Securities owned by such seller; provided, that Purchaser shall have no obligation to provide any document pursuant to this section that is available on the SEC’s EDGAR system;

(d)use reasonable best efforts to register or qualify such Registrable Securities under such securities or “blue sky” laws of such jurisdictions as the Holders of Registrable Securities reasonably request and do any and all other acts and things that may be reasonably necessary or advisable to enable the holders of Registrable Securities to consummate the disposition in such jurisdictions of the Registrable Securities owned by such Holders and keep such registration or qualification in effect for so long as the registration statement remains effective under the Securities Act (provided that Purchaser shall not be required to (x) qualify generally to do business in any jurisdiction in which it would not otherwise be required to qualify but for this subparagraph, (y) subject itself to taxation in any such jurisdiction in which it would not otherwise be subject to taxation but for this subparagraph, or (z) consent to the general service of process in any jurisdiction in which it would not otherwise be subject to general service of process but for this subparagraph);

(e)notify each seller of such Registrable Securities, at any time when a prospectus relating thereto is required to be delivered under the Securities Act and the rules and regulations thereunder, upon the discovery that, or of the happening of any event as a result of which, the registration statement covering such Registrable Securities, as then in effect, contains an untrue statement of a material fact or omits to state any material fact required to be stated therein or any fact necessary to make the statements therein not misleading, and as promptly as reasonably practicable prepare and furnish to each such seller a supplement or amendment to the Resale Prospectus Supplement to such registration statement so that such registration statement shall not, and such Resale Prospectus Supplement as thereafter delivered to the purchasers of such Registrable Securities shall not, contain an untrue statement of a material fact or omit to state any material fact required to be stated therein or any fact necessary to make the statements therein not misleading;

(f) use reasonable best efforts to cause all Registrable Securities so registered to be listed on The Nasdaq Global Select Market or such other national securities exchange or established over-the-counter market as is then the principal market on which or through which similar securities of Purchaser are then listed or traded; and

(g) use reasonable best efforts to comply with all applicable laws related to such registration statement and offering and sale of securities and all applicable rules and regulations of Governmental Authorities in connection therewith (including, without limitation, the Securities Act and the Exchange Act).

5. Expenses. All expenses incident to Purchaser’s performance of, or compliance with, its obligations under this Exhibit C, including, without limitation, all registration and filing fees, all fees and expenses of compliance with securities and “blue sky” laws, all printing and copying expenses, all messenger and delivery expenses, all fees and expenses of Purchaser’s independent counsel  shall be borne by Purchaser. Purchaser shall not be responsible for the fees and expenses of any counsel, or any of the accountants, agents, or experts retained by the Holders in connection with the sale of Registrable Securities. Purchaser will pay its internal expenses (including, without limitation, all salaries and expenses of its officers and employees performing legal or accounting duties, the expense of any annual

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[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

audit, and the expense of any liability insurance) and the expenses and fees for listing the securities to be registered on each securities exchange and included in each established over-the-counter market on which similar securities issued by Purchaser are then listed or traded or for listing on any other exchange or automated quotation system.

6. Termination. Purchaser’s obligations under Section 2.12 and Section 7.4 of the Agreement and this Exhibit C shall terminate, with respect to each Holder, on the earliest date on which all of such Holder’s Registrable Securities have: (a) been registered on a Resale Registration Statement and disposed of in accordance therewith; (b) become eligible to be sold, without volume or manner of sale limitations, pursuant to Rule 144 under the Securities Act or any successor rule or regulation thereto that may be adopted by the SEC, (c) ceased to be outstanding, whether as a result of redemption, repurchase, cancellation, exchange or otherwise, or (d) been sold to the public pursuant to Rule 144 under the Securities Act or any successor rule or regulation thereto that may be adopted by the SEC. Purchaser will take such action as may reasonably requested by any Holder to remove any restrictive legend or other restriction on resale with respect to any Registrable Securities for which Purchaser’s obligations under this Schedule shall have terminated pursuant to this subparagraph.

7. Selling Shareholder Information. Each of the Holders shall furnish such information as may reasonably be required by Purchaser with respect to itself and its Registrable Securities to be included in a Resale Prospectus Supplement. In connection therewith, each Holder shall be required to represent to Purchaser that all such information which is given is both complete and accurate in all material respects when made.

8. Adjustments.

(a)In the event that Purchaser is prohibited from filing a Resale Prospectus Supplement in respect of the Milestone Shares only as a result of a suspension period outlined in Section 2 within [*] following the issuance of such Milestone Shares and files the Resale Prospectus Supplement more than [*] following such issuance date [*] following the issuance date, the “Target Date” with respect to such Milestone Shares), the date on which such Resale Prospectus Supplement is actually filed and available for use shall be the “Measurement Date” with respect to such Milestone Shares, and the provisions of this Paragraph 8 shall apply.

(b)If the closing price of the uniQure Ordinary Shares on Nasdaq on the Trading Day immediately preceding the applicable Measurement Date is more than [*] lower than such price on the Trading Day immediately preceding the applicable Target Date, Purchaser shall pay to the Sellers, as additional consideration, an amount equal to such difference, in cash or additional uniQure Ordinary Shares, at the discretion of Purchaser. Such additional consideration shall be paid or delivered by no later than [*] following the Measurement Date and shall not be subject to the market restrictions of Sections 9 and 10 below.

69

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

EXHIBIT D: FORM OF FRENCH SHORT FORM TRANSFER AGREEMENT

[Signature Pages Follow]

70

[*] = Certain confidential information contained in this document, marked by brackets, has been omitted
and filed separately with the Securities and Exchange Commission pursuant to Rule 24b-2 of the
Securities Exchange Act of 1934, as amended.

IN WITNESS WHEREOF, each party has executed this Agreement or caused this Agreement to be duly executed on its behalf by its officer thereunto duly authorized, all as of the date first above written.

uniQure N.V.
	/s/ Matthew Kapusta
By:	Matthew Kapusta
Title:	Chief Executive Officer
SELLERS
	[*]
HOLDER REPRESENTATIVE
	[*] on behalf of Kurma Partners

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EX-3.1

HOLDINGA MATTHIJSSEN KRAAK

Exhibit 3.1

NOTE ABOUT TRANSLATION:

This document is an English translation of a document prepared in Dutch. In preparing this document, an attempt has been made to translate as literally as possible without jeopardizing the overall continuity of the text. Inevitably, however, differences may occur in translation and if they do, the Dutch text will govern by law.

In this translation, Dutch legal concepts are expressed in English terms and not in their original Dutch terms. The concepts concerned may not be identical to concepts described by the English terms as such terms may be understood under the laws of other jurisdictions.

ARTICLES OF ASSOCIATION OF

"uniQure N. V."

as these read after the execution of the deed of amendment of the articles of association executed on 16 June 2021 before C. Holdinga, civil-law notary in Amsterdam.

1. DEFINITIONS.

In the articles of association the following terms shall have the meaning as defined below:

Annual Accounts: the annual accounts referred to in section 2:361 DCC;
Annual Statement of Accounts: the Annual Accounts and, if applicable, the Annual Report as well as the additional information referred to in section 2:392 DCC;

Board: the corporate body of the Company consisting of the Executive Directors of the board in office and the Non-Executive Directors of the board in office;

Board Members: the Executive Directors of the Board in office and the Non-Executive Directors of the Board in office;

Chief Executive Officer: the Executive Director appointed as chief executive officer as referred to in article 7.3.;

Company: the public limited company which organisation is laid down in these articles of association;

Executive Director: a Board member appointed as executive director;

DCC: the Dutch Civil Code;

General Meeting: the corporate body that consists of Shareholders entitled to vote and all other persons entitled to vote/ the meeting in which Shareholders and all other persons entitled to attend general meetings assemble;

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Management Report: the annual report referred to in section 2:391 DCC;

Meeting Rights: the right to, either in person or by proxy authorised in writing, attend the General Meeting and to address such meeting;

Non-Executive Director: a Board member appointed as non-executive director;

Persons entitled to attend General Meetings: Shareholders as well as holders of a right of use and enjoyment (vruchtgebruik) and holders of a right of pledge with Meeting Rights;

Persons entitled to vote: Shareholders with voting rights as well as holders of a right of use and enjoyment (vruchtgebruik) and holders of a right of pledge with voting rights;

Secretary: the secretary of the Company as referred to in article 7.8.;

Share: a share in the share capital of the Company;

Shareholder: a holder of a Share;

Subsidiary: a subsidiary as referred to in section 2:24a DCC.

2. NAME. CORPORATE SEAT.

2.1. The name of the Company is: uniQure N.V.

Its corporate seat is in Amsterdam, the Netherlands, and it may establish branch offices elsewhere.

2.2. Objects.

The objects of the Company are:

(a) to research, develop, produce and commercialise products, services and technology in the(bio-)pharmaceutical sphere;

(b) to incorporate, participate in, conduct the management of and take any other financial interest in other companies and enterprises;

(c) to render administrative, technical, financial, economic or managerial services to other companies, persons or enterprises;

(d) to acquire, dispose of manage and exploit real and personal property, including patents, marks, licenses, permits and other intellectual property rights;

(e) to borrow and/or lend moneys, act as surety or guarantor in any other manner, and bind itself jointly and severally or otherwise in addition to or on behalf of others,

the foregoing, whether or not in collaboration with third parties, and inclusive of the performance and promotion of all activities which directly and indirectly relate to those objects, all this in the broadest sense.

3. SHARE STRUCTURE.

3.1. Authorised share capital

3.1.1. The authorised share capital of the Company amounts to three million euro(EUR 4.000.000,00) and is divided into sixty million (80,000,000) shares, each with a nominal value of five cent(€ 0.05).

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HOLDINGA MATTHIJSSEN KRAAK

3.1.2. The Shares shall be in registered form and shall be consecutively numbered from 1 onwards.

3.1.3. No share certificates shall be issued.

3.2. Issue of Shares.

3.2.1. Shares shall be issued pursuant to a resolution of the Board if by resolution of the General Meeting the Board has been authorised for a specific period not exceeding five (5) years to issue Shares. The resolution granting the aforesaid authorisation must determine the number and class of the Shares that may be issued. The authorisation may from time to time be extended for a period not exceeding five (5) years. Unless otherwise stipulated at its grant, the authorisation cannot be withdrawn.

3.2.2. If and insofar as an authorisation as referred to in article 3.2.1 is not in force, the General Meeting shall have the power, upon the proposal of the Board to resolve to issue Shares.

3.2.3. Article 3.2.1 and 3.2.2 shall equally apply to a grant of rights to subscribe for Shares, but shall not apply to an issue of Shares to a person who exercises a previously acquired right to subscribe for Shares.

3.2.4. Save for the provisions of section 2:80 DCC, the issue price may not be below nominal value of the Shares.

3.2.5. Shares shall be issued by deed in accordance with the provisions of sections 2:86c and 2:96 DCC.

3.3. Payment for Shares.

3.3.1. Shares may only be issued against payment in full of the amount at which such Shares are issued and with due observance of the provisions of sections 2:80a and 2:80b DCC.

3.3.2. Payment must be made in cash, unless an alternative contribution has been agreed. Payment other than in cash is made with due observance of the provisions of section 2:94b DCC.

3.3.3. Payment in cash may be made in a foreign currency if the Company agrees to this. In that case, the payment obligation shall be fulfilled for the amount up to which the amount paid up can be freely exchanged into euro. This rate of exchange shall be determined by the rate of exchange prevailing on the day of payment or, after application of the provisions of the next sentence, on the day referred to there. The Company may demand payment at the rate of exchange prevailing on a specific day within two (2) months prior to the last day on which payment must have been made, provided that the Shares shall be included on the official list of any stock exchange immediately following the issue.

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3.3.4. The Company may grant loans for the purpose of a subscription for or an acquisition of Shares in its share capital subject to any applicable statutory provisions.

3.3.5. The Board may perform legal acts as referred to in section 2:94 DCC without the prior approval of the General Meeting.

3.4. Pre-emptive rights.

3.4.1. Upon the issue of Shares, each Shareholder shall have a pre-emptive right to acquire such newly issued Shares in proportion to the aggregate amount of his Shares, it being understood that this pre­emptive right shall not apply to:

(a) any issue of Shares to employees of the Company or employees of a group Company;

(b) Shares which are issued against payment in kind.

3.4.2. Pre-emptive rights may be limited or excluded by resolution of the General Meeting upon proposal of the Board. The Board shall have the power to resolve upon the limitation or exclusion of the pre-emptive right, if and to the extent the Board has been designated by the General Meeting. Such designation shall only be valid for a specific period of not more than five (5) years and may from time to time be extended with a period of not more than five (5) years. Unless provided otherwise in the designation, the designation cannot be cancelled.

A resolution of the General Meeting to limit or exclude the pre­emptive rights as well as a resolution to designate the Board as referred to in this article 3.4.2 requires a two thirds majority of the votes cast if less than half the issued share capital is represented at a meeting.

3.4.3. Without prejudice to section 2:96a DCC, the General Meeting or the Board, as the case may be, shall, when adopting a resolution to issue Shares, determine the manner in which and the period within which such pre-emptive rights may be exercised.

3.4.4. The Company shall announce the issue with pre-emptive rights and the period within which such rights can be exercised in such manner as shall be prescribed by applicable law and applicable stock exchange regulations, including, but not limited to, an announcement published by electronic means of communication.

3.4.5. This article 3.4 shall equally apply to a grant of rights to subscribe for Shares, but shall not apply to an issue of Shares to a person who exercises a previously acquired right to subscribe for Shares.

3.5. Depositary receipts for shares

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HOLDINGA MATTHIJSSEN KRAAK

The Company is not authorised to cooperate in the issue of depositary receipts for Shares.

4. OWN SHARES. CAPITAL REDUCTION.

4.1. Acquisition of Shares.

4.1.1. Subject to authorisation by the General Meeting and with due observance of the applicable relevant statutory provisions, the Board may resolve on the acquisition by the Company of fully paid-up Shares. Such authorisation shall only be valid for a specific period of not more than eighteen (18) months and may from time to time be extended with a period of not more than eighteen (18) months. Acquisition by the Company of non-paid up Shares is null and void.

4.1.2. The authorisation of the General Meeting as referred to in article 4.1.1 shall not be required if the Company acquires fully paid-up Shares for the purpose of transferring such Shares, by virtue of an applicable employee stock purchase plan, to persons employed by the Company or by a group Company, provided such Shares are quoted on the official list of any stock exchange.

4.2. Capital reduction.

4.2.1. With due observance of the statutory requirements the General Meeting may resolve at the proposal of the Board to reduce the issued share capital by (i) reducing the nominal value of Shares by amending the articles of association, or (ii) cancelling:

(a) Shares in its own share capital which the Company holds itself in the Company's share capital, or

(b) all issued Shares against repayment of the amount paid-up on those Shares;

4.2.2. Partial repayment on Shares pursuant to a resolution to reduce their nominal value will be made proportionally.

5. TRANSFER.

5.1. Form of transfer of Shares.

5.1.1. The transfer of a Share shall require a deed executed for that purpose and, save in the event that the Company itself is a party to the transaction, written acknowledgement by the Company of the transfer. The acknowledgement is to be made either in the transfer deed, or by a dated statement endorsed upon the transfer deed or upon a copy of or extract from that deed certified by a notary (notaris) or bailiff (deurwaarder), or in the manner as referred to in article

5.1.2.Service of notice of the transfer deed or of the aforesaid copy or extract upon the Company shall be the equivalent of acknowledgement as stated in this paragraph.

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HOLDINGA MATTHIJSSEN KRAAK

5.1.2. The preceding paragraph shall apply mutatis mutandis to the transfer of any limited right to a Share, provided that a pledge may also be created without acknowledgement by or service of notice upon the Company and that section 3:239 DCC applies, in which case acknowledgement by or service of notice upon the Company shall replace the announcement referred to section 3:239, subsection 3 DCC.

6. REGISTERS. PLEDGE. USE AND ENJOYMENT (vruchtgebruik)

6.1. Shareholders register.

6.1.1. With due observance of the applicable statutory provisions in respect of registered shares, a shareholders register shall be kept by or on behalf of the Company, which register shall be regularly updated and, at the discretion of the Board, may, in whole or in part, be kept in more than one copy and at more than one address. Part of the shareholders register may be kept abroad in order to comply with applicable foreign statutory provisions or applicable listing rules.

6.1.2. Each Shareholder's name, his address and such further information as required by law or considered appropriate by the Board, shall be recorded in the shareholders register.

6.1.3. The form and the contents of the shareholders register shall be determined by the Board with due observance of the articles 6.1.1 and 6.1.2.

6.1.4. Upon his request a Shareholder shall be provided free of charge with written evidence of the contents of the shareholders register with regard to the Shares registered in his name, and the statement so issued may be validly signed on behalf of the Company by a person to be designated for that purpose by the Board.

6.1.5. The provisions of the articles 6.1.3 and article 6.1.4 shall equally apply to persons who hold a right of use and enjoyment (vruchtgebruik) or a right of pledge on one or more Shares.

6.2.Joint holding.

If through any cause whatsoever one or more Shares are jointly held by two or more persons, such persons may jointly exercise the rights arising from those Shares, provided that these persons be represented for that purpose by one from their midst or by a third party authorised by them for that purpose by a written power of attorney.

The Board may, whether or not subject to certain conditions, grant an exemption for the provision of the previous sentence.

6.3. Right of pledge.

6.3.1. Shares may be encumbered with a pledge as security for a debt.

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6.3.2. If a Share is encumbered with a pledge, the voting right attached to that Share shall vest in the Shareholder, unless at the creation of the pledge the voting right has been granted to the pledgee.

6.3.3. Shareholders who as a result of a right of pledge do not have voting rights, have Meeting Rights.

6.4. Right of use and enjoyment (vruchtgebruik).

6.4.1. Shares may be encumbered with a right of use and enjoyment.

6.4.2. If a Share is encumbered with a right of use and enjoyment, the voting right attached to that Share shall vest in the Shareholder, unless at the creation of the right of use and enjoyment the voting right has been granted to the holder of the right of use and enjoyment.

6.4.3. Shareholders who as a result of a right of use and enjoyment do not have voting rights, have Meeting Rights.

7. BOARD.

7.1. Board: composition.

7.1.1. The Company shall be managed by the Board.

7.1.2. The Board shall consist of one or more Executive Directors and one or more Non-Executive Directors The board shall determine the number of Executive Directors and the number of Non-Executive Directors, provided that the number of Executive Directors shall at all times be less than the number of Non-Executive Directors.

Only natural persons can be Non-Executive Director.

7.2. Board: appointment, suspension and dismissal.

7.2.1. The Executive Directors and the Non-Executive Directors shall be appointed as such by the General Meeting at the binding nomination of the Non-Executive Directors.

7.2.2. If an Executive Director or Non-Executive Director is to be appointed, the Non-Executive Directors shall make a binding nomination of at least the number of persons as prescribed by law.

The General Meeting may at all times overrule the binding nomination by a resolution adopted by at least a two thirds majority of the votes cast, provided such majority represents more than half the issued share capital. If the General Meeting overruled the binding nomination, the Non-Executive Directors shall make a new nomination.

The nomination shall be included in the notice of the General Meeting at which the appointment shall be considered.

If a nomination has not been made or has not been made in due time, this shall be stated in the notice and the General Meeting shall be free to appoint a Board Member at its discretion.

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7.2.3. A resolution to appoint a Board Member that was not nominated by the Non-Executive Directors may only be adopted by at least a two thirds majority of the votes cast, provided such majority represents more than half the issued share capital.

7.2.4. When a proposal for appointment of a person as Executive Director is made, the following particulars shall be stated: his age and the position he holds or has held, insofar as these are relevant for the performance of the duties of an Executive Director. The proposal must state the reasons on which it is based.

7.2.5. When a proposal for appointment of a person as Non-Executive Director is made, the following particulars shall be stated: his age, his profession, the number of shares he holds and the positions he holds or has held, insofar as these are relevant for the performance of the duties of a Non-Executive Director. Furthermore, the names of the legal entities of which he is already a non-executive director shall be indicated; if those include legal entities which belong to the same group, reference of that group will be sufficient. The proposal must state the reasons on which it is based.

7.2.6. Board Members are appointed for a maximum term of four (4) years, provided that, unless a Board Member resigns earlier, his term of appointment shall end at the close of the annual General Meeting to be held in the fourth year after the year of his appointment.

A Board Member may be reappointed with due observance of the preceding sentence. The Board shall draw up a retirement schedule for the Board Members.

7.2.7. The General Meeting shall at all times be entitled to suspend or dismiss a Board Member. The General Meeting may only adopt a resolution to suspend or dismiss a Board Member by at least a two thirds majority of the votes cast, provided such majority represents more than half the issued share capital.

A second General Meeting as referred to in section 2:120, subsection 3 DCC may not be convened.

The Board shall also at all times be entitled to suspend (but not to dismiss) an Executive Director. Within three (3) months after a suspension of a Board Member has taken effect, the General Meeting or the Board if the Board resolves to suspend the Board Member, will resolve to either terminate or extend the suspension for a maximum period of another three (3) months. The suspended Board Member shall be given the opportunity to account for his actions at that meeting.

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7.2.8. If neither such resolution is adopted or the General Meeting has resolved to dismiss the Board Member, the suspension shall terminate after the period of suspension has expired.

7.2.9. In the event of the absence or inability to act of one or more Board Members, the powers of the Board remain intact, provided that:

(i) the Non-Executive Directors shall be authorised to temporarily fill the vacant position for a period up to the first General Meeting or, in case of a Board Member unable to act, up to the moment he is no longer unable to act;

(ii) in the event of the absence or inability to act of all members of the Board, the Secretary shall temporarily be responsible for the management of the Company until the vacancies have been filled.

In the event of the absence or inability to act of all members of the Board, the Secretary shall as soon as possible take the necessary measures to make a definitive arrangement.

The term prevented from acting means:

(i) suspension;

(ii) illness;

(iii) inaccessibility,

in the events referred to under sub (ii) and (iii) without the possibility of contact between the Board Member concerned and the Company for a period of five (5) days, unless the Board or the Secretary sets a different term in the case at hand.

7.3. Chief Executive Officer. Chairman of the Board.

7.3.1. The Board shall appoint an Executive Director as Chief Executive Officer for such period as the Board may decide. In addition, the Board may grant other titles to an Executive Director.

7.3.2. The Board shall appoint a Non-Executive Director to be chairman of the Board for such period as the board may decide.

7.3.3. The Board may appoint one or more of the Non-Executive Directors as vice-chairman of the Board for such period as the Board may decide. If the chairman is absent or unwilling to take the chair, a vice-chairman shall be entrusted with such duties of the chairman as the Board may decide.

7.3.4. If no chairman has been appointed or if the chairman is absent or unwilling to take the chair, a meeting of the Board shall be presided over by a vice-chairman or in the event of his absence or unwillingness to take the chair, by a Board Member or another person present designated for such purpose by the meeting.

7.4. Board: remuneration.

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7.4.1. The Company must establish a policy in respect of the remuneration of the Board. The remuneration policy is adopted by the General Meeting upon the proposal of the Non-Executive Directors.

The remuneration of the Executive Directors shall be determined by the Non-Executive Directors with due observance of the remuneration policy adopted by the General Meeting. The remuneration of the Non­ Executive Directors shall be determined by the Board with due observance of the remuneration policy adopted by the General Meeting.

7.4.2. A proposal with respect to remuneration schemes in the form of Shares or rights to Shares is submitted by the Non-Executive Directors to the General Meeting for its approval.

This proposal must set out at least the maximum number of Shares or rights to Shares to be granted to members of the Board and the criteria for granting or amendment.

7.5. Board: meetings.

7.5.1. Meetings of the Board may be called at any time, either by one or more Board Members or, on his or their instructions, by the Secretary.

7.5.2. The Secretary may attend the meetings of the Board. The board may decide to permit others to attend a meeting as well.

7.5.3. Each Board Member will have the right to cast one (1) vote. The Board shall adopt its resolutions by an absolute majority of votes cast. In the event of a tie, the proposal shall be considered rejected.

7.5.4. A Board Member will not participate in deliberations and the adoption of resolutions in respect of which he has a personal direct or indirect conflict of interest with the company or its enterprise. If all Board Members have a conflict of interest, the resolution concerned will be adopted by the General Meeting.

7.5.5. The minutes of meetings of the Board shall be kept by the Secretary. The minutes shall be adopted by the Board at the same meeting or at a subsequent meeting.

If the Board has adopted resolutions without holding a meeting, the Secretary shall keep a record of each resolution adopted without holding a meeting. Such record shall be signed by the chairman and the Secretary.

7.6. Board: powers, division of duties, restrictions.

7.6.1. The Board shall be entrusted with the management of the Company and shall for such purpose have all the powers within the limits of the law that are not granted by the articles of association to others. The day to day management of the Company shall be entrusted to the Executive Directors. The task to supervise the performance by the

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Directors of their duties cannot be taken away from the Non-Executive Directors.

7.6.2. With due observance of the articles of association the Board shall adopt one or more sets of regulations dealing with such matters as its internal organisation, the manner in which decisions are taken, the composition, the duties and organisation of committees as referred to in article 7.6.4. and any other matters concerning the Board, the Chief Executive Officer, the Executive Directors, the Non-Executive Directors and the committees established by the Board.

7.6.3. The Executive Directors may adopt legally valid resolutions with respect to matters that fall within the scope of their duties referred to in article 7.6.1. and 7.6.2. The Non-Executive Directors may also adopt legally valid resolutions with respect to matters that fall within the scope of their duties referred to in article 7.6.1. and 7.6.2.

7.6.4. The Board may establish such committees as it may deem necessary which committees may consist of one or more Board Members or of other persons.

7.6.5. The Executive Directors shall timely provide the Non-Executive Directors with all information required for the exercise of their duties.

7.6.6. Without prejudice to any other applicable provisions of these articles of association, the Board shall require the approval of the General Meeting for resolutions of the Board regarding a significant change in the identity or nature of the Company or the enterprise, including in any event:

(a) the transfer of the enterprise or practically the entire enterprise to a third party;

(b) the entry into or termination of any long-lasting cooperation by the Company or a Subsidiary with any other legal person or company or as a fully liable general partner of a limited partnership or a general partnership, provided that such cooperation or the termination thereof is of significant importance to the Company; and

(c) the acquisition or disposal of a participating interest in the capital of a Company with a value of at least one-third of the sum of the assets according to the consolidated balance sheet with explanatory notes thereto according to the last adopted Annual Accounts of the Company, by the Company or a Subsidiary.

