10-Q

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, DC 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 September 30, 2022

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: 001-38707

LogicBio Therapeutics, Inc.

(Exact Name of Registrant as Specified in its Charter)

Delaware	47-1514975
(State or other jurisdiction of incorporation or organization) 65 Hayden Avenue, 2nd Floor, Lexington, MA (Address of principal executive offices)	(I.R.S. Employer Identification No.) 02421 (Zip code)

(617) 245-0399

(Registrant’s telephone number, including area code)

n/a

(Former name, former address and formal fiscal year, if changed since last report)

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

Title of each class	Trading Symbol(s)	Name of each exchange on which registered
Common Stock, par value $0.0001 per share	LOGC	The Nasdaq Global 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, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “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 November 8, 2022, the registrant had 32,962,733 shares of common stock, $0.0001 par value per share, outstanding.

Table of Contents

		Page
PART I.	FINANCIAL INFORMATION	7
Item 1.	Financial Statements (Unaudited)	7
	Condensed Consolidated Balance Sheets as of September 30, 2022 and December 31, 2021	7
	Condensed Consolidated Statements of Operations for the Three and Nine Months Ended September 30, 2022 and 2021	8
	Condensed Consolidated Statements of Comprehensive Loss for the Three and Nine Months Ended September 30, 2022 and 2021	9
	Condensed Consolidated Statements of Stockholders’ Equity for the Three and Nine Months Ended September 30, 2022 and 2021	10
	Condensed Consolidated Statements of Cash Flows for the Nine Months Ended September 30, 2022 and 2021	11
	Notes to Unaudited Condensed Consolidated Financial Statements	12
Item 2.	Management’s Discussion and Analysis of Financial Condition and Results of Operations	24
Item 3.	Quantitative and Qualitative Disclosures About Market Risk	36
Item 4.	Controls and Procedures	36
PART II.	OTHER INFORMATION	37
Item 1.	Legal Proceedings	37
Item 1A.	Risk Factors	37
Item 6.	Exhibits	91
Signatures		92

2

SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS AND INDUSTRY DATA

This Quarterly Report on Form 10-Q contains forward-looking statements. All statements other than statements of historical fact are “forward-looking statements” for purposes of this Quarterly Report on Form 10-Q. In some cases, you can identify forward-looking statements by terminology such as “anticipate,” “approach,” “believe,” “continue,” “designed,” “estimate,” “expect,”  “goal,” “intend,” “may,” “plan,” “potential,” “projection,” “strategy,” “will,” “would,” “should,” “seek,” “likely,” “become,” “develop,” “engage,” “expand,” “leverage,” “future” or similar expressions, or the negative or plural of these words or expressions. These forward-looking statements may include statements concerning the following:

• the proposed acquisition of the Company by Alexion, including the parties’ ability consummate the proposed transaction;

• the design, cost, initiation, timing, progress and results of our current and future research and development activities, including statements with respect to our Phase 1/2 SUNRISE clinical trial and other development activities for our product candidate, LB-001, in methylmalonic acidemia, or MMA;  

• early clinical results and the significance and interpretation thereof;

• potential attributes and benefits of our GeneRide^® and sAAVy^TM platforms, our proprietary manufacturing process, mAAVRx^TM, and our existing or future product candidates, including any potential benefit of such platforms and technologies over competing platforms and technologies;

• the direct or indirect impacts of the COVID-19 pandemic on our business, operations and the markets and communities in which we and our partners, collaborators and vendors operate;

• our ability to take advantage of the modular nature of our GeneRide platform to simplify and accelerate development of new product candidates;

• our ability to optimize certain components of our viral vector manufacturing process, including production yields, drug product purity, decreasing “empty” capsids and developing reliable characterization methods;

• the potential benefits of our collaboration and license agreements and our ability to enter into future collaboration and licensing arrangements;

• the timing of, and our ability to obtain and maintain, regulatory approvals for our existing or future product candidates;

• our ability to quickly and efficiently identify and develop additional product candidates;

• our ability to obtain the funding for our operations necessary to continue the advancement of any product candidates;

• our ability to advance any product candidate into and successfully complete clinical trials;

• our intellectual property position, including obtaining and maintaining patents, the duration of our patent protection and trade secret protection; and

• our estimates regarding expenses, future revenues, capital requirements, the sufficiency of our current and expected cash resources and our need for additional financing.

Any or all of these forward-looking statements in this Quarterly Report on Form 10-Q may turn out to be inaccurate. These forward-looking statements involve risks and uncertainties, including those that are discussed below under the heading “Risk Factors Summary”, and the risk factors identified further in Part II, Item 1A. "Risk Factors" included in this Quarterly Report on Form 10-Q and elsewhere in this Quarterly Report on Form 10-Q, that could cause our actual results, financial condition, performance or achievements to be materially different from those indicated in these forward-looking statements. In particular, whether the proposed Merger with Alexion is actually consummated, the actual time it may take to demonstrate clinical efficacy, the potential timing and cost impacts of the SUNRISE protocol amendments relating to the lifting of the clinical hold announced in May 2022, the impact of the ongoing COVID-19 pandemic on our ability to progress with our research, development, manufacturing and regulatory efforts, and the value of and market for our common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as safety concerns that the U.S. Food and Drug Administration, or FDA, and other regulatory bodies may continue to have and the ultimate duration of the pandemic. In addition we are subject to the following risks: existing preclinical or clinical data may not be predictive of the results of ongoing or later clinical trials; clinical trials may not be successful or may be discontinued or delayed for any reason; manufacturing and process development risks, including delays relating to continuously improving our manufacturing processes; risks associated with management and key personnel changes and transitional periods; the actual funding required to develop and commercialize product candidates, including for safety, tolerability, enrollment, manufacturing or economic reasons; the timing and content of decisions made by regulatory authorities; the actual time it takes to initiate and complete preclinical and clinical studies; the competitive landscape; changes in our economic and financial conditions; and our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates and technologies. Given these risks and uncertainties, you should not place undue reliance on these forward-looking statements. Forward-looking statements speak only as of the date of this Quarterly Report on Form 10-Q. Except as required by law, we assume no obligation to update or

3

revise these forward-looking statements for any reason. Unless otherwise stated, our forward-looking statements do not reflect the potential impact of any future acquisitions, mergers, dispositions, joint ventures or investments we may make.

In this Quarterly Report on Form 10-Q, unless the context otherwise requires, the terms “LogicBio,” “LogicBio Therapeutics, Inc.,” the “Company,” “we,” “us,” “our” and similar references in this Quarterly Report on Form 10-Q refer to LogicBio Therapeutics, Inc. and its subsidiaries.

LOGICBIO^®, GENERIDE^®, SAAVY^TM, MAAVRX^TM and any associated logos are trademarks of LogicBio and/or its affiliates. All other trademarks, trade names and service marks appearing in this Quarterly Report on Form 10-Q are the property of their respective owners. The use or display of other parties’ trademarks, trade dress or products in this Quarterly Report on Form 10-Q does not imply that we have a relationship with, or endorsement or sponsorship of, the trademark or trade dress owners. Any website addresses given in this Quarterly Report on Form 10-Q are for information only and are not intended to be an active link or to incorporate any website information into this document.

4

RISK FACTORS SUMMARY

The following is a summary of the principal risks that could adversely affect our business, financial condition and results of operations:

Risks Related to the Proposed Acquisition by Alexion

• We may not complete the pending transactions with Alexion within the time frame we anticipate, or at all, which could have an adverse effect on our business, financial results and/or operations.

• The announcement and pendency of the transactions with Alexion could adversely affect our business, financial results and/or operations.

• While the Merger Agreement is in effect, we are subject to restrictions on our business activities.

• The Merger Agreement limits our ability to pursue alternative transactions, which could deter a third party from proposing an alternative transaction.

• If the Merger occurs, our stockholders will not be able to participate in any upside to our business.

• Stockholder complaints have been filed, and others could be filed, against us and the members of our Board arising out of the proposed Merger, which may delay or prevent the proposed Merger, and could result in significant costs of defense, indemnification and liability.

• We have incurred, and will continue to incur, direct and indirect costs as a result of the pending transactions with Alexion.

Risks Related to Our Financial Position and Need for Additional Capital

• We have incurred significant losses since inception and anticipate that we will incur continued losses for the foreseeable future. We may never achieve or maintain profitability.

• Under our ASC 205-40 analysis, there is “substantial doubt” that we will have sufficient funds to satisfy our obligations through the next twelve months from the date of issuance of this Quarterly Report on Form 10-Q.

• We will require additional capital to fund our operations, and if we fail to obtain necessary financing, we may not be able to complete the development and commercialization of any product candidates.

Risks Related to Discovery, Development, Clinical Testing, Manufacturing and Regulatory Approval

• Our product candidates may cause serious adverse events or undesirable side effects or have other properties that may delay or prevent their regulatory approval, limit the commercial profile of an approved label or result in significant negative consequences following marketing approval, if any.

• We intend to identify and develop product candidates based on our novel GeneRide and sAAVy technology platforms, which makes it difficult to predict the time and cost of product candidate development.

• Because gene delivery is novel and the regulatory landscape that governs any product candidates we may develop is uncertain and may change, we cannot predict the time and cost of obtaining regulatory approval, if we receive it at all, for any product candidates we may develop.

• Clinical trials are expensive, time-consuming, difficult to design and implement and involve an uncertain outcome.

• If we encounter difficulties enrolling patients in our clinical trials, our clinical development activities could be delayed or otherwise adversely affected.

• Even if we complete the necessary clinical trials, we cannot predict when, or if, we will obtain regulatory approval to commercialize a product candidate we may develop, and any such approval may be for a more narrow indication than we seek.

• We may not be successful in our efforts to identify additional product candidates.

• The regulatory approval processes of the FDA, the EMA and other regulatory authorities are lengthy, time consuming and inherently unpredictable, and if we are ultimately unable to obtain regulatory approval for our product candidates, our business will be substantially harmed.

• We are heavily dependent on the success of LB-001 and if LB-001 does not receive regulatory approval in the United States or other jurisdictions, or is not successfully commercialized, our business will be harmed.

• Interim “top-line” and preliminary data from our clinical trials that we announce or publish from time to time may change as more patient data become available and are subject to audit and verification procedures that could result in material changes in the final data.

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Risks Related to Our Dependence on Third Parties

• Reliance on third-party manufacturers increases the risk that we will not have sufficient quantities of testing materials, product candidates or any medicines that we may develop and commercialize, or that such supply will not be available to us at an acceptable cost.

• If the third parties that conduct, supervise and monitor our clinical trials do not successfully carry out their contractual duties, or if they perform in an unsatisfactory manner, it may harm our business.

• Collaborations we enter into with third parties for the research, development and commercialization of certain of our product candidates or technologies may not be successful, we may not be able to capitalize on the market potential of those product candidates or technologies.

• Our collaborators or strategic partners may decide to adopt alternative technologies or may be unable to develop commercially viable products with our technology, which would negatively impact our revenues and our strategy to develop these products.

• If we fail to comply with obligations in the agreements under which we license intellectual property rights from third parties or otherwise experience disruptions to our business relationships with our licensors, we could lose license rights that are important to our business.

Risks Related to Our Intellectual Property

• If we are unable to obtain and maintain sufficient patent protection for any product candidates and for our technology, our competitors could develop and commercialize products and technology similar or identical to ours.

• If we fail to comply with our obligations in the agreements under which we license intellectual property rights from third parties or otherwise experience disruptions to our business relationships with our licensors, we could lose license rights that are important to our business.

• Patent terms and market exclusivity for our product candidates may be inadequate to protect our competitive position for an adequate amount of time.

• The intellectual property landscape around genetic medicines is highly dynamic, and third parties may initiate legal proceedings alleging that we are infringing, misappropriating, or otherwise violating their intellectual property rights, the outcome of which would be uncertain.

• We may be subject to claims challenging the inventorship of our patents and other intellectual property.

Risks Related to Healthcare Laws and Other Legal Compliance Matters

• Our current and future business operations are and will be subject to applicable healthcare regulatory laws, which could expose us to penalties and other sanctions.

• We are subject to stringent privacy laws, information security laws, regulations, policies and contractual obligations related to data privacy and security and changes in such laws, regulations, policies and contractual obligations could adversely affect our business.

Risks Related to Employee Matters and Managing Growth

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

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PART I—FINANCIAL INFORMATION

Item 1. Financial Statements (Unaudited).

LogicBio Therapeutics, Inc.

Condensed Consolidated Balance Sheets

(In thousands, except share and per share data)

	September 30, 2022			December 31, 2021
ASSETS
CURRENT ASSETS:
Cash and cash equivalents	$	30,780		$	53,480
Accounts receivable		—			30
Prepaid expenses and other current assets		1,996			2,156
Total current assets		32,776			55,666
Property and equipment, net		1,503			1,911
Restricted cash		622			622
Operating lease right-of-use asset		3,700			4,571
TOTAL ASSETS	$	38,601		$	62,770
LIABILITIES AND STOCKHOLDERS’ EQUITY
CURRENT LIABILITIES:
Accounts payable	$	1,111		$	718
Accrued expenses and other current liabilities		2,321			3,704
Operating lease liabilities		1,335			1,227
Current portion of long-term debt		3,306			3,295
Current portion of deferred revenue		8,360			10,639
Total current liabilities		16,433			19,583
Long-term debt, net of issuance costs and discount		2,626			5,006
Operating lease liabilities, net of current portion		2,711			3,725
Deferred revenue, net of current portion		1,092			3,729
Total liabilities		22,862			32,043
COMMITMENTS AND CONTINGENCIES (Note 11)
STOCKHOLDERS’ EQUITY:
Preferred stock, par value of $0.0001 per share; 25,000,000 shares authorized; no shares issued and outstanding as of September 30, 2022 and December 31, 2021		—			—
Common stock, par value of $0.0001 per share; 175,000,000 shares authorized; 32,962,733 and 32,952,306 shares issued and outstanding as of September 30, 2022 and December 31, 2021, respectively		3			3
Additional paid-in capital		173,237			170,744
Accumulated deficit		(157,501	)		(140,020	)
Total stockholders’ equity		15,739			30,727
TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY	$	38,601		$	62,770

See notes to unaudited condensed consolidated financial statements.

7

LogicBio Therapeutics, Inc.

Condensed Consolidated Statements of Operations

(In thousands, except share and per share data)

	Three Months Ended September 30,						Nine Months Ended September 30,
	2022			2021			2022			2021
REVENUE
Collaboration and service revenue	$	2,717		$	2,120		$	8,732		$	3,383
Total revenue		2,717			2,120			8,732			3,383
OPERATING EXPENSES
Research and development		5,051			7,806			15,524			21,482
General and administrative		3,410			4,257			10,293			12,081
Total operating expenses		8,461			12,063			25,817			33,563
LOSS FROM OPERATIONS		(5,744	)		(9,943	)		(17,085	)		(30,180	)
OTHER INCOME (EXPENSE):
Interest income		147			3			197			13
Interest expense		(176	)		(270	)		(591	)		(824	)
Other (expense) income, net		(2	)		(3	)		(2	)		(3	)
Total other expense, net		(31	)		(270	)		(396	)		(814	)
Loss before income taxes		(5,775	)		(10,213	)		(17,481	)		(30,994	)
Income tax benefit	$	—			28			—			28
Net loss	$	(5,775	)	$	(10,185	)	$	(17,481	)	$	(30,966	)
Net loss per share—basic and diluted	$	(0.18	)	$	(0.31	)	$	(0.53	)	$	(0.96	)
Weighted-average common stock outstanding—basic and diluted		32,962,733			32,443,960			32,962,221			32,181,912

See notes to unaudited condensed consolidated financial statements.

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LogicBio Therapeutics, Inc.

Condensed Consolidated Statements of Comprehensive Loss

(In thousands)

	Three Months Ended September 30,						Nine Months Ended September 30,
	2022			2021			2022			2021
Net loss	$	(5,775	)	$	(10,185	)	$	(17,481	)	$	(30,966	)
Other comprehensive income:
Unrealized gain on investments		—			—			—			—
Foreign currency translation adjustment		—			—			—			—
Comprehensive loss	$	(5,775	)	$	(10,185	)	$	(17,481	)	$	(30,966	)

See notes to unaudited condensed consolidated financial statements.

9

LogicBio Therapeutics, Inc.

Condensed Consolidated Statements of Stockholders’ Equity

(In thousands, except share and per share data)

	Common Stock $0.0001 Par Value				Additional Paid-in		Accumulated Other Comprehensive		Accumulated			Total Stockholders’
	Shares		Amount		Capital		Income (Loss)		Deficit			Equity
BALANCE, January 1, 2021		31,775,748	$	3	$	161,415	$	—	$	(99,991	)	$	61,427
Vesting of restricted stock		31,372		—		—		—		—			—
Issuance of common stock related to at-the-market offerings, net of issuance costs of $65		251,086		—		2,091		—		—			2,091
Stock-based compensation expense		—		—		989		—		—			989
Net loss		—		—		—		—		(10,282	)		(10,282	)
BALANCE, March 31, 2021		32,058,206		3		164,495		—		(110,273	)		54,225
Vesting of restricted stock		84,384		—		—		—		—			—
Exercise of options		70,620		—		52							52
Issuance of common stock related to at-the-market offerings, net of issuance costs of $1		9,156		—		45		—		—			45
Stock-based compensation expense		—		—		997		—		—			997
Net loss		—		—		—		—		(10,499	)		(10,499	)
BALANCE, June 30, 2021		32,222,366	$	3	$	165,589	$	—	$	(120,772	)	$	44,820
Vesting of restricted stock		11,489		—		—		—		—			—
Exercise of options		49,926		—		35							35
Issuance of common stock related to at-the-market offerings, net of issuance costs of $92		661,835		—		2,962		—		—			2,962
Stock-based compensation expense		—		—		1,132		—		—			1,132
Net loss		—		—		—		—		(10,185	)		(10,185	)
BALANCE, September 30, 2021		32,945,616	$	3	$	169,718	$	—	$	(130,957	)	$	38,764
BALANCE, January 1, 2022		32,952,306	$	3	$	170,744	$	—	$	(140,020	)	$	30,727
Vesting of restricted stock		10,427		—		—		—		—			—
Stock-based compensation expense		—		—		846		—		—			846
Net loss		—		—		—		—		(6,662	)		(6,662	)
BALANCE, March 31, 2022		32,962,733		3		171,590		—		(146,682	)		24,911
Stock-based compensation expense		—		—		849		—		—			849
Net loss		—		—		—		—		(5,044	)		(5,044	)
BALANCE, June 30, 2022		32,962,733	$	3	$	172,439	$	—	$	(151,726	)	$	20,716
Stock-based compensation expense		—		—		798		—		—			798
Net loss		—		—		—		—		(5,775	)		(5,775	)
BALANCE, September 30, 2022		32,962,733	$	3	$	173,237	$	—	$	(157,501	)	$	15,739

See notes to unaudited condensed consolidated financial statements.

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LogicBio Therapeutics, Inc.

Condensed Consolidated Statements of Cash Flows

(In thousands)

	Nine Months Ended September 30,
	2022			2021
CASH FLOWS FROM OPERATING ACTIVITIES:
Net loss	$	(17,481	)	$	(30,966	)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation expense		508			451
Stock-based compensation expense		2,493			3,118
Non-cash interest expense		131			173
Non-cash lease expense		836			811
Changes in operating assets and liabilities:
Prepaid expenses and other current assets		160			1,220
Accounts receivable		30			144
Accounts payable		393			456
Accrued expenses and other current liabilities		(2,254	)		26
Deferred revenue		(4,916	)		10,733
Net cash used in operating activities		(20,100	)		(13,834	)
CASH FLOWS FROM INVESTING ACTIVITIES:
Purchases of property and equipment		(100	)		(734	)
Net cash used in investing activities		(100	)		(734	)
CASH FLOWS FROM FINANCING ACTIVITIES:
Proceeds from exercise of stock options		—			87
Net proceeds from at-the-market common stock issuances		—			5,098
Principal repayments on term loan		(2,500	)		(1,111	)
Net cash (used in) provided by financing activities		(2,500	)		4,074
NET DECREASE IN CASH, CASH EQUIVALENTS AND RESTRICTED    CASH		(22,700	)		(10,494	)
Cash, cash equivalents and restricted cash at beginning of year		54,102			70,697
Cash, cash equivalents and restricted cash at end of period	$	31,402		$	60,203
RECONCILIATION OF CASH, CASH EQUIVALENTS AND RESTRICTED    CASH
Cash and cash equivalents	$	30,780		$	59,581
Long-term restricted cash		622			622
Total cash, cash equivalents and restricted cash	$	31,402		$	60,203
SUPPLEMENTAL DISCLOSURE OF CASH FLOW INFORMATION:
Cash paid for interest	$	460		$	651
SUPPLEMENTAL DISCLOSURE OF NON-CASH ACTIVITIES:
Property and equipment purchases in accrued expenses and accounts payable	$	—		$	48

See notes to unaudited condensed consolidated financial statements.

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LogicBio Therapeutics, Inc.

Notes to Unaudited Condensed Consolidated Financial Statements

(Dollars in thousands, except share and per share data)

1. NATURE OF BUSINESS AND BASIS OF PRESENTATION

Business Overview

LogicBio^® Therapeutics, Inc. (“LogicBio” or the “Company”) was incorporated in 2014 as a Delaware corporation. Its principal office is in Lexington, Massachusetts. LogicBio is a clinical-stage genetic medicine company pioneering genome editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood. The Company's genome editing platform, GeneRide^®, is a new approach to precise gene insertion harnessing a cell's natural deoxyribonucleic acid (“DNA”) repair process potentially leading to durable therapeutic protein expression levels. The Company's gene delivery platform, sAAVy™, is an adeno-associated virus (“AAV”) capsid engineering platform designed to optimize gene delivery for treatments in a broad range of indications and tissues. The Company’s proprietary manufacturing process, mAAVRx^TM, is a novel process aimed at improving production yields for viral vector manufacturing.

The Company’s lead product candidate, LB-001, is a single-administration, genome editing therapy developed using its GeneRide technology, currently in Phase 1/2 development for the treatment of methylmalonic acidemia (“MMA”) in pediatric patients. MMA is a rare and life-threatening genetic disorder affecting approximately 1 in 50,000 newborns in the United States that often results in developmental delays and other long-term complications and a high rate of hospitalizations.

In April 2021, the Company entered into an Exclusive Research Collaboration, License and Option Agreement (the “Original CANbridge Agreement”) with CANbridge Care Pharma Hong Kong Limited (“CANbridge”), pursuant to which LogicBio granted CANbridge (a) an exclusive worldwide license to certain of the Company’s intellectual property rights, including those relating to AAV sL65 (“sL65”), the first capsid produced from the sAAVy platform, to develop, manufacture and commercialize gene therapy candidates for the treatment of Fabry and Pompe diseases, (b) an option to obtain an exclusive worldwide license to certain of the Company’s intellectual property rights, including those relating to sL65, to develop and commercialize gene therapy candidates for the treatment of two additional indications, and (c) an exclusive option to obtain an exclusive license to develop and commercialize LB-001 for the treatment of MMA in China, Taiwan, Hong Kong and Macau. The Original CANbridge Agreement was amended and restated on October 3, 2022. For the description of the amended and restated agreement, see Note 13 in the accompanying notes to our unaudited consolidated financial statements. Also in April 2021, the Company announced a research collaboration with Daiichi Sankyo Company, Limited (“Daiichi”) for the development of treatments for two indications based on GeneRide. In addition, the Company entered into a research collaboration with Takeda Pharmaceutical Company Limited (“Takeda”) in January 2020 to develop LB-301, an investigational therapy leveraging GeneRide, for the treatment of Crigler-Najjar syndrome (“CN”), a rare pediatric disease. The work under the research plan was completed in 2021.  

Since its inception, the Company has devoted the majority of its efforts to research and development, including its preclinical and clinical development activities and its manufacturing and process development activities, raising capital, and providing general and administrative support for these operations. The Company is subject to a number of risks similar to those of other companies conducting high-risk, early-stage research and development. Principal among these risks are clinical and regulatory risks associated with drug development, dependency on key individuals and intellectual property, competition from other products and companies, and the technical risks associated with the successful research, development and clinical manufacturing of its product candidates. The Company’s success is dependent upon its ability to continue to raise additional capital in order to fund ongoing research and development, meet its obligations and, ultimately, obtain regulatory approval of its product candidates, successfully commercialize its products and technologies, and if approved, generate revenue and attain profitable operations.

Proposed Acquisition by Alexion Pharmaceuticals, Inc.

On October 3, 2022, the Company entered into an Agreement and Plan of Merger (as it may be amended from time to time, the “Merger Agreement”), by and among the Company, Alexion Pharmaceuticals, Inc., a Delaware corporation (“Alexion” or “Parent”), and Camelot Merger Sub, Inc., a Delaware corporation and a wholly-owned subsidiary of Parent (“Merger Sub”). Pursuant to the Merger Agreement, on October 18, 2022, Merger Sub commenced a tender offer to purchase, subject to the satisfaction or waiver of certain conditions set forth in the Merger Agreement, any and all of the issued and outstanding shares of common stock, par value $0.0001 per share, of the Company (the “Shares”), at a price of $2.07 per Share, to the seller in cash, without interest, less any applicable withholding taxes, upon the terms and subject to the conditions set forth in the Offer to Purchase, dated October 18, 2022 (the “Offer to Purchase,” and together with the Letter of Transmittal and other related materials, as may be amended, which together constitute the “Offer”).

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On the same date as the consummation of the Offer and subject to the satisfaction or waiver of certain conditions set forth in the Merger Agreement, Merger Sub will be merged with and into the Company (the “Merger”) without a vote of the Company’s stockholders to adopt the Merger Agreement and consummate the Merger, in accordance with Section 251(h) of the of the Delaware General Corporation Law (the “DGCL”), with the Company continuing as the surviving corporation and as a wholly-owned subsidiary of Parent. The Offer is subject to certain important conditions; see “Item 1A. Risk Factors” for more information.

COVID-19 Impact

The Company continues to closely monitor the COVID-19 pandemic in order to promote the safety of its personnel and to continue advancing its research and development activities. The Company is following federal, state and local requirements and guidelines with respect to the COVID-19 pandemic and has allowed its employees to work on-premises in accordance with those requirements and guidelines.

The COVID-19 pandemic did not have a material impact on the Company’s results of operations, cash flow and financial position as of and for the quarter ended September 30, 2022. However, the Company is aware that certain of its third-party vendors are being affected by import/export and other restrictions due to COVID-19, which may have an impact on certain of the Company’s research, development and manufacturing activities. The full extent to which the COVID-19 pandemic, including the development of new variants, could directly or indirectly impact the Company’s business, results of operations and financial position will depend on future developments that are uncertain and cannot be accurately predicted.

We cannot predict the impact of the progression of the COVID-19 pandemic on future results due to a variety of factors, including the health of our and our third-party vendors’, suppliers’ and collaborators’ employees, our ability to maintain operations, the ability of our third-party vendors, suppliers and collaborators to continue operations, any further government and/or public actions taken in response to the pandemic and ultimately the length of the pandemic. We plan to continue to closely monitor the COVID-19 pandemic in order to ensure the safety of our personnel and to continue advancing our research and development activities.

Liquidity and Capital Resources

The Company has had recurring losses since inception and incurred a loss of $17,481 during the nine months ended September 30, 2022. Net cash used in operations for the nine months ended September 30, 2022 was $20,100. The Company expects to continue to generate operating losses and use cash in operations for the foreseeable future. As of September 30, 2022, the Company had cash and cash equivalents of $30,780 which management believes will be sufficient to fund its operating expenses and capital expenditure requirements into the second quarter of 2023, however due to the uncertainties described below, there is substantial doubt about the Company’s ability to continue as a going concern. On a quarterly basis, the Company is required to conduct an accounting analysis under ASC 205-40, Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern, (“ASC 205-40”). The result of the Company’s ASC 205-40 analysis is that there is substantial doubt about the Company’s ability to continue as a going concern through the next twelve months from the date of issuance of these unaudited condensed consolidated financial statements.

The Company will require substantial additional capital to fund its research and development, including its preclinical and clinical development activities and its manufacturing and process development activities, and ongoing operating expenses.  In connection with the proposed Merger with Alexion, following a consummation of the Offer and subject to the satisfaction or waiver of certain conditions set forth in the Merger Agreement, the Company would continue as a wholly-owned subsidiary of Alexion. Without taking into account the proposed Merger with Alexion, management’s plans to mitigate the conditions that raise substantial doubt on its ability to fund ongoing R&D and other operating activities include raising additional capital through equity or debt financings, payments from its collaborators, strategic transactions, or a combination of those approaches. These plans may also include the possible elimination or deferral of certain research programs or operating expenses unless and until additional capital is received. There can be no assurance that the Company will be successful in raising additional capital or that such capital, if available, will be on terms that are acceptable to the Company, its Board of Directors, or that the Company will be successful in deferring certain operating expenses.

The accompanying unaudited interim condensed consolidated financial statements have been prepared on a going concern basis, which contemplates the realization of assets and satisfaction of liabilities in the ordinary course of business. The unaudited condensed consolidated financial statements do not include any adjustments relating to the recoverability and classification of recorded asset amounts or the amounts and classification of liabilities that might result from the outcome of this uncertainty.

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Basis of Presentation

The accompanying unaudited condensed consolidated financial statements have been prepared by the Company in conformity with accounting principles generally accepted in the United States of America (“U.S. GAAP”) and pursuant to the rules and regulations of the U.S. Securities and Exchange Commission (“SEC”) for interim financial statements. Certain information and footnote disclosures normally included in financial statements prepared in accordance with U.S. GAAP have been condensed or omitted pursuant to such rules and regulations. However, the Company believes that the disclosures are adequate to make the information presented not misleading. These unaudited condensed consolidated financial statements should be read in conjunction with the Company’s audited consolidated financial statements and the notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2021, filed with the SEC on March 4, 2022.

The unaudited interim condensed consolidated financial statements have been prepared on the same basis as the audited consolidated financial statements. In the opinion of management, the accompanying unaudited interim condensed consolidated financial statements contain all adjustments that are necessary for a fair statement of the Company’s financial position as of September 30, 2022, consolidated results of operations for the three and nine months ended September 30, 2022 and 2021 and cash flows for the nine months ended September 30, 2022 and 2021. Such adjustments are of a normal and recurring nature. The results of operations for the three and nine months ended September 30, 2022 are not necessarily indicative of the results of operations that may be expected for the year ending December 31, 2022.

2. SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES

Significant Accounting Policies

The Company’s significant accounting policies are disclosed in the audited consolidated financial statements and the notes thereto, which are included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2021, filed with the SEC on March 4, 2022. Since the date of those financial statements, there have been no material changes to its significant accounting policies.

Recently Issued Accounting Pronouncements

From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board (“FASB”) or other standard setting bodies that are adopted by the Company as of the specified effective date. The Company believes that the impact of recently issued standards that are not yet effective will not have a material impact on the Company’s consolidated financial statements upon adoption.

In March 2020, the FASB issued Accounting Standards Update (“ASU”) 2020-04, Facilitation of the Effects of Reference Rate Reform on Financial Reporting (Topic 848). This ASU provides optional expedients and exceptions for applying U.S. GAAP to transactions affected by reference rate (e.g., LIBOR) reform if certain criteria are met, for a limited period of time to ease the potential burden in accounting for (or recognizing the effects of) reference rate reform on financial reporting. The ASU is effective now through December 31, 2022. The Company will evaluate transactions or contract modifications, including any related to its July 2019 loan and security agreement that uses LIBOR as a reference rate, occurring as a result of reference rate reform and determine whether to apply the optional guidance on an ongoing basis. The ASU is currently not expected to have a material impact on the Company’s consolidated financial statements and related disclosures.

3. FAIR VALUE MEASUREMENTS

The following tables present information about the Company’s financial assets measured at fair value on a recurring basis and indicate the level of the fair value hierarchy utilized to determine such fair values:

Description	September 30, 2022		Quoted Prices in Active Markets for Identical Assets (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Other Observable Inputs (Level 3)
Assets
Money market funds and other cash equivalents	$	29,834	$	29,834	$	—	$	—
Total financial assets	$	29,834	$	29,834	$	—	$	—

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Description	December 31, 2021		Quoted Prices in Active Markets for Identical Assets (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Other Observable Inputs (Level 3)
Assets
Money market funds and other cash equivalents	$	52,866	$	52,866	$	—	$	—
Total financial assets	$	52,866	$	52,866	$	—	$	—

When developing fair value estimates, the Company maximizes the use of observable inputs and minimizes the use of unobservable inputs. When available, the Company uses quoted market prices to measure fair value. The valuation technique used to measure fair value for the Company’s financial assets is a market approach, using prices and other relevant information generated by market transactions involving identical or comparable assets. If market prices are not available, the fair value measurement is based on models that use primarily market-based parameters including yield curves, volatilities, credit ratings and currency rates. In certain cases where market rate assumptions are not available, the Company is required to make judgments about assumptions market participants would use to estimate the fair value of a financial instrument.

The Company did not have any transfers of assets between levels of the fair value measurement hierarchy during the nine months ended September 30, 2022.

4. ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES

Accrued expenses and other current liabilities at September 30, 2022 and December 31, 2021 consisted of the following:

	September 30, 2022		December 31, 2021
Accrued compensation and benefits	$	1,648	$	2,289
Accrued professional services		665		1,341
Other		8		74
Total accrued expenses and other current liabilities	$	2,321	$	3,704

Accrued compensation and benefits consists primarily of accrued bonuses. Accrued professional services consists primarily of consulting services, legal services and services provided by contract research organizations (“CRO”) and contract manufacturing organizations (“CMO”).

5. DEBT

On July 2, 2019 (the “Closing Date”), the Company entered into a loan and security agreement (the “Loan Agreement”), for term loans with Oxford Finance LLC (“Oxford”) and Horizon Technology Finance Corporation (“Horizon,” and, together with Oxford, the “Lenders”). The Loan Agreement allowed the Company to borrow up to $20,000 issuable in two equal tranches (the “Term Loans”). On the Closing Date, the first tranche of $10,000 was drawn down by the Company. In September 2020 and March 2021, the Company entered into amendments to the Loan Agreement, each of which extended the availability of the second $10,000 tranche subject to certain conditions. In the second quarter of 2021, the Company met the conditions necessary to initiate the drawdown of the second tranche but did not exercise its right to do so, and the option to draw down the second tranche of the Term Loans expired unused.

The outstanding loan balance will accrue interest at the greater of (i) the rate of the one-month U.S. LIBOR rate plus 6.25% and (ii) 8.75%. The Loan Agreement provides for an interest only period until July 1, 2021, followed by thirty-six equal monthly payments of principal and interest continuing through June 1, 2024 (the “Maturity Date”). The Company has the option to prepay the outstanding balance prior to maturity, subject to a prepayment fee ranging from 1.0% to 3.0% depending upon when the prepayment occurs. Upon repayment of the Term Loans, the Company is required to make a final payment to the Lenders equal to 4.5% of the original principal amount of the Term Loans funded which will be accrued by charges to interest expense over the term of the loans using the effective interest method.

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In conjunction with the Loan Agreement, the Company issued warrants to the Lenders to purchase 15,686 shares of common stock (“Warrants”) at a per share exercise price of $12.75, a maximum contractual term of 10 years and exercisable immediately. The fair value of the Warrants was accounted for as a debt discount and calculated to be approximately $136 using the Black-Scholes method. The Company determined the Warrants met the criteria for equity classification, and, as such, the fair value of the Warrants is recorded as additional paid-in capital on the condensed consolidated balance sheets. Finally, the Company incurred costs related to the issuance of the Term Loans of approximately $150. Both the debt discount and issuance costs will be accreted to Long-term debt, net of issuance costs and discount by charges to interest expense over the term of the loan using the effective interest method.

The Loan Agreement contains customary representations, warranties and covenants and also includes customary events of default. Events of default include, among other things, the non-payment of principal, interest, and other amounts when due, breach of covenants, a material adverse change event, cross-default on material indebtedness, misrepresentations, material judgments, bankruptcy and delisting. Upon the occurrence of an event of default, a default interest rate of an additional 5.0% per annum may be applied to the outstanding loan balances, and the Lenders may declare all outstanding obligations immediately due and payable. Borrowings under the Loan Agreement are collateralized by substantially all the Company’s assets, other than its intellectual property, which include maintaining certain cash balances in controlled accounts.

Interest expense related to the Loan Agreement was $176 and $591 for the three and nine months ended September 30, 2022, respectively. Interest expense related to the Loan Agreement was $270 and $824 for the three and nine months ended September 30, 2021, respectively. The effective rate on the Loan Agreement, including the amortization of the debt discount and issuance costs, and accretion of the final payment, was 11.34% at September 30, 2022. The components of the long-term debt balance are as follows:

	September 30, 2022			December 31, 2021
Notes payable, gross	$	5,556		$	8,055
Less: Unamortized debt discount and    issuance costs		(46	)		(93	)
Accretion of final payment fee		422			339
Carrying value of notes payable		5,932			8,301
Less: Current portion of long-term debt		(3,306	)		(3,295	)
Long-term debt, net of issuance costs and    discount	$	2,626		$	5,006

As of September 30, 2022, the estimated future principal payments due were as follows:

	As of September 30, 2022
2022	$	833
2023		3,333
2024		1,389
Thereafter		—
Total principal payments	$	5,555

6. STOCK-BASED COMPENSATION

Equity Incentive Plans

In December 2014, the Company adopted the LogicBio Therapeutics, Inc. 2014 Equity Incentive Plan, as amended (the “2014 Plan”), for the issuance of stock options and other stock-based awards. Subsequently, in October 2018, the Company’s 2018 Equity Incentive Plan (the “2018 Plan”) was adopted, superseding the 2014 Plan; no further awards have been, or will be, made under the 2014 Plan. The 2018 Plan was established to provide equity-based ownership opportunities for employees and directors, as well as outside consultants and advisors. Any awards granted under the 2014 Plan prior to the adoption of the 2018 Plan remained outstanding in accordance with their respective terms.

Under the 2018 Plan, there is an annual increase on January 1 of each year from 2019 until 2028, of the lesser of (i) 4% of the number of shares of common stock outstanding on December 31 of the prior year or (ii) a number of shares of common stock as determined by the Company’s Board of Directors (“Board”). Accordingly, on January 1, 2022, the number of shares available for future grant under the 2018 Plan increased by 1,318,271 shares. At September 30, 2022, there were 997,841 shares available for future grant under the 2018 Plan.

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The 2018 Plan is administered by the Compensation Committee of the Board, except with respect to such matters that are not delegated to the Compensation Committee by the Board (collectively, the “Administrator”). The exercise prices, vesting and certain other restrictions are determined at the discretion of the Administrator, except that the exercise price per share of stock options may not be less than 100% of the fair market value of the common stock on the date of grant. The term of stock options awarded under the 2018 Plan may not exceed 10 years from the grant date. Stock options, shares of restricted stock and restricted stock units (“RSUs”) granted to employees, officers, members of the Board, advisors, and consultants of the Company typically vest over one to four years.  

Stock Options

During the nine months ended September 30, 2022 and 2021, the Company granted options with time-based vesting to purchase 1,672,250   and 2,097,256 shares of common stock, respectively, with a weighted-average grant date fair value per share of $0.44 and $4.13, respectively. The Company recorded stock-based compensation expense for options granted of $2,491 and $2,812 during the nine months ended September 30, 2022 and 2021, respectively. As of September 30, 2022, there were 5,390,188 outstanding options, of which 2,931,063 were unvested, and $6,019 of unrecognized stock-based compensation expense to be recognized over a weighted-average period of 2.3 years.

Restricted Stock

The Company has granted shares of restricted stock with time-based and performance-based vesting conditions from time to time, and subsequent grants may be made. The Company did not grant any restricted stock during the nine months ended September 30, 2022 or 2021. During the nine months ended September 30, 2022, no expense was recorded for restricted stock previously granted. During the nine months ended September 30, 2021, the Company recorded stock-based compensation expense of $132 related to restricted stock previously granted. As of September 30, 2022, there were no unvested shares of restricted stock outstanding.

Restricted Stock Units

The Company has granted RSUs with time-based vesting conditions from time to time. Each RSU represents the right to receive one share of the Company’s common stock upon vesting. The fair value is calculated based upon the Company’s closing stock price on the date of grant, and the stock-based compensation expense is recognized over the vesting period. The Company did not grant any RSUs during the nine months ended September 30, 2022. The Company granted 5,939 RSUs during the nine months ended September 30, 2021. The Company recorded stock-based compensation for RSUs of $2 and $174 during the nine months ended September 30, 2022 and 2021, respectively. As of September 30, 2022, there were no unvested RSUs outstanding.

Stock-Based Compensation Expense

Total stock-based compensation expense recorded as research and development and general and administrative expenses, respectively, for employees, directors and non-employees for the nine months ended September 30, 2022 and 2021 is as follows:

	Nine Months Ended September 30,
	2022		2021
Research and development	$	872	$	1,051
General and administrative		1,621		2,067
Total stock-based compensation expense	$	2,493	$	3,118

7. STOCKHOLDERS’ EQUITY

Open Market Sale Agreement

On November 15, 2019, the Company entered into an Open Market Sale Agreement (the “Open Market Sale Agreement”) with Jefferies LLC, as agent (“Jefferies”), and filed a related prospectus supplement, pursuant to which the Company may issue and sell shares of its common stock at the then current market prices having an aggregate offering price of up to $50,000 (the “Open Market Shares”) from time to time through Jefferies (the “Open Market Offering”).

Under the Open Market Sale Agreement, Jefferies may sell the Open Market Shares by methods deemed to be an “at the market offering” as defined in Rule 415(a)(4) promulgated under the Securities Exchange Act of 1934, as amended. The Company may sell the Open Market Shares in amounts and at times to be determined by the Company from time to time subject to the terms and conditions of the Open Market Sale Agreement, but it has no obligation to sell any of the Open Market Shares in the Open Market Offering.

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The Company or Jefferies may suspend or terminate the offering of Open Market Shares upon notice to the other party and subject to other conditions. The Company has agreed to pay Jefferies commissions for its services in acting as agent in the sale of the Open Market Shares in the amount of up to 3.0% of gross proceeds from the sale of the Open Market Shares pursuant to the Open Market Sale Agreement. The Company has also agreed to provide Jefferies with customary indemnification and contribution rights.

During the nine months ended September 30, 2022, the Company did not issue any Open Market Shares. During the nine months ended September 30, 2021, the Company issued 922,077 Open Market Shares at a weighted-average price of $5.70 per share, resulting in net proceeds to the Company of $5,098. At September 30, 2022, the Company had $41,253 in aggregate gross offering amount available under the Open Market Sale Agreement.

8. REVENUE

Service Revenue

Takeda Agreement

In January 2020, the Company entered into a Research Collaboration and Option Agreement with Takeda (“Takeda Agreement”), which expired by its own terms in the year ended December 31, 2021.

The Company assessed the Takeda Agreement in accordance with ASC 606 and concluded that it represented a contract with a customer and was within the scope of ASC 606. The promised goods and services represented one combined performance obligation and the entire transaction price was allocated to that single combined performance obligation. In addition, the Company concluded that the exclusive option to obtain an exclusive license to LB-301 granted to Takeda under the Takeda Agreement (the “License Option”) did not provide any discounts or other rights. Terms related to an exclusive license negotiated after the exercise of the License Option would be part of a separate contract and reflect applicable standalone selling prices. As such, the Company concluded the License Option was not considered to be a material right.

Under the Takeda Agreement, Takeda was obligated to reimburse the Company for the costs incurred under the research plan. Costs incurred were billed by the Company to Takeda from time to time. The Company elected to recognize revenue under the "right to invoice" practical expedient based on the Company's right to invoice Takeda at an amount that approximated the value transferred to the customer and the performance completed to date. During 2022, there was no service revenue recognized under the Takeda Agreement. During the three and nine months ended September 30, 2021, the Company recognized $43 and $556, respectively, in service revenue under the Takeda Agreement.

Daiichi Sankyo Agreement

In April 2021, the Company entered into a Research Collaboration and Exclusive Option Agreement with Daiichi (the “Daiichi Agreement”) for the development of gene therapy candidates for two indications based on the GeneRide platform (each, a “Daiichi Candidate”). Under the terms of the Daiichi Agreement, Daiichi will fund all research and development activities related to the development of the Daiichi Candidates under a mutually agreed research plan (the “Daiichi Research Plan”). The Daiichi Agreement also provides Daiichi with an exclusive, non-binding option for each Daiichi Candidate to negotiate in good faith for a certain period of time to enter into a license agreement with respect to each such Daiichi Candidate (the “Daiichi License Options”).

The Company assessed the Daiichi Agreement in accordance with ASC 606 and concluded that it represents a contract with a customer and is within the scope of ASC 606. The Company concluded that its conduct of research services under the Daiichi Research Plan, which includes a research data package, participation in various joint oversight committees, a research license and a materials transfer, represents a single combined performance obligation. The Company determined the transaction price totaled $2,000, which included an upfront payment of $1,000 and an additional $1,000 prepayment of the first-year research and development fees. The entire transaction price will be allocated to the single combined performance obligation, which is transferred over the expected term of the conduct of the research services. Terms related to exclusive licenses negotiated after the exercise of the Daiichi License Options will be part of a separate contract and reflect applicable standalone selling prices. As such, the Company concluded the Daiichi License Options are not considered to be a material right.

The upfront payment of $2,000 was recorded as deferred revenue and is being recognized as revenue over time in conjunction with the Company’s conduct of research services as the research services are the primary component of the combined performance obligation. Revenue associated with the upfront payment and ongoing research services will be recognized using an input-based measurement of actual costs incurred as a percentage of the estimated total costs expected to be incurred over the expected term of conduct of the research services. The Company believes this input-based method to recognize revenue best reflects the transfer of value to Daiichi. During the three and nine months ended September 30, 2022, the Company recognized $382 and $1,124, respectively, as service revenue under the Daiichi Agreement. During the three and nine months ended September 30, 2021, the Company recognized $392

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and $572, respectively, as service revenue under the Daiichi Agreement. As of September 30, 2022, there was $959 in deferred revenue related to the Daiichi Agreement, all of which is classified as current deferred revenue.  