7.7. Representation.

7.7.1. The Board as well as two (2) Executive Directors acting jointly are authorised to represent the Company. In case only one Executive

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Director is in office, such Executive Director is authorised to represent the Company acting independently

7.7.2. The Board may grant one or more persons, whether or not employed by the Company, the power to represent the Company (procuratie) or grant the power to represent the Company on a continuing basis in a different manner.

7.8. Secretary.

7.8.1. The Board shall appoint a Secretary from outside its members.

7.8.2. The Secretary shall participate in the meetings of the Board, as well as the meetings of the committees established by the Board, this in conformity with the regulations to be decided upon.

7.8.3. The Secretary shall further have such powers as are assigned to him by the articles of association and, subject to the articles of association, by the Board on or after his appointment.

7.8.4. The Secretary may be removed from office at any time by the Board.

7.9. Indemnification Board Members.

7.9.1. Unless Dutch law provides otherwise, the following shall be reimbursed to current and former members of the Board:

(a) the reasonable costs of conducting a defence against claims based on acts or failures to act in the exercise of their duties or any other duties currently or previously performed by them at the Company's request;

(b) any damages or fines payable by them as a result of an act or failure to act as referred to under a;

(c) the reasonable costs of appearing in other legal proceedings in which they are involved as current or former members of the Board, with the exception of proceedings primarily aimed at pursuing a claim on their own behalf.

There shall be no entitlement to reimbursement as referred to above if and to the extent that:

(d) a Dutch court or, in the event of arbitration, an arbitrator has established in a final and conclusive decision that the act or failure to act of the person concerned can be characterised as wilful (opzettelijk), intentionally reckless (bewust roekeloos) or seriously culpable (ernstig verwijtbaar) conduct, unless Dutch law provides otherwise or this would, in view of the circumstances of the case, be unacceptable according to standards of reasonableness and fairness; or

(e) the costs or financial loss of the person concerned are covered by an insurance and the insurer has paid out the costs or financial loss.

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If and to the extent that it has been established by a Dutch court or, in the event of arbitration, an arbitrator in a final and conclusive decision that the person concerned is not entitled to reimbursement as referred to above, he shall immediately repay the amount reimbursed by the Company.

7.9.2. The Company may take out liability insurance for the benefit of the persons concerned.

7.9.3. The Board may by agreement give further implementation to the above.

8. GENERAL MEETINGS.

8.1. General Meetings.

8.1.l. General Meetings shall be held in Amsterdam or in the municipality of Haarlemmermeer (Schiphol Airport).

8.1.2. A General Meeting shall be held once a year, no later than six (6) months after the end of the financial year of the Company.

8.1.3. The Board shall provide the General Meeting with all requested information, unless this would be contrary to an overriding interest of the Company. If the Board invokes an overriding interest, it must give reasons.

8.2. Extraordinary General Meetings.

Extraordinary General Meetings shall be convened by the Board or by those who are authorised by law or pursuant to these articles of association to do so.

8.3. General Meetings: notice and agenda.

8.3.1. Notice of the General Meeting shall be given by the Board or by those who are authorised by law or pursuant to these articles of association to do so upon a term of at least such number of days prior to the day of the meeting as required by law, in accordance with law and the regulations of the stock exchange where the Shares in the share capital of the Company at the Company's request are officially listed.

8.3.2. The Board or the person who is authorised by law or pursuant to these articles of association to convene the meeting may decide that the convocation letter in respect of a person authorised to attend a General Meeting who agrees thereto, is replaced by a legible and reproducible message sent by electronic mail to the address indicated by him to the Company for such purpose.

8.3.3. The notice shall state the subjects on the agenda or shall inform the persons authorised to attend a General Meeting that they may inspect the agenda at the office of the Company and that copies thereof are obtainable at such places as are specified in the notice.

8.3.4. The agenda for the annual General Meeting shall in any case include the following items:

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(a) the consideration of Annual Statement of Accounts;

(b) the adoption of the Annual Accounts;

(c) the appropriation of profits;

(d) proposals relating to the composition of the Board, including the filling of any vacancies in the Board;

(e) the proposals placed on the agenda by the Board together with proposals made by Shareholders in accordance with provisions of the law and the provisions of the articles of association.

8.3.5. A matter, the consideration of which has been requested in writing by one or more Shareholders, representing solely or jointly at least the percentage prescribed by law of the issued share capital, will be placed on the notice or will be announced in the same manner if the Company has received the request not later than on the date as prescribed by law.

8.3.6. The Board shall inform the General Meeting by means of a shareholders' circular or explanatory notes to the agenda of all facts and circumstances relevant to the proposals on the agenda.

8.4. General Meetings: attendance of meetings.

8.4.1. The persons who are entitled to attend the General Meeting are persons who:

(i) are a Shareholder or a person who is otherwise entitled to attend the General Meeting as per a certain date, determined by the Board, such date hereinafter referred to as: the "record date";

(ii) are as such registered in a register (or one or more parts thereof) designated thereto by the Board, hereinafter referred to as: the "register''; and

(i)have given notice in writing to the Company prior to a date set in the notice that they will attend a General Meeting,

regardless of who will be Shareholder at the time of the meeting. The notice will contain the name and the number of Shares the person will represent in the meeting. The provision above under (iii) concerning the notice to the Company also applies to the proxy holder of a person authorised to attend a General Meeting.

8.4.2. The Board may decide that Persons entitled to attend General Meetings and vote thereat may, within a period prior to the General Meeting to be set by the Board, which period cannot begin prior to the record date as meant in article 8.4.1, cast their votes electronically in a manner to be decided by the Board. Votes cast in accordance with the previous sentence are equal to votes cast at the meeting.

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8.4.3. The Board may decide that the business transacted at a General Meeting can be taken note of by electronic means of communication.

8.4.4. The Board may decide that each person entitled to attend General Meetings and vote thereat may, either in person or by written proxy, vote at that meeting by electronic means of communication, provided that such person can be identified via the electronic means of communication and furthermore provided that such person can directly take note of the business transacted at the General Meeting concerned. The Board may attach conditions to the use of the electronic means of communication, which conditions shall be announced at the convocation of the General Meeting and shall be posted on the Company's website.

8.4.5. Board Members shall have admission to the General Meetings. They shall have an advisory vote at the General Meetings.

8.4.6. Furthermore, admission shall be given to the persons whose attendance at the General Meeting is approved by the chairman of the meeting.

8.4.7. All issues concerning the admittance to the General Meeting shall be decided by the chairman of the meeting.

8.5. General Meetings: order of the meeting, minutes.

8.5.1. The General Meeting will be chaired by the chairman of the Board or in his absence by one of the other Non-Executive Directors designated by the Board; if none of the Non-Executive Directors is present at the meeting, the meeting will be chaired by one of the Executive Directors designated by the Board. The chairman shall designate the secretary.

8.5.2. The chairman of the meeting shall determine the order of proceedings at the meeting with due observance of the agenda and he may restrict the allotted speaking time or take other measures to ensure orderly progress of the meeting.

8.5.3. All issues concerning the proceedings at the meeting, shall be decided by the chairman of the meeting.

8.5.4. Minutes shall be kept of the business transacted at the meeting unless a notarial record is prepared thereof. Minutes shall be adopted and in evidence of such adoption be signed by the chairman and the secretary of the meeting concerned.

8.5.5. A certificate signed by the chairman and the secretary of the meeting confirming that the General Meeting has adopted a particular resolution, shall constitute evidence of such resolution vis-à-vis third parties.

8.6. General Meetings: adoption of resolutions.

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8.6.1. Unless another majority of votes or quorum is required by virtue of the law, all resolutions of the General Meeting shall be adopted by at least a simple majority of the votes cast, in a meeting where more than one-third of the issued share capital is represented.

A second meeting referred to in article 2:120, subsection 3 DCC cannot be convened.

8.6.2. Each Share confers the right to cast one (1) vote at the General Meeting.

Blank votes and invalid votes shall be regarded as not having been cast.

8.6.3. No votes may be cast at the General Meeting in respect of Shares which are held by the Company or any of its Subsidiaries.

Holders of a right of use and enjoyment (vruchtgebruik) and pledgees of Shares which belong to the Company or its Subsidiaries shall not be excluded from the right to vote if the right of use and enjoyment or pledge was created before the Shares concerned were held by the Company or a Subsidiary of the Company and at the creation of the right of pledge or the right of use and enjoyment, the voting rights were granted to the pledgee or holder of the right of use and enjoyment.

8.6.4. The chairman of the General Meeting determines the method of

voting.

8.6.5. The ruling pronounced by the chairman of the General Meeting in respect of the outcome of any vote taken at a General Meeting shall be decisive. The same shall apply to the contents of any resolution passed.

8.6.6. Any and all disputes with regard to voting for which neither the law nor the articles of association provide shall be decided by the chairman of the General Meeting.

9. FINANCIAL YEAR. AUDITOR.

9.1. Financial year; Annual Statement of Accounts.

9.1.1. The financial year of the Company shall be the calendar year.

9.1.2. Annually, within the term set by law, the Board shall prepare Annual Accounts.

The Annual Accounts shall be accompanied by the auditor's statement referred to in article 9.2.1, if the instruction referred to in that article has been given, by the Report of the Board of Directors, unless section 2:391 DCC does not apply to the Company, as well as by the other particulars to be added to those documents by virtue of applicable statutory provisions.

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The Annual Accounts shall be signed by all Board Members; if the signature of one or more of them is lacking, this shall be disclosed, stating the reasons therefor.

9.1.3. The Company shall ensure that the Annual Accounts as prepared, the Report of the Board of Directors (if applicable) and the other particulars referred to in article 9.1.2 shall be made available at the office of the Company as of the date of the notice of the General Meeting at which they are to be discussed.

The Shareholders and other Persons entitled to attend General Meetings may inspect the above documents at the office of the Company and obtain a copy thereof free of charge.

9.2. Auditor.

9.2.1. The General Meeting shall instruct a registered accountant or another expert, as referred to in section 2:393, subsection 1 DCC, both hereinafter called: the "auditor", to audit the Annual Accounts prepared by the Board, in accordance with the provisions of section 2:393, subsection 3 DCC. The auditor shall report on his audit to the Board and shall present the results of his examination regarding the accuracy of the Annual Accounts in an auditor's statement.

9.2.2. If the General Meeting fails to give such instructions, then the Board shall be so authorised.

9.2.3. The instruction given to the auditor may be revoked by the General Meeting and by the corporate body which has given such instruction. The instruction may only be revoked for good reasons with due observance of section 2:393, subsection 2 DCC.

9.2.4. The Board may give instructions to the auditor or any other auditor at the expense of the Company.

10. PROFITS.

10.1. Profit and loss. Distributions on Shares.

10.1.1. The Board will keep a share premium reserve and profit reserve for the Shares.

10.1.2. The Company may make distributions on Shares only to the extent that its shareholders' equity exceeds the sum of the paid-up and called-up part of the capital and the reserves which must be maintained by law.

10.1.3. Distributions of profit, meaning the net earnings after taxes shown by the adopted Annual Accounts, shall be made after the adoption of the Annual Accounts from which it appears that they are permitted, without prejudice to any of the other provisions of these articles of association.

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10.1.4. The Board may determine that any amount out of the profit shall be added to the reserves.

10.1.5. The profit remaining after application of article 10.1.4 shall be at the disposal of the General Meeting, which may resolve to carry it to the reserves or to distribute it among the Shareholders.

10.1.6. On a proposal of the Board the General Meeting may resolve to distribute to the Shareholders a dividend in the form of Shares in the share capital of the Company.

10.1.7. Subject to the other provisions of this article 10.1 the General Meeting may, on a proposal made by the Board resolve to make distributions to the Shareholders to the debit of one (1) or several reserves which the Company is not prohibited from distributing by virtue of the law.

10.1.8. No dividends shall be paid on Shares held by the Company in its own share capital, unless such Shares are encumbered with a right of use and enjoyment (vruchtgebruik) or pledge.

10.2. Interim distributions.

10.2.1. The Board may resolve to make interim distributions to the Shareholders if an interim statement of assets and liabilities shows that the requirement of article 10.1.2 has been met.

10.2.2. The interim statement of assets and liabilities shall relate to the condition of the assets and liabilities on a date no earlier than the first day of the third month preceding the month in which the resolution to distribute is published. It shall be prepared on the basis of generally acceptable valuation methods. The amounts to be reserved under the law and these articles of association shall be included in the statement of assets and liabilities. It shall be signed by the Board Members. If the signature of one or more of them is lacking, this shall be disclosed, stating the reasons therefor.

10.2.3. Any proposal for distribution of dividend on Shares and any resolution to distribute an interim dividend on Shares shall immediately be published by the Board in accordance with the regulations of the stock exchange where the Shares at the Company's request are officially listed. The notification shall specify the date when and the place where the dividend shall be payable or - in the case of a proposal for distribution of dividend - is expected to be made payable.

10.2.4. Dividends shall be payable no later than thirty (30) days after the date they were declared, unless the body declaring the dividend determines a different date.

10.2.5. Dividends which have not been claimed upon the expiry of five (5) years and one (1) day after the date when they became payable shall be forfeited to the Company and shall be carried to the reserves.

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10.2.6. The Board may determine that distributions on Shares shall be made payable either in euro or in another currency.

11. AMENDMENT OF THE ARTICLES OF ASSOCIATION, DISSOLUTION OF THE COMPANY.

11.1. A resolution to amend the articles of association or to dissolve the Company may only be adopted at the proposal of the Board.

11.2. Liquidation.

11.2.1. On the dissolution of the Company, the liquidation shall be carried out by the Board, unless otherwise resolved by the General Meeting.

11.2.2. Pending the liquidation the provisions of these articles of association shall remain in force to the fullest extent possible.

11.2.3. The surplus assets of the Company remaining after satisfaction of its debts shall, in accordance with the provisions of section 2:23b DCC, be for the benefit of the Shareholders in proportion to the nominal value amount of the Shares held by each of them.

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EX-4.1

Exhibit 4.1

uniQure N.V.

2014 Share Incentive Plan

(Amended and Restated effective as of June 16, 2021)

1.                                      Purpose

The purpose of this 2014 Share Incentive Plan, as herein amended and restated (the “Plan”) of uniQure N.V., a public limited company incorporated under the laws of the Netherlands (the “Company”), is to advance the interests of the Company’s shareholders by enhancing the Company’s ability to attract, retain and motivate persons who are expected to make important contributions to the Company and by providing such persons with equity ownership opportunities and performance-based incentives that are intended to better align the interests of such persons with those of the Company’s shareholders. Except where the context otherwise requires, the term “Company” shall include any of the Company’s present or future parent or subsidiary corporations as defined in Sections 424(e) or (f) of the U.S. Internal Revenue Code of 1986, as amended, and any regulations thereunder (the “Code”) and any other business venture (including, without limitation, joint venture or limited liability company) in which the Company has a controlling interest, as determined by the Board of Directors of the Company (the “Board”). The Plan was initially effective as of January 9, 2014 and was amended and restated effective as of June 10, 2015, June 15, 2016, and June 13, 2018. This amended and restated Plan will be effective as of June 16, 2021, subject to the approval of the Company’s shareholders (the “Amendment Effective Date”).

Changes made pursuant to this amendment and restatement shall only apply to Awards granted on or after the Amendment Effective Date. Awards granted prior to the Amendment Effective Date shall continue to be governed by the applicable Award agreements and the terms of the Plan, without giving effect to changes made pursuant to this amendment and restatement, and the Board shall administer such Awards in accordance with the Plan, without giving effect to changes made pursuant to this amendment and restatement.

2.                                      Eligibility

All of the Company’s employees, executive directors and non-executive directors, as well as consultants and advisors to the Company (as such terms are defined and interpreted for purposes of Form S-8 under the Securities Act of 1933, as amended (the “Securities Act”), or any successor form) are eligible to be granted Awards under the Plan. Eligibility to participate in the Plan shall be determined at the sole discretion of the Board. Each person who is granted an Award under the Plan is deemed a “Participant.” “Award” means Options (as defined in Section 5), SARs (as defined in Section 6), Restricted Shares (as defined in Section 7), Restricted Share Units (as defined in Section 7) and Other Share-Based Awards (as defined in Section 8).

3.                                      Administration and Delegation

(a)                                 Administration by the Board. The Plan will be administered by the Board. The Board shall have authority to grant Awards and to adopt, amend and repeal such administrative rules, guidelines and practices relating to the Plan as it shall deem advisable. The Board may construe and interpret the terms of the Plan and any Award agreements entered into under the Plan. The Board may correct any defect, supply any omission or reconcile any inconsistency in the Plan or any Award in the manner and to the extent it shall deem expedient and it shall be the sole and final judge of such expediency. All decisions by the Board shall be made in the Board’s sole discretion and shall be final and binding on all persons having or claiming any interest in the Plan or in any Award.

(b)                                 Appointment of Committees. To the extent permitted by applicable law, the Board may delegate any or all of its powers under the Plan to one or more committees or subcommittees of the Board (a “Committee”). All references in the Plan to the “Board” shall mean the Board or a Committee of the Board to the extent that the Board’s powers or authority under the Plan have been delegated to such Committee.

4.                                      Shares Available for Awards

(a)                                 Number of Shares; Share Counting.

(1)                                 Authorized Number of Shares. Subject to adjustment under Section 9, the aggregate number of ordinary shares (€0.05 par value per share) of the Company (the “Ordinary Shares”) that may be issued on or after the Amendment Effective Date with respect to Awards granted under the Plan shall not exceed 12,601,471.

(2)                                 Share Counting. For purposes of counting the number of shares available for the grant of Awards under the Plan:

(A)                               the gross number of Ordinary Shares covered by SARs shall be counted against the number of shares available for the grant of Awards under the Plan; provided, however, that (i) SARs that may be settled only in cash shall not be so counted and (ii) if the Company grants a SAR in tandem with an Option for the same number of Ordinary Shares and provides that only one such Award may be exercised (a “Tandem SAR”), only the shares covered by the Option, and not the shares covered by the Tandem SAR, shall be so counted, and the expiration of one in connection with the other’s exercise will not restore shares to the Plan;

(B)                               if any Award (i) expires or is terminated, surrendered or canceled without having been fully exercised or is forfeited in whole or in part (including as the result of Ordinary Shares subject to such Award being repurchased by the Company at the original issuance price pursuant to a contractual repurchase right) or (ii) results in any Ordinary Shares not being issued (including as a result of a SAR that was settleable either in cash or in shares actually being settled in cash), the unused Ordinary Shares covered by such Award shall again be available for the grant of Awards; provided, however, that (1) in the case of Incentive Share Options, the foregoing shall be subject to any limitations under the Code, (2) in the case of the exercise of a SAR, the number of shares counted against the shares available under the Plan shall be the gross number of shares subject to the SAR multiplied by the percentage of the SAR actually exercised, regardless of the number of shares actually used to settle such SAR upon exercise and (3) the shares covered by a Tandem SAR shall not again become available for grant upon the expiration or termination of such Tandem SAR; and

(C)                               Ordinary Shares delivered (either by actual delivery, attestation, or net exercise) to the Company by a Participant to (i) purchase Ordinary Shares upon the exercise of an Award or (ii) satisfy tax withholding obligations with respect to Options and SARs (including shares retained from the Option or SAR creating the tax obligation) shall not be added back to the number of shares available for the future grant of Awards.

(b)                                 Substitute Awards. In connection with a merger or consolidation of an entity with the Company or the acquisition by the Company of property or stock of an entity, the Board may grant Awards in substitution for any options or other share or share-based awards granted by such entity or an affiliate thereof. Substitute Awards may be granted on such terms as the Board deems appropriate in the circumstances, notwithstanding any limitations on Awards contained in the Plan. Substitute Awards shall not count against the overall share limit set forth in Section 4(a), except as may be required by reason of Section 422 and related provisions of the Code.

5.                                      Share Options

(a)                                 General. The Board may grant options to purchase Ordinary Shares (each, an “Option”) and determine the number of Ordinary Shares to be covered by each Option, the exercise price of each Option and the conditions and limitations applicable to the exercise of each Option, including conditions relating to applicable securities laws, as it considers necessary or advisable.

(b)                                 Incentive Share Options. An Option that the Board intends to be an “incentive stock option” as defined in Section 422 of the Code (an “Incentive Share Option”) shall only be granted to employees of uniQure N.V., any of uniQure N.V.’s present or future parent or subsidiary corporations as defined in Sections 424(e) or (f) of the Code, and any other entities the employees of which are eligible to receive Incentive Share Options under the Code, and shall be subject to and shall be construed consistently with the requirements of Section 422 of the Code. An Option that is not intended to be an Incentive Share Option shall be designated a “Share Option.” The Company shall have no liability to a Participant, or any other party, if an Option (or any part thereof) that is intended to be an Incentive Share Option is not an Incentive Share Option or if the Company converts an Incentive Share Option to a Share Option. Awards with respect to a maximum of 200,000 Ordinary Shares may be granted in the form of Incentive Share Options under the Plan.

(c)                                  Exercise Price. The Board shall establish the exercise price of each Option and specify the exercise price in the applicable Option agreement which shall be not less than 100% of the Fair Market Value per Ordinary Share on the date the Option is granted; provided, however, that if the Board approves the grant of an Option with an exercise price to be determined on a future date, the exercise price shall be not less than 100% of the Fair Market Value on such future date. For purposes of the Plan, unless otherwise required by applicable law, the Fair Market Value per Ordinary Share as of any date shall be (A) if the Ordinary Shares are readily tradeable on a national securities exchange or other market system, either (I) or (II), as determined by the Board on or prior to the date of grant, where (I) is the average of the closing sales prices of the Ordinary Shares during regular trading hours for the ten trading days following the date of grant and (II) is the closing sales price of the Ordinary Shares during regular trading hours on the date of grant, or (B) if the Ordinary Shares are not readily tradeable on a national securities exchange or other market system, the amount determined in good faith by (or in a manner approved by) the Board (“Fair Market Value”). Notwithstanding the foregoing (x) for purposes of any Option intended to be an Incentive Share Option, Fair Market Value shall be determined in accordance with the applicable provisions of Section 422 of the Code and the corresponding regulations, (y) for purposes of any Share Option granted to a Participant who is subject to taxation in the United States, Fair Market Value shall be determined in accordance with the applicable provisions of Section 409A of the Code and the corresponding regulations and (z) in no event shall the exercise price of any Option be less than the nominal value per Ordinary Share.

(d)                                 Duration of Options. Each Option shall be exercisable at such times and subject to such terms and conditions as the Board may specify in the applicable option agreement; provided, however, that no Option will be granted with a term in excess of 10 years.

(e)                                  Exercise of Options. Options may be exercised by delivery to the Company of a notice of exercise in a form (which may be electronic) approved by the Company, together with payment in full (in the manner specified in Section 5(f)) of the exercise price for the number of shares for which the Option is exercised. Ordinary Shares subject to the Option will be delivered by the Company as soon as practicable following exercise.

(f)                                   Payment Upon Exercise. Ordinary Shares purchased upon the exercise of an Option granted under the Plan shall be paid for as follows:

(1)                                 By wire transfer, in cash or by check, payable to the order of the Company;

(2)                                 except as may otherwise be provided in the applicable Option agreement or approved by the Board, in its sole discretion, by (i) delivery of an irrevocable and unconditional undertaking by a creditworthy broker to deliver promptly to the Company sufficient funds to pay the exercise price and any required tax withholding or (ii) delivery by the Participant to the Company of a copy of irrevocable and unconditional instructions to a creditworthy broker to deliver promptly to the Company cash or a check sufficient to pay the exercise price and any required tax withholding;

(3)                                 to the extent provided for in the applicable Option agreement or approved by the Board, in its sole discretion, by delivery (either by actual delivery or attestation) of Ordinary Shares owned by the Participant valued at their Fair Market Value, provided (i) such method of payment is then permitted under applicable law, (ii) such Ordinary Shares, if acquired directly from the Company, were owned by the Participant for such minimum period of time, if any, as may be established by the Board in its discretion and (iii) such Ordinary Shares are not subject to any repurchase, forfeiture, unfulfilled vesting or other similar requirements;

(4)                                 to the extent provided for in the applicable Share Option agreement or approved by the Board in its sole discretion, by delivery of a notice of “net exercise” to the Company, as a result of which the Participant would receive (i) the number of shares underlying the portion of the Option being exercised, less (ii) such number of shares as is equal to (A) the aggregate exercise price for the portion of the Option being exercised divided by (B) the Fair Market Value on the date of exercise;

(5)                                 to the extent permitted by applicable law and provided for in the applicable Option agreement or approved by the Board, in its sole discretion, by payment of such other lawful consideration as the Board may determine; or

(6)                                 by any combination of the above permitted forms of payment.

6.                                      Share Appreciation Rights

(a)                                 General. The Board may grant Awards consisting of share appreciation rights (“SARs”) entitling the holder, upon exercise, to receive an amount of Ordinary Shares or cash or a combination thereof (such form to be determined by the Board) determined by reference to appreciation, from and after the date of grant, in the Fair Market Value of an Ordinary Share over the measurement price established pursuant to Section 6(b). The date as of which such appreciation is determined shall be the exercise date.

(b)                                 Measurement Price. The Board shall establish the measurement price of each SAR and specify it in the applicable SAR agreement. The measurement price shall not be less than 100% of the Fair Market Value on the date the SAR is granted; provided that if the Board approves the grant of a SAR effective as of a future date, the measurement price shall be not less than 100% of the Fair Market Value on such future date.

(c)                                  Duration of SARs. Each SAR shall be exercisable at such times and subject to such terms and conditions as the Board may specify in the applicable SAR agreement; provided, however, that no SAR will be granted with a term in excess of 10 years.

(d)                                 Exercise of SARs. SARs may be exercised by delivery to the Company of a notice of exercise in a form (which may be electronic) approved by the Company, together with any other documents required by the Board.

7.                                      Restricted Shares; Restricted Share Units

(a)                                 General. The Board may grant Awards entitling recipients to acquire Ordinary Shares (“Restricted Shares”), subject to the right of the Company to repurchase all or part of such shares at their issue price or other stated or formula price (or to require forfeiture of such shares if issued at no cost) from the recipient in the event that conditions specified by the Board in the applicable Award are not satisfied prior to the end of the applicable restriction period or periods established by the Board for such Award. The Board may also grant Awards entitling the recipient to receive Ordinary Shares or cash to be delivered at the time such Award vests (“Restricted Share Units”) (Restricted Shares and Restricted Share Units are each referred to herein as a “Restricted Share Award”).

(b)                                 Terms and Conditions for All Restricted Share Awards. The Board shall determine the terms and conditions of a Restricted Share Award, including the conditions for vesting and repurchase (or forfeiture) and the issue price, if any.

(c)                                  Additional Provisions Relating to Restricted Shares.