Collaboration Revenue

CANbridge Agreement

In April 2021, the Company entered into an Exclusive Research Collaboration, License and Option Agreement with CANbridge (the “Original CANbridge Agreement”). The Original CANbridge Agreement was amended and restated on October 3, 2022.  For the description of the amended and restated agreement, see Note 13 in the accompanying notes to our unaudited consolidated financial statements.

Under the terms of the Original CANbridge Agreement, the Company granted CANbridge (a) an exclusive worldwide license to certain of the Company’s intellectual property rights, including those relating to sL65, to develop, manufacture and commercialize gene therapy candidates for the treatment of Fabry and Pompe diseases (the “Fabry and Pompe License”), (b) an option to obtain an exclusive worldwide license to certain of the Company’s intellectual property rights, including those relating to sL65, to develop and commercialize gene therapy candidates for the treatment of two additional indications (the “Candidate Options”) and (c) an exclusive option to obtain an exclusive license to develop and commercialize LB-001 for the treatment of MMA (the “LB-001 Option”) in China, Taiwan, Hong Kong and Macau (“Greater China”). Pursuant to the Original CANbridge Agreement, LogicBio and CANbridge collaborated in the development of the gene therapy candidates referenced in (a) above for the treatment of Fabry and Pompe diseases plus, upon CANbridge’s exercise of the applicable Candidate Options, two additional indications under a mutually agreed research plan (the “CANbridge Research Plan”).

Under the Original CANbridge Agreement, the Company received an upfront, non-refundable and non-creditable payment of $10,000 from CANbridge a portion of which was paid to a third party under certain of the Company’s in-licensing obligations. In addition, CANbridge was obligated to reimburse the Company for research and development costs incurred by the Company for activities related to the development of the gene therapy candidates for Fabry and Pompe diseases under the CANbridge Research Plan.

In addition, under the Original CANbridge Agreement, the Company was eligible to receive up to $542,000 in aggregate from CANbridge contingent on the achievement of specified clinical, regulatory and sales milestones relating to the named gene therapy candidates for Fabry and Pompe diseases, the additional indications for which CANbridge exercises the Candidate Options, and the payment of any option exercise fees. The Company was also eligible to receive up to $49,000 in aggregate clinical, regulatory and sales milestones for LB-001, subject to the exercise of the LB-001 Option, and the payment of the LB-001 Option exercise fee. CANbridge was obligated to pay to the Company royalties based on an escalating tiered, mid- to high-single digit percentage of the annual worldwide net sales for each non-LB-001 indication pursued. In addition, CANbridge was obligated to pay to the Company royalties based on an escalating tiered, high-single digit to mid-double digit percentage of the annual Greater China net sales for LB-001 for the treatment of MMA, subject to the exercise of the LB-001 Option.

The Company applied ASC Topic 808, Collaborative Arrangements (“ASC 808”) and determined that the Original CANbridge Agreement was within the scope of ASC 808. Furthermore, the Company determined that certain aspects of the Original CANbridge Agreement represented a vendor-customer relationship as CANbridge represents a customer for certain activities. As such, the Company applied the relevant guidance from ASC 606 to evaluate the appropriate accounting for the vendor-customer aspects of the Original CANbridge Agreement. In accordance with ASC 606, the Company identified its performance obligation as a grant of a license to CANbridge for certain of its intellectual property rights, including those relating to sL65, and its conduct of research services under the CANbridge Research Plan, which included participation in various joint oversight committees and a technology transfer. The Company determined that its grant of a license to CANbridge to certain of its intellectual property subject to certain conditions was not distinct as it did not have stand-alone value to CANbridge apart from the services to be performed by the Company pursuant to the Original CANbridge Agreement. A third party would not be able to provide research and development services due to the specific nature of the intellectual property and knowledge required to perform the services, and CANbridge could not benefit from the license without the corresponding services. The Company also concluded that the LB-001 Option and Candidate Options were not provided to CANbridge at a significant discount. The terms of the options, including the upfront exercise fee and applicable milestone payments, reflected applicable standalone selling prices at the time of the Original CANbridge Agreement. As such, the Company concluded that none of the options were considered to be material rights and, as such, were not performance obligations.  

Accordingly, the Company determined that its grant of a license to CANbridge and its performance of research and development services under the CANbridge Research Plan should be accounted for as one combined performance obligation, and that the combined performance obligation is transferred over the expected term of the conduct of the research and development services. 

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In accordance with ASC 606, the Company determined that the initial transaction price under the Original CANbridge Agreement was $10,878, consisting of the upfront, non-refundable and non-creditable payment of $10,000 and an upfront payment of estimated quarterly research costs $878. The upfront payment of $10,878 was initially recorded as deferred revenue and, along with payments related to the Company’s performance of research services under the CANbridge Research Plan, will be recognized as revenue using an input-based measurement of actual costs incurred as a percentage of the estimated total costs expected to be incurred over the expected term of conduct of the research services. The Company believes this input-based method to recognize revenue best reflects the transfer of value to CANbridge. The Company recorded the initial $878 prepayment of the quarterly research and development fees as deferred revenue, and such fees will be recognized as revenue as the research services are delivered.

The Company also assessed the effects of variable elements including the likelihood of receiving (i) various clinical, regulatory and commercial milestone payments, and (ii) royalties on net sales of any product candidate. Based on its assessment, the Company concluded that, based on the likelihood of these uncertain events occurring, there was not a significant variable element included in the transaction price. Accordingly, the Company has not assigned a transaction price to these variable elements given the substantial uncertainty related to their achievement and has not assigned a transaction price to any CANbridge milestone or royalties.

During the three and nine months ended September 30, 2022, the Company recognized collaboration revenue of $2,335 and $7,608, respectively, under the Original CANbridge Agreement. During the three and nine months ended September 30, 2021, the Company recognized collaboration revenue of $1,685 and $2,255, respectively, under the Original CANbridge Agreement. As of September 30, 2022, aggregate deferred revenue related to the Original CANbridge Agreement was $8,493 of which $7,401 was classified as current. Both the current and non-current deferred revenue amounts will be recognized during the expected term of the conduct of research and development services. As a direct result of the Company’s entry into the Original CANbridge Agreement, the Company incurred $775 in sublicense fees to certain of its existing licensors which was expensed to research and development expense during the second quarter of 2021.

9. INCOME TAXES

For the nine months ended September 30, 2022 and the year ended December 31, 2021, the Company maintained a full valuation allowance on federal and state deferred tax assets since management does not forecast the Company to be in a taxable position in the near future.

10. LOSS PER SHARE

Basic loss per share is computed by dividing net loss by the weighted-average shares of common stock outstanding, without consideration to common stock equivalents:

	Three Months Ended September 30,						Nine Months Ended September 30,
	2022			2021			2022			2021
Numerator:
Net loss	$	(5,775	)	$	(10,185	)	$	(17,481	)	$	(30,966	)
Denominator:
Weighted-average common stock outstanding		32,962,733			32,443,960			32,962,221			32,181,912
Net loss per share — basic and diluted	$	(0.18	)	$	(0.31	)	$	(0.53	)	$	(0.96	)

The Company’s potentially dilutive shares, which include any outstanding stock options, warrants and unvested restricted stock, are considered to be common stock equivalents and are only included in the calculation of diluted net loss when their effect is dilutive.

The Company excluded the following potential common stock equivalents from the computation of diluted net loss per share attributable to common stockholders because including them would have had an anti-dilutive effect for the three and nine months ended September 30, 2022 and 2021.

	September 30, 2022		September 30, 2021
Unvested restricted common stock		—		11,153
Unvested restricted stock units		—		5,939
Options to purchase common stock		5,390,188		4,508,640
Loan warrants		15,686		15,686

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11. COMMITMENTS AND CONTINGENCIES

Operating Lease

The Company has historically entered into lease arrangements for its facilities and certain equipment. As of September 30, 2022, the Company had one operating lease with required future minimum payments related to its headquarters in Lexington, MA.

In November 2019, the Company entered into a lease agreement for office, laboratory and vivarium space located at 65 Hayden Avenue, Lexington, Massachusetts (“65 Hayden Ave Lease”) to replace the Company’s prior headquarters located at 99 Erie Street Cambridge, Massachusetts. Under the terms of the 65 Hayden Ave Lease, the Company leases approximately 23,901 square feet of space and initially paid an annual base rent of approximately $1,494, subject to scheduled annual increases, plus certain operating expenses and taxes. The Company took possession of the space on April 1, 2020 (“Lease Commencement Date”) and the lease will continue through July 1, 2025 (“Lease Termination Date”). The Company has an option to extend the lease for a single additional term of 5 years. Upon execution of the 65 Hayden Ave Lease, the Company executed a $622 cash-collateralized letter of credit. Lease payments are due in monthly installments through the Lease Termination Date.

At the Lease Commencement Date, the Company performed a lease assessment under the guidance prescribed in ASC Topic 842, Leases (“ASC 842”), and concluded that the 65 Hayden Ave Lease was an operating lease. As such, the Company recorded an operating lease right-of-use asset and corresponding operating lease liability on the consolidated balance sheets of $6,428 which reflected the net present value of future payments under the lease. The discount rate used to calculate the net present value of future payments was the Company’s incremental borrowing rate at the Lease Commencement Date, which was 7.6%.

The following table contains a summary of the lease costs recognized under ASC 842 and other information pertaining to the Company’s operating leases for the three and nine months ended September 30, 2022 and 2021:

	Three Months Ended September 30,				Nine Months Ended September 30,
	2022		2021		2022		2021
Operating leases
Lease cost
Operating lease cost	$	378	$	378	$	1,134	$	1,134
Variable lease cost		231		208		669		639
Total lease cost	$	609	$	586	$	1,803	$	1,773
Other year-to-date lease information
Operating cash flows used    for operating leases					$	1,166	$	385
Operating lease liabilities    arising from obtaining    right-of-use assets					$	—	$	—

The following table contains a summary of the lease liabilities recognized on the Company’s unaudited condensed consolidated balance sheet as of September 30, 2022 and on the Company’s audited consolidated balance sheet as of December 31, 2021:

	As of September 30, 2022			As of December 31, 2021
Other operating lease information
Operating lease liabilities — short-term	$	1,335		$	1,227
Operating lease liabilities — long-term	$	2,711		$	3,725
Weighted-average remaining lease term	2.8 years			3.5 years
Weighted-average discount rate		7.60	%		7.60	%

The variable lease costs for the three and nine months ended September 30, 2022 and 2021 include common area maintenance and other operating charges. As the Company’s leases do not provide an implicit rate, the Company utilized its incremental borrowing rate based on what it would normally pay to borrow on a collateralized basis over a similar term for an amount equal to the lease payments at the commencement date in determining the present value of lease payments.

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Future minimum lease payments under the Company’s operating lease as of September 30, 2022 and December 31, 2021, were as follows:

	As of September 30, 2022			As of December 31, 2021
Maturity of lease liabilities
2022	$	396		$	1,562
2023		1,609			1,609
2024		1,656			1,656
2025		841			841
Thereafter		—			—
Total lease payments		4,502			5,668
Less: imputed interest		(456	)		(716	)
Total operating lease liabilities	$	4,046		$	4,952

Legal Proceedings

Since the announcement of the Merger Agreement, three complaints have been filed, captioned: Bushansky v. LogicBio Therapeutics, Inc. et al., No. 1:22-CV-09145 (S.D.N.Y.) (the “Bushansky Complaint”), Wilhelm v. LogicBio Therapeutics, Inc. et al., No. 1:22-CV-09186 (S.D.N.Y.) (the “Wilhelm Complaint”), and Carlisle v. LogicBio Therapeutics, Inc. et al., No. 1:22-CV-01418 (D. Del.) (the “Carlisle Complaint”) (collectively, the “Complaints”).  Each of the Complaints names the Company and its directors as defendants.  The Company has also received eight stockholder demand letters, including one draft complaint.  These are the “Vugman Demand Letter,” the “Way Demand Letter,” the “Wheeler Demand Letter,” the “Barbeau Demand Letter,” the “Redfield Demand Letter,” the “Ortega Demand Letter,” the “Browning Demand Letter,” and the “Smith Demand Letter” (collectively, the “Demand Letters”).  The Barbeau Demand Letter contained a draft complaint, captioned Barbeau v. LogicBio Therapeutics, Inc. et al.  Moreover, the Company received a stockholder demand letter seeking access to the Company’s books and records pursuant to Delaware Code Title 8, Section 220 (the “Section 220 Demand”).

The Complaints allege that the Company and its directors violated federal securities laws by failing to disclose material information in the Solicitation/Recommendation Statement on Schedule 14D-9 filed by the Company with the SEC on October 18, 2022 (together with the exhibits thereto, the “Schedule 14D-9”).  The Complaints generally seek, among other things, to enjoin the Company from proceeding with closing the Offer, the Merger and the other transactions contemplated by the Merger Agreement (the “Transactions”) unless and until it discloses and disseminates the alleged material information or, in the event the Transactions are consummated, a rescission of the Transactions and a recovery of the rescissory damages.  In addition, the Complaints seek an award of plaintiffs’ costs, including reasonable allowance for attorneys’ fees and costs.  The Carlisle Complaint also seeks a declaration that the defendants violated federal securities laws. The Demand Letters generally seek that certain allegedly omitted information in the Schedule 14D-9 be disclosed. The Section 220 Demand seeks access to the Company’s books and records relevant to the Transactions and the Company’s board members. The Section 220 Demand cites substantially similar allegations to those in the Complaints and Demand Letters as the basis for seeking to inspect the information requested in the Section 220 Demand.

The Company believes that the allegations in the Complaints, Demand Letters and Section 220 Demand are without merit and intends to defend against them vigorously.

At this time, no assessment can be made as to the likely outcome of the Complaints, Demand Letters or Section 220 Demand or whether the outcome will be material to the Company. The Company has not recorded an expense related to the outcome of the foregoing matters because it is not yet possible to determine if a potential loss is probable nor reasonably estimable.

12. RELATED PARTIES

The Company is party to a consulting service agreement with Mark Kay, who is a co-founder of the Company and a member of the Board. Under the terms of this agreement, the Company has agreed to pay an annual fee of $68 for research and development consulting services. For each of the three- and nine- months ended September 30, 2022 and 2021, the Company recorded research and development expense of $17 and $51, respectively, related to consulting services received from Mark Kay.

In addition, as a result of his participation on the Scientific Advisory Board in June 2021, Mark Kay earned $5 and received a non-qualified stock option to purchase 5,000 shares of the Company’s common stock with a fair value of $13, which are being expensed over a two-year vesting period. Expenses related to Mark Kay’s participation on the Scientific Advisory Board are recorded in research and development expense.

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13. SUBSEQUENT EVENTS

Proposed Acquisition by Alexion Pharmaceuticals, Inc.

The description of the Merger Agreement with Alexion in Note 1 in the accompanying notes to our unaudited consolidated financial statements is incorporated by reference hereto.

A&R CANbridge Agreement

On October 3, 2022, the Company and CANbridge entered into the Amended and Restated Exclusive Research Collaboration, License and Option Agreement (the “A&R CANbridge Agreement”), dated as of October 2, 2022, which amends the Original CANbridge Agreement.

Under the A&R CANbridge Agreement, the Candidate Options and the LB-001 Option were removed as mutually agreed by both parties. Further, in connection with the Fabry and Pompe License, the A&R CANbridge Agreement grants CANbridge a non-exclusive license to certain Company intellectual property rights related to manufacturing for the Fabry and Pompe programs. Additionally, under the A&R CANbridge Agreement, the CANbridge Research Plan was amended to reflect that the full transfer of the technology pertaining to the Fabry and Pompe programs to CANbridge was effective as of October 31, 2022. Under the A&R CANbridge Agreement, CANbridge does not have an obligation to pay or reimburse additional research expenses.

Under the A&R CANbridge Agreement, the Company will be eligible to receive clinical, regulatory and commercial milestone payments of up to $224.5 million in the aggregate (assuming one product for each indication). In accordance with the removal of the two additional Candidate Options and LB-001 Option, milestones related thereto were removed in the A&R CANbridge Agreement. Under the A&R CANbridge Agreement, the tiered royalty rates payable to the Company will range from low single digit to mid-single digit rates. Under the A&R CANbridge Agreement, LogicBio and CANbridge will negotiate a product reversion agreement in good faith in the event of a termination of the A&R CANbridge Agreement under certain circumstances.

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

The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our unaudited condensed consolidated financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q. Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report on Form 10-Q, including information with respect to our plans and strategy for our business, includes forward-looking statements that involve risks and uncertainties. As a result of many factors, including those factors set forth in the “Risk Factors” section of this Quarterly Report on Form 10-Q, our actual results could differ materially from the results described in, or implied by, these forward-looking statements. Please also refer to the section under the heading “Special Note Regarding Forward-Looking Statements and Industry Data.”

Proposed Acquisition by Alexion Pharmaceuticals, Inc.

On October 3, 2022, the Company entered into an Agreement and Plan of Merger, or as it may be amended from time to time, the Merger Agreement, by and among the Company, Alexion Pharmaceuticals, Inc., a Delaware corporation, or Alexion or Parent, and Camelot Merger Sub, Inc., a Delaware corporation and a wholly-owned subsidiary of Parent, or Merger Sub. Pursuant to the Merger Agreement, on October 18, 2022, Merger Sub commenced a tender offer to purchase, subject to the satisfaction or waiver of certain conditions set forth in the Merger Agreement, any and all of the issued and outstanding shares of common stock, par value $0.0001 per share, of the Company, or the Shares, at a price of $2.07 per Share, to the seller in cash, without interest, less any applicable withholding taxes, upon the terms and subject to the conditions set forth in the Offer to Purchase, dated October 18, 2022, or the Offer to Purchase. The Offer to Purchase, together with the Letter of Transmittal and other related materials, as may be amended, constitute the Offer.

On the same date as the consummation of the Offer and subject to the satisfaction or waiver of certain conditions set forth in the Merger Agreement, Merger Sub will be merged with and into the Company, or the Merger, without a vote of the Company’s stockholders to adopt the Merger Agreement and consummate the Merger, in accordance with Section 251(h) of the of the Delaware General Corporation Law, or the DGCL, with the Company continuing as the surviving corporation and as a wholly-owned subsidiary of Parent. The Offer is subject to certain important conditions; see “Item 1A. Risk Factors” for more information.

Overview

We are a clinical-stage genetic medicine company pioneering genome editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood. Our genome editing platform, GeneRide^®, is a new approach to precise gene insertion harnessing a cell's natural deoxyribonucleic acid, or DNA, repair process potentially leading to durable therapeutic protein expression levels. Our gene delivery platform, sAAVy™, is an AAV capsid engineering platform designed to optimize gene delivery for treatments in a broad range of indications and tissues. Our proprietary manufacturing process, mAAVRx^TM, is a novel process aimed at improving production yields for viral vector manufacturing.

Our lead product candidate, LB-001, is a single-administration, genome editing therapy developed using our GeneRide technology, currently in Phase 1/2 development for the treatment of MMA in pediatric patients. MMA is a rare and life-threatening genetic disorder affecting approximately 1 in 50,000 newborns in the United States that often results in developmental delays and other long-term complications and a high rate of hospitalizations.

In December 2021, we announced the nomination of a new product candidate, LB-401, based on our GeneRide technology. LB-401 is a genome editing therapy being developed for the treatment of hereditary tyrosinemia type 1, or HT1. HT1 is a rare, genetic disorder characterized by elevated blood levels of the amino acid tyrosine, a building block of most proteins, and affects more than 1,200 patients in North America and Europe alone.

Also based on our GeneRide technology, we completed the first phase of preclinical development of our product candidate, LB-301, for the treatment of Crigler-Najjar syndrome, or CN, a rare pediatric disease affecting approximately 1 in 1,000,000 newborns globally, in collaboration with Takeda Pharmaceutical Company Limited, or Takeda. In addition, we are developing treatments based on our GeneRide technology for two indications in collaboration with Daiichi Sankyo Company, Limited, or Daiichi. We have also demonstrated proof of concept of our GeneRide platform in hemophilia B and alpha-1-antitrypsin deficiency, or A1ATD, and Wilson disease animal disease models.

Based on our sAAVy technology, we are developing gene therapy candidates utilizing, among other things, sL65, the first capsid produced from sAAVy, for the treatment of Fabry and Pompe diseases in collaboration with CANbridge. We also granted CANbridge an option to obtain an exclusive worldwide license to certain of our intellectual property rights, including those relating to sL65, to develop and commercialize gene therapy candidates for the treatment of two additional indications.

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LB-001 for the Treatment of Methylmalonic Acidemia (MMA) in Pediatric Patients

We are evaluating the safety, tolerability and preliminary efficacy of LB-001 in our Phase 1/2 SUNRISE clinical trial in pediatric patients with MMA. The SUNRISE trial is designed to enroll patients with ages ranging from six months to twelve years and evaluate a single administration of LB-001 at two dose levels (5e¹³ vg/kg and 1e¹⁴ vg/kg), with dose escalation subject to certain conditions. The primary endpoint of the SUNRISE trial is to assess the safety and tolerability of LB-001 at 52 weeks after a single infusion. Additional endpoints, which are secondary and exploratory, include changes in disease-related biomarkers, including serum methylmalonic acid, clinical outcomes such as growth and healthcare utilization, and the pharmacodynamic marker albumin-2A. 

In June 2021, we announced that the first patient was dosed in the SUNRISE trial. In accordance with the FDA-reviewed protocol, we initially enrolled patients in the three- to twelve-year-old age group at the lower dose. In October 2021, we announced early results from the SUNRISE trial, which showed measurable levels of albumin-2A, or ALB-2A, a technology-related biomarker indicating site-specific gene insertion and protein expression. Detection of albumin-2A is an indication that we have achieved the first ever in vivo genome editing in children. Following an evaluation of the safety data from the first two patients enrolled in the SUNRISE trial, we also announced in October 2021, that the independent Data Safety Monitoring Board, or the DSMB, overseeing the SUNRISE trial recommended the continuation of the trial without modification. Albumin-2A detection together with the DSMB continuation recommendation enabled us to begin enrolling two patients in the higher dose cohort (with ages ranging three to twelve years old), and two patients with ages ranging from six months to two years old at the lower dose.

More recently, in mid-August 2022, we announced additional early results from the SUNRISE trial from the first four pediatric patients treated with LB-001. These results showed that albumin-2A, a technology-related biomarker, had been detected in the serum of all four patients, which indicated site-specific integration of the MMUT gene. Additionally, in two of the four patients, we saw increasing levels of ALB-2A over time, which indicated selective advantage.

The disease-related biomarkers, including methylmalonic acid, methylcitric acid, propionate oxidation and FGF-21, were variable, and had not shown a clear trend to date.

As previously disclosed, the third and fourth patients dosed in the SUNRISE trial, each of whom received 5e¹³ vg/kg of LB-001, experienced a drug-related serious adverse event, or SAE, which was categorized as a case of thrombotic microangiopathy, or TMA. Additionally, prior to the TMA event, the fourth patient experienced a grade 1 drug-related SAE that was categorized as cytokine release syndrome and necessitated an additional day in the hospital post-dosing. Each TMA resolved within a few weeks, and these patients continue to be followed in accordance with the SUNRISE protocol. Following the occurrence of the TMA experienced by the third patient, we implemented a DSMB-endorsed response plan that included increased patient monitoring.

Following the occurrence of the TMA experienced by the fourth patient, we announced on February 2, 2022 that the FDA placed our Investigational New Drug Application, or IND, for LB-001 on clinical hold. We subsequently announced on May 9, 2022 that the hold was lifted. In its communication lifting the hold, the FDA indicated that all issues related to the clinical hold had been addressed. In connection with the lifting of the clinical hold, we amended the SUNRISE protocol. The amended protocol includes enhanced monitoring measures such as frequent testing for complement activation, a characteristic of TMA, as well as use of a complement inhibitor in the event there are laboratory findings indicating a potential or imminent TMA.

In addition to the Phase 1/2 SUNRISE trial, we have also completed a retrospective natural history study designed to evaluate disease progression in pediatric patients with MMA. These data helped to provide us with a better understanding of the natural progression of the disease, the impact of a liver transplant on the outcomes of MMA patients and potential endpoints such as the relevance of methylmalonic acid levels on clinical outcomes, with the goal of informing our future clinical development in MMA and our discussions with regulatory agencies as we look toward advancing our MMA program. We presented preliminary findings from our retrospective natural history study at the American College of Medical Genetics in April 2021.

In July 2019, the FDA granted rare pediatric disease designation for LB-001 for the treatment of MMA, and in April 2019, the FDA granted orphan drug designation for LB-001 for the treatment of MMA. In November 2020, the FDA granted fast track designation for LB-001 for the treatment of MMA, and in June 2021, the European Commission granted orphan drug designation to LB-001 for the treatment of MMA.

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LB-401 for the Treatment of Hereditary Tyrosinemia Type 1

In December 2021, we announced the nomination of a new development candidate, LB-401, for the treatment of HT1. This development candidate is based on our GeneRide platform. HT1 is a rare, genetic disorder characterized by elevated blood levels of the amino acid tyrosine that affects more than 1,200 patients in North America and Europe alone. This condition is caused by a shortage of the enzyme fumarylacetoacetate hydrolase, or FAH, one of the enzymes required for the multi-step process that breaks down tyrosine.

In preclinical studies presented at the European Society of Gene and Cell Therapy, or ESGCT, Annual Meeting in October 2021, the data in HT1 models with acute liver damage showed that GeneRide-edited hepatocytes repopulated the entire liver within four weeks post-administration, replacing the diseased hepatocytes with corrected hepatocytes. HT1 mice that received the GeneRide-FAH vector were no longer reliant on the current standard of care for the disease, and demonstrated restored normal body growth, liver function, and undetectable succinyl acetone levels. In preclinical studies to be presented at the ASGCT Annual Meeting in May 2022, additional data in HT1 models with acute liver damage highlighted the potential durability of LB-401 for at least 10 months. Additionally, compared to the standard of care, these data demonstrated that HT1 mice treated with LB-401 showed succinylacetone reduction, better tyrosine management, and a rapid decrease of alfa-fetoprotein, suggesting a lower risk of hepatocellular carcinoma.

Other GeneRide Development Programs

Also based on our GeneRide technology, we completed the first phase of preclinical development of our product candidate, LB-301, for the treatment of CN, a rare pediatric disease affecting approximately 1 in 1,000,000 newborns globally, in collaboration with Takeda. In initial proof-of-concept studies, a murine GeneRide construct of LB-301 was used to correct the gene deficiency in an animal model of CN. The introduction of UGT1A1 into the albumin locus in mouse liver cells resulted in normalization of bilirubin levels and long-term survival of mice deficient in UGT1A1 from less than twenty days to at least one year. Our Takeda collaboration to further develop LB-301 in CN, resulted in the extension of our proof-of-concept data and was reported at American Society of Gene & Cell Therapy, or ASGCT, in May 2020. In these reported studies, neonatal mice treated with a murine GeneRide construct of LB-301 resulted in a survival benefit and a dose-dependent reduction in total circulating bilirubin. The bilirubin levels achieved were within the range of that observed in patients with Gilbert’s syndrome (1-6mg/dL), a mild liver disorder usually requiring no medical treatment. Moreover, through a collaboration with Dr. Andrés Muro at the International Centre for Genetic Engineering and Biotechnology in Trieste, Italy, we demonstrated that GeneRide treatment could improve motor discoordination in preclinical mouse models of CN.

In addition, we are developing treatments based on our GeneRide technology for two indications in collaboration with Daiichi. We have also demonstrated proof of concept of our GeneRide platform in mouse models of hemophilia B and alpha-1-antitrypsin deficiency, or A1ATD, and Wilson disease.

Our sAAVy Platform

We are also developing sAAVy, a next generation AAV capsid platform, for use in gene editing and gene therapy. At the ASGCT Annual Meeting in May 2020, we presented data showing that the capsids delivered highly efficient functional transduction of human hepatocytes in a humanized mouse model. Based on these data, we believe the top-tier capsid candidates from this effort demonstrated the potential to achieve significant improvements over benchmark AAVs that are currently in clinical development. We are developing these highly potent vectors for use in our internal development candidates and collaborations. We announced data generated from translational animal models using these capsids at the ASGCT 2021 Annual Meeting in May 2021. In addition, in January 2021, we announced the extension of our collaboration with Children’s Medical Research Institute, or CMRI, to continue to develop next-generation capsids for gene therapy and gene editing applications in the liver as well as additional tissues.

Manufacturing  

Our genome editing and gene therapy product candidates are manufactured as viral vectors. Viral vector manufacturing is a complex process due to several factors, including the following:

• the use of complex biological raw materials, including plasmid DNA, and cell banks, that need to comply with stringent regulatory requirements;

• the use of complex manufacturing methods, such as transfection of suspension mammalian cell culture in large bioreactors (transfection is the process by which plasmid DNA enters the cells), and purification of intact viral particles;

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• the need to produce high-concentration, high purity drug product; and

• the challenges associated with scale-up, batch-to-batch consistency and product characterization, including measurement of potency and quality.

We have built extensive internal non-GMP process development, analytical development and vector development capabilities that are designed to optimize certain components of viral vector manufacturing, including the following:

• Improved production yields. One challenge facing gene editing and gene therapy product candidates manufactured as viral vectors is production yield. Improvements in production yields have the potential to make drug product available to more patients in need by decreasing the drug product cost of goods. Initial results from our proprietary manufacturing process, mAAVRx^TM, have demonstrated significantly improved production yields in comparison to published results.

• Increased drug product purity. Another challenge facing gene editing and gene therapy product candidates manufactured as viral vectors is that, over the last several years, the occurrence of undesirable side effects in clinical trials have led the industry stakeholders and regulatory authorities to look for higher levels of purity of drug product for use in humans. The proprietary methods developed by our internal team are designed to increase the purity of drug product.

• Decreasing “empty” capsids. A challenge facing AAV vector manufacturing in particular is the formation of an excess of what are known as “empty” capsids, which are unable to provide a therapeutic benefit and may contribute to exacerbating adverse effects. Our internal team has developed proprietary and scalable methods designed to decrease the proportion of empty capsids in our drug product.  

• Reliable characterization methods.  We have invested resources in developing a large panel of methods to enable accurate and consistent characterization of our clinical supply of our lead product, LB-001, that we can leverage for use in our other development programs if and as we optimize the drug product from those programs for use in humans.  

Operating Overview

Since our inception in 2014, we have devoted the majority of our efforts to research and development, including our preclinical and clinical development activities and our manufacturing and process development activities, raising capital, and providing general and administrative support for these operations. We do not have any products approved for sale and our only revenue recognized to date has been revenue related to upfront payments and research cost reimbursement under our strategic agreements with CANbridge, Daiichi and Takeda. Through September 30, 2022, we have raised approximately $126.0 million in equity capital through underwritten public offerings, at-the-market sales of our common stock and $33.1 million in net proceeds from the sale of preferred stock prior to our initial public offering. In July 2019, we entered into the Loan Agreement, under which term loans in an aggregate principal amount of $20.0 million were made available to us in two tranches, subject to certain terms and conditions. As of September 30, 2022, we had drawn down the $10.0 million first tranche. In 2021, we met the conditions to initiate drawdown of the second $10.0 million tranche but did not exercise our right to do so and the option to draw down the second tranche of the Term Loans expired. The Company expects all outstanding principal, interest, and fees owned under the Loan Agreement to be repaid in full if the Merger is consummated. The Company expects that prepayment in connection with the Merger Agreement will result in a 1% prepayment fee.

We have incurred significant operating losses since our inception. Our ability to generate product revenue sufficient to achieve profitability will depend on the successful development and commercialization of our current product candidates and any future product candidates. Our net loss was $17.5 million for the nine months ended September 30, 2022. As of September 30, 2022, we had an accumulated deficit of $157.5 million. We expect to continue to incur significant expenses and increasing operating losses for the foreseeable future in connection with our ongoing activities. While we intend to continue to evaluate ways to enhance our liquidity and capital position, our efforts will largely depend on future developments that are highly uncertain and cannot be predicted with confidence at this time.

Impact of COVID-19

The COVID-19 pandemic continues to present a substantial public health and economic challenge around the world. We continue to closely monitor the COVID-19 pandemic in order to promote the safety of our personnel and to continue advancing our research and development activities. We are following federal, state and local requirements and guidelines with respect to the COVID-19 pandemic and have allowed our employees to work on-premises in accordance with those requirements and guidelines.

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The COVID-19 pandemic did not have a material impact on our results of operations, cash flow and financial position as of and for the quarter ended September 30, 2022. However, we are aware that certain of our third-party vendors are being affected by import/export and other restrictions due to the COVID-19 pandemic, which may have an impact on certain of our research, development and manufacturing activities. Further, the pandemic could also potentially affect the business of the FDA, the EMA or other governmental authorities, which could result in delays in meetings, reviews, inspections and approvals, including those relating to LB-001. Any decision by the FDA, EMA or other governmental authorities to delay meeting with us or scheduling inspections in light of COVID-19 could have a material adverse effect on our clinical trials, which could increase our operating expenses and have a material adverse effect on our financial results, including the timing and amount of future regulatory milestones we could receive from our partners.

We cannot predict the impact of the progression of the COVID-19 pandemic on future results due to a variety of factors, including the health of our and our third-party vendors, suppliers and collaborators’ employees, our ability to maintain operations, the ability of our third-party vendors, suppliers and collaborators to continue operations, any further government and/or public actions taken in response to the pandemic and ultimately the length of the pandemic. We plan to continue to closely monitor the COVID-19 pandemic in order to ensure the safety of our personnel and to continue advancing our research and development activities.

Components of Results of Operations

Revenue

Our only revenue recognized to date has been collaboration and service revenue related to upfront payments and research cost reimbursement under our agreements with CANbridge, Daiichi and Takeda. We have not generated any revenue from product sales and do not expect to generate any revenue from product sales for the foreseeable future.

Operating Expenses

Research and Development Expenses

Research and development expenses consist primarily of costs incurred for our research activities, including our discovery efforts and the development of our product candidates and technologies, and include:

• salaries, benefits and other related costs, including stock-based compensation expense, for personnel engaged in research and development functions;

• license maintenance fees and milestone fees incurred in connection with various license agreements;

• the cost of laboratory supplies and acquiring, developing and manufacturing preclinical study and clinical trial materials;

• expenses incurred under agreements with contract research organizations, or CROs, contract manufacturing organizations, or CMOs, as well as academic institutions and consultants that conduct our preclinical studies and other scientific development services;

• facility-related expenses, which include direct depreciation costs and allocated expenses for rent and maintenance of facilities and other operating costs;

• costs of outside consultants, including their fees, stock-based compensation and related travel expenses; and

• costs related to compliance with regulatory requirements.

We expense research and development costs as incurred. Costs for external development activities are recognized based on an evaluation of the progress to completion of specific tasks. Payments for these activities are based on the terms of the individual agreements, which may differ from the pattern of costs incurred, and are reflected in our financial statements as prepaid or accrued research and development expenses.

Research and development activities are central to our business model. We will continue to incur significant expenses to conduct the clinical program for our product candidate, LB-001, and as we continue to discover and develop additional product candidates and technologies. If any of our product candidates enters into later stages of clinical development, we expect that they will generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials.

General and Administrative Expenses

General and administrative expenses consist primarily of salaries, benefits and other related costs, including stock-based compensation, for personnel in our executive, finance, legal, human resources, corporate and business development and administrative

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functions. General and administrative expenses also include professional fees for legal, patent, accounting, auditing, tax and consulting services, travel expenses, and facility-related expenses, which include direct depreciation costs and allocated expenses for rent and maintenance of facilities and other operating costs.

Other Income (Expense), Net

Interest income consists primarily of interest on our cash and cash equivalents and investments. Interest expense consists of interest expense related to the term loan under the Loan Agreement in July 2019. A portion of the interest expense on the term loan is non-cash expense relating to the accretion of the debt discount, amortization of issuance costs and accretion of the final payment fee. During the three and nine months ended September 30, 2022, we recorded $0.2 million and $0.6 million, respectively, in interest expense, of which $0.1 million and $0.5 million, respectively, related to cash interest paid and the remainder to the accretion of the debt discount and amortization of issuance costs. During the three and nine months ended September 30, 2021, we recorded $0.3 million and $0.8 million, respectively, in interest expense, of which $0.2 million and $0.7 million, respectively, related to cash interest paid and the remainder to the accretion of the debt discount and amortization of issuance costs.

Results of Operations

Comparison of the Three Months Ended September 30, 2022 and 2021

The following table summarizes our results of operations for the three months ended September 30, 2022 and 2021:

	Three Months Ended September 30,
	2022			2021
	(in thousands)
REVENUE
Collaboration and service revenue	$	2,717		$	2,120
Total revenue		2,717			2,120
OPERATING EXPENSES
Research and development		5,051			7,806
General and administrative		3,410			4,257
Total operating expenses		8,461			12,063
LOSS FROM OPERATIONS		(5,744	)		(9,943	)
OTHER (EXPENSE) INCOME:
Other expense, net		(31	)		(270	)
Loss before income taxes		(5,775	)		(10,213	)
Income tax benefit		—			28
Net loss	$	(5,775	)	$	(10,185	)

Revenue

Revenue for the three months ended September 30, 2022 consisted of $2.7 million in collaboration and service revenue largely driven by our Original CANbridge Agreement, and to a lesser extent, our agreement with Daiichi. Revenue for the three months ended September 30, 2021 consisted of $2.1 million in collaboration and service revenue related to our agreements with CANbridge, Daiichi, and Takeda. The year-over-year increase was primarily the result of higher collaboration related revenues from the Original CANbridge Agreement, $2.3 million versus $1.7 million, respectively; revenues related to the Research Collaboration and Option Agreement with Takeda did not re-occur in the quarter ended September 30, 2022, as such agreement expired by its own terms in the year ended December 31, 2021. Due to the amended and restated collaboration agreement with CANbridge executed in October 2022, we expect our collaboration and service revenue to increase for the fourth quarter of 2022 as the work under the collaboration is expected to end and the associated deferred revenue will be recognized.

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Research and Development Expenses

The following table summarizes our research and development expenses for the three months ended September 30, 2022 and 2021:

	Three Months Ended September 30,
	2022		2021		(Decrease)/Increase
	(in thousands)
LB-001 external development and manufacturing costs	$	513	$	2,207	$	(1,694	)
Personnel-related costs		2,812		2,778		34
Lab supplies		514		1,068		(554	)
Other research and development costs		1,212		1,753		(541	)
Total research and development expenses	$	5,051	$	7,806	$	(2,755	)

Research and development expenses for the three months ended September 30, 2022 were $5.1 million, compared to $7.8 million for the three months ended September 30, 2021. The year-over-year decrease of approximately $2.8 million was primarily due to a decrease of $1.7 million related to the LB-001 external development and manufacturing costs, as there was no enrollment in the Phase 1/2 SUNRISE clinical trial during the third quarter of 2022, costs to manufacture supply had primarily occurred in 2021 and work under the prospective natural history study had completed in 2021. In addition, there was a decrease of $0.6 million in lab supplies due to a decrease in headcount and a decrease of $0.5 million in other research and development costs primarily related to completing work under the collaboration with CMRI. We expect that our research and development expenses will decrease during the fourth quarter of 2022 compared to the third quarter of 2022 as our work with CANbridge and CMRI has been completed.

General and Administrative Expenses

General and administrative expenses were $3.4 million for the three months ended September 30, 2022, compared to $4.3 million for the three months ended September 30, 2021. The decrease of approximately $0.9 million was primarily driven by a decrease of approximately $0.3 million in professional service fees as we brought more professional work in-house and a decrease of $0.3 million in personnel expenses as headcount decreased during the quarter. The decrease was partially offset by transaction costs in connection with the proposed acquisition by Alexion, as well as the A&R CANbridge Agreement. We expect that general and administrative expenses will increase substantially during the fourth quarter of 2022 primarily as a result of transaction costs.

Other Expense, Net

Other expense, net was $31,000 for the three months ended September 30, 2022, compared to $0.3 million for the three months ended September 30, 2021. Other expense, net decreased due to a lower principal amount due on the term loan balance during the three months ended September 30, 2022 as well as higher interest rates on our cash and cash equivalents balance. We expect our other expense, net to decrease during the fourth quarter of 2022 as the principal balance on our term loan decreases.

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Comparison of the nine months ended September 30, 2022 and 2021

The following table summarizes our results of operations for the nine months ended September 30, 2022 and 2021

	Nine Months Ended September 30,
	2022			2021
	(in thousands)
REVENUE
Collaboration and service revenue	$	8,732			3,383
Total revenue		8,732			3,383
OPERATING EXPENSES
Research and development		15,524			21,482
General and administrative		10,293			12,081
Total operating expenses		25,817			33,563
LOSS FROM OPERATIONS		(17,085	)		(30,180	)
OTHER (EXPENSE) INCOME:
Other expense, net		(396	)		(814	)
Loss before income taxes		(17,481	)		(30,994	)
Income tax benefit		—			28
Net loss	$	(17,481	)	$	(30,966	)

Revenue

Our revenue for the nine months ended September 30, 2022 consisted of $8.7 million in revenue related to the Daiichi and CANbridge agreements. Our revenue for the nine months ended September 30, 2021consisted of $3.4 million in revenue related to the Takeda, Daiichi and CANbridge agreements. The increase in revenue for the nine months ended September 30, 2022 compared to the corresponding period in 2021 was related to an increase in revenue recognized under the April 2021 CANbridge and Daiichi agreements and partially offset by winding down activities under the January 2020 Takeda agreement.

Research and Development Expenses

The following table summarizes our research and development expenses for the nine months ended September 30, 2022 and 2021

	Nine Months Ended September 30,
	2022		2021		(Decrease)/Increase
	(in thousands)
LB-001 external development and manufacturing costs	$	2,128	$	5,905		(3,777	)
Personnel-related costs		7,247		7,270		(23	)
Lab supplies		1,844		2,662		(818	)
Other research and development costs		4,305		5,645		(1,340	)
Total research and development expenses	$	15,524	$	21,482	$	(5,958	)

Research and development expenses for the nine months ended September 30, 2022 were $15.5 million, compared to $21.5 million for the nine months ended September 30, 2021. The decrease of approximately $6.0 million was primarily due to a decrease of $3.8 million in LB-001 external development and manufacturing costs related to clinical trial start-up expenses, clinical supply manufacturing expenses and supporting study expenses that occurred during the nine months ended September 30, 2021, which did not re-occur during the nine months ended September 30, 2022. In addition, there was a decrease of $1.3 million in other research and development costs primarily related to one-time intellectual property costs that occurred as a result of entering the April 2021 collaboration agreement with CANbridge during the nine months ended September 30, 2021, which did not re-occur during the nine months ended September 30, 2022. Finally, there was a $0.8 million decrease in lab supplies mainly related to the decrease in headcount during the nine months ended September 30, 2022 as compared to the same period in 2021.

General and Administrative Expenses

General and administrative expenses were $10.3 million for the nine months ended September 30, 2022, compared to $12.1 million for the nine months ended September 30, 2021. The decrease of approximately $1.8 million was primarily driven by a decrease of $1.4 million in professional service fees as we brought more professional work in-house through key hires made during 2021. As

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such, personnel expenses increased approximately $0.1 million as we filled key open positions within the human resources, legal and business development functions. Finally, there was a decrease of $0.5 million in insurance and other general and administrative expenses.

Other Expense, Net

Other expense, net was $0.4 million for the nine months ended September 30, 2022 compared to other expense, net of $0.8 million for the nine months ended September 30, 2021. Other expense, net decreased primarily due to a lower principal amount due on the term loan balance during the nine months ended September 30, 2022 as well higher interest income on our cash and cash equivalents balance due to higher interest rates.

Liquidity and Capital Resources

Overview

Since our inception and through September 30, 2022, we have not generated any sales revenue and have incurred significant losses and negative cash flows from our operations.

As of September 30, 2022, we had cash and cash equivalents of $30.8 million, which we believe will be sufficient to fund our operating expenses and capital expenditure requirements into the second quarter of 2023. As such, and due to the uncertainties described below, there is substantial doubt about the Company’s ability to continue as a going concern. On a quarterly basis, we are required to conduct an accounting analysis under ASC 205-40, Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern, or ASC 205-40. The result of our ASC 205-40 analysis is that there is substantial doubt about our ability to continue as a going concern through the next twelve months from the date of issuance of these unaudited condensed consolidated financial statements.  We will require substantial additional capital to fund our research and development, including our preclinical and clinical development activities and our manufacturing and process development activities, and ongoing operating expenses. In connection with the proposed acquisition by Alexion, following a consummation of the Offer and subject to the satisfaction or waiver of certain conditions set forth in the Merger Agreement, the Company would continue as a wholly-owned subsidiary of Alexion. Without taking into account the proposed Merger with Alexion, management’s plans to mitigate the conditions that raise substantial doubt include raising additional capital through equity or debt financings, payments from our collaborators, strategic transactions, or a combination of those approaches. These plans may also include the possible elimination or deferral of certain operating expenses unless and until additional capital is received. There can be no assurance that we will be successful in raising additional capital or that such capital, if available, will be on terms that are acceptable to us, or that we will be successful in deferring certain operating expenses. 

Additionally, we are subject to a variety of specified restrictions under the Merger Agreement. Unless we obtain Alexion’s prior written consent, we may not, among other things, subject to certain exceptions, issue, split or repurchase securities; sell, lease, license, or dispose of any material portion of our assets, business or properties; pay dividends; or incur indebtedness.

Cash Flows

The following table summarizes our cash flows for the nine months ended September 30, 2022 and 2021:

	Nine Months Ended September 30,
	2022			2021
	(in thousands)
Net cash used in operating activities	$	(20,100	)	$	(13,834	)
Net cash used in investing activities		(100	)		(734	)
Net cash (used in) provided by financing activities		(2,500	)		4,074
Net decrease in cash, cash equivalents and    restricted cash	$	(22,700	)	$	(10,494	)

Operating Activities

During the nine months ended September 30, 2022, net cash used in operating activities was $20.1 million, primarily related to our net loss adjusted for non-cash charges and changes in the components of working capital. The $6.3 million increase in net cash used in operating activities during the nine months ended September 30, 2022, as compared to the nine months ended September 30, 2021, was primarily driven by a $15.6 million decrease in deferred revenue, as revenue recognized during the nine months ended September 30, 2022 was previously paid by our collaboration partners as well as a $3.5 million decrease in working capital based on the timing of

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payments. These decreases in cash were partially offset by a $13.5 million decrease in net loss during the nine months ended September 30, 2022 as compared to the same period in 2021.