(1)                                 Dividends. Unless otherwise provided in the applicable Award agreement, any dividends (whether paid in cash or shares) declared and paid by the Company with respect to shares of Restricted Shares (“Accrued Dividends”) shall be paid to the Participant only if and when such shares become free from the restrictions on transferability and forfeitability that apply to such shares. Each payment of Accrued Dividends will be made no later than the end of the calendar year in which the dividends are paid to shareholders of that class of shares or, if later, the 15th day of the third month following the lapsing of the restrictions on transferability and the forfeitability provisions applicable to the underlying shares of Restricted Share. For the avoidance of doubt, dividends declared and paid by the Company with respect to Restricted Shares that are subject to performance-based restrictions on transfer and forfeitability shall be paid if and to the extent that the restrictions on transfer and forfeitability with respect to the underlying Restricted Shares lapse, as determined by the Board.

(d)                                 Additional Provisions Relating to Restricted Share Units.

(1)                                 Settlement. Upon the vesting of and/or lapsing of any other restrictions (i.e., settlement) with respect to each Restricted Share Unit, the Participant shall be entitled to receive from the Company the number of shares of Ordinary Shares set forth in the applicable Award agreement or (if so provided in the applicable Award agreement) an amount of cash equal to the Fair Market Value of one of such number of Ordinary Shares. The Board may, in its discretion, provide that settlement of Restricted Share Units shall be deferred, on a mandatory basis or at the election of the Participant in a manner that complies with Section 409A of the Code.

(2)                                 Voting Rights. A Participant shall have no voting rights with respect to any Restricted Share Units.

(3)                                 Dividend Equivalents. The Award agreement for Restricted Share Units may provide Participants with the right to receive an amount equal to any dividends or other distributions declared and paid on an equal number of outstanding Ordinary Shares (“Dividend Equivalents”). Dividend Equivalents may be paid currently or credited to an account for the Participant, may be settled in cash and/or Ordinary Shares and may be subject to the same restrictions as the Restricted Share Units with respect to which paid, in each case to the extent provided in the Award agreement. Notwithstanding the foregoing, Dividend Equivalents with respect to Restricted Share Units that are subject to performance-based restrictions shall only be paid if and to the extent that the restrictions with respect to the underlying Restricted Share Units lapse, as determined by the Board.

8.                                      Other Share-Based Awards

(a)                                 General. Other Awards of Ordinary Shares, and other Awards that are valued in whole or in part by reference to, or are otherwise based on, Ordinary Shares or other property, may be granted hereunder to Participants (“Other Share-Based-Awards”). Such Other Share-Based Awards shall also be available as a form of payment in the settlement of other Awards granted under the Plan or as payment in lieu of compensation to which a Participant is otherwise entitled. Other Share-Based Awards may be paid in Ordinary Shares or cash, as the Board shall determine.

(b)                                 Terms and Conditions. Subject to the provisions of the Plan, the Board shall determine the terms and conditions of each Other Share-Based Award, including any purchase price applicable thereto.

9.                                      Adjustments for Changes in Ordinary Shares and Certain Other Events

(a)                                 Changes in Capitalization. In the event of any share split, share consolidation, share dividend, recapitalization, combination of shares, reclassification of shares, spin-off or other similar change in capitalization or event, or any dividend or distribution to holders of Ordinary Shares other than an ordinary cash dividend, (i) the number and class of securities available under the Plan, (ii) the share counting rules set forth in Section 4(a), (iii) the number and class of securities and exercise price per share of each outstanding Option, (iv) the share and per-share provisions and the measurement price of each outstanding SAR, (v) the number of shares subject to and the repurchase price per share subject to each outstanding Restricted Share Award and (vi) the share and per-share-related provisions and the purchase price, if any, of each outstanding Restricted Share Unit or Other Share-Based Award, shall be equitably adjusted by the Company (or substituted Awards may be made, if applicable) in the manner determined by the Board. Without limiting the generality of the foregoing and subject to compliance with Section 409A of the Code, if applicable, in the event the Company effects a split of the Ordinary Shares by means of a share dividend and the exercise price of and the number of shares subject to an outstanding Option are adjusted as of the date of the distribution of the dividend (rather than as of the record date for such dividend), then an optionee who exercises an Option between the record date and the distribution date for such share dividend shall be entitled to receive, on the distribution date, the share dividend with respect to the Ordinary Shares acquired upon such Option exercise, notwithstanding the fact that such shares were not outstanding as of the close of business on the record date for such share dividend.

(b)                                 Reorganization Events.

(1)                                 Definition. A “Reorganization Event” shall be deemed to have occurred upon any of the following events:

(A)                               any person or other entity (other than any of the Company’s subsidiaries or any employee benefit plan sponsored by the Company or any of its subsidiaries), including any person as defined in Section 13(d)(3) of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), becomes the beneficial owner, as defined in Rule 13d-3 under the Exchange Act, directly or indirectly, of more than 50% of the total combined voting power of all classes of capital stock of the Company normally entitled to vote for the election of directors of the Company (the “Voting Stock”);

(B)                               consummation of the sale of all or substantially all of the property or assets of the Company; or

(C)                               consummation of a consolidation or merger of the Company with another corporation (other than with any of the Company’s subsidiaries), which results in the stockholders of the Company immediately before the occurrence of the consolidation or merger owning, in the aggregate, less than 51% of the Voting Stock of the surviving entity.

Notwithstanding the foregoing, the Board may provide for a different definition of “Change in Control” in an Award agreement if it determines that such different definition is necessary or appropriate, including without limitation, to comply with the requirements of Section 409A of the Code.

(2)                                 Consequences of a Reorganization Event on Awards.

(A)                               In connection with a Reorganization Event where the Company is not the surviving corporation (or survives only as a subsidiary of another corporation), unless the Board determines otherwise, all outstanding Awards that are not exercised or paid at the time of the Reorganization Event shall be assumed by, or replaced with Awards that have comparable terms by, the surviving corporation (or a parent or subsidiary of the surviving corporation). After a Reorganization Event, references to the “Company” as they relate to employment matters shall include the successor employer, unless the Board provides otherwise.

(B)                               Unless the Award agreement provides otherwise, if a Participant’s employment or other service is terminated by the Company without cause (as determined by the Board) upon or within 12 months following a Reorganization Event, the Participant’s outstanding Awards shall become fully exercisable and any restrictions on such Awards shall lapse as of the date of such termination; provided that if the restrictions on any such Awards is based, in whole or in part, on performance, the applicable Award agreement shall specify how the portion of the Award that becomes vested pursuant to this Section 9(b)(2) shall be calculated.

(C)                               In connection with a Reorganization Event, if all outstanding Awards are not assumed by, or replaced with Awards that have comparable terms by, the surviving corporation (or a parent or subsidiary of the surviving corporation), the Board may take any one or more of the following actions as to all or any (or any portion of) outstanding Awards on such terms as the Board determines without the consent of any Participant (except to the extent specifically provided otherwise in an applicable Award agreement or another agreement between the Company and the Participant): (i) upon written notice to a Participant, provide that all of the Participant’s unexercised and/or unvested Awards will terminate immediately prior to the consummation of such Reorganization Event unless exercised by the Participant (to the extent then exercisable) within a specified period following the date of such notice, (ii) provide that outstanding Awards shall become exercisable, realizable, or deliverable, or restrictions applicable to an Award shall lapse, in whole or in part prior to or upon such Reorganization Event, (iii) in the event of a Reorganization Event under the terms of which holders of Ordinary Shares will receive upon consummation thereof a cash payment for each share surrendered in the Reorganization Event (the “Acquisition Price”), make or provide for a cash payment to Participants with respect to each Award held by a Participant equal to (I) the number of shares of Ordinary Shares subject to the vested portion of the Award (after giving effect to any acceleration of vesting that occurs upon or immediately prior to such Reorganization Event) multiplied by (II) the excess, if any, of (x) the Acquisition Price over (y) the exercise, measurement or purchase price of such Award and any applicable tax withholdings, in exchange for the termination of such Award, (iv) provide that, in connection with a liquidation or dissolution of the Company, Awards shall convert into the right to receive liquidation proceeds (if applicable, net of the exercise, measurement or purchase price thereof and any applicable tax withholdings) and (v) any combination of the foregoing. In taking any of the actions permitted under this Section 9(b)(2), the Board shall not be obligated by the Plan to treat all Awards, all Awards held by a Participant, or all Awards of the same type, identically. Such surrender, termination or payment shall take place as of the date of the Reorganization Event or such other date as the Board may specify. Without limiting the foregoing, (1) if the per share Acquisition Price does not exceed the per share Option exercise price or SAR measurement price, as applicable, the Company shall not be required to make any payment to the Participant upon surrender of the Option or SAR and (2) upon the occurrence of a Reorganization Event involving the liquidation or dissolution of the Company, except to the extent specifically provided to the contrary in the instrument evidencing any Restricted Shares or any other agreement between a Participant and the Company, all restrictions and conditions on all Restricted Shares then outstanding shall automatically be deemed terminated or satisfied.

(D)                               Notwithstanding the foregoing in this Section 9(b)(2), in the case of outstanding Restricted Share Units that are subject to Section 409A of the Code: (i) if the applicable Restricted Share Unit agreement provides that the Restricted Share Units shall be settled upon a “change in control event”

within the meaning of U.S. Treasury Regulation Section 1.409A-3(i)(5)(i), and the Reorganization Event constitutes such a “change in control event”, then no assumption or substitution shall be permitted pursuant to Section 9(b)(2)(A) and the Restricted Share Units shall instead be settled in accordance with the terms of the applicable Restricted Share Unit agreement; and (ii) the Board may only undertake the actions set forth in clauses (ii), (iii) or (iv) of Section 9(b)(2)(C) if the Reorganization Event constitutes a “change in control event” as defined under U.S. Treasury Regulation Section 1.409A-3(i)(5)(i) and such action is permitted or required by Section 409A of the Code; if the Reorganization Event is not a “change in control event” as so defined or such action is not permitted or required by Section 409A of the Code, and the acquiring or succeeding corporation does not assume or substitute the Restricted Share Units pursuant to Section 9(b)(2)(A), then the unvested Restricted Share Units shall terminate immediately prior to the consummation of the Reorganization Event without any payment in exchange therefor.

(E)                                For purposes of Section 9(b)(2)(A), an Award (other than Restricted Shares) shall be considered assumed if, following consummation of the Reorganization Event, such Award confers the right to purchase or receive pursuant to the terms of such Award, for each Ordinary Share subject to the Award immediately prior to the consummation of the Reorganization Event, the consideration (whether cash, securities or other property) received as a result of the Reorganization Event by holders of Ordinary Shares for each Ordinary Share held immediately prior to the consummation of the Reorganization Event (and if holders were offered a choice of consideration, the type of consideration chosen by the holders of a majority of the outstanding Ordinary Shares); provided, however, that if the consideration received as a result of the Reorganization Event is not solely ordinary shares or common stock of the acquiring or succeeding corporation (or an affiliate thereof), the Company may, with the consent of the acquiring or succeeding corporation, provide for the consideration to be received upon the exercise or settlement of the Award to consist solely of such number of ordinary shares or common stock of the acquiring or succeeding corporation (or an affiliate thereof) that the Board determined to be equivalent in value (as of the date of such determination or another date specified by the Board) to the per share consideration received by holders of outstanding Ordinary Shares as a result of the Reorganization Event.

10.                               General Provisions Applicable to Awards

(a)                                 Transferability of Awards. Awards shall not be sold, assigned, transferred, pledged or otherwise encumbered by the person to whom they are granted, either voluntarily or by operation of law, except by will or the laws of descent and distribution applicable to such Participant or, other than in the case of an Incentive Share Option, pursuant to a qualified domestic relations order, and, during the life of the Participant, shall be exercisable only by the Participant; provided, however, that the Board may permit or provide in an Award for the gratuitous transfer of the Award by the Participant to or for the benefit of any immediate family member, family trust or other entity established for the benefit of the Participant and/or an immediate family member thereof if the Company would be eligible to use a Form S-8 under the Securities Act for the registration of the sale of the Ordinary Shares subject to such Award to such proposed transferee; provided further, that the Company shall not be required to recognize any such permitted transfer until such time as such permitted transferee shall, as a condition to such transfer, deliver to the Company a written instrument in form and substance satisfactory to the Company confirming that such transferee shall be bound by all of the terms and conditions of the Award. References to a Participant, to the extent relevant in the context, shall include references to authorized transferees. For the avoidance of doubt, nothing contained in this Section 10(a) shall be deemed to restrict a transfer to the Company.

(b)                                 Documentation. Each Award shall be evidenced in such form (written, electronic or otherwise) as the Board shall determine. Each Award may contain terms and conditions in addition to those set forth in the Plan.

(c)                                  Board Discretion. Except as otherwise provided by the Plan, each Award may be made alone or in addition or in relation to any other Award. The terms of each Award need not be identical, and the Board need not treat Participants uniformly.

(d)                                 Termination of Status. The Board shall determine the effect on an Award of the disability, death, termination or other cessation of employment, authorized leave of absence or other change in the employment or other status of a Participant and the extent to which, and the period during which, the Participant, or the Participant’s legal representative, conservator, guardian or Designated Beneficiary, may exercise rights under the Award. “Designated Beneficiary” means (i) the beneficiary designated, in a manner determined by the Board, by a Participant to receive amounts due or exercise rights of the Participant in the

event of the Participant’s death or (ii) in the absence of an effective designation by a Participant, the Participant’s estate.

(e)                                  Withholding. The Participant must satisfy all applicable Dutch, United States and other applicable national, federal, state, and local or other income, national insurance, social and employment tax withholding obligations before the Company will deliver or otherwise recognize ownership of Ordinary Shares under an Award. The Company may decide to satisfy the withholding obligations through additional withholding on salary or wages. If the Company elects not to or cannot withhold from other compensation, the Participant must pay the Company the full amount, if any, required for withholding or have a broker tender to the Company cash equal to the withholding obligations. Payment of withholding obligations is due before the Company will issue any shares on exercise, vesting or release from forfeiture of an Award or at the same time as payment of the exercise or purchase price, unless the Company determines otherwise. If provided for in an Award or approved by the Board in its sole discretion, a Participant may satisfy such tax obligations in whole or in part by delivery (either by actual delivery or attestation) of Ordinary Shares, including shares retained from the Award creating the tax obligation, valued at their Fair Market Value; provided, however, except as otherwise provided by the Board, that the total tax withholding where shares are being used to satisfy such tax obligations cannot exceed the Company’s minimum statutory withholding obligations (based on minimum statutory withholding rates for Dutch, United States and other applicable national, federal and state tax purposes, including payroll taxes, that are applicable to such supplemental taxable income). Shares used to satisfy tax withholding requirements cannot be subject to any repurchase, forfeiture, unfulfilled vesting or other similar requirements.

(f)                                   Amendment of Award. Subject to Section 11(c), the Board may amend, modify or terminate any outstanding Award, including but not limited to, substituting therefor another Award of the same or a different type, changing the date of exercise or realization, and converting an Incentive Share Option to a Share Option. The Participant’s consent to such action shall be required unless (i) the Board determines that the action, taking into account any related action, does not materially and adversely affect the Participant’s rights under the Plan or (ii) the change is permitted under Section 9.

(g)                                  Conditions on Delivery of Ordinary Shares. The Company will not be obligated to deliver any Ordinary Shares pursuant to the Plan or to remove restrictions from shares previously issued or delivered under the Plan until (i) all conditions of the Award have been met or removed to the satisfaction of the Company, (ii) in the opinion of the Company’s counsel, all other legal matters in connection with the issuance and delivery of such shares have been satisfied, including any applicable securities laws and regulations and any applicable stock exchange or stock market rules and regulations, and (iii) the Participant has executed and delivered to the Company such representations or agreements as the Company may consider appropriate to satisfy the requirements of any applicable laws, rules or regulations.

(h)                                 Acceleration. Notwithstanding Section 10(i), the Board may at any time provide that any Award shall become immediately exercisable in whole or in part, free of some or all restrictions or conditions, or otherwise realizable in whole or in part, as the case may be.

(i)                                     Minimum Vesting. Awards granted under the Plan shall vest or become exercisable over a period that is not less than one year from the date of grant. Subject to any adjustments made in accordance with Section 9(a) above, up to 5% of the Ordinary Shares subject to the share reserve set forth in Section 4(a)(1) may be granted without regard to the minimum vesting requirement of this Section 10(i).

11.                               Miscellaneous

(a)                                 No Right To Employment or Other Status. No person shall have any claim or right to be granted an Award by virtue of the adoption of the Plan, and the grant of an Award shall not be construed as giving a Participant the right to continued employment or any other relationship with the Company. The Company expressly reserves the right at any time to dismiss or otherwise terminate its relationship with a Participant free from any liability or claim under the Plan, except as expressly provided in the applicable Award. This Plan will not be considered a part of any employment agreement in force between the Participant and the Company and/or a group company. The grant of an Award does not qualify as an employment condition and shall not be included in the calculation of any severance payment or any other payments in connection with the Participant’s employment agreement or the termination thereof. The granting of an Award or the vesting thereof does not in any way affect the scope or level of the Participant’s pension rights, pension entitlements and/or of

any other entitlements vis-a-vis the Company and/or a group company. The granting of an Award is at the sole discretion of the Board and does not entitle the Participant to any future Awards.

(b)                                 No Rights As Shareholder. Subject to the provisions of the applicable Award, no Participant or Designated Beneficiary shall have any rights as a shareholder with respect to any Ordinary Shares to be distributed with respect to an Award until becoming the record holder of such shares.

(c)                                  No Repricing. Except in connection with a corporate transaction involving the Company (including, without limitation, any stock dividend, distribution (whether in the form of cash, Ordinary Shares, other securities or property), stock split, extraordinary cash dividend, recapitalization, change in control, reorganization, merger, consolidation, split-up, spin-off, combination, repurchase or exchange of Ordinary Shares or other securities, or similar transactions), the Company may not, without obtaining shareholder approval, (i) amend the terms of outstanding Options or SARs to reduce the exercise price of such outstanding Options or measurement price of such SARs, (ii) cancel outstanding Options or SARs in exchange for Options or SARs with an exercise price or measurement price, as applicable, that is less than the exercise price or measurement price of the original Options or SARs or (iii) cancel outstanding Options or SARs with an exercise price or measurement price, as applicable, above the current stock price in exchange for cash or other securities.

(d)                                 Effective Date and Term of Plan. The Plan became effective on January 9, 2014, which is the date the Plan is approved by the Company’s shareholders (the “Effective Date”). No Awards shall be granted under the Plan after the expiration of 10 years from the Effective Date, but Awards previously granted may extend beyond that date.

(e)                                  Amendment of Plan. Subject to Section 11(c), the Board may amend, suspend or terminate the Plan or any portion thereof at any time provided that no amendment that would require shareholder approval under the rules of the NASDAQ Stock Market may be made effective unless and until the Company’s shareholders approve such amendment. In addition, if at any time the approval of the Company’s shareholders is required as to any other modification or amendment under Section 422 of the Code or any successor provision with respect to Incentive Share Options, the Board may not effect such modification or amendment without such approval. Unless otherwise specified in the amendment, any amendment to the Plan adopted in accordance with this Section 11(e) shall apply to, and be binding on the holders of, all Awards outstanding under the Plan at the time the amendment is adopted, provided the Board determines that such amendment, taking into account any related action, does not materially and adversely affect the rights of Participants under the Plan. No Award shall be made that is conditioned upon shareholder approval of any amendment to the Plan unless the Award provides that (i) it will terminate or be forfeited if shareholder approval of such amendment is not obtained within no more than 12 months from the date of grant and (ii) it may not be exercised or settled (or otherwise result in the issuance of Ordinary Shares) prior to such shareholder approval.

(f)                                   Authorization of Sub-Plans. The Board may from time to time establish one or more sub-plans under the Plan for purposes of satisfying applicable securities, tax or other laws of various jurisdictions. The Board shall establish such sub-plans by adopting supplements to the Plan containing (i) such limitations on the Board’s discretion under the Plan as the Board deems necessary or desirable or (ii) such additional terms and conditions not otherwise inconsistent with the Plan as the Board shall deem necessary or desirable. All supplements adopted by the Board shall be deemed to be part of the Plan, but each supplement shall apply only to Participants within the affected jurisdiction and the Company shall not be required to provide copies of any supplement to Participants in any jurisdiction which is not the subject of such supplement.

(g)                                  Compliance with Section 409A of the Code. Except as provided in individual Award agreements initially or by amendment, if and to the extent (i) any portion of any payment, compensation or other benefit provided to a Participant pursuant to the Plan in connection with his or her employment termination constitutes “nonqualified deferred compensation” within the meaning of Section 409A of the Code and (ii) the Participant is a specified employee as defined in Section 409A(a)(2)(B)(i) of the Code, in each case as determined by the Company in accordance with its procedures, by which determinations the Participant (through accepting the Award) agrees that he or she is bound, such portion of the payment, compensation or other benefit shall not be paid before the day that is six months plus one day after the date of “separation from service” (as determined under Section 409A of the Code) (the “New Payment Date”), except as Section 409A of the Code may then permit. The aggregate of any payments that otherwise would have been paid to the Participant during the period between the date of separation from service and the New Payment Date shall be paid to the Participant in a lump sum on such New Payment Date, and any remaining payments will be paid on their original schedule.

The Company makes no representations or warranty and shall have no liability to the Participant or any other person if any provisions of or payments, compensation or other benefits under the Plan are determined to constitute nonqualified deferred compensation subject to Section 409A of the Code but do not to satisfy the conditions of that section.

(h)                                 Limitations on Liability. Notwithstanding any other provisions of the Plan, no individual acting as a supervisory director, managing director, employee or agent of the Company will be liable to any Participant, former Participant, spouse, beneficiary, or any other person for any claim, loss, liability, or expense incurred in connection with the Plan, nor will such individual be personally liable with respect to the Plan because of any contract or other instrument he or she executes in his or her capacity as a supervisory director, managing director, employee or agent of the Company. The Company will indemnify and hold harmless each supervisory director, managing director, employee or agent of the Company to whom any duty or power relating to the administration or interpretation of the Plan has been or will be delegated, against any cost or expense (including attorneys’ fees) or liability (including any sum paid in settlement of a claim with the Board’s approval) arising out of any act or omission to act concerning the Plan unless arising out of such person’s own fraud or bad faith.

(i)                                     Data Protection. The Participant hereby fully consents to the processing and transfer of all relevant data in the context of the administration of this Plan and the Award Agreement. The Participant shall keep the Company fully informed of any changes in the relevant data.

(j)                                    Share Trading, Recoupment and Other Policies. All Awards made under the Plan shall be subject to any applicable clawback and recoupment policies, share trading policies and other policies that may be implemented by the Board from time to time, including, without limitation, the Company’s right to recover Awards, Ordinary Shares or any gains upon the sale of Ordinary Shares issued under the Plan in the event of a financial restatement due in whole or in part to fraud or misconduct by one or more of the Company’s executives or in the event a Participant violates any applicable restrictive covenants in favor of the Company to which the Participant is subject.

(k)                                 Governing Law. The provisions of the Plan and all Awards made hereunder shall be governed by and interpreted in accordance with the laws of the Netherlands, excluding choice-of-law principles of the law of such state that would require the application of the laws of a jurisdiction other than the Netherlands. Any disputes arising out of or in connection with the Plan shall, to the extent permitted by law, be submitted exclusively to the competent court of Amsterdam, the Netherlands.

EX-10.1

Exhibit 10.1

Employment Agreement

dated 17 May 2021

by and between

uniQure biopharma B.V., (the Company)

Paasheuvelweg 25a

1105 BP Amsterdam

The Netherlands

and

Pierre Caloz (the Employee)

(The Company and the Employee are also referred to as Party or Parties)

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Employment Agreement

Table of Contents

1.	Condition Precedent	5
2.	Commencement Date	5
3.	Function and Job Title	5
4.	Group Structure	5
5.	Duties and Responsibilities	5
6.	Work for Third Parties	6
7.	Officer Position	6
8.	Conflict of Interests	7
9.	Gifts	7
10.	Place of Work	7
	10.1.           Remote Working	7
	10.2.           Standards for Remote Working	8
	10.3.           Equipment and Data Safety regarding Remote Working	8
	10.4.           Remote Working Allowance	9
11.	Compensation	9
	11.1.           Base Salary	9
	11.2.           Bonus	10
	11.3.           Participation Plan	10
	11.4.           Sign-On Bonus	11
	11.5.           Acknowledgements of the Employee	12
	11.6.           No other Compensation	12
	11.7.           Social security, tax and deductions	12
12.	Expenses	13
13.	Probation Period and Termination	14
	13.1.           Probation Period	14

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Employment Agreement

	13.2.           Termination	14
	13.3.           Severance Generally	14
	13.4.           Severance on a Change of Control	15
	13.5.           Release from Work (Garden Leave)	17
14.	Work Equipment and Obligation to Return Work Equipment	18
15.	Working Time	18
	15.1.           General	18
	15.2.           Additional Work	18
16.	Vacation	19
17.	Public Holidays and Short Absences	19
	17.1.           Public Holidays	19
	17.2.           Short Absences	20
18.	Incapacity to Work and Insurances	20
	18.1.           Medical Certificate	20
	18.2.           Salary in case of Employee’s Incapacity to Work	20
	18.3.           Occupational and Non-occupational Accidents	21
	18.4.           Health Insurance (Illness)	21
	18.5.           Pension Plan	21
19.	Intellectual Property Rights and Work Results	22
20.	Data Protection	23
21.	Non-Competition and Non-Solicitation	23
	21.1.General	23
	21.2.           Non-Competition during Employment	24
	21.3.           Post-Contractual Non-Competition	24
	21.4.           Post-Contractual Non-Solicitation	25
	21.5.           Penalty	25
22.	Confidentiality	26
23.	Regulations and Policies	27
24.	Miscellaneous	27
	24.1.           Entire Agreement	27

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Employment Agreement

24.2.           Severability	28
24.3.           Amendments	28
24.4.           Applicable Law	28
24.5.           Place of Jurisdiction	28
24.6.           Execution	28

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Employment Agreement

1. Condition Precedent

None.

2. Commencement Date

This Employment shall start on 17 May 2021 (the Commencement Date). It shall be concluded for an indefinite period.

3. Function and Job Title

The Employee shall assume the function as Chief Operations Officer and shall be working 100%. The function and/or the job title may be adjusted by the Company at any time to reflect current circumstances.

The detailed duties of the Employee are set out in Job Description as per Annex A, which may be unilaterally amended by the Company at any time to provide additional or different duties consistent with the customary role of a Chief Operations Officer.