Investing Activities

During the nine months ended September 30, 2022, net cash used in investing activities was $0.1 million related to the purchases of property and equipment. During the nine months ended September 30, 2021, net cash used in investing activities was $0.7 million related to the purchases of property and equipment.  

Financing Activities

During the nine months ended September 30, 2022, net cash used in financing activities was $2.5 million, which was related to the repayment of principal on our term loans. During the nine months ended September 30, 2021 net cash provided by financing activities was $4.1 million primarily related to net proceeds from sales of our common stock under an Open Market Sale Agreement with Jefferies LLC as the sales agent, or the Open Market Sale Agreement, of $5.1 million, partially offset by the repayment of principal on our term loans of $1.1 million.

Funding Requirements

We expect to continue to incur a significant amount of expenses in connection with our ongoing activities for the foreseeable future. In particular, we will incur significant expenses related to the preclinical activities and clinical trials of our product candidates and any future product candidates.

We expect that we will continue to incur significant expenses if and as we:

• continue our current research programs and our preclinical development of any product candidates from our current research programs;

• take actions necessary to consummate the Merger;

• continue to conduct our clinical program for LB-001, including our Phase 1/2 SUNRISE clinical trial. In connection with the lifting of the LB-001 clinical hold described above, we amended the SUNRISE protocol. Implementation of the protocol amendments is expected to increase clinical trial costs primarily due to increased screening time, an intensified monitoring regimen, prolonged hospitalization and potential acute use of a complement inhibitor;

• seek to identify, assess, acquire and/or develop additional research programs and additional product candidates, and initiate and conduct clinical trials for such product candidates;

• engage in transactions, including strategic, merger, collaboration, acquisition and licensing transactions;

• seek regulatory approvals for any product candidates that successfully complete clinical trials; develop, optimize, scale and validate a manufacturing process and analytical methods for any product candidates we may develop;

• obtain market acceptance of any product candidates we may develop as viable treatment options;

• address competing technological and market developments;

• maintain, expand and protect our intellectual property portfolio and provide reimbursement of third-party expenses related to our patent portfolio;

• further develop our GeneRide and sAAVy technology platforms, as well as our proprietary manufacturing process, mAAVRx;

• hire additional scientific, clinical, technical, quality, regulatory, general and administrative, and commercial personnel and add operational, financial and management information systems and personnel, including personnel to support our process and analytical development, manufacturing and planned future commercialization efforts;

• make royalty, milestone or other payments under current or future in-license agreements;

• establish and maintain supply chain and manufacturing relationships with third parties that can provide adequate products and services, in both amount, timing and quality, to support our development programs and the market demand for any product candidate for which we obtain regulatory and marketing approval;

• lease and build new facilities, including offices and labs, as necessary to support any organizational growth;

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• comply with good practice quality guidelines and regulations, or GXP, including good laboratory practice, good clinical practice, or GCP, or current good manufacturing practice, and cGMP;

• build out and validate clinical and commercial-scale cGMP manufacturing capabilities;

• establish a sales, marketing and distribution infrastructure to commercialize any product candidates for which we may obtain marketing approval; and

• experience any delays or encounter issues with any of the above.

We are unable to estimate the timing and amounts of increased capital outlays and operating expenses associated with completing the research and development of our product candidates because of the numerous risks and uncertainties associated with the development of our lead product candidate, LB-001, and any other product candidates and programs we may develop and because the extent to which we may enter into collaborations with third parties for the development of LB-001 and any other product candidates we may develop is unknown.

At September 30, 2022, we had $30.8 million of cash and cash equivalents on hand, which we currently expect will be sufficient to fund our operating expenses and capital expenditure requirements into the second quarter of 2023. We have based these estimates on assumptions that may prove to be wrong, and we could use our available capital resources sooner than we currently expect. Our future funding requirements, both near and long-term, will depend on many factors, including:

• the scope, progress, timing, costs and results of drug discovery, preclinical development, laboratory testing, and clinical trials for our product candidates, including the ongoing development program for LB-001 (including the Phase 1/2 SUNRISE clinical trial), and process development and manufacturing activities for LB-001;

• the implementation of amendments to the Phase 1/2 SUNRISE clinical trial protocol made in connection with the lifting of the clinical hold, which include increased screening time, an intensified monitoring regimen, prolonged hospitalization and potential acute use of a complement inhibitor;

• the outcome, timing and cost of following the advice of and complying with regulatory requirements and decisions made by the FDA, EMA and other regulatory authorities, including relating to any potential clinical holds that may be imposed on us in the future;

• the impact of the COVID-19 pandemic on our ability to progress with our research, development, manufacturing and regulatory efforts, including our ability to advance and complete our Phase 1/2 SUNRISE clinical trial of LB-001;

• the cost of filing, prosecuting, defending and enforcing our patent claims and other intellectual property rights;

• the cost of defending potential intellectual property disputes, including patent infringement actions;

• the achievement of milestones or occurrence of other developments that trigger payments under any of our current agreements or other agreements we may enter into;

• the extent to which we are obligated to reimburse, or entitled to reimbursement of, clinical trial and other research and development costs under future collaboration agreements, if any;

• the effect of competing technological and market developments;

• the cost and timing of completion of process and analytical development and manufacturing activities;

• the extent to which we engage in transactions, including collaboration, merger, acquisition and licensing transactions;

• our ability to establish and maintain collaborations on favorable terms, if at all;

• the cost of establishing sales, marketing and distribution capabilities for LB-001 and any other product candidates in regions where we choose to commercialize our product candidates, if approved;  

• the initiation, progress, timing and results of our commercialization of LB-001 and any other product candidates, if approved, for commercial sale;

• our ability to repay outstanding debt; and

• our ability to attract, hire and retain qualified personnel.

A change in the outcome of any of the above variables that are applicable to the development of a product candidate could mean a significant change in the costs and timing associated with the research and development of that product candidate. For example, if the

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FDA or another regulatory authority were to require us to conduct clinical trials beyond those that we anticipate will be required for the completion of clinical development of a product candidate, or if we experience significant trial delays due to actions taken by regulatory authorities, institutional review boards, or IRBs, patient enrollment or other reasons, we would be required to expend significant additional financial resources and/or time on the completion of clinical development. Any significant delays in our programs may also require us to reevaluate our corporate strategy, resulting in the expenditure of significant resources and time. We may never succeed in obtaining regulatory approval for our product candidates or any future product candidates.

Until such time, if ever, that we can generate product revenue sufficient to achieve profitability, we expect to finance our future cash needs through equity or debt financings, payments from our collaborators, strategic transactions, or a combination of those approaches. There is no assurance that we will be successful in obtaining any additional financing on terms acceptable to the Company, if at all. Additionally, the terms of any financing may adversely affect the holdings or the rights of our stockholders. If we are unable to obtain funding, we may be required to delay, reduce or eliminate some or all of our research and development programs, preclinical and clinical development programs or future commercialization efforts, or we may be unable to continue operations.

At-the-Market Sales of Common Stock

In November 2019, we entered into the Open Market Sale Agreement. Under the terms of the Open Market Sale Agreement and the related prospectus supplement we filed with the Securities and Exchange Commission, or the SEC, in November 2019, we may sell shares of our common stock at the then current market prices, from time to time, having an aggregate value of up to $50.0 million through Jefferies LLC. We pay up to a 3% cash commission to Jefferies LLC on the proceeds from sales under the program. During the nine months ended September 30, 2022, we did not sell any shares under the Open Market Sale Agreement and the related prospectus supplement. During the nine months ended September 30, 2021, we issued 922,077 shares of our common stock at a weighted-average price of $5.70 per share, resulting in net proceeds to us of $5.1 million. At September 30, 2022, we had $41.3 million in aggregate gross offering amount of shares available under the Open Market Sale Agreement and the related prospectus supplement. For information relating to the rules known as the “baby shelf” rules, please see “—Raising additional capital may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidates.”

Policies and Significant Judgments and Estimates

Our unaudited condensed consolidated financial statements are prepared in accordance with generally accepted accounting principles in the United States, or U.S. GAAP. The preparation of our unaudited condensed consolidated financial statements and related disclosures requires us to make estimates and judgments that affect the reported amounts of assets, liabilities, costs and expenses, and the disclosure of contingent assets and liabilities in our unaudited condensed consolidated financial statements. We base our estimates on historical experience, known trends and events and various other factors that we believe are 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 readily apparent from other sources. We evaluate our estimates and assumptions on an ongoing basis. Our actual results may differ from these estimates under different assumptions or conditions.

There have been no significant changes to our critical accounting policies from those described in “Management’s Discussion and Analysis of Financial Condition and Results of Operations,” included in our Annual Report on Form 10-K for the year ended December 31, 2021, filed with the SEC on March 4, 2022, or our Form 10-K.

Emerging Growth Company Status

The Jumpstart Our Business Startups Act of 2012 permits an “emerging growth company” such as our company to take advantage of an extended transition period to comply with new or revised accounting standards applicable to public companies until those standards would otherwise apply to private companies. We have irrevocably elected to “opt out” of this provision and, as a result, we will comply with new or revised accounting standards when they are required to be adopted by public companies that are not emerging growth companies.

Recently Issued Accounting Pronouncements

Refer to Note 2 in the accompanying notes to our unaudited condensed consolidated financial statements appearing elsewhere in this Quarterly Report on Form 10-Q for a discussion of recent accounting pronouncements.

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Item 3. Quantitative and Qualitative Disclosures About Market Risk.

We are a smaller reporting company, as defined in Rule 12b-2 under the Exchange Act for this reporting period and are not required to provide the information required under this item.

Item 4. Controls and Procedures.

Evaluation of Disclosure Controls and Procedures

Our management, with the participation of our Chief Executive Officer (principal executive officer) and Chief Financial Officer (principal financial officer), evaluated the effectiveness of our disclosure controls and procedures as of September 30, 2022. The term “disclosure controls and procedures,” as defined in Rules 13a-15(e) and 15d-15(e) under the Exchange Act means controls and other procedures of a company that are designed to ensure that information required to be disclosed by a company in the reports that it files or submits under the Exchange Act are recorded, processed, summarized and reported, within the time periods specified in the SEC’s rules and forms. Disclosure controls and procedures include, without limitation, controls and procedures designed to ensure that information required to be disclosed by a company in the reports that it files or submits under the Exchange Act is accumulated and communicated to the Company’s management, including its principal executive and principal financial officers, as appropriate to allow timely decisions regarding required disclosure. Management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving their objectives and management necessarily applies its judgment in evaluating the cost-benefit relationship of possible controls and procedures. Based on the evaluation of our disclosure controls and procedures as of September 30, 2022, our Chief Executive Officer and our Chief Financial Officer concluded that, as of such date, our disclosure controls and procedures were effective at the reasonable assurance level.

Changes in Internal Control Over Financial Reporting

There were no changes in our internal control over financial reporting during the quarter ended September 30, 2022 that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.

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PART II—OTHER INFORMATION

Item 1. Legal Proceedings.

Since the announcement of the Merger Agreement, three complaints have been filed, captioned: Bushansky v. LogicBio Therapeutics, Inc. et al., No. 1:22-CV-09145 (S.D.N.Y.) (the “Bushansky Complaint”), Wilhelm v. LogicBio Therapeutics, Inc. et al., No. 1:22-CV-09186 (S.D.N.Y.) (the “Wilhelm Complaint”), and Carlisle v. LogicBio Therapeutics, Inc. et al., No. 1:22-CV-01418 (D. Del.) (the “Carlisle Complaint”) (collectively, the “Complaints”).  Each of the Complaints names the Company and its directors as defendants.  The Company has also received eight stockholder demand letters, including one draft complaint.  These are the “Vugman Demand Letter,” the “Way Demand Letter,” the “Wheeler Demand Letter,” the “Barbeau Demand Letter,” the “Redfield Demand Letter,” the “Ortega Demand Letter,” the “Browning Demand Letter,” and the “Smith Demand Letter” (collectively, the “Demand Letters”).  The Barbeau Demand Letter contained a draft complaint, captioned Barbeau v. LogicBio Therapeutics, Inc. et al.  Moreover, the Company received a stockholder demand letter seeking access to the Company’s books and records pursuant to Delaware Code Title 8, Section 220 (the “Section 220 Demand”).

The Complaints allege that the Company and its directors violated federal securities laws by failing to disclose material information in the Solicitation/Recommendation Statement on Schedule 14D-9 filed by the Company with the SEC on October 18, 2022 (together with the exhibits thereto, the “Schedule 14D-9”).  The Complaints generally seek, among other things, to enjoin the Company from proceeding with closing the Offer, the Merger and the other transactions contemplated by the Merger Agreement (the “Transactions”) unless and until it discloses and disseminates the alleged material information or, in the event the Transactions are consummated, a rescission of the Transactions and a recovery of the rescissory damages.  In addition, the Complaints seek an award of plaintiffs’ costs, including reasonable allowance for attorneys’ fees and costs.  The Carlisle Complaint also seeks a declaration that the defendants violated federal securities laws. The Demand Letters generally seek that certain allegedly omitted information in the Schedule 14D-9 be disclosed. The Section 220 Demand seeks access to the Company’s books and records relevant to the Transactions and the Company’s board members. The Section 220 Demand cites substantially similar allegations to those in the Complaints and Demand Letters as the basis for seeking to inspect the information requested in the Section 220 Demand.

The Company believes that the allegations in the Complaints, Demand Letters and Section 220 Demand are without merit and intends to defend against them vigorously.

Item 1A. Risk Factors.

We face a variety of risks and uncertainties in our business. Additional risks and uncertainties not presently known to us or that we currently believe to be immaterial may also become important factors that affect our business. If any of the following risks occurs, our business, financial statements and future growth prospects could be materially and adversely affected.

Risks Related to the Proposed Acquisition by Alexion

We may not complete the pending transactions with Alexion within the time frame we anticipate, or at all, which could have an adverse effect on our business, financial results and/or operations.

On October 3, 2022, the Company entered into the Merger Agreement by and among the Company, Parent and Merger Sub. Pursuant to the Merger Agreement, on October 18, 2022, Merger Sub commenced the Offer to purchase, subject to the satisfaction or waiver of certain conditions set forth in the Merger Agreement, any and all of the issued and outstanding Shares, at a price of $2.07 per Share to the seller in cash, without interest, less any applicable withholding taxes, upon the terms and subject to the conditions set forth in the Offer to Purchase and in the related Letter of Transmittal. On the same date as the consummation of the Offer and subject to the satisfaction or waiver of certain conditions set forth in the Merger Agreement, Merger Sub will be merged with and into the Company without a vote of the Company’s stockholders to adopt the Merger Agreement and consummate the Merger, in accordance with Section 251(h) of the of the DGCL, with the Company continuing as the surviving corporation and as a wholly-owned subsidiary of Parent.

The obligations of Merger Sub to, and Parent to cause Merger Sub to, accept for payment, and pay for, any Shares validly tendered (and not validly withdrawn) pursuant to the Offer are subject to the satisfaction or waiver of the following conditions:

(1) that the number of Shares validly tendered and not validly withdrawn as of one minutes following 11:59 p.m. (12:00 midnight), New York City time, on Tuesday, November 15, 2022 (the “Offer Expiration Time”), considered together with all other Shares (if any) otherwise beneficially owned by Parent or any of its wholly-owned subsidiaries (including Purchaser) (but excluding Shares tendered pursuant to guaranteed delivery procedures that have not yet been “received,” as

37

defined by Section 251(h)(6) of the DGCL, represents one more than 50% of the total number of the outstanding Shares at the Offer Expiration Time (the “Minimum Tender Condition”);

(2) the accuracy of the Company’s representations and warranties (subject to customary materiality qualifiers as more fully described in the Merger Agreement);

(3) the Company having complied with, or performed, in all material respects all of the Company’s covenants and agreements it is required to comply with or perform at or prior to the Offer Expiration Time;

(4) no Company Material Adverse Effect (as defined in the Merger Agreement) has occurred since the date of the Merger Agreement and is continuing;

(5) the delivery to Parent and Purchaser by the Company of a certificate executed on behalf of the Company by an executive officer of the Company confirming that the conditions in (2), (3) and (4) above have been duly satisfied;

(6) there has not been issued by any court of competent jurisdiction and remaining in effect any temporary restraining order, preliminary or permanent injunction or other order preventing the consummation of the Transactions, or imposing a Burdensome Condition (as defined in the Merger Agreement) as a condition or consequence of consummating the Transactions (including any decision by the European Commission (the “EC”) to examine the Transactions under Article 22(3) of the EU Merger Regulation, and any notification of a referral request under Article 22 (2) of the EU Merger Regulation prior to such a decision having been made, each of which would prevent or make unlawful the consummation of the Transactions while the standstill obligation is in effect), nor is there any applicable law or order promulgated, entered, enforced, enacted, issued or deemed applicable to the Transactions by any governmental entity of competent jurisdiction directly or indirectly prohibiting, or making illegal, the consummation of the Transactions or imposing a Burdensome Condition as a condition or consequence on consummating the Transactions (the “Injunction Condition”); provided, that no party to the Merger Agreement will be permitted to invoke this condition unless it will have taken all actions required under the Merger Agreement to have any such law or order lifted;

(7) (a) the offer letter and each restrictive covenant agreement (or, in the case of any Key Employee (as defined below) that has previously entered into a restrictive covenant agreement with the Company that includes a post-termination non-competition covenant, reaffirming their obligations under such restrictive covenant agreement in their offer letter) of each Key Employee entered into and delivered to Parent or the Company, as applicable, concurrently with the execution and delivery of the Merger Agreement continues to be in full force and effect, and no breach nor repudiation of such agreements by any such Key Employee having occurred or been imminent or threatened, and (b) each of Frederic Chereau, Mariana Nacht and Matthias Hebben (together, the “Key Employees”) will be an employee of the Company or any subsidiary of the Company immediately prior to the Effective Time (clauses (a) and (b) together, the “Key Employee Conditions”); and

(8) the Merger Agreement has not been terminated in accordance with its terms.

We cannot predict with certainty whether and when the conditions to the Offer will be satisfied. If one or more of these conditions are not satisfied, and as a result, we do not complete the Offer and the Merger, we would remain liable for significant transaction costs, and the focus of our management would have been diverted from seeking other potential strategic opportunities, in each case without realizing any benefits of the Offer and the Merger. Certain costs associated with the Offer and the Merger have already been incurred or may be payable even if the Offer and the Merger are not consummated. Finally, any disruptions to our business resulting from the announcement and pendency of the Offer and the Merger, including any adverse changes in our relationships with our collaborators, partners, vendors and employees, could continue or accelerate in the event that we fail to consummate the Offer and Merger.

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In addition, the Merger Agreement may be terminated under certain specified circumstances, including, but not limited to, a change in the recommendation of our Board or a termination of the Merger Agreement by us to enter into an agreement for a Superior Offer (as defined in the Merger Agreement). As a result, we cannot assure you that the Merger will be completed, or that, if completed, it will be exactly on the terms set forth in the Merger Agreement or within the expected time frame.

If the Merger is not completed within the expected time frame or at all, we may be subject to a number of material risks. The price of our common stock may decline to the extent that current market prices reflect a market assumption that the Merger will be completed. The failure to complete the Merger also may result in negative publicity and negatively affect our relationship with our stockholders, employees, collaborators, vendors, regulators and other business partners. We may also be required to devote significant time and resources to litigation related to any failure to complete the Merger. Any of these events could have a material adverse effect on our business, operating results and financial condition and could cause a decline in the price of our common stock.

The announcement and pendency of the Transactions with Alexion could adversely affect our business, financial results and/or operations.

Our efforts to complete the Transactions could cause substantial disruptions in, and create uncertainty surrounding, our business, which may materially adversely affect our results of operation and our business. Uncertainty as to whether the Transactions will be completed may affect our ability to recruit prospective employees or to retain and motivate existing employees. Employee retention may be particularly challenging while the Transactions are pending because employees may experience uncertainty about their roles following the Transactions. A substantial amount of our management’s and employees’ attention is being directed toward the completion of the Transactions and thus is being diverted from our day-to-day operations. Uncertainty as to our future could adversely affect our business and our relationship with collaborators, vendors, suppliers, regulators and other business partners. For example, vendors, suppliers, collaborators and other counterparties may defer decisions concerning working with us, or seek to change existing business relationships with us. Changes to or termination of existing business relationships could adversely affect our results of operations and financial condition, as well as the market price of our common stock. The adverse effects of the pendency of the Transactions could be exacerbated by any delays in completion of the Transactions or termination of the Merger Agreement.

While the Merger Agreement is in effect, we are subject to restrictions on our business activities.

While the Merger Agreement is in effect, we are subject to restrictions on our business activities, generally requiring us to conduct our business in the ordinary course in all material respects, consistent with past practice, and subjecting us to a variety of specified limitations absent Alexion’s prior consent. These limitations include, among other things, restrictions on our ability to issue, split or repurchase securities; sell, lease, license, or dispose of any material portion of our assets, business or properties (subject to certain exceptions); pay dividends; amend our organizational documents; enter into or amend certain agreements; settle a material proceeding; incur indebtedness; commence or terminate a clinical trial or terminate an IND-enabling preclinical study; sell, license or dispose of material intellectual property; and other restrictions listed in the Merger Agreement. These restrictions could prevent us from pursuing strategic business opportunities, taking actions with respect to our business that we may consider advantageous and responding effectively and/or timely to competitive pressures and other developments. As a result, these restrictions may materially and adversely affect our business, results of operations and financial condition, our stock price, regardless of whether the Merger is completed.

The Merger Agreement limits our ability to pursue alternative transactions, which could deter a third party from proposing an alternative transaction.

The Merger Agreement contains provisions that, subject to certain exceptions, limit our ability to, (a) continue any direct or indirect solicitation, knowing encouragement, knowing facilitation (including by way of providing non-public information), discussions or negotiations with any persons that may be ongoing with respect to any Company Acquisition Proposal (as defined below) and (b) directly or indirectly, (i) solicit, initiate or knowingly facilitate or knowingly encourage (including by way of furnishing non-public information) any inquiries regarding, or the making of any proposal or offer that constitutes, or would reasonably be expected to lead to, a Company Acquisition Proposal, (ii) engage in, continue or otherwise participate in any discussions or negotiations regarding, or furnish to any other person any non-public information in connection with or for the purpose of encouraging or facilitating, a Company Acquisition Proposal or any proposal or offer that would reasonably be expected to lead to a Company Acquisition Proposal, (iii) enter into any letter of intent, acquisition contract, contract in principle or other contract with respect to a Company Acquisition Proposal or any proposal or offer that would reasonably be expected to lead to a Company Acquisition Proposal (other than a customary confidentiality agreement that contains provisions (other than standstill provisions) that are no less favorable in the aggregate than those contained in the confidentiality agreements between the Company and Parent and that does not prohibit the Company from providing any information to Parent in accordance with, or otherwise prohibit the Company from complying with, its obligations under the terms of the Merger Agreement or (iv) waive,

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terminate, modify or fail to enforce any provision of any “standstill” or similar obligation of any person (other than Parent) with respect to the Company (unless the Board concludes in good faith, after consultation with its outside legal advisors, that the failure to do so would be inconsistent with its fiduciary duties under applicable laws). Under certain circumstances set forth in the Merger Agreement, we are permitted to terminate the Merger Agreement to accept a Superior Offer (as defined in the Merger Agreement); however, we would be required to pay Alexion a termination fee equal to $2.1 million in connection with such termination.