4. Group Structure

The Employee acknowledges that the Company is part of a group of companies ultimately controlled by uniQure N.V. (each such company including the holding company a Group Company, together the Group). The Employee acknowledges that the Employee will need to work with and/or report to other employees and/or officers of other Group Companies. The Employee acknowledges that this does not create separate employment relationships with other Group Companies.

5. Duties and Responsibilities

It is understood that the duties and responsibilities arising out of the above function include all tasks customarily or reasonably incidental to such function and those expressly mentioned in the Job Profile.

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Employment Agreement

The Company may assign to the Employee any other, additional or new duties or responsibilities as deemed reasonable or appropriate by the Company in the course and fulfilment of its business.

The Employee undertakes to use the Employee’s entire working ability to fulfill the Employee’s contractual obligations and to loyally safeguard and foster the business and the interests of the Company. The Employee shall carefully perform all work and tasks assigned to the Employee.

6. Work for Third Parties

The Employee is not entitled to work for any third party or to engage in any gainful or unpaid employment, whether full-time or part-time, for the duration of the Employment Agreement without the prior written approval of the Company.

Membership of the boards of directors of other companies and other institutions that are related to the business purpose of the Company or otherwise affect the interests of the Company or a Group Company also requires the prior consent of the Company.

7. Officer Position

In fulfilment of the Employee’s duties, the Employee may have to act as officer, director or in any other corporate function within the Company or any Group Company. The Company may decide at its full discretion when such function shall end, and the Employee will retire from such functions and sign the necessary documentation upon first request.

The base salary as defined in Section 11.1 includes any and all remuneration for such functions and positions. In case the law provides for a mandatory remuneration the Company will decide whether such compensation shall be forwarded to the Company or be set off against the base salary as defined in Section 11.1 paid to the Employee by the Company.

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Employment Agreement

8. Conflict of Interests

The Employee shall avoid any conflict of interest and inform the Company immediately if any potential conflict of interest arises.

A conflict of interest arises especially in case of a participation in suppliers or clients of the Company or in a Group Company.

9. Gifts

In connection with the performance of his duties, the Employee is prohibited from accepting or stipulating, either directly or indirectly, any commission, reimbursement or payment, in whatever form, or gifts from third parties. The foregoing does not apply to standard promotional gifts having little monetary value.

10. Place of Work

10.1. Remote Working

The Employee is allowed to work mainly from the Employee’s home (but not in any other countries should the Employee change residence). At the request of the Company, the Employee must be present at the Company's business premises.

Nevertheless, the Employee understands and agrees that the Employee may, in the course of the Employment and where reasonably requested by the Company, be required to travel to and work in other places and countries in order to perform the Employee’s obligations and duties under the Employment Agreement. In particular, the Employee will need to be regularly present in the head offices of the Company in the Netherlands.

The Employee is obliged to track any days not worked in Switzerland, including place of work, and submit such schedule regularly to the Company upon request.

The Company will work with you to determine a reasonable budget for costs associated with travel between your home, and Company facilities

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Employment Agreement

in Amsterdam, Netherlands and Lexington, Massachusetts to include airfare, lodging and associated expenses while doing business on behalf of the Company.

In the event that you relocate to the Amsterdam, Netherlands area, the Company intends to provide executive relocation financial support.

10.2. Standards for Remote Working

The Employee confirms that the Employee has sufficient office space in the Employee’s residential home which is suitable for the performance of the Employee’s tasks. The Employee’s office at home must meet the applicable Swiss standards and legal requirements of work safety and accident prevention. The suitability of the Employee’s office at home may be verified by the Company.

The furnishing of the residential place of work with office furniture falls strictly to the Employee. ln the process, it must be ensured that the office furniture complies with the requirements of occupational ergonomics and work safety.

The Employee undertakes under no circumstances to give the impression that the Company has a branch in the Employee’s place of residence. Accordingly, the Employee may not, for example, mark the name of the Company on the Employee’s letterbox or affix a Company sign at the Employee’s place of residence.

10.3. Equipment and Data Safety regarding Remote Working

The Employee is obliged to have the necessary technical connection devices available in the Employee’s office at home.

The necessary technical work equipment for the Employee’s office at home shall not be provided by the Company, except for a laptop and mobile phone.

The work equipment provided to the Employee may be used solely for business purposes. It must neither be made accessible nor be left to third parties. The Employee is responsible for ensuring that the loaned work equipment is protected against unauthorized access by third parties. Passwords and access paths to the data network of the Company must not be disclosed to third parties. The Company may restrict the use of the communication devices provided

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to the Employee by means of appropriate technical measures and may verify usage on the basis of the monthly fees.

At the Employee’s office at home, the Employee is required to pay particular attention to the protection of data and information with respect to third parties. The Employee undertakes to observe and apply the statutory provisions and the Company's internal regulations on data protection and data security. ln particular, the Employee must ensure that third parties cannot gain access to confidential information and passwords.

The Employee agrees to store and safeguard all work equipment and documents provided in such a way that any access by third parties, including in particular persons living in the common household with the Employee, is excluded.

Confidential documents must be kept locked up or otherwise sufficiently safeguarded.

Any work equipment and documents no longer required shall be returned and/or destroyed safely by the Employee.

10.4. Remote Working Allowance

The Employee shall receive a flat allowance of CHF 275 gross per month to cover all costs of the office at home (rent, electricity, phone, internet, use of furniture, work equipment not provided, etc.) (Remote Working Allowance). During any release from work (garden leave), the Employee shall not be entitled to any Remote Working Allowance. The allowance may be deducted from the Employee’s base salary at the discretion of the Company.

11. Compensation

11.1. Base Salary

The Employee shall receive an annual base salary of CHF 463,760 gross (the Base Salary), payable in 12 monthly instalments at the end of the month to a bank or postal account to be specified by the Employee.

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11.2. Bonus

The Employee may, if applicable, participate in a bonus program of the Company or a Group Company, which the Company or the issuing Group Company shall determine at its full discretion. If no bonus program is issued, any bonus shall be determined at the discretion of the Company.

The Company may set targets for the bonus, according to which the amount of the bonus is determined. However, the Company is free to deviate at its own discretion upwards or downwards from the targets set in its final determination of the bonus. If no targets are set, any bonus shall be determined at the discretion of the Company.

Initially, the target bonus (“Target Bonus”) shall correspond to 50% of the Base Salary for a full financial year.

The Target Bonus will be prorated in the first calendar year of employment based on the total number of days worked during the calendar year.

In the event of termination of the Employment, the Employee has no entitlement to a bonus, not even on a pro rata basis. To be eligible for any bonus pursuant to this Agreement or otherwise pursuant to Employee’s employment with Employer, Employee must be in service of Employer on the date any bonus is paid.

11.3. Participation Plan

The Employee may be given the opportunity by uniQure N.V. to participate in the growth of the Group pursuant to a participation plan such as, for instance, an employee share option plan or a share plan, and as amended from time to time (the Participation Plan). It is in the full discretion of uniQure N.V. to issue and/or to unilaterally amend such Participation Plan at any time.

The Employee expressly acknowledges that the Employee does not have any right or claim under the Participation Plan against the Company, but only against uniQure N.V. or the Group Company issuing the Participation Plan. The Employee also confirms that any participation in the Participation Plan does not constitute an employment relationship with uniQure N.V. or the issuing Group Company.

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11.4. Sign-On Bonus

The Company will pay the Employee a one-off cash sign-on bonus of CHF 180,103.00 gross, payable within 30 days of the Commencement Date (the Due Date).

The Employee is not entitled to receive the sign-on bonus if (for whatever reason and howsoever caused), at the Due Date, the Employee is no longer an employee of the Company or has given or received notice to terminate the Employment.

If, within twelve months after the Commencement Date (the receipt of notice of termination is relevant in this respect), the Employment:

(a) has been terminated by the Employee;

(b) has been terminated without notice for good cause by the Company (in accordance with Article 337 CO); or

(c) has been terminated by the Company due to the Employee having given the Company good cause to do so (in accordance with Article 340c(2) CO)

the Employee shall either repay the sign-on bonus to the Company or the Company shall be entitled to set off its claim. The amount of the repayment or offsetting shall be in accordance with the following scale. Such repayment shall be made within 30 days after the date of termination of the Employment:

(a) Termination less than 12 months after the Commencement Date – 100% of sign-on bonus;

(b) Termination at least 12 months, but less than 18 months, after the Commencement Date – 50% of sign-on bonus;

(c) Termination at least 18 months, but less than 24 months, after the Commencement Date – 25% of sign-on bonus

The Employee accepts any set-off and accepts that this provision to repay or set-off the sign-on bonus to the Company is fair and reasonable under the circumstances.

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11.5. Acknowledgements of the Employee

The Employee acknowledges and agrees that any entitlements granted and payments made in addition to the Base Salary, including, but not limited to any bonuses, participations, or gratuities of the Company or any Group Company (the Additional Payments) are not part of the salary legally or contractually owed by the Company and are made at full discretion of the Company or the Group Company granting such bonus, participation or gratuity. Any Additional Payments shall not create any obligation of the Company or any Group Company to make such Additional Payments in the future and shall not create any right or claim of the Employee to such Additional Payments in the future even if paid over consecutive years and without express reservation.

11.6. No other Compensation

The Employee acknowledges and agrees that the Employee shall not be entitled to receive any other compensation or benefit of any nature from the Company except as expressly provided for in this Employment Agreement.

11.7. Social security, tax and deductions

As long as legally possible, the Employee is insured according to the Federal Laws on old age (AHVG), disability (IVG), compensation for the loss of earnings (EOG), unemployment insurance (AVIG), accident insurance (UVG), sickness benefits insurance, if any, and pension plan (BVG). From any and all gross compensation hereunder – if provided by law, regulations or policies – any portions of the Employee’s contributions to the sickness benefits insurance, if any, and withholding taxes, if any, will be deducted and withheld by the Company from the payments made to the Employee.

Any portions of the Employee’s social security contributions in accordance with the AHVG, IVG, EOG, AVIG, UVG and premiums to pension schemes (BVG) must be paid by the Employee to the respective insurance institutions in Switzerland in accordance with the separately signed agreement according to art. 21 para. 2 of Regulation (EC) No. 987/09 as per Annex B to this Employment Agreement. In accordance with said agreement is the Employee further obliged to pay the Company’s social security contributions portions in accordance with the AHVG, IVG, EOG, AVIG, UVG and premiums to pension schemes (BVG). Therefore, the Company transfers any and all compensation hereunder after having deducted

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any portions of the Employee’s contributions to the sickness benefits insurance, if any, and withholding taxes, if any. In addition, the Company transfers the Company’s social security contributions portions in accordance with the AHVG, IVG, EOG, AVIG, UVG and premiums to pension schemes (BVG) as provided by law, regulations or policies, to the Employee.

The Company reserves the right to unilaterally terminate this agreement according to art. 21 para. 2 of Regulation (EC) No. 987/09 at any time, to register as a company and thus to pay the social security contributions due (employee and employer contributions) itself. In case of such unilateral termination of said agreement, the Employee shall be notified by the Company in due time. In this case, from any and all gross compensation hereunder – if provided by law, regulations or policies – any portions of the Employee’s social security contributions in accordance with the AHVG, IVG, EOG, AVIG, UVG, sickness benefits insurance, if any, premiums to pension schemes and withholding taxes, if any, will be deducted and withheld by the Company from the payments made to the Employee.

Where it will not be possible anymore to remain in the Swiss social security system, the Employee will be insured according to the applicable social security system in accordance with the respective applicable legislation, Company policies, as well as insurance policies and regulations.

12. Expenses

The Employee shall be entitled to reimbursement by the Company of out-of-pocket business expenses reasonably incurred by the Employee during the Employment in the performance of the Employee’s duties under this Employment Agreement. The provisions in Section 10.4 shall be reserved. However, the reimbursement is subject to (i) the submission of relevant vouchers and receipts indicating the amount and purpose of the expenses, and (ii) the compliance with the reimbursement policies of the Company issued and unilaterally amended from time to time.

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13. Probation Period and Termination

13.1. Probation Period

The first 2 months of the Employment are deemed to be the probation period. During the probation period, either Party may terminate the Employment at any time with a notice period of seven days to the end of any calendar day.

13.2. Termination

After expiration of the probation period, the Employment may be terminated by either Party with a notice period of 4 months (the “Notice Period”).

Upon observance of the notice period, termination shall be effective as of the end of a calendar day and not the end of a calendar month.

The Employment is being terminated automatically at the end of the month in which the Employee reaches the retirement age according to the Federal Law on Old-age and Survivors’ Insurance (AHVG). In case of a permanent disability to work the same applies. In case of a partial permanent disability the Employment ends to the same extent as the Employee is declared disabled.

13.3. Severance Generally

lf the Employment is terminated by the Company, except

(i) in case of a summary dismissal for good cause by the Company (in accordance with Article 337 CO);

(ii) in case the Employee having given the Company good cause to do so (in accordance with Article 340c(2) CO); or

(iii) in case the Employee the Employee is in breach of any duties and failed to remedy such breach within 30 days after having been asked in writing to do so;

(iv) in case the Employee is terminated after an illness lasting for at least 6 months; or

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(v) in case of poor performance after having been put on a performance improvement plan for at least 90 days and having failed to meet the set targets,

then the Company shall grant the Employee severance pay (Severance) equal to

(i) 100% of the annual Base Salary (less any pay received during the Notice Period);

(ii) 100% an amount corresponding to the target Bonus (i.e. 50% of Base Salary) amount of the Target Bonus; plus

(iii) an amount corresponding to a prorated target Bonus amount for the year of termination as provided. The pro-rata Bonus shall be the product of the formula B x D/365 where B represents the target Bonus (i.e. 50% of the Base Salary), and D represents the number of days elapsed in the calendar year through the date of notice to the Employee),

Any severance payment is subject to the condition that the Employee signs a termination agreement with the Company including a full waiver of any other claims and the reinstatement of all restrictive covenants. For the avoidance of any doubt, no other bonus or severance shall become payable in such case. Except as expressly provided in this clause, bonus payments, if any, will not be taken into account for the calculation of any possible severance payment upon termination of the Agreement.

13.4. Severance on a Change of Control

lf the Employment is terminated by the Company within the period beginning ninety (90) days before and continuing until twelve (12) months after a Change of Control (as defined below), and subject to the conditions for Severance as per Section 13.3 above being fulfilled, the Company shall grant the Employee a severance pay (Change of Control Severance) equal to

(i) 150% of the annual Base Salary (less any pay received during the Notice Period);

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(ii) 150% an amount corresponding to the Target Bonus (i.e. 50% of Base Salary) amount of the Target Bonus; plus

(iii) an amount corresponding to a prorated Target Bonus amount for the year of termination as provided. The pro-rata Bonus shall be the product of the formula B x D/365 where B represents the Target Bonus (i.e. 50% of the Base Salary), and D represents the number of days elapsed in the calendar year through the date of notice to the Employee).

The Change of Control Severance is subject to the condition that the Employee signs a termination agreement with the Company including a full waiver of any other claims and the reinstatement of all restrictive covenants. For the avoidance of any doubt, no other bonus or severance shall become payable in such case. Except as expressly provided in this clause, bonus payments, if any, will not be taken into account for the calculation of any possible severance payment upon termination of the Agreement.

In the event of a Change of Control as defined below, the vesting conditions that may apply to any stock options, restricted shares, restricted stock units, performance stock units or other grants of equity held by Employee pursuant to this Agreement and the Company’s Amended and Restated 2014 Share Incentive Plan will be automatically waived and shall be deemed fully vested immediately prior to the Change of Control event. All Stock Options will be deemed to be fully exercisable commencing on the date of and immediately prior to the Change of Control and ending on the eighteen (18) month anniversary of the Change of Control or, if earlier, the expiration of the term of such Stock Options

For the purposes of this Employment Agreement, Change of Control shall mean the date on which any of the following events occurs:

a) any “person,” as such term is used in Sections 13(d) and 14(d) of the United States Securities Exchange Act of 1934, as amended (the Act) (other than uniQure N.V., any of its subsidiaries, or any trustee, fiduciary or other person or entity holding securities under any employee benefit plan or trust of the uniQure N.V. or any of its subsidiaries), together with all “affiliates” and “associates” (as such terms are defined in Rule 12b-2 under the Act) of such person, shall become the “beneficial owner” (as such term is defined in Rule 13d-3 under the Act), directly or indirectly, of securities of the uniQure N.V. representing forty (40) percent or more

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of the combined voting power of uniQure N.V.’s then outstanding securities having the right to vote in an election of the Board (“Voting Securities”) (in such case other than as a result of an acquisition of securities directly from the uniQure N.V.); or

b) a majority of the members of the Board is replaced during any 12-month period by directors whose appointment or election is not endorsed by a majority of the members of the Board before the date of the appointment or election; or

c) the consummation of (i) any consolidation or merger of uniQure N.V. where the stockholders of uniQure N.V., immediately prior to the consolidation or merger, would not, immediately after the consolidation or merger, beneficially own (as such term is defined in Rule 13d-3 under the Act), directly or indirectly, shares representing in the aggregate more than fifty (50) percent of the voting shares of uniQure N.V. issuing cash or securities in the consolidation or merger (or of its ultimate parent corporation, if any), or (ii) any sale or other transfer (in one transaction or a series of transactions contemplated or arranged by any party as a single plan) of all or substantially all of the assets of uniQure N.V.

13.5. Release from Work (Garden Leave)

The Company may at any time (including during the Notice Period) and with immediate effect release the Employee from the duty to work. In such case, the Employee continues to be paid the Base Salary. Vacation, any overtime and time compensation entitlements, if any, shall be offset against the time of release from work. The Company may set forth further conditions applying to the release from duties.

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14. Work Equipment and Obligation to Return Work Equipment

The Company shall provide the Employee with the necessary work equipment such as laptops or mobile phones. The work equipment shall remain the property of the Company and the Company shall have the right to replace and/or reclaim the work equipment at any time.

At the Company’s first request, but no later than upon termination of this Employment Agreement for any reason, the Employee shall return to the Company everything the Employee produced in the course of the Employee’s work for the Company, everything which was given to the Employee throughout the course of this Employment and everything which otherwise fell into the Employee’s possession. The obligation to return work equipment includes in particular but is not limited to keys, mobile phones, laptops, badges as well as data carriers and records of any kind, including any copies. Any possible retention right of the Employee is explicitly waived.

15. Working Time

15.1. General

The weekly working time depends on the needs to perform the position successfully but is at least 40 hours per week on an average basis (100% position).

15.2. Additional Work

The Employee shall work overtime, if this is necessary to fulfil the Employee’s duties under this Employment Agreement. Considering the Employee’s independent position and duties the Swiss Labour Act is not applicable to the Employment. The Employee shall therefore have no entitlement to additional compensation for any such extra work (overtime, extra hours, Sunday work, work on public holidays, or night work). All such extra and overtime work is already compensated by the Base Salary and the vacation days exceeding the statutory minimum.

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16. Vacation

The Employee is entitled to 30 business days of vacation per calendar year (for a 100% stint). The 10 additional, days of vacation granted exceeding the statutory minimum entitlement of 20 days shall be granted expressly as compensation for any overtime worked and may be offset against any time off entitlements.

The Company has the right to determine by giving one (1) month advance notice when the Employee shall take vacation days. In exceptional situations, this advance notice period is shortened to up to one week. Nevertheless, the Company will consider wishes of the Employee. If the Employee requests to take vacation, the Employee shall, reasonably prior to the intended vacation, inform the responsible executive. In any event the Employee shall provide for suitable internal representation during the Employee’s vacation.

For the year in which the Employment begins or ends, the vacation entitlement is calculated pro rata temporis.

It is the Employee’s duty to refund to the Company any vacation salary received for vacation days in excess of the vacation entitlement of the Employee.

The Employee shall take vacation days within the calendar year for which such entitlement accrues. The Employee shall not, without prior consultation and approval of the Company and/or the responsible executive or otherwise as allowed pursuant to Company policy, roll such days over to the subsequent calendar year.

17. Public Holidays and Short Absences

17.1. Public Holidays

The Employee is not obliged to work on federal and cantonal public holidays at the primary place of work in Switzerland. The Employee is not entitled to any compensation whether in cash or in kind for such public holidays when such public holidays are on weekends.

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17.2. Short Absences

The Employee shall, upon request, be granted the usual hours or days off without deduction from the salary, provided that they necessarily fall within working hours. The extent of such absences shall be determined in accordance with the Company's current practice.

Such short absences shall not be grounds for a deduction of the Employee’s entitlements to the Base Salary or vacation days, unless the absence exceeds the time period as set forth above.

Such absences shall not be grounds for a deduction of the Employee’s entitlements to the Base Salary or vacation days, unless the absence exceeds the time period as set forth above.

18. Incapacity to Work and Insurances

The Employee shall notify the Company immediately about any incapacity to work and its probable duration, stating the respective reasons.

18.1. Medical Certificate

If the Employee's incapacity to work due to illness or accident exceeds 3 business days, the Employee shall without request by the Company furnish a medical certificate in an ongoing Employment. The Company reserves the right to request a medical certificate even in the event of a shorter duration of incapacity to work. If the Employment has been terminated, the Employee shall in any case be obliged to furnish a medical certificate to the Company from the first day of incapacity to work. In all cases of illness and accident, the Company is entitled to ask the Employee to be examined by an independent medical examiner at the Company’s expense.

18.2. Salary in case of Employee’s Incapacity to Work

If the Employee is prevented from work due to illness or accident, the Company shall continue to pay the remuneration hereunder in accordance with the law (Berne scale) unless a daily sickness benefits insurance exists.

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In case a daily sickness benefits insurance exists, the Employee will receive the benefits due under such insurance in accordance with the corresponding insurance policy and the respective Company regulations and policies, as amended from time to time. The Employee will be notified accordingly by the Company in such case.

In any case, continued salary payment obligation of the Company ceases at the end of the employment relationship.

18.3. Occupational and Non-occupational Accidents

If the Employee works for the Company for an average of less than 8 hours per week, the Employee is only insured for certain medical expenses for occupational accidents. However, if the Employee works for the Company for an average of more than 8 hours per week, the Employee is insured for certain medical expenses for both occupational and non-occupational accidents. Premiums for occupational accident insurance and occupational sickness insurance are paid by the Company. Premiums for non-occupational accident insurance are paid by the Employee.

18.4. Health Insurance (Illness)

Health insurance is compulsory in Switzerland and needs to be obtained by the Employee. The Company is not providing any coverage of costs related to illness.

18.5. Pension Plan

Provided that the Employee meets the regulatory requirements, the Employee is, through a pension plan (the Pension Plan), insured against the economic consequences of retirement, disability and death.

The Employee will be covered by the Pension Plan as amended from time to time.

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19. Intellectual Property Rights and Work Results

The Company shall own all work results (including but not limited to data, know-how, documentation, concepts, drafts, inventions, works, applications, software, etc.) and all intellectual property rights therein, irrespective of their protectability under the applicable law, (including but not limited to trademarks, patents, designs, and copyrights) (the foregoing all together “Work Results”) created by the Employee in the course of the Employment (regardless of whether within or outside agreed office or workplaces and within or outside working hours).

All such Work Results shall vest automatically in the Company upon their creation. If the Company has not become the automatic owner of the Work Results and/or if the Work Results are not transferred to the Company by law, the Employee is obliged to irrevocably transfer and assign and hereby transfers and assigns said Work Results to the Company. If such Work Product cannot be transferred to the Company for any reason whatsoever, the Employee grants the Company an exclusive, worldwide, transferable, unlimited, irrevocable, sub-licensable and royalty-free license to use and exploit the Work Result.

Further, the Employee waives the right (i) to be mentioned as inventor, author or creator of a Work Result, (ii) to object to any change, modification, revision, translation or alteration of the Work Result or (iii) to determine the first publication of any Work Result.

The Employee is obliged to take all steps reasonably requested by the Company in order to fulfil the Employee’s obligations according to the above sections. This obligation continues even after termination of the Employment.

If Employee has created the Work Result with the assistance of another individual or legal entity that is not legally or contractually obliged to transfer the Work Result to the Company, the Employee ensures to take the required actions to have such third party’s share in the Work Result transferred to the Company or (if a transfer is not possible) to have it licensed to the Company according to the terms above. In addition, the Employee ensures that the third party waives the right (i) to be mentioned as inventor, author or creator of a Work Result, (ii) to object to any change, modification, revision, translation or alteration of the Work Result or (iii) to determine the first publication of any Work Result.

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Compensation for the transfer or licensing of any and all Work Results according to the above sections, in particular intellectual property rights and/or licensing rights, is included in the Employee’s Base Salary according to Section 11.1.

If a Work Result is created by the Employee in the course of the Employment but outside of the duties under the Employment Agreement, the Employee shall immediately inform the Company thereof in writing. The Company shall have the right to acquire ownership of such Work Result for a reasonable additional compensation, provided that the Company notifies the Employee in writing of its will to exercise this option within six (6) months as of the Employee’s notice of the creation of the Work Result.

20. Data Protection

The Company informs the Employee about the processing of the Employee’s personal information in a privacy notice (Personal Data).

The Company may amend the privacy notice and respective policies at any time.

21. Non-Competition and Non-Solicitation

21.1. General

The Employee acknowledges and agrees to adhere to undertakings in this Section 21 as the Company and the Group have a serious business interest in binding the Employee to the non-competition and non-solicitation undertakings, due to the fact that (i) within the organization of the Company and the Group competition-sensitive information as well as confidential information related to the Company, the Group and their clients and relations, such as but not limited to products, or research or development or commercialization of the Company and the Group (Sensitive Business Information) are available and (ii) in the position of Chief Operations Officer the Employee has access to this Sensitive Business Information and/or will become aware of this Sensitive Business Information and/or will maintain (commercial) contacts with clients, suppliers, competitors etc. Given the aforesaid considerations (i) and (ii) in this clause, combined with the education and capacities of the Employee, the Company and the Group have a well-founded fear that their business interests will be harmed

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substantially if the Employee performs competing activities as set forth in this Section 21.

21.2. Non-Competition during Employment

The Employee shall refrain from competing with the Company during the Employment, i.e. the Employee is obliged in particular not to:

– directly or indirectly, once, occasionally or professionally, under the Employee’s name or under a third-party name, on behalf of the Employee’s own or on behalf of third parties’ account compete with the Company or any Group Company; or

– engage in any way in any enterprise competing with the Company or any Group Company, and the Employee also agrees not to found, assist or promote any business being active in the same line of business as the Company or any Group Company.

Any solicitation or referral of clients and/or employees of the Company or any Group Company is prohibited.

In the event of a breach of this non-competition or non-solicitation obligation as set out in this Section 21.2, the Employee agrees to pay to the Company a penalty equal to one (1) month's Base Salary (including salary increases as granted from time to time) for each breach.

21.3. Post-Contractual Non-Competition

The Employee agrees that for a period of 12 months after termination of the Employment the Employee will neither:

– directly or indirectly, once, occasionally or professionally, under the Employee’s name or under a third-party name, on behalf of the Employee’s own or on behalf of third parties’ account compete with the Company or any Group Company; nor

– engage in any way in any enterprise competing with the Company or any Group Company, and the Employee also agrees not to found, assist or

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promote any business being active in the same line of business as the Company or any Group Company.

Particularly, any gene therapy activity (including the manufacture or development of a gene therapy) shall be considered as competing activity.

This non-competition obligation shall apply to the whole territory for which the Employee was responsible during the Employment and/or to the whole territory in which the Employee was working with products of the Company or any Group Company during the Employment, but at least to the territories and in relation to the markets of Switzerland, the European Union, Australia, and the United States.

21.4. Post-Contractual Non-Solicitation

For a period of 12 months after termination of the Employment the Employee shall abstain directly or indirectly from:

– enticing away, soliciting or interfering with any personnel from the Company or any Group Company with whom the Employee was in contact during the Employment; or

– enticing away, soliciting or interfering with clients or contacts of the Company or any Group Company with whom the Employee was in contact during the last three years prior to termination of the Employment or about whom the Employee gained knowledge during the Employment.

21.5. Penalty

If the Employee violates the post-contractual non-competition obligation according to Section 21.3, the Employee shall pay to the Company a penalty in the amount of 3 monthly Base Salaries Salary (incl. salary increases as granted from time to time) for each violation.

If the Employee violates the post-contractual non-solicitation obligation with respect to co-workers according to Section 21.4, the Employee shall pay the Company a penalty of 1 monthly Base Salary (incl. salary increases as granted from time to time) for each violation.

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If the Employee violates the post-contractual non-solicitation obligation with respect to clients according to Section 21.4, the Employee shall pay to the Company a penalty in the amount of 2 monthly Base Salaries (including salary increases as granted from time to time) for each violation.

If the breach consists in non-authorized participation in a competing company or in entering into a long-term obligation (such as an employment, service, agency or consultant contract), the penalty shall be increased by EUR 1’000 for each month or part thereof in which the breach continues (the Continuous Breach).

Multiple breaches of the obligations each trigger separate penalties, if necessary several times within one month. If individual breaches occur within a Continuous Breach, they shall be covered by the penalty which has to be paid for the Continuous Breach.

The payment of the penalty does not release the Employee from the obligation to comply with the non-competition and/or non-solicitation obligations. The Company shall be entitled to seek injunctive measures or any other type of immediate relief to stop the infringement as soon as possible, regardless of whether any penalty is offered or paid.

Further, the Company reserves the right to claim compensation for damages (in addition to the penalty or penalties).

22. Confidentiality

The Employee will have access to confidential and proprietary information relating to the business and operations of the Company, any Group Companies and their clients, in particular to business and manufacturing secrets. Such confidential and proprietary information constitutes a unique and valuable asset of the Company and any Group Companies and their acquisition required great time and expense. The disclosure or any other use of such confidential or proprietary information, other than for the sole benefit of the Company or any Group Company, would cause irreparable harm to the Company.

The Employee is under a strict duty to keep all confidential and proprietary information strictly and permanently confidential and, accordingly, shall not during the Employment or after termination of the Employment directly or indirectly for any purpose other than for the sole benefit of the Company or any

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Group Company disclose or permit to be disclosed to any third party any confidential or proprietary information without first obtaining the written consent of the responsible executive and the party concerned, if applicable, except if required to do so by law.

The Employee may not make any statement to the media, as far as the Employee is not authorized to do so by the Company and/or the responsible executive.

In the event the Employee breaches the obligations pursuant to this Section a disciplinary penalty of one monthly Base Salary (including salary increases as granted from time to time) shall be owed by the Employee to the Company for each breach.

However, the payment of the penalty does not release the Employee from further complying with the confidentiality obligation.

The Company reserves the right to claim compensation for damages in addition to the penalty.

23. Regulations and Policies

The Employee confirms that he is familiar with the regulations and policies of the Company and will comply with them at all times. The Employee acknowledges that the Company may amend existing regulations and policies from time to time and may issue new regulations and policies from time to time.

24. Miscellaneous

24.1. Entire Agreement

This Employment Agreement constitutes the complete Employment Agreement between the Parties regarding its subject matter and supersedes all prior oral and/or written agreements, representations and/or communications concerning the subject matter hereof.

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24.2. Severability

Should any of the provisions of this Employment Agreement be or become legally invalid, such invalidity shall not affect the validity of the remaining provisions. Any gap resulting from such invalidity shall be filled by a provision consistent with the spirit and purpose of the Employment Agreement. In the same way shall be proceeded if a contractual gap appears.

24.3. Amendments

Any amendments or supplementation of this Employment Agreement shall require written form and must be signed by both Parties. The written form may be dispensed only in writing.

Upon 30 day written notice to the Employee, the Company may convert any amounts owed under this agreement (including any Base Salary, allowance or other payment) from CHF (Swiss Francs) to Euros using a generally accepted exchange rate at the time of such conversion.

24.4. Applicable Law

This Employment Agreement shall be construed in accordance with and governed by Swiss law (without giving effect to the principles of conflicts of law).

24.5. Place of Jurisdiction

Any dispute arising out of or in connection with this Employment Agreement and the Employment resulting therefrom shall be exclusively submitted to and determined by the ordinary courts at the domicile of the Company, subject to mandatory places of jurisdiction.

24.6. Execution

The Parties have duly executed this Employment Agreement in two originals, each Party receiving one original.

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Signatures

uniQure biopharma B.V. (Company)

/s/ Lilly Burggraaf

Lilly Burggraaf

VP, Global Human Resources

Employee

May 17, 2021	/s/ Pierre Caloz
Date	Pierre Caloz

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Annex A - Job Description

Chief Operations Officer (COO)

Reporting to the CEO and based either in Amsterdam, Netherlands or in Lexington, MA, the Chief Operations Officer (COO) will be responsible for all cGMP relevant activities within uniQure including oversight of cGMP manufacturing, supply chain, facilities and all technical operations, including process and analytical development. While cGMP manufacturing is consolidated in the company’s Lexington, MA facility, this is an end-to-end global role that includes early product development functions located in Amsterdam, Netherlands. Areas of specific responsibility and accountability include:

◾Designing the Company’s CMC strategy and tactical execution, including oversite of cGMP manufacturing, supply chain, facilities, and all technical operations, including process and analytical development.

◾Supplying drug substance and drug product for large animal studies, clinical trials, and commercial applications in compliance with all relevant regulatory requirements.

◾Ensuring appropriate CMC capacity and delivering high quality and cost-effective development and manufacturing of the Company’s products and product candidates.

◾Investing in and maintaining the Company’s premises, utilities, and equipment.

◾Developing and directing strategies for upstream and downstream process development, analytical development, formulation development and manufacturing in alignment with Clinical, Quality, Regulatory and Commercial requirements.

◾Collaborating with Quality and other functional areas to efficiently manage technology transfer, process scale-up and design of validation strategies with regards to routine/non-routine development and cGMP activities (e.g., deviations/investigations, process improvements, change controls, shelf-life extensions, CAPAs, etc.).

◾Developing batch records, experimental data, protocols, reports, analytical methods, SOPs, etc.

◾Authoring and reviewing technical sections for company’s global regulatory submissions (INDs, IMPDs, BLAs, MAAs, Meeting Requests, Briefing Documents, responses to Health Authority questions, etc.) related to assigned programs.

◾Conducting any process verification and qualification studies.

◾Contributing to the generation and protection of company intellectual property.

◾Overseeing the development and maintenance of the budget for assigned program activities, including the preparation of cost estimates for new work.

◾Defining, implementing, maintaining, and improving processes and systems, supported by meaningful Key Performance Indicators (KPI’s).

◾Building best-in-class operations teams and leaders, including recruitment, performance management, mentorship, career development and succession planning.

◾Collaborating seamlessly with other functional disciplines to ensure day-to-day cooperation and advance the Company’s business objectives.

30 of 31

Employment Agreement

Annex B - Employer-Employee Agreement in accordance with Article 21, para. 2

31 of 31

EX-10.2

Exhibit 10.2

Jamie Brady

Chief Human Resources Officer

May 17, 2021

Pierre Caloz

RE: Side Letter Agreement re Equity Grants

Dear Pierre,

Subject to Board of Directors approval at the next regularly scheduled uniQure N.V. Board meeting after the execution of this Agreement and subject to the commencement of employment pursuant to your employment agreement with uniQure biopharma B.V., uniQure N.V. (the “Company”) agrees to grant you:

· Twenty-Five Thousand (25,000) restricted stock units of the Company (RSUs), such RSUs vesting pro-rata on each of the first three anniversaries of the grant date;

· An option to purchase Seventy-Five Thousand (75,000) ordinary shares of the Company, having an exercise price as of the closing share price on the date of grant, such option vesting over a period of four years, with one-quarter of the shares vesting on the first anniversary of the grant date and the remaining shares vesting quarterly on a pro-rata basis during the remainder of the vesting period; and

· A supplemental grant of Ten Thousand (10,000) RSUs vesting in full on the first anniversary of the grant date.

Each grant will be pursuant to uniQure N.V.’s Amended and Restated 2014 Share Incentive Plan, and the terms will otherwise reflect the standard vesting and other terms and conditions contained in the Share Incentive Plan and the Company’s standard equity grant agreements.

Such option will be approved by the Board of Directors of uniQure N.V. not later than at its next regularly scheduled meeting. If the Board fails to make the grants at such regularly scheduled meeting, it shall be deemed a good reason for termination of the employment agreement by you subject to the payment of severance.

You will be eligible for future equity grants pursuant to the Company’s policies and procedures. Any additional grants during the first year of employment (not including those provided above) will be prorated based on hire date, and all future grants of equity shall be subject to the provisions of this Agreement, including, without limitation, provisions related to change of control and termination.

Sincerely,	Agreed and Acknowledged:
/s/ Jamie Brady	/s/ Pierre Caloz
Jamie Brady	Pierre Caloz

uniQure |Paasheuvelweg 25a |1105BP Amsterdam

EX-10.3

Exhibit 10.3

EMPLOYMENT AGREEMENT PURSUANT TO SECTION 7:610 (et seq.) of the Dutch Civil Code (DCC)

June 15, 2021

Employment agreement between

(1) uniQure biopharma B.V., a company with limited liability (besloten vennootschap met beperkte aansprakelijkheid), with registered office at Amsterdam and principal place of business at Paasheuvelweg 25a, (1105 BP) Amsterdam (the Employer); and

(2) Christian Klemt (the Employee);

each “a Party”, collectively, “the Parties”.

The Parties agree as follows:

1 Commencement date Agreement and position

1.1 This employment agreement (the “Agreement”) is effective as of June 15, 2021 (the “Effective Date”).  As of the Effective Date, Employee shall continue his employment with Employer in the position of Chief Financial Officer. Employee undertakes to perform all the duties and responsibilities customary or reasonable of a Chief Financial Officer of a public company listed on the Nasdaq stock exchange as well as other duties that may reasonably be assigned to him by or on behalf of the Employer and which are related to position of Chief Financial Officer and the Employer's business. To the best of his ability in doing so, the Employee will comply with the instructions given to him by or on behalf of the Employer. Employee’s role includes, without limitation, serving as the principal financial officer of uniQure N.V. and encompasses performing duties as Chief Financial Officer for Employer and its affiliates (the “uniQure Group Companies”). Employee shall report to the Chief Executive Officer of the uniQure Group Companies.

1.2 This Agreement replaces and supersedes the prior Employment Agreement dated March 1, 2020, which is contemporaneously terminated as of the Effective Date of this Agreement. No agreements other than this Agreement govern the employment relationship between Employer and Employee. As of the Effective Date, Employee no longer holds the title of Chief Accounting Officer.

1.3 The Employer shall be entitled to assign other duties than the usual activities of the Employee, or to alter the position of the Employee if in the reasonable opinion of the Employer the business circumstances so require.

1.4 The Employee shall not be engaged in any business activity which, in the judgment of the Employer, conflicts with Employee’s ability to carry out his duties for the Employer.

1.5 The work will be performed at the office of the Employer at Paasheuvelweg 25a (1105 BP) in Amsterdam provided, however, that the Employee shall be required to travel from time to time for business purposes. The Employer reserves the right to change the location where the work is performed after consultation with the Employee.

1.6 The normal working hours for a full-time, 1.0 FTE position are 40 hours per week. The working hours are normally 8.5 hours a day with a thirty (30) minute lunch break.

Paasheuvelweg 25a	Chamber of Commerce:	1
P.O. Box 22506	34275365
1100 DA Amsterdam, NL	legal entity: uniQure biopharma B.V.
T: +31 20 240 6000	www.uniQure.com
F: +31 20 240 6020	info@uniQure.com

Exhibit 10.3

2Term and termination Agreement

2.1 The Agreement has been entered into for an indefinite period of time.

2.2 The Agreement will in any event, without notice being required, terminate as of the first day of the month following the date the Employee reaches the State pension age (AOW-gerechtigde leeftijd).

2.3 The Agreement can be terminated by each of the Parties with due observance of the statutory notice period of 4 months for the Employer and 2 months for the Employee.

2.4 Severance Payments.

2.4.1 lf the Agreement is terminated on the initiative of Employer, other than in the case of summary dismissal as referred to in article 7:677 of the Dutch Civil Code, long-term illness (article 7:669 section 3 under b Dutch Civil Code) or severely culpable acts or omissions by Employee as referred to in article 7:669 section 3 under the Dutch Civil Code, Employer shall grant the Employee severance pay equal to 100% of (i) the annual Base Salary, plus (ii) the amount of the Target Bonus (i.e., Forty percent (40%) of the annual Base Salary (as defined in Clause 3)), (hereinafter: 'Severance Pay') subject to deductions that are authorized by Employee and/or required by applicable laws and regulations.

2.4.2 lf the Agreement is terminated on the initiative of Employer within the period beginning ninety (90) days before and continuing until twelve (12) months after a Change of Control (as defined below), long-term illness (article 7:669 section 3 under b Dutch Civil Code) or severely culpable acts or omissions by Employee as referred to in article 7:669 section 3 under the Dutch Civil Code, clause 2.4.1 shall not apply and, instead, Employer shall grant the Employee severance pay equal to 150% of (i) the annual Base Salary, plus (ii) the amount of the Target Bonus (i.e., Forty percent (40%) of the annual Base Salary), (hereinafter: 'Severance Pay on a Change of Control') subject to deductions that are authorized by Employee and/or required by applicable laws and regulations.  Employer shall not owe any payments or partial payments of any kind under both clauses 2.4.1 and 2.4.2, which are mutually exclusive.

2.4.3 If Severance Pay under clause 2.4.1 or Severance Pay on a Change of Control under clause 2.4.2 is owed to the Employee, then the Employer shall grant the Employee additional severance pay equal to a prorated Target Bonus amount for the year of termination as provided in this clause (the “Pro-rata Bonus”). The Pro-rata Bonus shall be the product of the formula B x D/365 where B represents the Target Bonus (i.e., Forty percent (40%)  of the annual Base Salary)), and D represents the number of days elapsed in the calendar year through the date of the separation of Executive’s employment from the Employer.

2.4.4 lf and insofar as Employee is entitled to the transition payment as referred to in article 7:673 of the Dutch Civil Code, this transition payment shall be deemed to be factored into the Severance pay, the Severance pay on a Change of Control, and the Pro-Rata Bonus.

2.4.5 In the event of a termination under this clause 2.4, the Employer shall provide 4 months' notice to the Employee. The Employer, in its sole discretion subject to applicable law, may choose to put the Employee on garden leave at any time during the notice period. Garden leave will be considered equal to continuation of full-time employment, but the Employee will be released from his working duties. In the event that the Employee is placed on garden

Paasheuvelweg 25a	Chamber of Commerce:	2
P.O. Box 22506	34275365
1100 DA Amsterdam, NL	legal entity: uniQure biopharma B.V.
T: +31 20 240 6000	www.uniQure.com
F: +31 20 240 6020	info@uniQure.com

Exhibit 10.3

leave, the amount of the severance pay under this clause 2.4 will be reduced by an amount equivalent to the Base Salary during the Employee's garden leave, and the effective date of the garden leave shall be the date of the separation of Executive’s employment from the Employer for purposes of calculating the Pro-rata Bonus of clause 2.4.3.

2.4.6 In the event of a Change of Control as defined below, the vesting conditions that may apply to any stock options, restricted shares, restricted stock units, performance stock units or other grants of equity held by Executive pursuant to this Agreement and the Company’s Amended and Restated 2014 Share Incentive Plan will be automatically waived and shall be deemed fully vested immediately prior to the Change of Control event. All Stock Options will be deemed to be fully exercisable commencing on the date of and immediately prior to the Change of Control and ending on the eighteen (18) month anniversary of the Change of Control or, if earlier, the expiration of the term of such Stock Options.

2.4.7 For purposes of this Agreement, “Change of Control” shall mean the date on which any of the following events occurs:

2.4.7.1 any “person,” as such term is used in Sections 13(d) and 14(d) of the United States Securities Exchange Act of 1934, as amended (the “Act”) (other than uniQure N.V. (the “Company”), any of its subsidiaries, or any trustee, fiduciary or other person or entity holding securities under any employee benefit plan or trust of the Company or any of its subsidiaries), together with all “affiliates” and “associates” (as such terms are defined in Rule 12b-2 under the Act) of such person, shall become the “beneficial owner” (as such term is defined in Rule 13d-3 under the Act), directly or indirectly, of securities of the Company representing forty (40) percent or more of the combined voting power of the Company’s then outstanding securities having the right to vote in an election of the Board (“Voting Securities”) (in such case other than as a result of an acquisition of securities directly from the Company); or

2.4.7.2 a majority of the members of the Board is replaced during any 12-month period by directors whose appointment or election is not endorsed by a majority of the members of the Board before the date of the appointment or election; or

2.4.7.3 the consummation of (i) any consolidation or merger of the Company where the stockholders of the Company, immediately prior to the consolidation or merger, would not, immediately after the consolidation or merger, beneficially own (as such term is defined in Rule 13d-3 under the Act), directly or indirectly, shares representing in the aggregate more than fifty (50) percent of the voting shares of the Company issuing cash or securities in the consolidation or merger (or of its ultimate parent corporation, if any), or (ii) any sale or other transfer (in one transaction or a series of transactions contemplated or arranged by any party as a single plan) of all or substantially all of the assets of the Company.

3 Salary, bonus, equity and holiday allowance

3.1 The Employee’s annual salary will be EUR Three Hundred Twenty-Five Thousand(€ 325,000) (the “Base Salary”), including an 8% holiday allowance on the basis of full-time employment. The salary, including the holiday allowance, shall be paid in 12 equal, monthly instalments ultimately by the end of each calendar month. The Employee will work on a fulltime basis, 1,0 FTE and, therefore, the actual monthly salary is EUR 27,083,33.  In case of part time employment, all earnings will be pro-rated.

Paasheuvelweg 25a	Chamber of Commerce:	3
P.O. Box 22506	34275365
1100 DA Amsterdam, NL	legal entity: uniQure biopharma B.V.
T: +31 20 240 6000	www.uniQure.com
F: +31 20 240 6020	info@uniQure.com

Exhibit 10.3

3.2 The Employee shall be eligible for a bonus payment amounting to a target amount of Forty percent (40%) of his Base Salary (the “Target Bonus”). The Employee’s eligibility for a Target Bonus or any other bonus payment shall be dependent on the Employer guidelines and is at the full discretion of the Board.

3.3 Except as provided in clause 2.4, bonus payments, if any, will not be taken into account for the calculation of any possible severance payment upon termination of the Agreement. To be eligible for any bonus pursuant to this Agreement or otherwise pursuant to Employee’s employment with Employer, Employee must be in service of Employer on the date any bonus is paid.

4 Overtime

The Employee undertakes to work overtime at the request of the Employer. The Employer does not pay any compensation for overtime.

5 Expenses

5.1 The costs for travelling from home to office shall be compensated in accordance with the Employer policy.

5.2 To the extent that the Employer has given prior approval for business travels, the Employer shall reimburse reasonable travel and accommodation expenses relating to such business travel incurred by the Employee in the performance of his duties upon submission of all the relevant invoices and vouchers within 30 days following completion of the business travel.

6 Holidays

6.1 The Employee is entitled to 30 business days holiday per year or a pro rata portion thereof if the Agreement commences and/or terminates during the calendar year and/or the Employee works part-time.

6.2 The statutory holiday days (20 days of the 30 per year on full time employment) shall be forfeited after 6 months after the end of the year in which the holiday days were accrued.

6.3 The Employer shall determine the commencement and the end of the holiday in consultation with the Employee. The Employee shall take his holidays in the period that the activities best allow this.

7 Illness

In the event of illness in the sense of section 7:629 Dutch Civil Code, the Employee must report sick to the Employer as soon as possible, but no later than 9 a.m. on the first day of illness. The Employee undertakes to comply with the rules related to reporting and inspection in the case of illness, as adopted from time to time by the Employer.

8 Insurance

The Employer will comply with the obligations under the Dutch Health Care Insurance Act.

Paasheuvelweg 25a	Chamber of Commerce:	4
P.O. Box 22506	34275365
1100 DA Amsterdam, NL	legal entity: uniQure biopharma B.V.
T: +31 20 240 6000	www.uniQure.com
F: +31 20 240 6020	info@uniQure.com

Exhibit 10.3

9 Pension

The Employee shall be entitled to participate in the pension scheme of the Employer following Employer guidelines.

10 Confidentiality obligation

10.1 Both during the term of the Agreement and after the Agreement has been terminated for any reason whatsoever, the Employee shall not make any statements in any way whatsoever to anyone whomsoever (including other personnel of the Employer, unless these should be informed of anything in connection with the work they perform for the Employer), regarding matters, activities and interests of a confidential nature related to the business of the Employer and/or the Employer’s affiliates, of which the Employee became aware within the scope of his work for the Employer and the confidential nature of which he is or should be aware (“Confidential Information”). The Confidential Information includes, inter alia, information about the Employer’s products, processes and services, including but not limited to, information relating to research, development, inventions, manufacture, purchasing, engineering, marketing, merchandising and selling.

10.2 For all oral and written publications by the Employee, which can or could harm the interests of the Employer, prior approval from the Employer has to be obtained. This approval shall only be refused on sincere grounds based on those interests.

10.3 All information exchanged via the Employer’s email system is considered to be Employer’s proprietary information and should be taken care of accordingly.

10.4 The Employee agrees that the confidentiality obligations set forth in this clause 10 supersede the Employee’s obligations to any other company, fund or other organization with which the Employee may have a relationship (“Affiliated Entities”) and that any Confidential Information that Employee receives will only be used within the scope of his employment under this Agreement or any successor agreement with Employer and will not be used during the course of his relationship, or communicated through by any means to, any Affiliated Entity.

11 Documents

The Employee is prohibited from in any way having documents and/or correspondence and/or other information carriers and/or copies thereof in his possession that belong to the Employer and/or to the Employer’s affiliates, with the exception of the extent to which and as long as required for the performance of his activities for the Employer. In any event, the Employee is required, even without any request being made to that end, to return such documents and/or correspondence and/or other information carriers and/or copies thereof to the Employer immediately upon the end of the Agreement, or in the event the Employee is on non-active duty for any reason whatsoever.

12 Ban on ancillary jobs

During the term of the Agreement, without the prior written consent of the Employer, the Employee shall not accept any paid work or time-consuming unpaid work at or for third parties and will refrain from doing business for his own account.

Paasheuvelweg 25a	Chamber of Commerce:	5
P.O. Box 22506	34275365
1100 DA Amsterdam, NL	legal entity: uniQure biopharma B.V.
T: +31 20 240 6000	www.uniQure.com
F: +31 20 240 6020	info@uniQure.com

13 Non-competition and business relationship clause

13.1 Both during the term of the Agreement and for a period of one year after the Agreement has been terminated for any reason whatsoever, without the prior written consent of the Employer, the Employee shall not be engaged or involved or have any share in any manner whatsoever, directly or indirectly, whether on his own behalf or for third parties, in any enterprise which conducts activities in a field similar to or otherwise competing with that of the Employer and/or the Employer’s affiliates, nor act, in any manner whatsoever, directly or indirectly, whether on his own behalf or for third parties, as an intermediary in relation to such activities.

13.2 Both during the term of the Agreement and for a period of one year after the Agreement has been terminated for any reason whatsoever, without the prior written consent of the Employer, the Employee shall not perform or have performed professional services in connection with any product or research or development or commercialization that competes with products, or research or development or commercialization of Employer, directly or indirectly, whether on his own behalf or for third parties, nor enter into contacts, in that respect, directly or indirectly, whether on his own behalf or for third parties, with clients and/or relations of the Employer and/or the Employer’s affiliates and/or purchasers of products and/or services of the Employer and/or the Employer's affiliates.

13.3 Clients and/or relations of the Employer and/or the Employer’s affiliates such as set out in article 13.2 of this Agreement shall in all events mean relations of the Employer and/or the Employer’s affiliates with which the Employer has or has had (business) contact in any manner whatsoever throughout the course of, or otherwise prior to the termination of, the Agreement.

13.4 Both during the term of the Agreement and for a period of one year after the Agreement has been terminated for any reason whatsoever, without the prior written consent of the Employer, the Employee shall refrain from becoming engaged or involved in any manner whatsoever, directly or indirectly, whether on his own behalf or for third parties, in actively enticing away, taking (or causing to have taken) into employment, nor make use of, in any manner whatsoever, directly or indirectly, whether on his own behalf or for third parties, the type of work of employees or persons who in a period of one year prior to the termination of the Agreement of the Employee are or have been in the employment of the Employer and/or the Employer’s affiliates.

13.5 Employee acknowledges and agrees to adhere to this clause as the Employer has a serious business interest in binding the Employee to the non-competition and business relationship clause, due to the fact that (i) within the organization of the Employer competition-sensitive information as well as confidential information related to the Employer and its clients and relations, such as but not limited to products, or research or development or commercialization of Employer (“Sensitive Business Information”) are available and (ii) in the position of Chief Financial Officer the Employee has access to this Sensitive Business Information and/or will become aware of this Sensitive Business Information and/or will maintain (commercial) contacts with clients, suppliers, competitors etc. Given the aforesaid considerations (i) and (ii) in this clause, combined with the education and capacities of the Employee, the Employer has a well-founded fear that its business interest will be harmed substantially if the Employee performs competing activities as set forth in clauses 13.1 up to and including 13.5 of the Agreement within a period of 12 months after termination of the Agreement.