Under the Merger Agreement, “Company Acquisition Proposal” means any inquiry, proposal or offer from, or indication of interest in making a proposal or offer by, any person (other than Parent and its affiliates) or “group”, within the meaning of Section 13(d) of the Exchange Act, whether written or oral, contemplating or otherwise relating to, in a single transaction or series of related transactions, any (a) acquisition, disposition, sale, lease, exchange, transfer or license of the assets or the businesses of the Company or its subsidiaries equal to 20% or more of the Company’s consolidated assets or to which 20% or more of the Company’s consolidated net revenues or net earnings are attributable, (b) issuance or acquisition of 20% or more of the outstanding capital stock of the Company or its subsidiaries, (c) recapitalization, tender offer or exchange offer that if consummated would result in any person or group beneficially owning 20% or more of the outstanding capital stock of the Company or (d) merger, consolidation, amalgamation, share exchange, business combination, recapitalization, liquidation, dissolution or similar transaction involving the Company or its subsidiaries that if consummated would result in any person or group beneficially owning 20% or more of the outstanding capital stock of the Company, in each case other than the Transactions.

It is possible that these or other provisions in the Merger Agreement, including the termination fee of approximately $2.1 million payable to Alexion, might discourage a potential competing acquirer that might have an interest in acquiring all or a significant part of our outstanding common stock from considering an acquisition or might result in a potential competing acquirer proposing an overall lower per-share consideration amount than it might otherwise have proposed to offer.

If the Transactions are consummated, our stockholders will not be able to participate in any upside to our business.

Pursuant to the Merger Agreement, on October 18, 2022, Merger Sub commenced the Offer to purchase, subject to the satisfaction or waiver of certain conditions set forth in the Merger Agreement, any and all of the issued and outstanding Shares, at a price of $2.07 per Share, to the seller in cash, without interest, less any applicable withholding taxes, upon the terms and subject to the conditions set forth in the Offer to Purchase and in the related Letter of Transmittal. Our stockholders will not receive any shares of Alexion common stock in connection with the Offer. As a result, if our business following the consummation of the Transactions performs well, our current stockholders will not receive any benefit from the performance of our business following the consummation of the Transactions.

Stockholder complaints have been filed, and others could be filed, against us and the members of our Board arising out of the proposed Merger, which may delay or prevent the proposed Merger, and could result in significant costs of defense, indemnification and liability.

Putative stockholder complaints have been filed against us and the members of our Board and other complaints, including stockholder class action complaints, could be filed against us, our Board, Alexion, Alexion’s board of directors, and others in connection with the Transactions contemplated by the Merger Agreement. The outcome of litigation is uncertain, and we may not be successful in defending against any such claims. Lawsuits that have been or may be filed against us, our Board, Alexion, or Alexion’s board of directors could delay or prevent the consummation of the Merger, divert the attention of our management and employees from our day-to-day business, and otherwise adversely affect us financially.

We have incurred, and will continue to incur, direct and indirect costs as a result of the pending Transactions with Alexion.

We have incurred, and will continue to incur, significant costs and expenses, including fees for professional services and other transaction costs, in connection with the pending Transactions with Alexion. We must pay a material portion of these costs and expenses whether or not the Merger is completed. There are a number of factors beyond our control that could affect the total amount or the timing of these costs and expenses, any of which could materially and adversely affect our business, prospects, financial condition and results of operations. Many of the expenses that will be incurred, by their nature, are difficult to estimate accurately.

The following risk factors do not take into account the proposed Merger and assume that we remain a stand-alone company except as otherwise noted.

Risks Related to Our Financial Position and Need for Additional Capital

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We have incurred significant losses since inception and anticipate that we will incur continued losses for the foreseeable future. We may never achieve or maintain profitability.

Investment in pharmaceutical product development and commercialization is highly speculative because it entails upfront capital expenditures and significant risk that a product candidate will fail to gain marketing approval or that an approved product will not be commercially viable. We have incurred net losses in each year since our inception, including net losses of $17.5 million for the nine months ended September 30, 2022. As of September 30, 2022, we had an accumulated deficit of $157.5 million. In addition, we have not commercialized any products and have never generated any revenue from product sales. Since our inception, we have devoted most of our resources to research and development, including our preclinical and clinical development activities, as well as to building out our team and infrastructure. We expect to continue to incur significant additional operating losses for the foreseeable future as we seek to continue to advance LB-001, our lead product candidate, through clinical development, expand our research and development capabilities and activities, develop new product candidates and advance them through preclinical and clinical development, advance the development of our GeneRide and sAAVy technology platforms, conduct process and analytical development, conduct manufacturing activities, conduct clinical trials, seek regulatory approval and, if we receive approval from the FDA, the European Medicines Agency, or EMA, or other regulatory authorities, commercialize our product candidates. Our net losses may fluctuate significantly from quarter to quarter and year to year. Because of the numerous risks and uncertainties associated with genetic medicine product development, we are unable to accurately predict the timing or amount of increased expenses, when, if ever, we will generate revenue from the commercialization of products or whether we will achieve or maintain profitability. We anticipate that we will continue to incur significant expenses if and as we:

• continue our current research programs and our preclinical development of any product candidates from our current research programs;

• take actions necessary to consummate the Merger;

• continue to conduct our clinical program for LB-001, including our Phase 1/2 SUNRISE clinical trial. In connection with the lifting of the LB-001 clinical hold described above, we amended the SUNRISE protocol. Implementation of the protocol amendments is expected to increase clinical trial costs primarily due to increased screening time, an intensified monitoring regimen, prolonged hospitalization and potential acute use of a complement inhibitor;

• seek to identify, assess, acquire and/or develop additional research programs and additional product candidates, and initiate and conduct clinical trials for such product candidates;

• engage in transactions, including strategic, merger, collaboration, acquisition and licensing transactions;

• seek regulatory approvals for any product candidates that successfully complete clinical trials; develop, optimize, scale and validate a manufacturing process and analytical methods for any product candidates we may develop;

• obtain market acceptance of any product candidates we may develop as viable treatment options;

• address competing technological and market developments;

• maintain, expand and protect our intellectual property portfolio and provide reimbursement of third-party expenses related to our patent portfolio;

• further develop our GeneRide and sAAVy technology platforms, as well as our proprietary manufacturing process, mAAVRx;

• hire additional scientific, clinical, technical, quality, regulatory, general and administrative, and commercial personnel and add operational, financial and management information systems and personnel, including personnel to support our process and analytical development, manufacturing and planned future commercialization efforts;

• make royalty, milestone or other payments under current or future in-license agreements;

• establish and maintain supply chain and manufacturing relationships with third parties that can provide adequate products and services, in both amount, timing and quality, to support our development programs and the market demand for any product candidate for which we obtain regulatory and marketing approval;

• lease and build new facilities, including offices and labs, as necessary to support any organizational growth;

• comply with GXP, including good laboratory practice, GCP, and cGMP;

• build out and validate clinical and commercial-scale cGMP manufacturing capabilities;

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• establish a sales, marketing and distribution infrastructure to commercialize any product candidates for which we may obtain marketing approval; and

• experience any delays or encounter issues with any of the above.

Furthermore, our ability to successfully develop, commercialize and license our product candidates and potentially generate product revenue is subject to substantial additional risks and uncertainties. Each program and any product candidate we develop, along with our GeneRide and sAAVy platforms, will require additional preclinical and clinical development, regulatory approval in one or more jurisdictions, process and analytical development, securing supply and manufacturing capacity, building of a commercial organization, substantial investment and significant marketing efforts before we generate any revenue from product sales. See “—Risks Related to Discovery, Development, Clinical Testing, Manufacturing and Regulatory Approval” and “—Risks Related to Commercialization.”

As a result of all of the above, as well as other potential factors, we expect to continue to incur net losses and negative cash flows for the foreseeable future. These net losses and negative cash flows have had, and will continue to have, an adverse effect on our stockholders’ equity and working capital. The amount of our future net losses will depend, in part, on the rate of future growth of our expenses and our ability to generate revenue. If we are unable to develop and commercialize one or more product candidates either alone or with collaborators, or if revenue from any product candidate that receives marketing approval is insufficient, we will not achieve profitability. Even if we do achieve profitability, we may not be able to sustain or increase profitability.

Under our ASC 205-40 analysis, there is “substantial doubt” that we will have sufficient funds to satisfy our obligations through the next twelve months from the date of issuance of this Quarterly Report on Form 10-Q.

At September 30, 2022, we had $30.8 million of cash and cash equivalents on hand. On a quarterly basis, we are required to conduct an accounting analysis under ASC 205-40, Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern, or ASC 205-40. The result of our ASC 205-40 analysis is that there is “substantial doubt” that we will have sufficient funds to satisfy our obligations through the next twelve months from the date of issuance of the unaudited condensed consolidated financial statements. Our ability to continue as a going concern is dependent on our ability to obtain the necessary financing to meet our obligations and repay our liabilities arising from the ordinary course of business operations when they become due. The outcome of these matters cannot be predicted with any certainty at this time. If we are unable to raise sufficient capital when needed, our business, financial condition and results of operations will be materially and adversely affected, and we will need to significantly modify or terminate our operational plans. Our conclusion, in accordance with ASC 205-40, that there is “substantial doubt” that we will have sufficient funds to satisfy our obligations through the next twelve months from the date of issuance of this Quarterly Report on Form 10-Q may materially adversely affect our business, prospects, financial condition, results of operations, share price and our ability to raise capital or to enter into agreements with third parties that may be beneficial to us.

We will require additional capital to fund our operations, and if we fail to obtain necessary financing, we may not be able to complete the development and commercialization of any product candidates.

We expect to spend substantial amounts to complete the development of, seek regulatory approvals for and commercialize LB-001 and any other product candidate we may identify and develop. We will require additional capital, which we may seek to raise through equity offerings, debt financings, marketing and distribution arrangements, collaborations, strategic alliances, licensing arrangements or other sources, to enable us to complete the development and potential commercialization of LB-001 and any other product candidate. Adequate additional financing may not be available to us on acceptable terms, or at all. Our failure to raise capital as and when needed would have a negative effect on our financial condition and our ability to pursue our business strategy. For more information, see the risk factor titled “– Under our ASC 205-40 analysis, there is ‘substantial doubt’ that we will have sufficient funds to satisfy our obligations through the next twelve months from the date of issuance of this Quarterly Report on Form 10-Q.” In addition, attempting to secure additional financing may divert the time and attention of our management from day-to-day activities and harm our product candidate development efforts.

Changing circumstances could cause us to consume capital significantly faster than we currently anticipate, and we may need to spend more than currently expected because of circumstances beyond our control. Because the length of time and activities associated with successful development of LB-001 and any other product candidates is highly uncertain, we are unable to estimate the actual funds we will require for development and, if applicable, any approved marketing and commercialization activities. Our future funding requirements, both near and long-term, will depend on many factors, including:  

• the scope, progress, timing, costs and results of drug discovery, preclinical development, laboratory testing, and clinical trials for our product candidates, including the ongoing development program for LB-001 (including the Phase 1/2 SUNRISE clinical trial), and process development and manufacturing activities for LB-001;

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• the implementation of amendments to the Phase 1/2 SUNRISE clinical trial protocol made in connection with the lifting of the clinical hold, which include increased screening time, an intensified monitoring regimen, prolonged hospitalization and potential acute use of a complement inhibitor;

• the outcome, timing and cost of following the advice of and complying with regulatory requirements and decisions made by the FDA, EMA and other regulatory authorities, including relating to any potential clinical holds that may be imposed on us in the future;

• the impact of the COVID-19 pandemic on our ability to progress with our research, development, manufacturing and regulatory efforts, including our ability to advance and complete our Phase 1/2 SUNRISE clinical trial of LB-001;

• the cost of filing, prosecuting, defending and enforcing our patent claims and other intellectual property rights;

• the cost of defending potential intellectual property disputes, including patent infringement actions;

• the achievement of milestones or occurrence of other developments that trigger payments under any of our current agreements or other agreements we may enter into;

• the extent to which we are obligated to reimburse, or entitled to reimbursement of, clinical trial and other research and development costs under future collaboration agreements, if any;

• the effect of competing technological and market developments;

• the cost and timing of completion of process and analytical development and manufacturing activities;

• the extent to which we engage in transactions, including collaboration, merger, acquisition and licensing transactions;

• our ability to establish and maintain collaborations on favorable terms, if at all;

• the cost of establishing sales, marketing and distribution capabilities for LB-001 and any other product candidates in regions where we choose to commercialize our product candidates, if approved;  

• the initiation, progress, timing and results of our commercialization of LB-001 and any other product candidates, if approved, for commercial sale;

• our ability to repay outstanding debt; and

• our ability to attract, hire and retain qualified personnel.

Identifying potential product candidates and conducting preclinical testing, process and analytical development, manufacturing activities and clinical trials is a time-consuming, expensive and uncertain process that takes years to complete, and we may never generate the necessary data or results required to obtain marketing approval and achieve product sales. In addition, even if we successfully identify and develop product candidates and one or more are approved, we may not achieve commercial success. Our commercial revenues, if any, will be derived from sales of medicines that we do not expect to be commercially available for many years, if at all.

Accordingly, we will need to continue to rely on additional financing to achieve our business objectives. We cannot be certain that additional funding will be available on acceptable terms, or at all. If we are unable to raise additional capital in sufficient amounts or on terms acceptable to us, we may be required to delay, reduce or eliminate some or all of our research and development programs, preclinical and clinical development programs or future commercialization efforts, or we may be unable to continue operations.

Any significant delays in our programs may also require us to reevaluate our corporate strategy, resulting in the expenditure of significant resources and time, or potentially resulting in our discontinuing our operations.

Raising additional capital may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidates.

To the extent that we raise additional capital through the sale of convertible debt securities or equity, including through our existing at-the-market equity facility, the ownership interests of our stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our common stockholders. In addition, under U.S. securities laws, a company with a public float of less than $75 million measured at certain time periods may not issue securities under Registration Statements on Form S-3 in excess of one-third of its public float in a 12-month period, which may limit the amount of funds we can raise using Registration Statements on Form S-3. For purposes of the prior sentence, “public float” means the aggregate market value of a company’s common stock held by non-affiliates.

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Debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends. In addition, debt financing would result in increased fixed payment obligations. For information about our current outstanding debt, see Note 5 in the accompanying notes to our unaudited consolidated financial statements appearing elsewhere in this Quarterly Report on Form 10-Q and the risk factor titled “The terms of our Loan and Security Agreement place restrictions on our operating and financial flexibility.” If we raise additional funds through collaborations, strategic alliances or marketing, distribution or licensing arrangements with third parties, we may be required 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.

We have never generated revenue from product sales and may never be profitable.

Our ability to generate revenue from product sales and achieve profitability depends on our ability, alone or with collaborative partners, to successfully complete the development of, and obtain the regulatory approvals necessary to commercialize, product candidates we may identify for development. To date, we have not generated any revenue from product sales and do not anticipate generating revenues from product sales for the foreseeable future. Our ability to generate future revenues from product sales depends heavily on our, or our collaborators’, ability to successfully:

• identify product candidates and complete research and development of any product candidates we may identify;

• obtain sufficient financial and other resources to complete the LB-001 development program and the development programs of any other product candidate;

• obtain data from clinical programs of our product candidates that supports an acceptable risk-benefit profile of each such product candidate in the intended populations, including LB-001 in pediatric patients with MMA;

• develop safe and effective delivery mechanisms for our genetic medicine product candidates;

• achieve desirable medicinal properties for the intended indications;

• seek and obtain regulatory approvals for any product candidate for which we complete clinical trials;

• launch and commercialize any product candidate for which we obtain regulatory approval by establishing a sales force, marketing and distribution infrastructure and/or collaborating with a commercialization partner;

• qualify for adequate healthcare coverage and reimbursement by government and third-party payors for any product candidate for which we obtain regulatory approval;

• develop, enhance, scale and validate a manufacturing process and analytical methods for any product candidates we may develop;

• implement effective strategies and knowledge management systems to ensure the integrity of data, specifically the completeness, consistency and accuracy of data used to ensure the safety, efficacy and quality of products manufactured;

• establish and maintain supply and manufacturing relationships with third parties that remain compliant with all relevant health authority and legal requirements and can provide adequate, in amount, timing and quality, products and services to support clinical development and the market demand for any product candidate for which we obtain regulatory approval;

• compete with other therapies and treatment options;

• obtain market acceptance of any product candidates we may develop as viable treatment options;

• obtain a continued acceptable safety profile of any approved medicines following approval;

• address competing technological and market developments;

• implement internal systems and infrastructure, as needed;

• enter into collaborations to further the development of any product candidate;

• negotiate favorable terms in any collaboration, licensing or other arrangements into which we may enter and performing our obligations in such collaborations;

• maintain, protect and expand our portfolio of intellectual property rights, including patents, trade secrets, know-how and non-patent exclusivity for our medicines;

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• avoid and defend against third-party interference or infringement claims; and

• attract, hire and retain qualified personnel.

Additionally, because our technology involves genome editing and gene therapy we are subject to the following additional challenges and risks with respect to our ability to generate future product revenue, including:

• regulatory requirements that govern gene and cell therapy products, which have changed frequently and may continue to change in the future, and few products that involve the genetic modification of patient cells have been approved in the United States or the European Union, or the EU;  

• the FDA’s recommendation of a follow-up observation period of up to 15 years or longer for all patients who receive treatment using gene delivery, necessitating us to adopt such an observation period for any product candidate we may develop;

• gene delivery technologies are novel approaches and may present additional challenges and risks, including obtaining approval from regulatory authorities that have limited experience with the development of such technologies; and

• reports may arise from preclinical or clinical testing of the gene delivery technologies of our competitors or our product candidates that may raise safety or efficacy concerns about our product candidates.

Risks Related to Discovery, Development, Clinical Testing, Manufacturing and Regulatory Approval

Our product candidates may cause serious adverse events or undesirable side effects or have other properties that may delay or prevent their regulatory approval, limit the commercial profile of an approved label or result in significant negative consequences following marketing approval, if any.

It is impossible to predict when or if any product candidates we may develop will prove safe in humans. In the genomic medicine field, there have been several significant adverse events from gene therapy treatments in the past, including reported cases of leukemia and death, some of which took years to present. There can be no assurance that our genomic medicine technologies will not cause undesirable side effects. Results of our clinical trials could reveal a high and unacceptable severity and prevalence of side effects, toxicities or unexpected characteristics, including death. A significant risk in many gene editing products is that the edit will be “off-target” (or “on-target,” but unwanted) and cause serious adverse events, undesirable side effects, toxicities or unexpected characteristics. For example, off-target cuts could lead to disruption of a gene or a genetic regulatory sequence at an unintended site in the DNA, or, in those instances where we also provide a segment of DNA to serve as a repair template, it is possible that following off-target cut events, DNA from such repair template could be integrated into the genome at an unintended site, potentially disrupting another important gene or genomic element. While we believe our GeneRide technology obviates this through the use of homologous recombination, or HR, we cannot be certain that off-target editing will not occur in any of our planned or future clinical trials. There is also the potential risk of delayed adverse events following exposure to gene editing therapy, due to the potential for persistent biological activity of the genetic material or other product components used to carry the genetic material.  In addition, cases of TMA have been previously reported in association with AAV genetic therapies. As previously disclosed, certain patients in our SUNRISE trial have experienced TMAs following dosing, as well as what has been categorized as cytokine release syndrome.  For more information on these cases, please see “Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations—Overview—LB-001 for the Treatment of Methylmalonic Acidemia (MMA) in Pediatric Patients.” In addition to SAEs or side effects caused by any product candidate we may develop, the administration process or related procedures also can cause undesirable side effects. If any such events occur, our clinical trials could be suspended or terminated.

If SAEs or undesirable side effects arise in the development of any of our product candidates, we, the DSMB, the FDA or other regulatory authorities, IRBs, or ethics committees at the institutions in which our studies are conducted, could suspend or terminate our clinical trials, and the FDA or other regulatory authorities could order us to cease clinical trials, or delay or deny approval of our product candidates for any or all targeted indications, or approve our product candidates with a restrictive label. For example, we announced in February 2022 that the FDA placed our LB-001 IND on clinical hold after we reported an SAE in our Phase 1/2 SUNRISE clinical trial of LB-001 in pediatric MMA patients. We announced that the hold was lifted in May 2022. For more information, please see “Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations—Overview—LB-001 for the Treatment of Methylmalonic Acidemia (MMA) in Pediatric Patients.” Even though the FDA has now lifted the clinical hold, there is no guarantee that we will be able to continue to enroll the SUNRISE trial in a timely manner or at all. SAEs or undesirable side effects that arise in the development of any of our product candidates and any resulting action taken by us, the DSMB, the FDA or other regulatory authorities, or the IRBs, may result in our needing to abandon their development or limit development to certain uses or subpopulations in which the SAEs, undesirable side effects or other characteristics are less prevalent,

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less severe or more acceptable from a risk-benefit perspective, any of which would harm our business, financial condition, results of operations and prospects. Many product candidates that initially showed promise in early-stage testing have later been found to cause side effects that prevented further clinical development of the product candidates. Moreover, if we elect, or are required, to delay, suspend or terminate any clinical trial of any product candidate we may develop, the commercial prospects of such product candidates may be harmed and our ability to generate product revenues from any of these product candidates may be delayed or eliminated. Treatment-related side effects also could affect patient recruitment or the ability of enrolled patients to complete the trial or result in potential product liability claims. In addition, these side effects may not be appropriately recognized or managed by the treating medical staff. We may have to train medical personnel using our product candidates to understand the side effect profiles for our clinical trials and upon any commercialization of any of our product candidates. Inadequate training in recognizing or managing the potential side effects of our product candidates could result in patient injury or death. Any of these occurrences may harm our ability to identify and develop product candidates, which could harm our business, financial condition and prospects significantly.

If any of our product candidates receives marketing approval, and we or others later identify undesirable side effects caused by any such product, including during any long-term follow-up observation period recommended or required for patients who receive treatment using our products, a number of potentially significant negative consequences could result, including:

• regulatory authorities may withdraw approvals of such product;

• we may be required to recall a product or change the way such product is administered to patients;

• additional restrictions may be imposed on the marketing of the particular product or the manufacturing processes for the product;

• regulatory authorities may require additional warnings on the label, such as a “black box” warning or contraindication;

• if we successfully develop a product candidate and it receives marketing approval, the FDA could require us to implement a risk evaluation and mitigation strategy, or REMS, which may include a medication guide outlining the risks of such side effects for distribution to patients, a communication plan to health care practitioners, extensive patient monitoring or distribution systems and processes that are highly controlled, restrictive and more costly than what is typical for the industry;  

• the product could become less competitive;

• we could be sued and held liable for harm caused to patients; and

• our reputation may suffer.

Any of these events could prevent us from achieving or maintaining market acceptance of the particular product candidate, if approved, which could harm our business, financial condition, results of operations and prospects.

We intend to identify and develop product candidates based on our novel GeneRide and sAAVy technology platforms, which makes it difficult to predict the time and cost of product candidate development.