Paasheuvelweg 25a	Chamber of Commerce:	6
P.O. Box 22506	34275365
1100 DA Amsterdam, NL	legal entity: uniQure biopharma B.V.
T: +31 20 240 6000	www.uniQure.com
F: +31 20 240 6020	info@uniQure.com

13.6 13.1. to 13.5 shall not apply in the case of summary dismissal as referred to in article 7:677 of the Dutch Civil Code.

14 Intellectual and industrial property

14.1 The Employer is or will be considered to be, to the fullest extent allowed by law, the maker/producer/designer/breeder of all that which is made, created, improved, produced, designed, invented or discovered by the Employee during his activities performed for the Employer (the Works).

14.2 The Employee is obliged to fully and comprehensibly disclose all Works to the Employer in writing immediately after they are created or after the creation becomes known to the Employee, and in any case at the request of the Employer.

14.3 The Employee hereby transfers and assigns all his rights to and in connection with the Works to the Employer in advance.

14.4 The Employee is obliged, at first request of the Employer, to transfer and assign to the Employer all rights to and in connection with the Works that do not belong to the Employer by operation of law (van rechtswege), and that are not transferred to the Employer pursuant to article 14.3 of this Agreement. This concerns all rights, anywhere in the world, to and arising from or in connection with the Works. This obligation of the Employee remains in force even after the end of this Agreement.

14.5 The Employee agrees to perform, to the extent necessary and/or at the request of the Employer, such further acts as may be necessary or desirable to apply for, obtain and/or maintain protection for the Works, inter alia by means of the establishment of intellectual and industrial property rights. The Employee hereby grants permission and power of attorney to the Employer to the extent necessary to carry out every required act on behalf of the Employee to obtain protection for the Works, or to transfer the Works and any rights relating thereto, to the Employer. The Employer will compensate the reasonable costs made in respect hereof, in so far as the payment that the Employee receives pursuant to article 3.1 of this Agreement cannot be considered as compensation for such costs. This obligation of the Employee remains in force even after the end of the Agreement.

14.6 The Employee acknowledges that the payment ex article 3.1 of this Agreement includes a reasonable compensation for any possible deprivation of any intellectual and industrial property rights. To the extent legally possible, the Employee hereby waives his right to any additional compensation with respect to the Works.

15 Gifts

In connection with the performance of his duties, the Employee is prohibited from accepting or stipulating, either directly or indirectly, any commission, reimbursement or payment, in whatever form, or gifts from third parties. The foregoing does not apply to standard promotional gifts having little monetary value.

16 Penalty clause

In the event the Employee acts in violation of any of the obligations under the articles 10 through 15 of this Agreement, the Employee shall, contrary to section 7:650 paragraphs 3, 4

Paasheuvelweg 25a	Chamber of Commerce:	7
P.O. Box 22506	34275365
1100 DA Amsterdam, NL	legal entity: uniQure biopharma B.V.
T: +31 20 240 6000	www.uniQure.com
F: +31 20 240 6020	info@uniQure.com

and 5 Dutch Civil Code, without notice of default being required, forfeit to the Employer for each such violation, a penalty in the amount of EUR 10.000,00 as well as a penalty of EUR 1.000,00 for each day such violation has taken place and continues. Alternatively, the Employer will be entitled to claim full damages.

17 Transfer of an undertaking

The Employee shall remain under the obligation to adhere the set out in the articles 10 through 16 of this Agreement vis-à-vis the Employer, if the enterprise of the Employer or a part thereof is transferred to a third party within the meaning of section 7:662 and onwards Dutch Civil Code and this Agreement terminates before or at the time of such transfer, whereas in the event of continuation of the Agreement the Employee would have entered the employment of the acquirer by operation of law.

18 Other arrangements

Subject to the provisions in this Agreement, the arrangements related to employment conditions adopted by the Employer from time to time, as laid down in the Employee Handbook are applicable. A copy of these arrangements has been provided to the Employee. By signing this agreement, the Employee acknowledges to have received and understood the Employee Handbook and the Insider Trading Policy.

19 Employment costs regulation

The conditions of employment costs regulation determined by the Employer apply. In this context, the Employer reserves the right at its sole discretion to modify certain fringe benefits, without any compensation in return.

20 Amendment clause

20.1 The Employer reserves the right to unilaterally amend the Agreement and the arrangements referred to in article 18 of this Agreement if it has such a serious interest in that respect entailing that the interests of the Employee must yield to that in accordance with standards of reasonableness and fairness.

20.2 The Employer reserves the right to unilaterally amend the Agreement and the arrangements referred to in article 18 of this Agreement in the event of a relevant amendment of the law.

21 Applicable law, no collective labour agreement

21.1 This Agreement is governed by Dutch law.

21.2 The Agreement is not subject to any collective labour agreement.

Paasheuvelweg 25a	Chamber of Commerce:	8
P.O. Box 22506	34275365
1100 DA Amsterdam, NL	legal entity: uniQure biopharma B.V.
T: +31 20 240 6000	www.uniQure.com
F: +31 20 240 6020	info@uniQure.com

THIS AGREEMENT has been entered into on the date stated at the beginning of this Agreement

uniQure biopharma B.V.		Employee
	/s/ Lilly Burggraaf		/s/ Christian Klemt
By:	Lilly Burggraaf	By:	Christian Klemt
Title:	VP, Global Human Resources

Paasheuvelweg 25a	Chamber of Commerce:	9
P.O. Box 22506	34275365
1100 DA Amsterdam, NL	legal entity: uniQure biopharma B.V.
T: +31 20 240 6000	www.uniQure.com
F: +31 20 240 6020	info@uniQure.com

EX-31.1

Exhibit 31.1

Certification of Chief Executive Officer

I, Matthew Kapusta, certify that:

1.                                      I have reviewed this Quarterly Report on Form 10-Q of uniQure N.V.;

2.                                      Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;

3.                                      Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;

4.                                      The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:

(a)                                 Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;

(b)                                 Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;

(c)                                  Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation;

(d)                                 Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting;

5.                                      The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):

(a)                                 All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and

(b)                                 Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.

By:	/s/ MATTHEW KAPUSTA
	Matthew Kapusta
	Chief Executive Officer
	(Principal Executive Officer)
	July 26, 2021

EX-31.2

Exhibit 31.2

Certification of Chief Financial Officer

I, Christian Klemt, certify that:

1.                                      I have reviewed this Quarterly Report on Form 10-Q of uniQure N.V.;

2.                                      Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;

3.                                       Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;

4.                                      The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:

(a)                                 Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;

(b)                                 Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;

(c)                                  Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation;

(d)                                 Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting;

5.                                      The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):

(a)                                 All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and

(b)                                 Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.

By:	/s/ CHRISTIAN KLEMT
	Christian Klemt
	Chief Financial Officer
	(Principal Financial Officer)
	July 26, 2021

EX-32.1

Exhibit 32.1

CERTIFICATION PURSUANT TO 18 U.S.C. SECTION 1350

AS ADOPTED PURSUANT TO SECTION 906 OF THE SARBANES-OXLEY ACT OF 2002

In connection with this Quarterly Report of uniQure N.V. (the “Company”) on Form 10-Q for the period ended June 30, 2021, as filed with the Securities and Exchange Commission on the date hereof (the “Report”), the undersigned, Matthew Kapusta, Chief Executive Officer, and Christian Klemt, Chief Financial Officer of the Company, hereby certify, pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002, that:

1                                         the Report fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934; and

2                                         the information contained in the Report fairly presents, in all material respects, the financial condition and results of operations of the Company.

By:	/s/ MATTHEW KAPUSTA
	Matthew Kapusta
	Chief Executive Officer
	(Principal Executive Officer)
	July 26, 2021

By:	/s/ CHRISTIAN KLEMT
	Christian Klemt
	Chief Financial Officer
	(Principal Financial Officer)
	July 26, 2021

A signed original of this written statement required by Section 906 has been provided to uniQure N.V. and will be retained by uniQure N.V. and furnished to the SEC or its staff upon request.

XML

Document and Entity Information - shares	6 Months Ended
	Jun. 30, 2021	Jul. 21, 2021
Document and Entity Information
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Jun. 30, 2021
Document Transition Report	false
Entity File Number	001-36294
Entity Registrant Name	uniQure N.V.
Entity Incorporation, State or Country Code	P7
Entity Address, Address Line One	Paasheuvelweg 25
Entity Address, City or Town	1105 BP Amsterdam
Entity Address, Country	NL
City Area Code	31
Local Phone Number	20-240-6000
Title of 12(b) Security	Ordinary Shares, par value €0.05
Trading Symbol	QURE
Security Exchange Name	NASDAQ
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		46,073,373
Entity Central Index Key	0001590560
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2021
Document Fiscal Period Focus	Q2
Amendment Flag	false

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CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands	Jun. 30, 2021	Dec. 31, 2020
Current assets
Cash and cash equivalents	$ 677,330	$ 244,932
Accounts receivables	2,705	6,618
Prepaid expenses	10,861	4,337
Other current assets and receivables	3,328	3,024
Total current assets	694,224	258,911
Non-current assets
Property, plant and equipment, net of accumulated depreciation of $37.3 million as of June 30, 2021 and $35.2 million as of December 31, 2020, respectively	38,371	32,328
Operating lease right-of-use assets	26,782	26,086
Intangible assets, net	2,637	3,361
Goodwill	525	542
Deferred tax assets	15,965	16,419
Other non-current assets	5,760	2,748
Total non-current assets	90,040	81,484
Total assets	784,264	340,395
Current liabilities
Accounts payable	16,817	3,772
Accrued expenses and other current liabilities	23,526	18,038
Current portion of operating lease liabilities	5,752	5,524
Total current liabilities	46,095	27,334
Non-current liabilities
Long-term debt	70,780	35,617
Operating lease liabilities, net of current portion	30,534	30,403
Other non-current liabilities	3,247	3,136
Total non-current liabilities	104,561	69,156
Total liabilities	150,656	96,490
Commitments and contingencies
Shareholders' equity
Ordinary shares, 0.05 par value: 80,000,000 shares authorized as of June 30, 2021 and 60,000,000 shares authorized as of December 31, 2020 and 46,050,250 and 44,777,799 ordinary shares issued and outstanding as of June 30, 2021 and December 31, 2020, respectively	2,788	2,711
Additional paid-in-capital	1,062,234	1,016,018
Accumulated other comprehensive (loss) / income	(4,595)	9,907
Accumulated deficit	(426,819)	(784,731)
Total shareholders' equity	633,608	243,905
Total liabilities and shareholders' equity	$ 784,264	$ 340,395

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CONSOLIDATED BALANCE SHEETS (Parenthetical) $ in Millions	Jun. 30, 2021 USD ($) shares	Jun. 30, 2021 € / shares	Dec. 31, 2020 USD ($) shares	Dec. 31, 2020 € / shares
CONSOLIDATED BALANCE SHEETS
Accumulated depreciation | $	$ 37.3		$ 35.2
Ordinary shares, par value (in euros per share) | € / shares		€ 0.05		€ 0.05
Ordinary shares, authorized	80,000,000		60,000,000
Ordinary shares, issued	46,050,250		44,777,799
Ordinary shares, outstanding	46,050,250		44,777,799

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CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020
Total revenues	$ 463,868	$ 1,535	$ 464,322	$ 1,639
Operating expenses:
Cost of contract revenues	(23,178)		(23,178)
Research and development expenses	(32,747)	(28,401)	(64,777)	(54,414)
Selling, general and administrative expenses	(17,299)	(11,511)	(30,300)	(20,583)
Total operating expenses	(73,224)	(39,912)	(118,255)	(74,997)
Other income	7,590	669	7,942	1,526
Other expense	(226)	(500)	(459)	(839)
Income / (loss) from operations	398,008	(38,208)	353,550	(72,671)
Interest income	37	81	77	903
Interest expense	(1,902)	(970)	(3,453)	(1,945)
Foreign currency gains / (losses), net	6,583	(3,645)	11,209	957
Other non-operating gains, net		191		2,206
Income / (loss) before income tax expense	402,726	(42,551)	361,383	(70,550)
Income tax expense	(3,258)	0	(3,471)	0
Net income / (loss)	399,468	(42,551)	357,912	(70,550)
Other comprehensive (loss) / gain:
Foreign currency translation adjustments	(6,942)	4,647	(14,502)	(630)
Total comprehensive gain / (loss)	$ 392,526	$ (37,904)	$ 343,410	$ (71,180)
Earnings per ordinary share - basic
Basic net income / (loss) per ordinary share	$ 8.68	$ (0.96)	$ 7.82	$ (1.59)
Earnings per ordinary share - diluted
Diluted net income / (loss) per ordinary share	$ 8.51	$ (0.96)	$ 7.67	$ (1.59)
Weighted average shares - basic	46,037,900	44,387,463	45,754,766	44,333,460
Weighted average shares - diluted	46,929,870	44,387,463	46,678,835	44,333,460
License revenues
Total revenues	$ 462,400		$ 462,400
License revenues from related party
Total revenues		$ 1,530		$ 1,577
Collaboration revenues
Total revenues	$ 1,468		$ 1,922
Collaboration revenues from related party
Total revenues		$ 5		$ 62

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CONSOLIDATED STATEMENTS OF SHAREHOLDERS' EQUITY - USD ($) $ in Thousands	Ordinary shares	Additional paid-in capital	Accumulated other comprehensive (loss)/income	Accumulated deficit	Total
Beginning balance at Dec. 31, 2019	$ 2,651	$ 986,803	$ (6,689)	$ (659,707)	$ 323,058
Beginning balance (in shares) at Dec. 31, 2019	43,711,954
Increase (decrease) in shareholders' equity
Income (Loss) for the period				(70,550)	(70,550)
Other comprehensive Income (loss)			(630)		(630)
Exercise of share options	$ 12	3,390			3,402
Exercise of share options (in shares)	203,940
Restricted and performance share units distributed during the period	$ 29	(29)
Restricted and performance share units distributed during the period (in shares)	525,506
Share-based compensation expense		10,078			10,078
Issuance of ordinary shares relating to employee stock purchase plan		147			147
Issuance of ordinary shares relating to employee stock purchase plan (in shares)	3,005
Ending balance at Jun. 30, 2020	$ 2,692	1,000,389	(7,319)	(730,257)	265,505
Ending balance (in shares) at Jun. 30, 2020	44,444,405
Beginning balance at Mar. 31, 2020	$ 2,683	992,136	(11,966)	(687,706)	295,147
Beginning balance (in shares) at Mar. 31, 2020	44,299,596
Increase (decrease) in shareholders' equity
Income (Loss) for the period				(42,551)	(42,551)
Other comprehensive Income (loss)			4,647		4,647
Exercise of share options	$ 9	2,461			2,470
Exercise of share options (in shares)	139,178
Restricted and performance share units distributed during the period (in shares)	4,427
Share-based compensation expense		5,723			5,723
Issuance of ordinary shares relating to employee stock purchase plan		69			69
Issuance of ordinary shares relating to employee stock purchase plan (in shares)	1,204
Ending balance at Jun. 30, 2020	$ 2,692	1,000,389	(7,319)	(730,257)	265,505
Ending balance (in shares) at Jun. 30, 2020	44,444,405
Increase (decrease) in shareholders' equity
Accumulated deficit					(784,731)
Beginning balance at Dec. 31, 2020	$ 2,711	1,016,018	9,907	(784,731)	$ 243,905
Beginning balance (in shares) at Dec. 31, 2020	44,777,799				44,777,799
Increase (decrease) in shareholders' equity
Income (Loss) for the period				357,912	$ 357,912
Other comprehensive Income (loss)			(14,502)		(14,502)
Issuance of ordinary shares	$ 55	29,509			29,564
Issuance of ordinary shares (in shares)	921,730
Tax benefit past share issuance cost		2,977			2,977
Exercise of share options	$ 5	906			911
Exercise of share options (in shares)	71,976
Restricted and performance share units distributed during the period	$ 17	(17)
Restricted and performance share units distributed during the period (in shares)	277,571
Share-based compensation expense		12,792			12,792
Issuance of ordinary shares relating to employee stock purchase plan		49			49
Issuance of ordinary shares relating to employee stock purchase plan (in shares)	1,174
Ending balance at Jun. 30, 2021	$ 2,788	1,062,234	(4,595)	(426,819)	$ 633,608
Ending balance (in shares) at Jun. 30, 2021	46,050,250				46,050,250
Beginning balance at Mar. 31, 2021	$ 2,780	1,049,850	2,347	(826,287)	$ 228,690
Beginning balance (in shares) at Mar. 31, 2021	45,924,729
Increase (decrease) in shareholders' equity
Income (Loss) for the period				399,468	399,468
Other comprehensive Income (loss)			(6,942)		(6,942)
Issuance of ordinary shares	$ 3	1,862			1,865
Issuance of ordinary shares (in shares)	61,845
Tax benefit past share issuance cost		2,977			2,977
Exercise of share options	$ 4	515			519
Exercise of share options (in shares)	55,194
Restricted and performance share units distributed during the period	$ 1	(1)
Restricted and performance share units distributed during the period (in shares)	8,482
Share-based compensation expense		7,031			7,031
Ending balance at Jun. 30, 2021	$ 2,788	$ 1,062,234	$ (4,595)	$ (426,819)	$ 633,608
Ending balance (in shares) at Jun. 30, 2021	46,050,250				46,050,250
Increase (decrease) in shareholders' equity
Accumulated deficit					$ (426,819)

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CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020
Cash flows from operating activities
Net income (loss)	$ 357,912	$ (70,550)
Adjustments to reconcile net income / (loss) to net cash generated from / used in operating activities:
Depreciation and amortization	3,667	3,505
Share-based compensation expense	12,792	10,078
Change in fair value of derivative financial instruments		(2,206)
Unrealized foreign exchange gains, net	(12,279)	(908)
Other non-cash items, net	539	(1,600)
Changes in operating assets and liabilities:
Accounts receivable, prepaid expenses, and other current assets and receivables	(3,355)	1,050
Accounts payable	10,444	(981)
Accrued expenses, other liabilities, and operating leases	5,486	(1,332)
Net cash generated from / (used) in operating activities	375,206	(62,944)
Cash flows from investing activities
Purchases of intangible assets		(2,214)
Purchases of property, plant and equipment	(6,191)	(2,392)
Net cash used in investing activities	(6,191)	(4,606)
Cash flows from financing activities
Proceeds from loan increment, net of debt issuance costs	34,603
Proceeds from issuance of ordinary shares	30,899
Share issuance costs from issuance of ordinary shares	(1,334)
Proceeds from issuance of ordinary shares related to employee stock option and purchase plans	960	3,549
Net cash generated from financing activities	65,128	3,549
Currency effect on cash, cash equivalents and restricted cash	(1,665)	223
Net increase / (decrease) in cash, cash equivalents and restricted cash	432,478	(63,778)
Cash, cash equivalents and restricted cash at beginning of period	247,680	380,726
Cash, cash equivalents and restricted cash at the end of period	680,158	316,948
Cash and cash equivalents	677,330	314,265
Restricted cash related to leasehold and other deposits	2,828	2,683
Total cash, cash equivalents and restricted cash	680,158	316,948
Supplemental cash flow disclosures:
Cash paid for interest	(2,943)	(2,545)
Non-cash increase in accounts payables and accrued expenses and other current liabilities related to purchases of intangible assets and property, plant, and equipment	$ 3,488	$ 1,021

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General business information

6 Months Ended

Jun. 30, 2021

General business information

General business information

1General business information

uniQure (the “Company”) was incorporated on January 9, 2012 as a private company with limited liability (besloten vennootschap met beperkte aansprakelijkheid) under the laws of the Netherlands. The Company is a leader in the field of gene therapy and seeks to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results. The Company’s business was founded in 1998 and was initially operated through its predecessor company, Amsterdam Molecular Therapeutics (AMT) Holding N.V (“AMT”). In 2012, AMT undertook a corporate reorganization, pursuant to which uniQure B.V. acquired the entire business and assets of AMT and completed a share-for-share exchange with the shareholders of AMT. Effective February 10, 2014, in connection with its initial public offering, the Company converted into a public company with limited liability (naamloze vennootschap) and changed its legal name from uniQure B.V. to uniQure N.V.

The Company is registered in the trade register of the Chamber of Commerce (Kamer van Koophandel) in Amsterdam, the Netherlands under number 54385229. The Company’s headquarters are in Amsterdam, the Netherlands, and its registered office is located at Paasheuvelweg 25, Amsterdam 1105 BP, the Netherlands and its telephone number is +31 20 240 6000.

The Company’s ordinary shares are listed on the Nasdaq Global Select Market and trade under the symbol “QURE”.

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Summary of significant accounting policies

6 Months Ended

Jun. 30, 2021

Summary of significant accounting policies

Summary of significant accounting policies

2Summary of significant accounting policies

2.1Basis of preparation

The Company prepared these unaudited consolidated financial statements in compliance with generally accepted accounting principles in the United States of America (“U.S. GAAP”) and applicable rules and regulations of the Securities and Exchange Commission (“SEC”) regarding interim financial reporting. Any reference in these notes to applicable guidance is meant to refer to authoritative U.S. GAAP as found in the Accounting Standards Codification (“ASC”) and Accounting Standards Update (“ASU”) of the Financial Accounting Standards Board (“FASB”).

The unaudited consolidated financial statements are presented in U.S. dollars, except where otherwise indicated. Transactions denominated in currencies other than U.S. dollars are presented in the transaction currency with the U.S. dollar amount included in parenthesis, converted at the foreign exchange rate as of the transaction date.

2.2Unaudited interim financial information

The interim financial statements and related disclosures are unaudited, have been prepared on the same basis as the annual financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for a fair statement of the financial position, results of operations and changes in financial position for the period presented.

Certain information and footnote disclosures normally included in annual financial statements prepared in accordance with U.S. GAAP have been omitted. The results of operations for the six months ended June 30, 2021, are not necessarily indicative of the results to be expected for the full year ending December 31, 2021 or for any other future year or interim period. The accompanying financial statements should be read in conjunction with the audited financial statements and the related notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, filed with the SEC on March 1, 2021.

2.3Use of estimates

The preparation of the financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities at the date of the financial statements and reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates.

2.4Accounting policies

The principal accounting policies applied in the preparation of these unaudited consolidated financial statements are described in the Company’s audited financial statements as of and for the year ended December 31, 2020, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, filed with the SEC on March 1, 2021. There have been no material changes in the Company’s significant accounting policies during the six months ended June 30, 2021 except as noted below.

The Company determined its revenue recognition policies related to the CSL Behring Agreement (defined below) when the CSL Behring Agreement became fully effective on May 6, 2021. Refer to Note 3 (“Collaboration arrangements and concentration of credit risk”) for further detail.

The Company for the first time recognized net income in the three and six months ended June 30, 2021. Net income / (loss) per ordinary share was calculated in accordance with ASC 260, Earnings Per Share. Refer to Note 11 (“Basic and diluted earnings per share”) for further detail.

2.5Recent accounting pronouncements

There have been no new accounting pronouncements or changes to accounting pronouncements during the six months ended June 30, 2021, as compared to the recent accounting pronouncements described in Note 2.3.22 of the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, which could be expected to materially impact the Company’s unaudited consolidated financial statements.

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Collaboration arrangements and concentration of credit risk

6 Months Ended

Jun. 30, 2021

Collaboration arrangements and concentration of credit risk

Collaboration arrangements and concentration of credit risk

3 Collaboration arrangements and concentration of credit risk

CSL Behring collaboration

On June 24, 2020, (“Signing”), uniQure biopharma B.V., a wholly-owned subsidiary of uniQure N.V., entered into a commercialization and license agreement (the “CSL Behring Agreement”) with CSL Behring LLC, (“CSL Behring”), pursuant to which CSL Behring received exclusive global rights to etranacogene dezaparvovec, the Company’s investigational gene therapy for patients with hemophilia B, (the “Product”). On May 6, 2021, the CSL Behring Agreement became fully effective (“Closing”) upon the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 on May 5, 2021.

Pursuant to the CSL Behring Agreement, the Company has received a $450.0 million upfront cash payment and $12.4 million in other payments related to the transfer of the license, during the three and six months ended June 30, 2021. The Company is eligible to receive up to $1.6 billion in additional payments based on regulatory and commercial milestones. The CSL Behring Agreement also provides that the Company will be eligible to receive tiered double-digit royalties in a range of up to a low-twenties percent of net sales of the Product based on sales thresholds.

On Signing, the Company and CSL Behring entered into a development and commercial supply agreement, pursuant to which, among other things, the Company will supply the Product to CSL Behring at an agreed-upon price commensurate with the stand-alone selling price (“SSP”). The Company will be responsible for supplying development and commercial Product until such time that these capabilities may be transferred to CSL Behring or a designated contract manufacturing organization. The Company will be completing the HOPE-B clinical trial and the validation of the manufacturing process on behalf of CSL Behring, as well as provide further development services if requested by CSL Behring. Activities related to on-demand development services as well as activities related to the completing the HOPE-B clinical trial will be reimbursed by CSL Behring at an agreed full-time-employee rate (“FTE-rate”) and CSL Behring will also reimburse agreed third-party expenses incurred in relation to performing these activities. The validation of the manufacturing process as well as the development and validation of a next generation manufacturing process (“Manufacturing Development Services”) will be reimbursed through a future milestone payment. If completed after certain contractually agreed dates, the milestone payment will be reduced in accordance with a contractually agreed mechanism.

The Company concluded that CSL Behring is a customer in accordance with Topic 606.

The Company identified two material performance obligations related to the CSL Behring Agreement:

(i) Sale of the exclusive global rights to the Product (“License”); and

(ii) Generate information to support the regulatory approval of the current and next generation manufacturing process of Product and to provide any such information generated to CSL Behring (“Manufacturing Development”).

These performance obligations are considered distinct from one another, as CSL Behring can benefit from the identified service either on its own or together with other resources that are readily available to CSL Behring, and as the performance obligation is separately identifiable from other performance obligations in the CSL Behring Agreement. The Company continued to develop the Product between Signing and Closing and performed certain reimbursable activities to fulfill the transfer of the global rights (“Additional Covenants” and together with the License the “License Sale”). The Additional Covenants are not considered distinct from the performance obligation to sell the license to CSL Behring as CSL Behring could not benefit from the Additional Covenants on their own, or have these activities be performed with readily available resources.