We have concentrated our research and development efforts on product candidates utilizing our GeneRide and sAAVy technology platforms. Our future success depends on the successful development of these novel therapeutic approaches. Any product candidates we may develop will act at the level of DNA, and, because non-human animal DNA differs from human DNA, results of tests of our product candidates in preclinical animal models for either safety or efficacy may not be predictive of results that may be observed in humans. Also, preclinical animal models may not exist for some of the diseases we expect to pursue. Our GeneRide genome editing technology harnesses HR a naturally occurring DNA repair process. The mechanism of action of this technology is still not completely understood. Therefore, it is and will be difficult for us to predict whether any of our product candidates will be able to successfully and consistently integrate corrective DNA in or deliver gene transfer constructs to enough tissue cells or otherwise result in sufficient expression of the target protein to reach therapeutic levels. We cannot be certain that any of our product candidates will be able to meet safety and efficacy levels needed to be therapeutic in humans or that they will not cause SAEs, such as the SAEs referenced in the prior risk factor, or toxicities. As a result of these factors, it is difficult for us to predict the time and cost of product candidate development, and we cannot predict whether the application of our GeneRide or sAAVy technology platforms, or any similar or competitive gene delivery platforms, will result in the identification, development and regulatory approval of any medicines, or that other genetic medicine technologies will not be considered better or more attractive for the development of medicines. Any development problems we experience in the future related to our GeneRide or sAAVy technology platforms or any of our research programs may cause significant delays or unanticipated costs, or we may not be able to solve for the issue. We may also experience delays in developing a capable and scalable manufacturing process or transferring that process to collaboration partners. Any of these

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factors may prevent us from completing our preclinical studies or clinical trials that we may initiate or prevent us from commercializing any product candidates we may develop on a timely or profitable basis, if at all.

Because gene delivery is novel and the regulatory landscape that governs any product candidates we may develop is uncertain and may change, we cannot predict the time and cost of obtaining regulatory approval, if we receive it at all, for any product candidates we may develop.

Because gene delivery is novel, the regulatory requirements governing any gene delivery product candidates we develop are uncertain and subject to change. For example, in January 2020, the FDA issued guidance documents to reflect recent advances in the field, and to set forth the framework for the development, review and approval of gene therapies. These guidance documents pertain to the development of gene therapies for the treatment of specific disease categories, including rare diseases, and to manufacturing and long-term follow-up issues relevant to gene therapy, among other topics. The FDA also issued a guidance document in January 2021 addressing manufacturing considerations for licensed and investigational gene therapy products during the COVID-19 pandemic. In

addition, the FDA issued a draft guidance document in March 2022 providing recommendations for developing human gene therapy

products that incorporate genome editing of human somatic cells. Moreover, there is substantial, and sometimes uncoordinated, overlap in those responsible for global regulation of existing gene therapy products and cell therapy products. For example, in the United States, the FDA has established the Office of Tissues and Advanced Therapies within its Center for Biologics Evaluation and Research, or CBER, to consolidate the review of genome editing, gene therapy and related products, and the Cellular, Tissue and Gene Therapies Advisory Committee to advise CBER on its review. Genome editing and gene therapy clinical trials are also subject to review and oversight by an institutional biosafety committee, or IBC, a local institutional committee that reviews and oversees basic and clinical research conducted at the institution participating in the clinical trial. Although the FDA decides whether individual genome editing or gene therapy protocols may proceed, the review process and determinations of other reviewing bodies can impede or delay the initiation of a clinical trial, even if the FDA has reviewed the trial and approved its initiation. The same applies to regulatory bodies in many other jurisdictions. The EMA’s Committee for Advanced Therapies, or CAT, is responsible for assessing the quality, safety and efficacy of advanced-therapy medicinal products. The role of the CAT is to prepare a draft opinion on an application for marketing authorization for a gene therapy medicinal candidate that is submitted to the EMA. In the European Union, the development and evaluation of a gene therapy medicinal product must be considered in the context of the relevant European Union guidelines. The EMA may issue new guidelines concerning the development and marketing authorization for gene therapy medicinal products and require that we comply with these new guidelines.

Adverse developments in preclinical studies or clinical trials conducted by others in the field of gene therapy products, cell therapy products or products developed through the application of gene editing technology may cause the FDA, the EMA and other regulatory bodies to revise the requirements for approval of any product candidates we may develop or limit the use of products utilizing genome editing technologies, either of which could materially harm our business. In addition, the clinical trial requirements of the FDA, the EMA and other regulatory authorities and the criteria these regulators use to determine the safety and efficacy of a product candidate vary substantially according to the type, complexity, novelty and intended use and market of the potential products.

The regulatory approval process for product candidates such as ours can be more expensive and take longer than for other, better known or more extensively studied pharmaceutical or other product candidates. Furthermore, during the regulatory review process, we will need to identify success criteria and endpoints such that the FDA, the EMA or other regulatory authorities will be able to determine the clinical efficacy and safety profile of any product candidates we may develop. As we are initially seeking to identify and develop product candidates to treat diseases in which there is little clinical experience using new technologies, there is heightened risk that the FDA, the EMA or other regulatory authorities may not consider the clinical trial endpoints that we propose to provide clinically meaningful results (reflecting a tangible benefit to patients). In addition, the resulting clinical data and results may be difficult to analyze. Even if the FDA does find our success criteria to be sufficiently validated and clinically meaningful, we may not achieve the pre-specified endpoints to a degree of statistical significance. This may be a particularly significant risk for many of the genetically defined diseases for which we plan to develop product candidates because many of these diseases have small patient populations, and designing and executing a rigorous clinical trial with appropriate statistical power is more difficult than with diseases that have larger patient populations. Further, even if we do achieve the pre-specified criteria, we may produce results that are unpredictable or inconsistent with the results of the non-primary endpoints or other relevant data. The FDA also weighs the benefits of a product against its risks, and the FDA may view the efficacy results in the context of safety as not being supportive of regulatory approval. Other regulatory authorities in the European Union and other countries, such as the CAT, may make similar comments with respect to these endpoints and data. Any product candidates we may develop will be based on a novel technology that makes it difficult to predict the time and cost of development and of subsequently obtaining regulatory approval. To date, no product that utilizes our GeneRide or sAAVy technology has been approved. There have been a limited number of clinical trials of gene editing and gene therapy technologies, however no genome editing product candidates have been approved and only a few gene therapy

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products have been approved in the United States and Europe. Our Phase 1/2 SUNRISE clinical trial is the first clinical trial of a product candidate that utilizes our technology.

Regulatory agencies administering existing or future regulations or legislation may not allow production and marketing of products utilizing genome editing or gene therapy technology in a timely manner or under technically or commercially feasible conditions. In addition, regulatory action or private litigation could result in expenses, delays or other impediments to our research and development programs or the commercialization of resulting products.

The regulatory review committees and advisory groups described above and the new guidelines they promulgate may lengthen the regulatory review process, increase the scope of process and analytical development, require us to perform additional preclinical studies or clinical trials, increase our development costs, lead to changes in regulatory positions and interpretations, delay or prevent approval and commercialization of these treatment candidates, or lead to significant post-approval limitations or restrictions. As we advance our research programs and develop future product candidates, we will be required to consult with these regulatory and advisory groups and to comply with applicable guidelines. If we fail to do so, we may be required to delay or discontinue development of any product candidates we identify and develop.

We have limited experience conducting clinical trials and no history commercializing genetic medicine product candidates and we may encounter difficulties transitioning from a research-stage to clinical-stage company to ultimately a commercial-stage company, which may make it difficult to evaluate the prospects for our future viability.

We were founded in 2014 and began operations in 2015. Our operations to date have been limited to financing and staffing our company, developing our technology such as our GeneRide and sAAVy technology platforms, identifying and developing LB-001 and other product candidates, undertaking preclinical studies, conducting the SUNRISE clinical trial for LB-001, business planning and raising capital. Other than LB-001, all of our programs are still in the preclinical or research stage of development.

The risk of failure in the biopharmaceutical industry for programs or product candidates at such stage of development is high. We have not yet demonstrated an ability to successfully complete any interventional clinical trials, including pivotal clinical trials, obtain marketing approval, manufacture a commercial scale product or arrange for a third party to do so on our behalf or conduct sales and marketing activities necessary for successful product commercialization. Typically, it takes about six to ten years to develop a new drug from the time it enters Phase 1 clinical trials to when it is approved for treating patients, but it may take longer, in particular in the case of genetic medicines. Consequently, predictions about our future success or viability may not be as accurate as they could be if we had a longer operating history or a history of successfully developing and commercializing genetic medicine product candidates.

In addition, as a business with a limited operating history, we may encounter unforeseen expenses, difficulties, complications, delays and other known and unknown factors. We will eventually need to plan to transition from a company with a research and development focus to a company capable of supporting commercial activities, if any of our product candidates is approved. We may not be successful in such a transition.

Preclinical drug development is uncertain. Some or all of our preclinical programs may experience delays or may never advance to clinical trials, which would adversely affect our ability to obtain regulatory approvals or commercialize these product candidates on a timely basis or at all, which would have an adverse effect on our business.

In order to obtain FDA approval to market a new product, we must demonstrate proof of safety and efficacy in humans. To satisfy these requirements, we will have to conduct adequate and well-controlled clinical trials. Before we can commence clinical trials for a product candidate, we must complete extensive preclinical testing and studies that support our planned IND in the United States. We cannot be certain of the timely completion or outcome of our preclinical testing and studies, and we cannot predict if the FDA will accept our proposed clinical programs, if any, or if the outcome of our preclinical testing and studies will ultimately support the further development of any of our product candidates. As a result, we cannot be sure that we will be able to submit INDs or similar applications for any preclinical programs on the timelines we expect, if at all, and we cannot be sure that submission of INDs or similar applications will result in the FDA or other regulatory authorities allowing clinical trials to begin. For example, our IND for LB-001 was placed on clinical hold by the FDA in February 2020 prior to the dosing of the first patient with LB-001, which was lifted in August 2020.

Conducting preclinical testing is a lengthy, time-consuming and expensive process. The length of time may vary substantially according to the type, complexity, novelty and intended use of the product candidate, and often can be several years or more per product candidate. Delays associated with product candidates for which we are conducting preclinical testing and studies ourselves may cause us to incur additional operating expenses. Moreover, we may be affected by delays associated with the preclinical testing and studies of certain product candidates conducted by our current and potential future partners over which we have no control. The

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commencement and rate of completion of preclinical studies and initiation of clinical trials for a product candidate may be delayed by many factors, including, for example:

• inability to generate sufficient preclinical data to support clinical development;

• delays in reaching a consensus with regulatory agencies on preclinical and clinical study design;

• inability to obtain appropriate or sufficient test agents or preclinical animal models in connection with the indication the product candidate is meant to address;

• recruiting and retaining the appropriate scientific and technical personnel; and

• the product candidate may not be safe or efficacious for the indication we intend to address.

Moreover, even if we obtain positive results from preclinical studies or clinical trials, including interim data from a clinical trial, we may not achieve the same success in ongoing or future trials.

Clinical trials are expensive, difficult to design and implement, and involve an uncertain outcome.

Before obtaining marketing approval from regulatory authorities for the sale of any product candidates we may identify and develop, we must complete preclinical development and then conduct extensive clinical trials to demonstrate the safety and efficacy in humans of any such product candidates. Clinical testing is expensive and can take many years to complete, and its outcome is inherently uncertain. Failure can occur at any time during the clinical trial process, and there is a high failure rate for product candidates proceeding through clinical trials. The results of preclinical studies and clinical trials, including interim data from a clinical trial, of our product candidates may not be predictive of the results of ongoing or future clinical trials. Product candidates in later stages of clinical trials may fail to show the desired safety and efficacy traits despite having progressed through preclinical studies and initial clinical trials. A number of companies in the biotechnology and genetic medicine industries have suffered significant setbacks in advanced clinical trials due to lack of efficacy or adverse safety profiles, notwithstanding promising results in earlier trials. Even if our future clinical trials are completed as planned, we cannot be certain that their results will support the safety and effectiveness of our product candidates for particular indications, including LB-001 for MMA or any other potential indication.

Moreover, preclinical and clinical data are often susceptible to varying interpretations and analyses. Many companies that have believed their product candidates performed satisfactorily in preclinical studies and clinical trials have nonetheless failed to obtain marketing approval of their product candidates.

A clinical trial may be suspended or terminated by us, either independently or based on a recommendation by the DSMB, for such trial, by IRBs of the institutions in which such trials are being conducted, or by the FDA or other regulatory authorities. We or such authorities may impose such a suspension or termination due to a number of factors, including (1) failure to conduct the clinical trial in accordance with regulatory requirements or our clinical protocols; (2) findings from inspection of the clinical trial operations or trial site by the FDA or other regulatory authorities; (3) safety issues or adverse side effects; (4) failure to demonstrate a benefit from using a drug; (5) changes in governmental regulations; (6) administrative actions or lack of adequate funding to continue the clinical trial; or (7) imposition of a clinical hold by the FDA or other regulatory authorities. Furthermore, we may rely on CROs and clinical trial sites to ensure the proper and timely conduct of clinical trials and while we would have agreements governing their committed activities, we would have limited influence over their actual performance, as described in “—Risks Related to Our Dependence on Third Parties.”

In addition, LB-001 will require extensive additional clinical testing before we are prepared to submit a Biologic License Application, or BLA, for regulatory approval in the United States or submit a comparable application for regulatory approval in other jurisdictions. We cannot predict with any certainty if or when we might complete the development of LB-001 and submit a BLA for regulatory approval of LB-001 or whether any such BLA will be approved by the FDA or a comparable application for regulatory approval may be approved in other jurisdictions. We may also seek feedback from the FDA or other regulatory authorities on our clinical development program, and the FDA or such regulatory authorities may not provide such feedback on a timely basis, or such feedback may not be favorable, which could further delay our development programs.

Clinical trials are time consuming and subject to potential delays.

To date, we have not completed any clinical trials for any of our product candidates, including LB-001. We may experience delays in conducting any clinical trials, and we do not know whether planned clinical trials will begin on time, need to be redesigned to address clinical holds imposed by regulatory authorities or for other reasons, recruit and enroll patients on time or be completed on schedule, or at all.

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Clinical trials can be delayed or terminated for a variety of reasons, including delays or failures related to:

• the FDA or other regulatory authorities disagreeing as to the design or implementation of our clinical trials, including alignment with regulatory authorities on registrational endpoints for indications where there are not generally accepted registrational endpoints;

• determining achievable endpoints in connection with our clinical trials that are accepted by the FDA or other regulatory authorities, including with respect to the number of patients and the length of time;

• obtaining regulatory approval to commence a trial or to restart a trial following a clinical hold;

• reaching an agreement on acceptable terms with CMOs, CROs, and clinical trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CMOs, CROs and trial sites;

• obtaining IRB approval at each site;

• regulators, IRBs, DSMBs, safety committees, or ethics committees, may require that we suspend or terminate our clinical trials for various reasons, including noncompliance with regulatory requirements, unforeseen safety issues or adverse side effects, failure to demonstrate a benefit from using our product candidate, or a finding that the participants are being exposed to unacceptable health risk;

• recruiting suitable patients to participate in a trial, particularly in light of the COVID-19 pandemic;

• availability of competing therapies and clinical studies, and clinicians’ and patients’ perceptions as to the potential advantages of the product or product candidate being studied in relation to available therapies or other product candidates in development;

• developing and validating the companion diagnostic to be used in a clinical trial, if applicable;

• having patients complete a trial or return for post-treatment follow-up, which could be especially challenging in light of the COVID-19 pandemic;

• selection of clinical endpoints that require prolonged periods of clinical observation or analysis of the resulting data;

• clinical sites deviating from trial protocol or subjects dropping out of a trial;

• addressing patient safety concerns that arise during the course of a trial;

• occurrence of SAEs associated with any product candidates we may develop, such as the TMAs experienced by patients in our SUNRISE trial of LB-001 in pediatric MMA patients as further described above;

• occurrence of SAEs in trials of the same class of agents conducted by other sponsors;

• adding a sufficient number of clinical trial sites; or

• manufacturing sufficient quantities of product candidate for use in clinical trials.

For example, in May 2022, we subsequently announced the FDA lifted the clinical hold placed on the LB-001 IND. In connection with the lifting of the clinical hold, we made amendments to the protocol of our Phase 1/2 SUNRISE clinical trial of LB-001, one of which increases the screening time for each patient, and as a result, will increase the overall time to completion of the trial.

We may experience numerous unforeseen events during, or as a result of, clinical trials that could delay or prevent our ability to receive marketing approval or commercialize our product candidates or significantly increase the cost of such trials, including:

• we may receive feedback from regulatory authorities that requires us to modify the design of our clinical trials, such as the amendments we made to the protocol of our Phase 1/2 SUNRISE clinical trial, described above, in connection with the lifting of the clinical hold;

• clinical trials of our product candidates may produce negative or inconclusive results, and we may decide, or regulators may require us, to conduct additional clinical trials or abandon development programs;

• difficulty in designing well-controlled clinical trials due to ethical considerations which may render it inappropriate to conduct a trial with a control arm that can be effectively compared to a treatment arm;

• difficulty in designing clinical trials and selecting endpoints for diseases that have not been well-studied and for which the natural history and course of the disease is poorly understood;

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• the number of patients required for clinical trials of our product candidates may be larger than we anticipate, enrollment in these clinical trials may be slower than we anticipate and/or participants may drop out of these clinical trials at a higher rate than we anticipate;

• our third-party contractors may fail to comply with regulatory requirements or meet their contractual obligations to us in a timely manner, or at all;

• we or any sponsor investigators might have to suspend or terminate clinical trials of our product candidates for various reasons, including non-compliance with regulatory requirements, a finding that our product candidates have undesirable side effects or other unexpected characteristics, or a finding that the participants are being exposed to unacceptable health risks;

• the cost of clinical trials of our product candidates may be greater than we anticipate, and we may not have funds to cover the costs;

• the supply or quality of our product candidates or other materials necessary to conduct clinical trials or receive regulatory approval of our product candidates may be insufficient or inadequate;

• regulators may revise the requirements for approving our product candidates, or such requirements may not be as we anticipate;

• changes in regulatory requirements and guidance that require amending or submitting new clinical protocols; and

• any of our collaborators that conduct clinical trials may face any of the above issues, and may conduct clinical trials in ways they view as advantageous to them but that are suboptimal for us.

If we are required to conduct additional clinical trials or other testing of our product candidates beyond those that we currently contemplate, if we are unable to successfully complete clinical trials of our product candidates or other testing, if the results of these trials or tests are not positive or are only modestly positive or if there are safety concerns, we may:

• incur unplanned costs;

• be delayed in obtaining marketing approval for our product candidates or not obtain marketing approval at all;

• obtain marketing approval in some countries and not in others;

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

• obtain marketing approval with labeling that includes significant use or distribution restrictions or safety warnings, including boxed warnings;

• be subject to changes in 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;

• be required to implement a REMS, or create a medication guide outlining the risks of such side effects for distribution to patients;

• have the product removed from the market after obtaining marketing approval;

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

• be sued; or

• experience damage to our reputation.

If we experience delays in the commencement or completion of our clinical trials, or if we terminate a clinical trial prior to completion, the commercial prospects of LB-001 or any other product candidate we develop could be harmed. In addition, any delays in our clinical trials could increase our costs, slow down the development and approval process and jeopardize our ability to commence product sales and generate revenues. Any of these occurrences may harm our business, financial condition and results of operations. In addition, many of the factors that cause, or lead to, a delay in the commencement or completion of clinical trials also ultimately may lead to the denial of regulatory approval of our product candidates.

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Product development costs also will increase if we or our collaborators experience delays in testing or marketing approvals. We do not know whether any clinical trials will begin as planned, will need to be restructured, or will be completed on schedule, or at all. Significant clinical trial delays also could shorten any periods during which we may have the exclusive right to commercialize any product candidates we may develop, could allow our competitors to bring products to market before we do and could impair our ability to successfully commercialize any product candidates we may develop, any of which may harm our business, financial condition, results of operations and prospects.

If we encounter difficulties enrolling patients in our clinical trials, our clinical development activities could be delayed or otherwise adversely affected.

We or our collaborators may not be able to initiate or continue clinical trials for any product candidates we identify or develop if we are unable to locate and enroll a sufficient number of eligible patients to participate in these trials as required by the FDA or analogous regulatory authorities outside the United States, or as needed to provide appropriate statistical power for a given trial. The timely completion of clinical trials in accordance with their protocols depends, among other things, on our ability to enroll a sufficient number of patients who remain in the trial until its conclusion. In addition, if patients are unwilling to participate in our clinical trials because of negative publicity from adverse events related to the biotechnology, gene therapy or genome editing fields, competitive clinical trials for similar patient populations, clinical trials in competing products or for other reasons, the timeline for recruiting patients, conducting trials and obtaining regulatory approval of any product candidates we may develop may be delayed.

We may encounter delays in enrolling, or be unable to enroll, a sufficient number of patients to complete any of our clinical trials, and even once enrolled we may be unable to retain a sufficient number of patients to complete any of our trials. Our ability to enroll patients may be significantly delayed by the evolving COVID-19 pandemic, and we do not know the extent and scope of such delays at this point. The enrollment of patients depends on many factors, including:

• perceived risks and benefits of the product candidate under investigation or the method by which such product candidate will be administered to patients, in particular from patients, their caregivers and physicians;

• the patient inclusion and exclusion criteria defined in the protocol;

• the size of the patient population required for analysis of the trial’s primary endpoints;

• severity of the disease under investigation;

• the proximity of patients to trial sites and any barriers on patients' ability to travel (such as travel restrictions patients may encounter due to the COVID-19 pandemic or global conflict or restrictions imposed by clinical trial sites relating to patients traveling to such site);

• the design of the trial;

• restrictions on our ability to conduct clinical trials, including full or partial clinical holds on ongoing or planned clinical trials;

• availability and efficacy of approved medications for the disease under investigation;

• our ability to recruit clinical trial investigators with the appropriate competencies and experience;

• clinicians’ and patients’ perceptions as to the potential advantages of the product candidate being studied in relation to other available therapies, including any potentially competitive product candidates being studied in clinical trials and new products that may be approved for the indications we are investigating;

• our ability to obtain and maintain patient consents;

• the risk that patients enrolled in clinical trials will drop out of the trials before completion;

• the risk that the patients we screen may not be eligible to advance to the clinical trial and that the size of the qualifying patient population;

• perceived risks and benefits of genome editing as a therapeutic approach;

• perceived risks and benefits of the product candidate based on any interim data or updates that we may announce;

• efforts to facilitate timely enrollment in clinical trials;

• patient referral practices of physicians; and

• ability to monitor patients adequately during and after treatment.

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In February 2022, we announced that the FDA has placed our IND for LB-001 on clinical hold after we reported an SAE in our Phase 1/2 SUNRISE clinical trial of LB-001 in pediatric MMA patients. In May 2022, we subsequently announced that the FDA lifted the hold. In connection with the lifting of the clinical hold, we made amendments to the SUNRISE clinical trial protocol, which increase the amount of screening time for each patient, intensify each patient’s monitoring regimen, and prolong hospitalization, among other changes. Although the FDA has lifted the clinical hold, the clinical hold and related SAEs and the amended protocol, including the increase in the screening time for each patient, may affect our ability to enroll patients.