The Company determined that the fixed upfront payment of $450.0 million and the $12.4 million that the Company received in relation to the Additional Covenants should be allocated to the License Sale. In addition, the Company concluded that variable milestone payments, sales milestone payments and royalties should be allocated to the License Sale performance obligation as well. The Company determined that the sale of the License was completed on May 6, 2021, when it transferred the License and CSL Behring assumed full responsibility for the development and commercialization of the Product. At Closing, the Company evaluated the amount of potential payments and the likelihood that the payments will be received. The Company utilized the most likely amount method to estimate the variable consideration to be included in the transaction price. Since the Company cannot control the achievement of regulatory and first commercial sales milestones, the Company concluded that the potential payments are constrained as of Closing. The Company determined that it will recognize revenue related to these payments only to the extent that it becomes probable that no significant reversal of recognized cumulative revenue will occur thereafter. The Company will include payments related to sales milestones in the transaction price when their achievement becomes probable, and it will include royalties on the sale of Product once these have been earned. The Company recognized $462.4 million of revenues related to the License Sale in the three and six months ended June 30, 2021. The costs associated with fulfilling this performance obligation are presented as “Cost of contract revenue” with the exception of the cost incurred prior to the Closing, which had been presented as “Research and development expenses”.

The Company determined that the variable milestone payment related to Manufacturing Development Services should be allocated to the Manufacturing Development performance obligation. The Company concluded that this milestone payment represents the SSP of the services based on the estimated cost of providing the services including a reasonable margin. The services related to Manufacturing Development will be provided between Closing and the completion of an agreed manufacturing development plan. The variable consideration will be reduced if the Company does not complete the development by pre-agreed dates. The Company utilized the most likely amount method to estimate the variable consideration to be included in the transaction price. Completion of Manufacturing Development is partially dependent on the timing of regulatory submissions by CSL Behring as well as regulatory approvals of the developed manufacturing processes. Since the Company cannot control the timing or outcome of any regulatory decisions, the Company concluded that it would recognize revenue related to this payment when it becomes probable that the milestone has been achieved. The Company did not recognize any revenue related to Manufacturing Development during the three and six months ended June 30, 2021.

The Company recognized $0.4 million of collaboration revenue in the three and six months ended June 30, 2021, compared to nil in the same periods in the prior year.  The Company generates such collaboration revenue from services rendered in relation to completing the HOPE-B clinical trial on behalf of CSL Behring. CSL Behring may request additional development services or to request the Company to support the transfer of manufacturing to a party designated by CSL Behring. These collaboration services will be reimbursed at the pre-agreed FTE-rate. The Company concluded that these rights at Closing do not represent material rights.

As of June 30, 2021, the Company has an accounts receivable of $1.3 million from CSL Behring. The Company collected the $2.1 million of accounts receivable related to reimbursable contract fulfillment costs that was outstanding as of December 31, 2020.

Bristol-Myers Squibb collaboration

In May 2015, the Company and Bristol-Myers Squibb (“BMS”) entered into a collaboration and license agreement and various related agreements with BMS (“BMS CLA”).

The initial four-year research term under the collaboration terminated on May 21, 2019. On December 1, 2020, the Company and BMS amended the BMS CLA (“amended BMS CLA”). Under the amended BMS CLA, BMS is limited to four Collaboration Targets. BMS may until November 30, 2021 replace up to two of these four Collaboration Targets with up to two new targets in the field of cardiovascular disease. The Company continues to be eligible to receive research, development, and regulatory milestone payments of up to $217.0 million for each Collaboration Target if defined milestones are achieved.

For as long as any of the four Collaboration Targets are being advanced, BMS may place a purchase order to be supplied with research, clinical and commercial supplies. Subject to the terms of the amended BMS CLA, BMS has the right to terminate the research, clinical and commercial supply relationships, and has certain remedies for failures of supply, up to and including technology transfer for any such failure that otherwise cannot be reasonably resolved. Both BMS and the Company may agree to a technology transfer of manufacturing capabilities pursuant to the terms of the amended BMS CLA.

The amended BMS CLA does not extend the initial four-year research term. BMS may place purchase orders to provide limited services primarily related to analytical and development efforts in respect of the four Collaboration Targets. BMS may request such services for a period not to exceed the earlier of (i) the completion of all activities under a Research Plan and (ii) either (A) three years after the last replacement target has been designated by BMS during the one-year replacement period ending on November 30, 2021, or (B) November 30, 2023 if no replacement targets are designated. BMS continues to reimburse the Company for these services.

The Company evaluated the impact of the amended BMS CLA in relation to its performance obligation to provide access to BMS to its technology and know-how in the field of gene therapy and to participate in joint steering committee and other governing bodies (“License Revenue”).

The Company determined that its remaining performance obligation under the amended BMS CLA was immaterial and recognized the remaining balance of unrecognized License Revenue as of November 30, 2020. The Company includes variable consideration related to any research, development, and regulatory milestone payments, in the transaction price once it is considered probable that including these payments in the transaction price would not result in the reversal of cumulative revenue recognized. Due to the significant uncertainty surrounding the development of gene-therapy product candidates and the dependence on BMS’s performance and decisions, the Company does not generally consider this probable and did not record any License Revenue during the six months ended June 30, 2021.

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Fair value measurement

6 Months Ended

Jun. 30, 2021

Fair value measurement

Fair value measurement

4  Fair value measurement

The Company measures certain financial assets and liabilities at fair value, either upon initial recognition or for subsequent accounting or reporting. U.S. GAAP requires disclosure of methodologies used in determining the reported fair values, and establishes a hierarchy of inputs used when available. The three levels of the fair value hierarchy are described below:

● Level 1 – Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company can access at the measurement date.

● Level 2 – Valuations based on quoted prices for similar assets or liabilities in markets that are not active or models for which the inputs are observable, either directly or indirectly.

● Level 3 – Valuations that require inputs that reflect the Company’s own assumptions that are both significant to the fair value measurement and are unobservable.

To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized as Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement.

The carrying amount of cash and cash equivalents, accounts receivable, prepaid expenses, other assets, accounts payable, accrued expenses and other current liabilities reflected in the Consolidated balance sheets approximate their fair values due to their short-term maturities.

The following table sets forth the Company’s assets and liabilities that are required to be measured at fair value on a recurring basis as of June 30, 2021, and December 31, 2020:

	Quoted prices in active markets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)		Total		Classification in Consolidated balance sheets
		(in thousands)
At December 31, 2020
Assets:
Cash, cash equivalents and restricted cash	$	247,680	$	—	$	—	$	247,680	Cash and cash equivalents; other non-current assets
Total assets	$	247,680	$	—	$	—	$	247,680
Liabilities:
Derivative financial instruments		—		—		2,645		2,645	Other non-current liabilities
Total liabilities	$	—	$	—	$	2,645	$	2,645
At June 30, 2021
Assets:
Cash, cash equivalents and restricted cash	$	680,158	$	—	$	—	$	680,158	Cash and cash equivalents; other non-current assets
Total assets	$	680,158	$	—	$	—	$	680,158
Liabilities:
Derivative financial instruments	$	—	$	—	$	2,645	$	2,645	Other non-current liabilities
Total liabilities	$	—	$	—	$	2,645	$	2,645

The Company recognized no gains or losses related to changes in the fair market value of derivative financial instruments during the three and six months ended June 30, 2021.

Derivative financial instruments

The Company issued derivative financial instruments related to its collaboration with BMS.

In 2015, the Company granted BMS two warrants that were subsequently terminated in connection with the amendment to the BMS CLA on December 1, 2020.

On December 1, 2020, the Company and BMS agreed that upon the consummation of a change of control transaction of uniQure that occurs prior to December 1, 2026 or BMS’ delivery of a target cessation notice for all four Collaboration Targets, the Company (or its third party acquirer) shall pay to BMS a one-time, non-refundable, non-creditable cash payment of $70.0 million, provided that (x) if $70.0 million is greater than five percent (5.0%) of the net proceeds (as contractually defined) from such change of control transaction, the payment shall be an amount equal to five percent of such net proceeds, and (y) if $70.0 million is less than one percent of such net proceeds, the change of control payment shall be an amount equal to one percent of such net proceeds (“CoC-payment”). The Company has not consummated any change of control transaction as of June 30, 2021 that would obligate it to make a CoC-payment.

The Company determined that the CoC-payment should be recorded as a derivative financial liability as of December 1, 2020 and that subsequent changes in the fair market value of this derivative financial liability should be recorded in profit and loss. The fair market value of the derivative financial liability is materially impacted by probability that market participants assign to the likelihood of the occurrence of a change of control transaction that would give rise to a CoC-payment. This probability represents an unobservable input. The Company determined the fair market value of the derivative financial liability by using a present value model based on expected cash flow. The expected cash flows are materially impacted by the probability that market participants assign to the likelihood of the occurrence of a change of control transaction within the biotechnology industry. The Company estimated this unobservable input using the best information available as of June 30, 2021 and December 31, 2020. The Company obtained reasonably available market information that it believed market participants would use in determining the likelihood of the occurrence of a change-of control transaction within the biotechnology industry. Selecting and evaluating market information involves considerable judgement and uncertainty. Based on all such information and its judgment the Company estimated that the fair market value of the derivative financial liability (presented within “Other non-current liabilities”) as of June 30, 2021 and December 31, 2020 was $2.6 million.

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Accrued expenses and other current liabilities

6 Months Ended

Jun. 30, 2021

Accrued expenses and other current liabilities

Accrued expenses and other current liabilities

5 Accrued expenses and other current liabilities

Accrued expenses and other current liabilities include the following items:

	June 30,		December 31,
	2021		2020
	(in thousands)
Accruals for services provided by vendors-not yet billed	$	16,734	$	8,269
Personnel related accruals and liabilities		6,792		7,687
Contract liability		—		2,082
Total	$	23,526	$	18,038

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Long-term debt

6 Months Ended

Jun. 30, 2021

Long-term debt

Long-term debt

6 Long-term debt

On June 14, 2013, the Company entered into a venture debt loan facility with Hercules Capital, Inc. (formerly known as Hercules Technology Growth Capital, Inc.) (“Hercules”), which was amended and restated on June 26, 2014, and again on May 6, 2016 (“2016 Amended Facility”). On December 6, 2018, the Company signed an amendment that both refinanced the then-existing $20.0 million 2016 Amended Facility and allowed the Company to draw down an additional $15.0 million (“2018 Amended Facility”). The 2018 Amended Facility extended the loan’s maturity date from May 1, 2020 until June 1, 2023. The interest-only period was initially extended from November 2018 to January 1, 2021 and was further extended to January 1, 2022 as a result of raising more than $90.0 million in equity financing in September 2019. The interest only period was again further extended to June 1, 2023 as a result of the January 2021 amendment (see below).  The interest rate is adjustable and is the greater of (i) 8.85% and (ii) 8.85% plus the prime rate less 5.50% per annum. Under the 2018 Amended Facility, the Company owes a back-end fee of 4.95% of the outstanding debt. In addition, in May 2020 the Company paid a back-end fee of $1.0 million in relation to the 2016 Amended Facility.

On January 29, 2021, the Company and Hercules amended the 2018 Amended Facility (“2021 Amended Facility”). Pursuant to the 2021 Amended Facility, Hercules agreed to an additional Facility of $100.0 million (“Tranche B”), increasing the aggregate principal amount of the term loan facilities from $35.0 million to up to $135.0 million. On January 29, 2021, the Company drew down $35.0 million of the Tranche B. The Company may draw down the remaining $65.0 million under the Tranche B in a series of one or more advances of not less than $20.0 million each until December 15, 2021. Advances under Tranche B bear interest at a rate equal to the greater of (i) 8.25% or (ii) 8.25% plus the prime rate, less 3.25% per annum. The principal balance and all accrued but unpaid interest on advances under Tranche B is due on June 1, 2023, which date may be extended by the Company by up to two twelve-month periods. Advances under Tranche B may not be prepaid prior to July 29, 2021, following which the Company may prepay all such advances without charge. The Company owes a back-end fee of 4.85% of amounts outstanding under Tranche B. The back-end fee is reduced if prepayment occurs at an earlier date.

The amortized cost (including interest due presented as part of accrued expenses and other current liabilities) of the 2018 Amended Facility and 2021 Amended Facility was $71.3 million as of June 30, 2021, compared to $35.9 million amortized cost for the 2018 Amended Facility as of December 31, 2020, and is recorded net of discount and debt issuance costs. The foreign currency gain on the facilities in the three months ended June 30, 2021, was $0.9 million and the foreign currency loss on the facilities in the six months ended June 30, 2021, was $2.2 million, compared to a foreign currency gain of $0.7 million and $0.0 million during the same periods in 2020 for the 2018 Amended Facility.

Interest expense associated with the 2018 Amended Facility and 2021 Amended Facility during the three and six months ended June 30, 2021, was $1.8 million and $3.4 million, compared to $0.9 million and $1.8 million, respectively, during the same periods in 2020 for the 2018 Amended Facility.

As a covenant in the 2018 Amended Facility and 2021 Amended Facility, the Company has periodic reporting requirements and is required to keep a minimum cash balance deposited in bank accounts in the United States, equivalent to the lesser of (i) 65% of the outstanding balance of principal due or (ii) 100% of worldwide cash and cash equivalents. This restriction on cash and cash equivalents only relates to the location of the cash and cash equivalents, and such cash and cash equivalents can be used at the discretion of the Company. In combination with other covenants, the 2018 Amended Facility and 2021 Amended Facility restricts the Company’s ability to, among other things, incur future indebtedness and obtain additional debt financing, to make investments in securities or in other companies, to transfer assets, to perform certain corporate changes, to make loans to employees, officers, and directors, and to make dividend payments and other distributions to its shareholders. The Company secured the facilities by directly or indirectly pledging its total assets of $784.3 million with the exception of $103.5 million of cash and cash equivalents and other current assets held by uniQure N.V.

The 2018 Amended Facility and 2021 Amended Facility contain provisions that include the occurrence of a material adverse effect, as defined therein, which would entitle Hercules to declare all principal, interest and other amounts owed by the Company immediately due and payable. As of June 30, 2021, the Company was in material compliance with all covenants and provisions.

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Shareholders' equity

6 Months Ended

Jun. 30, 2021

Shareholders' equity

Shareholders' equity

7Shareholders’ Equity

On March 1, 2021, the Company entered into a Sales Agreement with SVB Leerink LLC (“SVB Leerink”) with respect to an at-the-market (“ATM”) offering program, under which the Company may, from time to time in its sole discretion, offer and sell through SVB Leerink, acting as agent, its ordinary shares, up to an aggregate offering price of $200.0 million. The Company will pay SVB Leerink a commission equal to 3% of the gross proceeds of the sales price of all ordinary shares sold through it as sales agent under the Sales Agreement. In March and April 2021, the Company issued 921,730 ordinary shares at a weighted average price of $33.52 per share, with net proceeds of $29.6 million, after deducting underwriting discounts and net of offering expenses. The Company defers direct, incremental costs associated to this offering, except for the commission costs to SVB Leerink, which are a reduction to additional paid-in capital, and will deduct these costs from additional paid-in capital in the consolidated balance sheets proportionately to the amount of proceeds raised. During the three and six months ended June 30, 2021, $0.3 million and $1.3 million of direct, incremental costs were deducted from additional paid-in capital.

Following the release of the valuation allowance related to the years 2011 to 2018, the Company recognized the tax benefits of share issuance costs incurred in 2014, 2015, 2017 and 2018. This resulted in an increase of additional paid-in capital of $3.0 million in the three and six months ended June 30, 2021.

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Share-based compensation

6 Months Ended

Jun. 30, 2021

Share-based compensation

Share-based compensation

8Share-based compensation

The Company’s share-based compensation plans include the 2014 Amended and Restated Share Option Plan (the “2014 Plan”) and inducement grants under Rule 5653(c)(4) of the Nasdaq Global Select Market with terms similar to the 2014 Plan (together the “2014 Plans”). At the annual general meeting of shareholders in June 2021, the Company’s shareholders approved amendments of the 2014 Plan, increasing the shares authorized for issuance by 4,000,000 to a total of 12,601,471.

a) 2014 Plans

Share-based compensation expense recognized by classification included in the Consolidated statements of operations and comprehensive loss in relation to the 2014 Plans for the periods indicated below was as follows:

	Three months ended June 30,				Six months ended June 30,
	2021		2020		2021		2020
	(in thousands)				(in thousands)
Research and development	$	3,286	$	2,889	$	5,960	$	5,271
Selling, general and administrative		3,735		2,825		6,815		4,783
Total	$	7,021	$	5,714	$	12,775	$	10,054

Share-based compensation expense recognized by award type was as follows:

	Three months ended June 30,				Six months ended June 30,
	2021		2020		2021		2020
	(in thousands)				(in thousands)
Award type
Share options	$	3,350	$	2,746	$	6,190	$	4,954
Restricted share units		3,222		2,209		5,782		3,653
Performance share units		449		759		803		1,447
Total	$	7,021	$	5,714	$	12,775	$	10,054

As of June 30, 2021, the unrecognized share-based compensation expense related to unvested awards under the 2014 Plans were:

	Unrecognized		Weighted average
	share-based		remaining
	compensation		period for
	expense		recognition
	(in thousands)		(in years)
Award type
Share options	$	34,855	2.99
Restricted share units		24,960	2.25
Performance share units		792	0.58
Total	$	60,607	2.66

The Company satisfies the exercise of share options and vesting of Restricted Share Units (“RSUs”) and Performance Share Units (“PSUs”) through newly issued ordinary shares.

Share options

Share options are priced on the date of grant and, except for certain grants made to non-executive directors, vest over a period of four years. The first 25% vests after one year from the initial grant date and the remainder vests in equal quarterly installments over years two, three and four. Certain grants to non-executive directors vest in full after one year. Any options that vest must be exercised by the tenth anniversary of the initial grant date.

The following tables summarize option activity under the Company’s 2014 Plans for the six months ended June 30, 2021:

	Options
	Number of	Weighted average
	ordinary shares	exercise price
Outstanding at December 31, 2020	2,659,279	$	28.13
Granted	914,118	$	36.36
Forfeited	(74,397)	$	43.19
Expired	(9,093)	$	44.14
Exercised	(71,976)	$	12.65
Outstanding at June 30, 2021	3,417,931	$	30.29
Thereof, fully vested and exercisable at June 30, 2021	1,774,059	$	21.68
Thereof, outstanding and expected to vest after June 30, 2021	1,643,872	$	39.58

Total weighted average grant date fair value of options issued during the period (in $ millions)	$	19.4
Proceeds from option sales during the period (in $ millions)	$	0.9

The fair value of each option issued is estimated at the respective grant date using the Hull & White option pricing model with the following weighted-average assumptions:

	Three months ended June 30,		Six months ended June 30,
Assumptions	2021	2020	2021	2020
Expected volatility	75%	70%	75%	70%
Expected terms	10 years	10 years	10 years	10 years
Risk free interest rate	1.72% - 1.85%	0.76% - 0.83%	1.21% - 1.85%	0.76% - 1.44%
Expected dividend yield	0%	0%	0%	0%

Restricted share units (“RSUs”)

The following table summarizes the RSUs activity for the six months ended June 30, 2021:

	RSU
		Weighted average
	Number of	grant-date fair
	ordinary shares	value
Non-vested at December 31, 2020	467,344	$	43.56
Granted	481,821	$	36.50
Vested	(145,203)	$	39.89
Forfeited	(31,067)	$	42.86
Non-vested at June 30, 2021	772,895	$	39.87
Total weighted average grant date fair value of RSUs granted during the period (in $ millions)		$	17.6

RSUs vest over one to three years. RSUs granted to non-executive directors vest one year from the date of grant.

Performance share units (“PSUs”)

The following table summarizes the PSUs activity for the six months ended June 30, 2021:

	PSU
		Weighted average
	Number of	grant-date fair
	ordinary shares	value
Non-vested at December 31, 2020	212,614	$	42.32
Vested	(132,368)	$	33.09
Forfeited	(2,916)	$	57.56
Non-vested at June 30, 2021	77,330	$	57.56

The PSUs will vest on the third anniversary of the grant, subject to the grantee’s continued employment.

b) Employee Share Purchase Plan (“ESPP”)

In June 2018, the Company’s shareholders adopted and approved an ESPP allowing the Company to issue up to 150,000 ordinary shares. The ESPP is intended to qualify under Section 423 of the Internal Revenue Code of 1986. Under the ESPP, employees are eligible to purchase ordinary shares through payroll deductions, subject to any plan limitations. The purchase price of the ordinary shares on each purchase date is equal to 85% of the lower of the closing market price on the offering date and the closing market price on the purchase date of each three-month offering period. During the six months ended June 30, 2021, 1,174 ordinary shares were issued under the ESPP compared to 3,005 during the same period in 2020. As of June 30, 2021, a total of 130,852 ordinary shares remain available for issuance under the ESPP plan compared to a total of 135,202 as of June 30, 2020.

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Other income

6 Months Ended

Jun. 30, 2021

Other income.

Other income

9 Other income

Other income during the three and six months ended June 30, 2021, was $7.6 million and $7.9 million, compared to $0.7 million and $1.5 million, respectively, during the same periods in 2020.

Other income in 2020 and 2021 includes income from payments received from European authorities to subsidize the Company’s research and development efforts in the Netherlands as well as income from subleasing part of the Amsterdam facility.

In addition, other income during the three and six months ended June 30, 2021 includes $1.3 million of employee retention credit received under the U.S. Coronavirus Aid, Relief, and Economic Security Act, as well as $3.0 million related to 69,899 shares in VectorY B.V. received in conjunction with a settlement agreement entered into in April 2021. No such income was recorded in 2020.

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Income taxes

6 Months Ended

Jun. 30, 2021

Income taxes

Income taxes

10 Income taxes

The Company recorded $3.3 million in income tax expense in the three months ended June 30, 2021, and $3.5 million in the six months ended June 30, 2021. The Company recorded no income tax expense during the same periods in 2020.

As of December 31, 2020, the Company had recorded a full valuation allowance against its Dutch net deferred tax assets. On May 6, 2021, the CSL Behring Agreement became effective (refer to Note 3 “Collaboration arrangements and concentration of credit risk”). The Company recorded $462.4 million of license revenue related to closing the transaction. The Company intends to record such revenue in its Dutch tax return related to the 12-month period ended December 31, 2020. As such, the Company expects to file a return showing a taxable profit in the Netherlands in 2020, which would result in the consumption of substantially all of its Dutch net operating losses for the years 2011 to 2018. The Company’s remaining Dutch net operating tax losses carried forward would relate to 2019. The Dutch government on June 4, 2021 enacted legislation, whereby such net operating tax losses can be carried forward indefinitely. The Company expects to continue incurring tax losses for the foreseeable future including 2021. As such, the Company retained its full valuation allowance related to the net deferred assets as of June 30, 2021. The adjustment to the Company’s 2020 filing position resulting in the release and consumption of prior year net operating tax losses carried forward was treated as a discrete event in the three months ended June 30, 2021. The Company allocated part of the release to the tax benefit of share issuance cost incurred in 2014, 2015, 2017 and 2018. This resulted in an increase of additional paid-in capital as well as deferred tax expenses of $3.0 million.

The Company released its valuation allowance against the Company’s deferred tax assets in the United States as of December 31, 2020. The Company recorded $0.2 million and $0.5 million deferred tax expense in relation to its operations in the United States during the three and six months ended June 30, 2021, respectively. The Company recorded a nil net deferred tax expense in the prior year as it had recorded a valuation allowance against its net deferred tax assets in the United States as of June 30, 2020.

The effective income tax rates of respectively -0.8% and -1.0% during the three and six months ended June 30, 2021, is substantially lower than the enacted rated of 25% in the Netherlands as the Company recorded a valuation allowance against its remaining net deferred tax assets in the Netherlands as of December 31, 2020, and June 30, 2021.

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Basic and diluted earnings per share

6 Months Ended

Jun. 30, 2021

Basic and diluted earnings per share

Basic and diluted earnings per share

11Basic and diluted earnings per share

Basic net income / (loss) per ordinary share is computed by dividing net income / (loss) for the period by the weighted average number of ordinary shares outstanding during the period. Diluted earnings per ordinary share are calculated by adjusting the weighted average number of ordinary shares outstanding, assuming conversion of all potentially dilutive ordinary shares. For the three months and six months ended June 30, 2021, dilutive net income per ordinary share is computed using the treasury method.

The following table provides the calculation of basic and diluted earnings per ordinary share:

	Three months ended June 30,				Six months ended June 30,
	2021		2020		2021		2020
	(in thousands)				(in thousands)
Numerator:
Net income / (loss) attributable to ordinary shares	$	399,468	$	(42,551)	$	357,912	$	(70,550)
		399,468		(42,551)		357,912		(70,550)
Denominator:
Weighted-average number of ordinary shares outstanding - basic		46,037,900		44,387,463		45,754,766		44,333,460
Stock options under 2014 Plans and previous plan		797,165		—		824,101		—
Non-vested RSUs and PSUs		92,681		—		98,514		—
Employee share purchase plan		2,124		—		1,454		—
Weighted-average number of ordinary shares outstanding - diluted		46,929,870		44,387,463		46,678,835		44,333,460

The following table presents ordinary share equivalents that were excluded from the calculation of diluted net income / (loss) per ordinary share for the three and six months ended June 30, 2021 and 2020 as the effect of their inclusion would have been anti-dilutive.

	Three months ended June 30,				Six months ended June 30,
	2021		2020		2021		2020
	(in thousands)				(in thousands)
Anti-dilutive ordinary share equivalents
Stock options under 2014 Plans and previous plan		2,634,766		2,893,733		2,607,830		2,893,733
Non-vested RSUs and PSUs		757,544		645,636		751,711		645,636
Employee share purchase plan		1,414		254		1,802		254
BMS warrants (derecognized as of December 1, 2020 - see Note 4, "Fair value measurement")		—		8,060,500		—		8,060,500
Total anti-dilutive ordinary share equivalents		3,393,724		11,600,123		3,361,343		11,603,143

The anti-dilutive ordinary shares are presented without giving effect to the application of the treasury method or exercise prices that exceeded the price of the Company’s ordinary shares as of June 30, 2020. In addition, the BMS warrants were not exercisable as of June 30, 2020. This would have resulted in a lower number of potentially dilutive ordinary shares as some stock option grants as well as the BMS warrants would have been excluded.

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Subsequent events

6 Months Ended

Jun. 30, 2021

Subsequent events

Subsequent events

12Subsequent event

Business combination

On June 22, 2021, the Company announced that it entered into a definitive transaction agreement under which the Company will acquire Corlieve Therapeutics S.A.S. (“Corlieve”, together the “Corlieve Transaction”). The Corlieve Transaction has been approved by the Boards of both companies and does not require the Company’s shareholder approval. The transaction is subject to customary closing conditions as well as review by the French Ministry of Economy, Finance and Recovery (Ministère de l’Economie, des Finances et de la Relance) pursuant to articles L.151-3 and R.151-1 and seq. of the French Code Monétaire et Financier. Currently, the transaction is anticipated to be completed early in the third quarter of 2021.

The Corlieve Transaction strengthens the Company’s pre-clinical program pipeline. Corlieve’s lead gene therapy program employs miRNA silencing technology to target suppression of aberrantly expressed kainate receptors in the hippocampus of patients with temporal lobe epilepsy (“TLE”) and will be known as AMT-260.

Upon closing of the Corlieve Transaction, the Company expects to pay EUR 46.3 million in cash. The Company will fund the acquisition from its cash position. Corlieve’s shareholders are eligible to receive the following additional payments of which up to 25% will be payable in the Company’s ordinary shares at the Company’s election: up to EUR 43.7 million in development milestones through Phase I/II and EUR 160.0 million in milestones associated with Phase III development and the approvals of AMT-260 in the U.S and European Union.

The Corlieve Transaction is expected to be accounted for as a business combination, which requires that assets acquired and liabilities assumed be recognized at their fair value as of the acquisition date.

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Summary of significant accounting policies (Policies)

6 Months Ended

Jun. 30, 2021

Summary of significant accounting policies

Basis of preparation

2.1Basis of preparation

The Company prepared these unaudited consolidated financial statements in compliance with generally accepted accounting principles in the United States of America (“U.S. GAAP”) and applicable rules and regulations of the Securities and Exchange Commission (“SEC”) regarding interim financial reporting. Any reference in these notes to applicable guidance is meant to refer to authoritative U.S. GAAP as found in the Accounting Standards Codification (“ASC”) and Accounting Standards Update (“ASU”) of the Financial Accounting Standards Board (“FASB”).

The unaudited consolidated financial statements are presented in U.S. dollars, except where otherwise indicated. Transactions denominated in currencies other than U.S. dollars are presented in the transaction currency with the U.S. dollar amount included in parenthesis, converted at the foreign exchange rate as of the transaction date.

Unaudited interim financial information

2.2Unaudited interim financial information

The interim financial statements and related disclosures are unaudited, have been prepared on the same basis as the annual financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for a fair statement of the financial position, results of operations and changes in financial position for the period presented.

Certain information and footnote disclosures normally included in annual financial statements prepared in accordance with U.S. GAAP have been omitted. The results of operations for the six months ended June 30, 2021, are not necessarily indicative of the results to be expected for the full year ending December 31, 2021 or for any other future year or interim period. The accompanying financial statements should be read in conjunction with the audited financial statements and the related notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, filed with the SEC on March 1, 2021.

Use of estimates

2.3Use of estimates

The preparation of the financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities at the date of the financial statements and reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates.

Accounting policies

2.4Accounting policies

The principal accounting policies applied in the preparation of these unaudited consolidated financial statements are described in the Company’s audited financial statements as of and for the year ended December 31, 2020, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, filed with the SEC on March 1, 2021. There have been no material changes in the Company’s significant accounting policies during the six months ended June 30, 2021 except as noted below.

The Company determined its revenue recognition policies related to the CSL Behring Agreement (defined below) when the CSL Behring Agreement became fully effective on May 6, 2021. Refer to Note 3 (“Collaboration arrangements and concentration of credit risk”) for further detail.

The Company for the first time recognized net income in the three and six months ended June 30, 2021. Net income / (loss) per ordinary share was calculated in accordance with ASC 260, Earnings Per Share. Refer to Note 11 (“Basic and diluted earnings per share”) for further detail.

Recent accounting pronouncements

2.5Recent accounting pronouncements

There have been no new accounting pronouncements or changes to accounting pronouncements during the six months ended June 30, 2021, as compared to the recent accounting pronouncements described in Note 2.3.22 of the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, which could be expected to materially impact the Company’s unaudited consolidated financial statements.

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Fair value measurement (Tables)

6 Months Ended

Jun. 30, 2021

Fair value measurement

Schedule of assets and liabilities measured at fair value on recurring basis

	Quoted prices in active markets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)		Total		Classification in Consolidated balance sheets
		(in thousands)
At December 31, 2020
Assets:
Cash, cash equivalents and restricted cash	$	247,680	$	—	$	—	$	247,680	Cash and cash equivalents; other non-current assets
Total assets	$	247,680	$	—	$	—	$	247,680
Liabilities:
Derivative financial instruments		—		—		2,645		2,645	Other non-current liabilities
Total liabilities	$	—	$	—	$	2,645	$	2,645
At June 30, 2021
Assets:
Cash, cash equivalents and restricted cash	$	680,158	$	—	$	—	$	680,158	Cash and cash equivalents; other non-current assets
Total assets	$	680,158	$	—	$	—	$	680,158
Liabilities:
Derivative financial instruments	$	—	$	—	$	2,645	$	2,645	Other non-current liabilities
Total liabilities	$	—	$	—	$	2,645	$	2,645

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Accrued expenses and other current liabilities (Tables)

6 Months Ended

Jun. 30, 2021

Accrued expenses and other current liabilities

Schedule of accrued expenses and other current liabilities

	June 30,		December 31,
	2021		2020
	(in thousands)
Accruals for services provided by vendors-not yet billed	$	16,734	$	8,269
Personnel related accruals and liabilities		6,792		7,687
Contract liability		—		2,082
Total	$	23,526	$	18,038

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Share-based compensation (Tables)

6 Months Ended

Jun. 30, 2021

Share-based compensation

Schedule of share-based compensation expense by classification included in consolidated statements of operations and comprehensive loss

	Three months ended June 30,				Six months ended June 30,
	2021		2020		2021		2020
	(in thousands)				(in thousands)
Research and development	$	3,286	$	2,889	$	5,960	$	5,271
Selling, general and administrative		3,735		2,825		6,815		4,783
Total	$	7,021	$	5,714	$	12,775	$	10,054

Schedule of share-based compensation expense

	Three months ended June 30,				Six months ended June 30,
	2021		2020		2021		2020
	(in thousands)				(in thousands)
Award type
Share options	$	3,350	$	2,746	$	6,190	$	4,954
Restricted share units		3,222		2,209		5,782		3,653
Performance share units		449		759		803		1,447
Total	$	7,021	$	5,714	$	12,775	$	10,054

Schedule of unrecognized compensation cost related to unvested awards

	Unrecognized		Weighted average
	share-based		remaining
	compensation		period for
	expense		recognition
	(in thousands)		(in years)
Award type
Share options	$	34,855	2.99
Restricted share units		24,960	2.25
Performance share units		792	0.58
Total	$	60,607	2.66

Schedule of weighted-average assumptions for fair value of option issued

	Three months ended June 30,		Six months ended June 30,
Assumptions	2021	2020	2021	2020
Expected volatility	75%	70%	75%	70%
Expected terms	10 years	10 years	10 years	10 years
Risk free interest rate	1.72% - 1.85%	0.76% - 0.83%	1.21% - 1.85%	0.76% - 1.44%
Expected dividend yield	0%	0%	0%	0%

Summary of RSUs activity

	RSU
		Weighted average
	Number of	grant-date fair
	ordinary shares	value
Non-vested at December 31, 2020	467,344	$	43.56
Granted	481,821	$	36.50
Vested	(145,203)	$	39.89
Forfeited	(31,067)	$	42.86
Non-vested at June 30, 2021	772,895	$	39.87
Total weighted average grant date fair value of RSUs granted during the period (in $ millions)		$	17.6

Summary of PSUs activity

	PSU
		Weighted average
	Number of	grant-date fair
	ordinary shares	value
Non-vested at December 31, 2020	212,614	$	42.32
Vested	(132,368)	$	33.09
Forfeited	(2,916)	$	57.56
Non-vested at June 30, 2021	77,330	$	57.56

2014 Plan

Share-based compensation

Summary of option activity

	Options
	Number of	Weighted average
	ordinary shares	exercise price
Outstanding at December 31, 2020	2,659,279	$	28.13
Granted	914,118	$	36.36
Forfeited	(74,397)	$	43.19
Expired	(9,093)	$	44.14
Exercised	(71,976)	$	12.65
Outstanding at June 30, 2021	3,417,931	$	30.29
Thereof, fully vested and exercisable at June 30, 2021	1,774,059	$	21.68
Thereof, outstanding and expected to vest after June 30, 2021	1,643,872	$	39.58

Total weighted average grant date fair value of options issued during the period (in $ millions)	$	19.4
Proceeds from option sales during the period (in $ millions)	$	0.9

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Basic and diluted earnings per share (Tables)

6 Months Ended

Jun. 30, 2021

Basic and diluted earnings per share

Schedule of bsic and diluted earnings per share

	Three months ended June 30,				Six months ended June 30,
	2021		2020		2021		2020
	(in thousands)				(in thousands)
Numerator:
Net income / (loss) attributable to ordinary shares	$	399,468	$	(42,551)	$	357,912	$	(70,550)
		399,468		(42,551)		357,912		(70,550)
Denominator:
Weighted-average number of ordinary shares outstanding - basic		46,037,900		44,387,463		45,754,766		44,333,460
Stock options under 2014 Plans and previous plan		797,165		—		824,101		—
Non-vested RSUs and PSUs		92,681		—		98,514		—
Employee share purchase plan		2,124		—		1,454		—
Weighted-average number of ordinary shares outstanding - diluted		46,929,870		44,387,463		46,678,835		44,333,460

Schedule of potential dilutive common shares

	Three months ended June 30,				Six months ended June 30,
	2021		2020		2021		2020
	(in thousands)				(in thousands)
Anti-dilutive ordinary share equivalents
Stock options under 2014 Plans and previous plan		2,634,766		2,893,733		2,607,830		2,893,733
Non-vested RSUs and PSUs		757,544		645,636		751,711		645,636
Employee share purchase plan		1,414		254		1,802		254
BMS warrants (derecognized as of December 1, 2020 - see Note 4, "Fair value measurement")		—		8,060,500		—		8,060,500
Total anti-dilutive ordinary share equivalents		3,393,724		11,600,123		3,361,343		11,603,143

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Collaboration arrangements and concentration of credit risk - BMS collaboration - Narrative (Details) $ in Thousands				3 Months Ended		6 Months Ended
	Jun. 24, 2021 USD ($)	Dec. 01, 2020 USD ($) item	Jun. 24, 2020 USD ($)	Jun. 30, 2021 USD ($)	Jun. 30, 2020 USD ($)	Jun. 30, 2021 USD ($) item	Jun. 30, 2020 USD ($)	Dec. 31, 2020 USD ($)
Collaboration arrangements
Number of collaboration targets | item						4
Revenue				$ 463,868	$ 1,535	$ 464,322	$ 1,639
Accounts receivables				$ 2,705		$ 2,705		$ 6,618
Tax rate enacted in the Netherlands				25.00%		25.00%
Tax benefit past share issuance cost				$ 2,977		$ 2,977
CSL Behring collaboration
Collaboration arrangements
Revenue				462,400		462,400
Upfront cash to receive upon the closing of the agreement	$ 450,000		$ 450,000
Other payments received	$ 12,400
Accounts receivables				1,300		1,300		$ 2,100
Regulatory and commercial milestone payments received			$ 1,600,000
License revenues from related party
Collaboration arrangements
Revenue					1,530		1,577
Collaboration revenues from related party
Collaboration arrangements
Revenue					5		62
Collaboration revenues
Collaboration arrangements
Revenue				1,468		$ 1,922
Collaboration revenues | CSL Behring collaboration
Collaboration arrangements
Revenue				$ 400	$ 0		$ 0
BMS arrangement | Bristol Myers Squibb
Collaboration arrangements
Initial research term						4 years
Number of collaboration targets | item		4
Collaboration Agreement, Threshold Number Of Collaboration Targets That Can Be Replaced | item		2
Threshold Number Of New Collaboration Targets In The Field Of Cardiovascular Disease | item		2
License revenue | Bristol Myers Squibb
Collaboration arrangements
Revenue						$ 0
Bristol Myers Squibb | BMS arrangement
Collaboration arrangements
Collaboration Agreement, Threshold Research, Development And Regulatory Milestone Payments Receivable		$ 217,000

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Fair value measurement - Assets and liabilities measured on a recurring basis (Details) - Recurring - USD ($) $ in Thousands	Jun. 30, 2021	Dec. 31, 2020
Assets, Fair Value Disclosure [Abstract]
Cash and cash equivalents	$ 680,158	$ 247,680
Total assets	680,158	247,680
Liabilities, Fair Value Disclosure [Abstract]
Fair value of derivative liability	2,645	2,645
Total liabilities	2,645	2,645
Fair value hierarchy Level 1
Assets, Fair Value Disclosure [Abstract]
Cash and cash equivalents	680,158	247,680
Total assets	680,158	247,680
Level 3
Liabilities, Fair Value Disclosure [Abstract]
Fair value of derivative liability	2,645	2,645
Total liabilities	$ 2,645	$ 2,645

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Fair value measurement - BMS warrants - Narrative (Details)	3 Months Ended	6 Months Ended
	Jun. 30, 2021 USD ($) item	Jun. 30, 2021 USD ($) item	Dec. 31, 2020 USD ($)
Collaboration arrangements
Number of warrant types | item	2	2
Number of Collaboration Targets | item		4
Collaboration Agreement, Cash Payable	$ 70,000,000.0	$ 70,000,000.0
Gain (Loss) from the recognition of the derivative financial liability	0	0
Non-current liabilities
Collaboration arrangements
Fair value of the derivative financial liability	$ 2,600,000	$ 2,600,000	$ 2,600,000
Collaboration Agreement, If 70 Million Is Greater Than Five Percent Of Net Proceeds | Derecognition Of Warrants
Collaboration arrangements
Threshold Percentage Of Net Proceeds	5.0	5.0
Percentage Of Net Proceeds Payable	5	5
Collaboration Agreement, If 70 Million Is Lesser Than One Percent Of Net Proceeds | Derecognition Of Warrants
Collaboration arrangements
Threshold Percentage Of Net Proceeds	1	1
Percentage Of Net Proceeds Payable	1	1

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Accrued expenses and other current liabilities (Details) - USD ($) $ in Thousands	Jun. 30, 2021	Dec. 31, 2020
Accrued expenses and other current liabilities
Accruals for services provided by vendors-not yet billed	$ 16,734	$ 8,269
Personnel related accruals and liabilities	6,792	7,687
Contract liability		2,082
Total	$ 23,526	$ 18,038

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Long-term debt (Details) - USD ($) $ in Thousands			3 Months Ended		6 Months Ended
	Jan. 29, 2021	Dec. 06, 2018	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020	Dec. 15, 2021	Dec. 31, 2020	May 31, 2020
Long-term Debt
Proceeds from loan increment, net of debt issuance costs					$ 34,603
2016 Amended Facility | Hercules | Second Amended and Restated Loan and Security Agreement [Member]
Long-term Debt
Outstanding debt		$ 20,000
Back-end fees due									$ 1,000
2018 Amended Facility | Hercules
Long-term Debt
Aggregate amount of Equity Financing					90,000
Foreign currency loss				$ 700
2018 Amended Facility | Hercules | Second Amended and Restated Loan and Security Agreement [Member]
Long-term Debt
Interest rate (as a percent)		8.85%
Back-end fee (as a percent)		4.95%
Proceeds from loan increment, net of debt issuance costs		$ 15,000
2018 Amended Facility | Prime Rate | Hercules | Second Amended and Restated Loan and Security Agreement [Member]
Long-term Debt
Variable interest rate basis		8.85%
Discount rate (as a percent)		5.50%
2021 Amended Facility
Long-term Debt
Outstanding debt	$ 35,000
Proceed from drew down	$ 35,000
Interest rate (as a percent)	8.25%
Back-end fee (as a percent)	4.85%
Maximum borrowing capacity	$ 135,000
Additional unconditional borrowing capacity	$ 100,000
2021 Amended Facility | Prime Rate
Long-term Debt
Variable interest rate basis	3.25%
2018 and 2021 Amended Facility | Hercules
Long-term Debt
Foreign currency loss					$ 2,200
Foreign currency gain			$ 900			$ 0
Venture debt loan facility | 2018 Amended Facility
Aggregate maturities of loan
Long-term debt								$ 35,900
Venture debt loan facility | 2018 Amended Facility | Hercules
Long-term Debt
Interest expense recorded				$ 900
Venture debt loan facility | 2018 and 2021 Amended Facility
Long-term Debt
Minimum cash and cash equivalents in U.S. bank accounts					65.00%
Assets pledged to secure facilities by directly or indirectly					$ 784,300
Assets not being pledged to secure facilities by directly or indirectly					103,500
Aggregate maturities of loan
Long-term debt			71,300		71,300
Venture debt loan facility | 2018 and 2021 Amended Facility | Hercules
Long-term Debt
Interest expense recorded			$ 1,800		$ 3,400	$ 1,800
Subsequent events. | 2021 Amended Facility
Long-term Debt
Outstanding debt							$ 65,000
Subsequent events. | 2021 Amended Facility | Minimum
Long-term Debt
Outstanding debt							$ 20,000

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Shareholders' equity (Details) - USD ($) $ / shares in Units, $ in Thousands		2 Months Ended	3 Months Ended	6 Months Ended
	Mar. 01, 2021	Apr. 30, 2021	Jun. 30, 2021	Jun. 30, 2021	Dec. 31, 2020
Aggregate offering price			$ 1,865	$ 29,564
Additional paid-in-capital			1,062,234	1,062,234	$ 1,016,018
Cash consideration paid				46,300
Sales Agreement with SVB Leerink LLC
Percentage of gross proceeds	3.00%
Net proceeds		$ 29,600
Sales Agreement with SVB Leerink LLC | Direct Incremental Labor
Adjustments to Additional Paid in Capital, Stock Issued, Issuance Costs			$ 300	$ 1,300
Sales Agreement with SVB Leerink LLC | Ordinary shares
Shares issued in connection with offering		921,730
Share price (in dollars per share)		$ 33.52
Sales Agreement with SVB Leerink LLC | Maximum
Aggregate offering price	$ 200,000

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Share-based compensation - Summary of share-based compensation expense and unrecognized costs (Details) - 2014 Plan - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020	Dec. 31, 2020
Share-based compensation
Authorized shares	12,601,471		12,601,471
Increase in authorized shares			4,000,000
Share-based compensation expense	$ 7,021	$ 5,714	$ 12,775	$ 10,054
Unrecognized compensation costs	60,607		$ 60,607
Weighted-average remaining period for recognition (in years)			2 years 7 months 28 days
Research and development expenses
Share-based compensation
Share-based compensation expense	3,286	2,889	$ 5,960	5,271
Selling, general and administrative expense
Share-based compensation
Share-based compensation expense	3,735	2,825	6,815	4,783
Share options
Share-based compensation
Share-based compensation expense	$ 3,350	2,746	$ 6,190	4,954
Outstanding of fully vested share options	3,417,931		3,417,931		2,659,279
Unrecognized compensation costs	$ 34,855		$ 34,855
Weighted-average remaining period for recognition (in years)			2 years 11 months 26 days
Restricted share units ("RSUs")
Share-based compensation
Share-based compensation expense	3,222	2,209	$ 5,782	3,653
Unrecognized compensation costs	24,960		$ 24,960
Weighted-average remaining period for recognition (in years)			2 years 3 months
Performance share units ("PSUs")
Share-based compensation
Share-based compensation expense	449	$ 759	$ 803	$ 1,447
Unrecognized compensation costs	$ 792		$ 792
Weighted-average remaining period for recognition (in years)			6 months 29 days

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Share-based compensation - Option activity and weighted-average assumptions (Details) - USD ($) $ / shares in Units, $ in Millions	3 Months Ended		6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020
Weighted average exercise price
Total weighted average grant date fair value of options issued during the period (in $ millions)			$ 19.4
Proceeds from option sales during the period (in $ millions)			$ 0.9
2014 Plan
Weighted average exercise price
Vesting period (in years)			4 years
Weighted-average assumptions used to estimate fair value of share options granted during year
Increase in authorized shares			4,000,000
2014 Plan | Share options
Options
Outstanding at beginning of the period (in shares)			2,659,279
Granted (in shares)			914,118
Forfeited (in shares)			(74,397)
Expired (in shares)			(9,093)
Exercised (in shares)			(71,976)
Outstanding at end of the period (in shares)	3,417,931		3,417,931
Thereof, fully vested and exercisable at end of period (in shares)	1,774,059		1,774,059
Thereof, outstanding and expected to vest at end of period (in shares)	1,643,872		1,643,872
Weighted average exercise price
Outstanding at beginning of the period (in dollars per share)			$ 28.13
Granted (in dollars per share)			36.36
Forfeited (in dollars per share)			43.19
Expired (in dollars per share)			44.14
Exercised (in dollars per share)			12.65
Outstanding at end of period (in dollars per share)	$ 30.29		30.29
Thereof, fully vested and exercisable at end of period (in dollars per share)	$ 21.68		21.68
Outstanding and expected to vest at end of the period (in dollars per share)			$ 39.58
Weighted-average assumptions used to estimate fair value of share options granted during year
Expected volatility (as a percent)	75.00%	70.00%	75.00%	70.00%
Expected terms (in years)	10 years	10 years	10 years	10 years
Risk free interest rate, minimum (as a percent)	1.72%	0.76%	1.21%	0.76%
Risk free interest rate, maximum (as a percent)	1.85%	0.83%	1.85%
Expected dividend (as a percent)	0.00%	0.00%	0.00%	0.00%
2014 Plan | Non-executive directors
Weighted average exercise price
Vesting period (in years)			1 year
2014 Plan | One year from grant date
Weighted average exercise price
Vesting percentage per year			25.00%
Vesting period (in years)			1 year

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Share-based compensation - RSU activity (Details) - 2014 Plan - USD ($) $ / shares in Units, $ in Thousands	1 Months Ended	6 Months Ended
	Mar. 31, 2017	Jun. 30, 2021
Other disclosure
Vesting period (in years)		4 years
Non-executive directors
Other disclosure
Vesting period (in years)		1 year
Restricted share units ("RSUs")
Number of shares
Non-vested at beginning of period (in shares)		467,344
Restricted stock granted (in shares)		481,821
Vested (in shares)		(145,203)
Forfeited (in shares)		(31,067)
Non-vested at end of period (in shares)		772,895
Weighted average grant-date fair value
Non-vested at beginning of period (in dollars per share)		$ 43.56
Granted (in dollars per share)		36.50
Vested (in dollars per share)		39.89
Forfeited (in dollars per share)		42.86
Non-vested at end of period (in dollars per share)		$ 39.87
Other disclosure
Total weighted average grant date fair value of RSUs granted during the period (in millions)		$ 17,600
Restricted share units ("RSUs") | Non-executive directors
Other disclosure
Vesting period (in years)	1 year
Minimum | Restricted share units ("RSUs")
Other disclosure
Vesting period (in years)		1 year
Maximum | Restricted share units ("RSUs")
Other disclosure
Vesting period (in years)		3 years

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Share-based compensation - PSU activity (Details) - 2014 Plan	6 Months Ended
	Jun. 30, 2021 $ / shares shares
Other disclosure
Vesting period (in years)	4 years
Performance share units ("PSUs")
Number of shares
Non-vested at beginning of period (in shares) | shares	212,614
Vested (in shares) | shares	(132,368)
Forfeited (in shares) | shares	(2,916)
Non-vested at end of period (in shares) | shares	77,330
Weighted average grant-date fair value
Non-vested at beginning of period (in dollars per share) | $ / shares	$ 42.32
Vested (in dollars per share) | $ / shares	33.09
Forfeited (in dollars per share) | $ / shares	57.56
Non-vested at end of period (in dollars per share) | $ / shares	$ 57.56

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Share-based compensation - Employee Share Purchase Plan - Narrative (Details) - ESPP - Employee Stock [Member] - shares	1 Months Ended	6 Months Ended
	Jun. 30, 2018	Jun. 30, 2021	Jun. 30, 2020
Share-based compensation
Ordinary shares available for issue	150,000	130,852	135,202
Discounted rate for purchase of shares	85.00%
Number of shares issued		1,174	3,005

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Other income (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020
Schedule of Equity Method Investments [Line Items]
Other Income	$ 7,590	$ 669	$ 7,942	$ 1,526
Employee retention income	$ 1,300		1,300
VectorY B.V
Schedule of Equity Method Investments [Line Items]
Employee retention income		$ 0		$ 0
Settlement Income			$ 3,000
Balance shares	69,899		69,899

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Income taxes - Effective Tax Rate (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020
Income tax expense	$ (3,258)	$ 0	$ (3,471)	$ 0
Effective income tax rate	0.80%		1.00%
Tax rate enacted in the Netherlands	25.00%		25.00%
Deferred income taxes (benefits)	$ 200	0	$ 500	0
Revenue	463,868	$ 1,535	464,322	$ 1,639
CSL Behring collaboration
Revenue	$ 462,400		$ 462,400

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Basic and diluted earnings per share - Ordinary share (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Income (Loss) for the period	$ 399,468	$ (42,551)	$ 357,912	$ (70,550)
Weighted Average Number of Shares Outstanding, Basic	46,037,900	44,387,463	45,754,766	44,333,460
Weighted-average number of ordinary shares outstanding - diluted	46,929,870	44,387,463	46,678,835	44,333,460
Stock options | Previous plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
ShareBased payment arrangements	797,165		824,101
Stock options | ESPP
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
ShareBased payment arrangements	2,124		1,454
Non-vested and earned RSUs and PSUs
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
ShareBased payment arrangements	92,681		98,514

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Basic and diluted earnings per share (Details) - shares	3 Months Ended		6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020
Basic and diluted earnings per share
Total potential dilutive ordinary shares	3,393,724	11,600,123	3,361,343	11,603,143
BMS Warrants | Bristol Myers Squibb
Basic and diluted earnings per share
Total potential dilutive ordinary shares		8,060,500		8,060,500
Stock options | Previous plan
Basic and diluted earnings per share
Total potential dilutive ordinary shares	2,634,766	2,893,733	2,607,830	2,893,733
Stock options | ESPP
Basic and diluted earnings per share
Total potential dilutive ordinary shares	1,414	254	1,802	254
Non-vested and earned RSUs and PSUs
Basic and diluted earnings per share
Total potential dilutive ordinary shares	757,544	645,636	751,711	645,636

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Subsequent events (Details) - 6 months ended Jun. 30, 2021 € in Millions, $ in Millions	USD ($)	EUR (€)
Subsequent events
Payments to Acquire Businesses, Gross	$ 46.3
Business Combination, Consideration Transferred, Equity Interests Issued and Issuable, Percentage	25.00%
Definitive Transaction Agreement, Development Milestone, Phase I and II	$ 43.7
Definitive Transaction Agreement, Development Milestone, Phase III | €		€ 160.0