Our ability to successfully initiate, enroll, and complete a clinical trial in any foreign country is subject to numerous risks unique to conducting business in foreign countries, including:

• difficulty in establishing and managing relationships with CROs and other vendors, as well as clinical trial sites and investigators;

• different standards for the conduct of clinical trials;

• different standard-of-care for patients with a particular disease;

• inability to locate qualified local consultants, physicians and partners;  

• potential burden of complying with a variety of foreign laws, medical standards and regulatory requirements, including the regulation of pharmaceutical and biotechnology products and treatment; and

• any heightened restrictions in foreign countries relating to the COVID-19 pandemic (such as travel restrictions).

In addition, our clinical trials will compete with other clinical trials for product candidates that are in the same therapeutic areas as our product candidates, and this competition will reduce the number and types of patients available to us, because some patients who might have opted to enroll in our trials may instead opt to enroll in a trial being conducted by one of our competitors. Since the number of qualified clinical investigators is limited, we expect to conduct some of our clinical trials at the same clinical trial sites that some of our competitors use, which will reduce the number of patients who are available for our clinical trials in such clinical trial sites.

Delays or failures in planned patient enrollment or retention may result in increased costs, program delays or both, which could have a harmful effect on our ability to develop LB-001 or any other product candidates, or could render further development impossible.

Even if we complete the necessary clinical trials, we cannot predict when, or if, we will obtain regulatory approval to commercialize a product candidate we may develop, and any such approval may be for a more narrow indication than we seek.

We cannot commercialize a product candidate until the appropriate regulatory authorities have reviewed and approved the product candidate. Even if any product candidates we may develop meet their safety and efficacy endpoints in clinical trials, the regulatory authorities may not complete their review processes in a timely manner, or we may not be able to obtain regulatory approval. Additional delays may result if an FDA advisory committee or other regulatory authority recommends non-approval or restrictions on approval. In addition, we may experience delays or rejections based upon additional government regulation from future legislation or administrative action, or changes in regulatory authority policy during the period of product development, clinical trials and the review process.

Regulatory authorities also may approve a product candidate for more limited indications than requested or they may impose significant limitations in the form of narrow indications, warnings or a REMS. These regulatory authorities may require precautions or contra-indications with respect to conditions of use, or they may grant approval subject to the performance of costly post-marketing clinical trials. In addition, regulatory authorities may not approve the labeling claims that are necessary or desirable for the successful commercialization of any product candidates we may develop. Any of the foregoing scenarios could materially harm the commercial prospects for any product candidates we may develop and materially adversely affect our business, financial condition, results of operations and prospects.

Adverse public perception of gene delivery technologies may negatively impact our potential products.

We are developing therapies using genome editing and gene therapy technologies. The clinical and commercial success of our potential products will depend in part on public acceptance of the use of genome editing and gene therapy for the prevention or treatment of human diseases. Public attitudes may be influenced by claims that gene editing and gene therapy are unsafe, unethical or immoral, and, consequently, our products may not gain the acceptance of the public or the medical community. Public perception of

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the cost, durability and potential harm from over-expression of genome editing and gene therapy may also affect public acceptance of our product candidates. Adverse public attitudes may adversely impact our ability to enroll clinical trials. Moreover, our success will depend upon physicians prescribing, and their patients being willing to receive, treatments that involve the use of product candidates we may develop in lieu of, or in addition to, existing treatments with which they are already familiar and for which greater clinical data may be available.

In addition, gene editing technology is subject to public debate and heightened regulatory scrutiny due to ethical concerns relating to the application of gene editing technology to human embryos or the human germline. The Alliance for Regenerative Medicine in Washington has issued principles setting forth a bioethical framework for the use of gene editing in therapeutic applications that states that the use of gene editing technologies in research that involved altering human embryos or human germline cells is currently not appropriate. Similarly, the National Institutes of Health, or NIH, has announced that it would not fund any use of gene editing technologies in human embryos, noting that there are multiple existing legislative and regulatory prohibitions against such work, including the Dickey-Wicker Amendment, which prohibits the use of appropriated funds for the creation of human embryos for research purposes or for research in which human embryos are destroyed. Laws in the United Kingdom prohibit genetically modified embryos from being implanted into women, but embryos can be altered in research labs under license from the Human Fertilisation and Embryology Authority. Research on embryos is more tightly controlled in many other European countries.

Although we do not use our technologies to edit human embryos or the human germline, such public debate about the use of gene editing technologies in human embryos and heightened regulatory scrutiny could prevent or delay our development of product candidates. More restrictive government regulations or negative public opinion would have a negative effect on our business or financial condition and may delay or impair our development and commercialization of product candidates or demand for any products we may develop.

Serious adverse events in our clinical trials, such as the two cases of TMA experienced by two patients in our Phase 1/2 SUNRISE clinical trial described above, or other clinical trials involving gene delivery technology, particularly AAV genetic medicines, such as candidates based on the same capsid serotypes as our product candidates, or occurring during use of 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, difficulties in enrolling patients, 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. For example, certain gene therapy trials led to several well-publicized adverse events, including cases of leukemia and death, some of which took years to present. Additionally, cases of TMA have been reported in association with other AAV genetic therapies. SAEs, such as these, whether in our clinical trials or other clinical trials involving gene therapy or genome editing products or our competitors’ products, even if not ultimately attributable to the relevant product candidates, and the resulting publicity could result in increased regulation and regulatory scrutiny, unfavorable public perception, potential regulatory delays in the testing or approval of our product candidates.

We may not be successful in our efforts to identify additional product candidates.

Part of our strategy involves identifying novel product candidates. Other than LB-001, all of our product candidates are still in the preclinical or research stage of development. The process by which we identify product candidates may fail to yield product candidates for clinical development for a number of reasons, including:

• we may not be able to generate sufficient preclinical data to support the initiation of clinical trials;

• we may not be able to assemble sufficient resources to acquire or discover additional product candidates;

• competitors may develop alternatives that render our potential product candidates obsolete or less attractive;

• potential product candidates we develop may nevertheless be covered by third parties’ patents or other exclusive rights;

• potential product candidates may, on further study, be shown to have harmful side effects, toxicities or other characteristics that indicate that they are unlikely to be products that will receive marketing approval and achieve market acceptance;

• negative results in the clinical trials for any of our product candidates could negatively impact other product candidates based on the same or similar technologies;

• potential product candidates may not be effective in or applicable to treating their targeted diseases;

• the market for a potential product candidate may change so that the continued development of that product candidate is no longer reasonable;

• a potential product candidate may not be commercially viable;

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• we may experience difficulties reaching a consensus with regulatory agencies on preclinical study design;

• a potential product candidate may not be capable of being produced in commercial quantities at an acceptable cost, or at all; and/or

• the regulatory pathway for a potential product candidate is too complex and difficult to navigate successfully or economically.

In addition, we may choose to focus our efforts and resources on a potential product candidate that ultimately proves to be unsuccessful. As a result, we may fail to capitalize on viable commercial products or profitable market opportunities, be required to forego or delay pursuit of opportunities with other product candidates or other diseases that may later prove to have greater commercial potential or relinquish valuable rights to such product candidates through collaboration, licensing or other royalty arrangements in cases in which it would have been advantageous for us to retain sole development and commercialization rights. If we are unable to identify additional suitable product candidates for clinical development, this could harm our business, financial condition, results of operations and prospects.

We and our contract manufacturers are subject to significant regulation with respect to manufacturing our product candidates. The manufacturing facilities on which we rely may not continue to meet regulatory requirements and have limited capacity.

We currently have relationships with a limited number of suppliers for the manufacturing of our product candidates. Each supplier may require licenses to manufacture such components if such processes are not owned by the supplier or in the public domain and we may be unable to transfer or sublicense the intellectual property rights we may have with respect to such activities.

All entities involved in the preparation of therapeutics for clinical trials or commercial sale, including our existing contract manufacturers for our product candidates, are subject to extensive regulation. Components of a finished therapeutic product approved for commercial sale or used in clinical trials must be manufactured in accordance with cGMP. These regulations govern manufacturing processes and procedures and the implementation and operation of quality systems to control and assure the quality of investigational products and products approved for sale. Poor control of production processes can lead to the introduction of adventitious agents or other contaminants, or to inadvertent changes in the properties or stability of our product candidates that can impact safety, efficacy and quality. We or our contract manufacturers must supply all necessary documentation in support of a BLA, or an application for regulatory approval in another jurisdiction, on a timely basis and must adhere to the FDA’s, or the applicable regulatory authority's, GLP, and cGMP regulations enforced by the FDA through its facilities inspection program. None of our contract manufacturers has produced a commercially-approved gene editing product, and some of our contract manufacturers have not produced a commercially-approved gene therapy product and therefore have not yet obtained the requisite FDA approvals to do so. The facilities and quality systems of some or all of our third-party contractors, as well as any facilities and quality systems we may have in the future, must pass a pre-approval inspection for compliance with the applicable regulations as a condition of regulatory approval of our product candidates or any of our other potential products. In addition, the regulatory authorities may, at any time, audit or inspect a manufacturing facility involved with the preparation of our product candidates or our other potential products or the associated quality systems for compliance with the regulations applicable to the activities being conducted. If these facilities do not pass a pre-approval plant inspection, FDA approval, or approval by regulatory authorities in other jurisdictions, will not be granted.

The regulatory authorities also may, at any time following approval of a product for sale, audit our manufacturing facilities or those of our third-party contractors. If any such inspection or audit identifies a failure to comply with applicable regulations or if a violation of our product specifications or applicable regulations occurs independent of such an inspection or audit, we or the relevant regulatory authority may require remedial measures that may be costly and/or time-consuming for us or a third party to implement and that may include the temporary or permanent suspension of a clinical trial or the temporary or permanent closure of a facility. Any such remedial measures imposed upon us or third parties with whom we contract could materially harm our business.

If we or any of our third-party manufacturers fail to maintain regulatory compliance, the FDA or regulatory authorities in other jurisdictions can impose regulatory sanctions including, among other things, refusal to approve a pending application for a new drug product or biologic product, or revocation of a pre-existing approval. As a result, our business, financial condition and results of operations may be materially harmed.

Additionally, if supply from one approved manufacturer is interrupted, there could be a significant disruption in commercial supply. In the United States, an alternative manufacturer would need to be qualified through a BLA supplement which could result in further delay, and there may be similar requirements in other jurisdictions. The regulatory agencies may also require additional studies if a new manufacturer is relied upon for commercial production. Switching manufacturers may involve substantial costs and is likely to result in a delay in our desired clinical and commercial timelines.

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These factors could cause the delay of clinical trials, regulatory submissions, required approvals or commercialization of our product candidates, cause us to incur higher costs and prevent us from commercializing our products successfully. Furthermore, if our suppliers fail to meet contractual requirements, and we are unable to secure one or more replacement suppliers capable of production at a substantially equivalent cost, our clinical trials may be delayed or we could lose potential revenue.

The regulatory approval processes of the FDA, the EMA and other regulatory authorities are lengthy, time consuming and inherently unpredictable, and if we are ultimately unable to obtain regulatory approval for our product candidates, our business will be substantially harmed.

The time required to obtain approval by the FDA, the EMA and other regulatory authorities is unpredictable, but typically takes many years following the commencement of clinical trials and depends upon numerous factors, including the type, complexity and novelty of the product candidates involved and the substantial discretion of the regulatory authorities. In addition, approval policies, regulations or the type and amount of clinical data necessary to gain approval may change during the course of a product candidate’s clinical development and may vary among jurisdictions. We have not obtained regulatory approval for any product candidate and it is possible that neither LB-001 nor any other product candidate will ever obtain regulatory approval. Neither we nor any future collaborator is permitted to market any of our product candidates in the United States, the EU or any other jurisdiction until we receive regulatory approval from the applicable regulatory authority. It is possible that the FDA, the EMA or other regulatory authorities may refuse to accept for substantive review any applications for marketing approval that we submit for our product candidates or may conclude after review of our data that our application is insufficient to obtain marketing approval of our product candidates.

Prior to obtaining approval to commercialize a product candidate in the United States or abroad, we or our collaborators must demonstrate with substantial evidence from well-controlled clinical trials, and to the satisfaction of the FDA, the EMA or other regulatory authorities, that such product candidates are safe and effective for their intended uses. The FDA, the EMA and other regulatory authorities have substantial discretion in the approval process and may refuse to accept any application or may decide that our data is insufficient for approval and require additional preclinical, clinical or other studies. Results from preclinical studies and clinical trials can be interpreted in different ways. Even if we believe the preclinical or clinical data for our product candidates are promising, such data may not be sufficient to support approval by the FDA, the EMA and other regulatory authorities. The FDA, the EMA or other regulatory authorities may also require us to conduct additional preclinical studies or clinical trials for our product candidates either prior to or post-approval, or it may object to elements of our clinical development program. Depending on the extent of these or any other studies required by regulatory authorities, approval of any marketing application that we submit may be delayed by several years, or may require us to expend significantly more resources than we have available.

Of the large number of potential products in development, only a small percentage successfully completes the FDA, the EMA or regulatory approval processes in other jurisdictions and are commercialized. The lengthy approval process as well as the unpredictability of future clinical trial results may result in our failing to obtain regulatory approval to market our product candidates, which would significantly harm our business, results of operations and prospects.

We are heavily dependent on the success of LB-001, our lead product candidate, and if LB-001 does not receive regulatory approval in the United States or other jurisdictions, or is not successfully commercialized, our business will be harmed.

To date, we have invested a significant portion of our efforts and financial resources in the development of LB-001. Our future success and ability to generate product revenue is substantially dependent on our ability to successfully develop, obtain regulatory approval for and successfully commercialize LB-001 and/or other product candidates. We currently have no products that are approved for commercial sale and may never be able to develop marketable products.

If we receive a positive data readout from our Phase 1/2 SUNRISE clinical trial that supports advancing LB-001 to Phase 3, we expect that a substantial portion of our efforts and expenditures over the subsequent years will be devoted to LB-001. Accordingly, our business currently depends heavily on the successful development, regulatory approval and commercialization of LB-001, which may never occur. We cannot be certain that LB-001 will be successful in clinical trials, receive regulatory approval or be successfully commercialized even if we receive regulatory approval. Even if we receive approval to market LB-001 from the FDA or other regulatory authorities, we cannot be certain that our product candidate will be successfully commercialized, widely accepted in the marketplace or more effective than other commercially available alternatives. Additionally, the research, testing, manufacturing, labeling, approval, sale, marketing and distribution of genetic medicine products are and will remain subject to extensive regulation by the FDA, the EMA and regulatory authorities in other jurisdictions. We are not permitted to market LB-001 in the United States until it receives approval of a BLA from the FDA, or in any foreign jurisdictions until it receives the requisite approval from such jurisdictions. We have not submitted a BLA to the FDA or comparable applications to other regulatory authorities and do not expect to be in a position to do so for the foreseeable future.

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LB-001 is our lead product candidate, and because other product candidates may be based on similar technology, if LB-001 shows unexpected adverse events or a lack of efficacy in the indications we intend to treat, or if we experience other regulatory or developmental issues, our development plans and business could be significantly harmed. Further, competitors may be developing products with similar technology and may experience problems with their products that could identify problems that would potentially harm our business. For example, as previously disclosed, two patients in our Phase 1/2 SUNRISE clinical trial of LB-001 in pediatric patients experienced cases of TMA.

In order to market any products in any particular jurisdiction, we must establish and comply with numerous and varying regulatory requirements on a country-by-country basis regarding safety and efficacy. Approval by the FDA in the United States does not ensure approval by regulatory authorities in other countries or jurisdictions. However, the failure to obtain approval in one jurisdiction may negatively impact our ability to obtain approval elsewhere. In addition, clinical trials conducted in one country may not be accepted by regulatory authorities in other countries, and regulatory approval in one country does not guarantee regulatory approval in any other country.

Approval processes vary among jurisdictions and can involve additional product testing and validation and additional administrative review periods. Seeking foreign regulatory approval could result in difficulties and increased costs for us and require additional preclinical studies or clinical trials which could be costly and time consuming. Regulatory requirements can vary widely from country to country and could delay or prevent the introduction of our products in those countries. We do not have any product candidates approved for sale in any jurisdiction, including in international markets, and we do not have experience in obtaining regulatory approval in international markets. If we fail to comply with regulatory requirements in international markets or to obtain and maintain required approvals, or if regulatory approvals in international markets are delayed, our target market will be reduced and our ability to realize the full market potential of any product we develop will be unrealized.

We may expend our limited resources to pursue a particular product candidate or indication and fail to capitalize on product candidates or indications that may be more profitable or for which there is a greater likelihood of success.

Because we have limited financial and managerial resources, we focus on research programs and product candidates that we identify for specific indications. As a result, we may forego or delay pursuit of opportunities with other product candidates or for other indications that later prove to have greater commercial potential. Our resource allocation decisions may cause us to fail to timely capitalize on viable commercial products or profitable market opportunities. Our spending on current and future research and development programs and product candidates for specific indications may not yield any commercially viable products. If we do not accurately evaluate the commercial potential or target market for a particular product candidate, we may relinquish valuable rights to that product candidate through collaboration, licensing or other royalty arrangements in cases in which it would have been more advantageous for us to retain sole development and commercialization rights to such product candidate. Any such event could harm our business, financial condition, results of operations and prospects.

Genomic medicines are novel, and any product candidates we develop may be complex and difficult to manufacture. We could experience production problems that result in delays in our development or commercialization programs, limit the supply of our products or otherwise harm our business.

Due to the novel nature of our platform, any product candidates we may develop will likely require processing and manufacturing steps that are more complex than those required for most chemical pharmaceuticals. Moreover, unlike chemical pharmaceuticals, the physical and chemical properties of a biologic such as the product candidates we are developing generally cannot be fully characterized. As a result, assays of the finished product may not be sufficient to ensure that the product will perform in the intended manner.

Problems with the manufacturing process, even minor deviations from the normal process, could result in product defects or manufacturing failures that result in lot failures, product recalls, product liability claims or insufficient inventory. If we successfully develop product candidates, we may encounter problems achieving adequate quantities and quality of clinical-grade materials that meet FDA, EMA or other comparable applicable foreign standards or specifications with consistent and acceptable production yields and costs.

In addition, the FDA, the EMA and other regulatory authorities may require us to submit samples of any lot of any approved product together with the protocols showing the results of applicable tests at any time. Under some circumstances, the FDA, the EMA or other regulatory authorities may require that we not distribute a lot until the agency authorizes its release. Slight unplanned deviations in the manufacturing process, including those affecting quality attributes and stability, may result in unacceptable changes in the product that could result in lot failures or product recalls. Lot failures or product recalls could cause us to delay clinical trials or product launches, which could be costly to us and otherwise harm our business, financial condition, results of operations and prospects.

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We or our third-party contractors also may encounter problems hiring and retaining the experienced scientific, quality control and manufacturing personnel needed to manage and/or provide the necessary oversight of our manufacturing process, which could result in delays in our production or difficulties in maintaining compliance with applicable regulatory requirements.

Given the nature of biologics manufacturing, there is a risk of contamination during manufacturing. Any contamination could materially harm our ability to produce product candidates on schedule and could harm our results of operations and cause reputational damage. Some of the raw materials that we anticipate will be required in our manufacturing process are derived from biologic sources. Such raw materials are difficult to procure and may be subject to contamination or recall. A material shortage, contamination, recall or restriction on the use of biologically derived substances in the manufacture of any product candidates we may develop could adversely impact or disrupt the commercial manufacturing or the production of clinical material, which could materially harm our development timelines and our business, financial condition, results of operations and prospects.

Any problems in our manufacturing process or the facilities with which we contract could make us a less attractive collaborator for potential partners, including larger pharmaceutical companies and academic research institutions, which could limit our access to additional attractive development programs. Problems in third-party manufacturing process or facilities also could restrict our ability to meet market demand for any products we develop and commercialize.

The manufacturing processes for our product candidates are complex and changes in our manufacturing processes may cause delays in our clinical development and commercialization plans.

The manufacturing processes for our product candidates, including viral vector manufacturing, are complex. Some of the factors that contribute to the complexity of this process include:

• the use of complex biological raw materials, including plasmid DNA, and cell banks, that need to comply with stringent regulatory requirements;

• the use of complex manufacturing methods, such as transfection of suspension mammalian cell culture in large bioreactors (transfection is the process by which plasmid DNA enters the cells), and purification of intact viral particles;

• the need to produce high-concentration, high purity drug product; and

• the challenges associated with scale-up, batch-to-batch consistency and product characterization, including measurement of potency and quality.

We explore improvements to the manufacturing process for our product candidates on a continual basis. Changes to the manufacturing process may occur in connection with efforts to optimize our process in preparation for the potential advancement of our clinical trial or commercialization of our product candidates. Changes to this process could induce a change in the purity or potency of a product candidate. In some circumstances, changes in the manufacturing process may require us to perform additional comparability studies or to collect additional data from patients prior to undertaking additional clinical studies, or to perform a bridging study.  The FDA could also require us to file a new IND with respect to such changes in our manufacturing process. These requirements may lead to delays in our clinical development and commercialization plans.

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Our employees and independent contractors, including investigators, CROs, CMOs, consultants, vendors and any third parties we may engage in connection with development and commercialization, may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements, which could harm our business.

Misconduct by our employees and independent contractors, including investigators, CROs or CMOs, consultants, vendors, and any third parties we may engage in connection with development and commercialization, could include intentional, reckless or negligent conduct or unauthorized activities that violate: (i) the laws and regulations of the FDA, EMA rules and regulations and other similar regulatory requirements, including those laws that require the reporting of true, complete and accurate information to such authorities; (ii) GXP; (iii) data privacy, security, fraud and abuse and other healthcare laws and regulations; or (iv) laws that require the reporting of true, complete and accurate financial information and data. Activities subject to these laws could also involve the improper use or misrepresentation of information obtained in the course of clinical trials, creation of fraudulent data in preclinical studies or clinical trials or illegal misappropriation of drug product, which could result in regulatory sanctions and cause serious harm to our reputation. It is not always possible to identify and deter misconduct by employees and other third parties, and the precautions we take to detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to comply with such laws or regulations. Additionally, we are subject to the risk that a person or government could allege such fraud or other misconduct, even if none occurred. If any such actions are instituted against us, and we are not successful in defending ourselves or asserting our rights, those actions could have a significant impact on our business and results of operations, including the imposition of significant civil, criminal and administrative penalties, damages, monetary fines, disgorgements, possible exclusion from participation in Medicare, Medicaid, other U.S. federal healthcare programs or healthcare programs in other jurisdictions, individual imprisonment, additional integrity oversight and reporting obligations, other sanctions, contractual damages, reputational harm, diminished profits and future earnings, and curtailment of our operations.

Our business and operations would suffer in the event of system failures.

Our computer systems, as well as those of our CROs, CMOs, suppliers and other contractors and consultants, are vulnerable to damage from computer viruses, unauthorized access, natural disasters, terrorism, war and telecommunication and electrical failures. If such an event were to occur and cause interruptions in our operations, it could result in a material disruption of our product candidate development programs. For example, the loss of preclinical study or clinical trial data from completed, ongoing or planned studies or trials could result in delays in our regulatory approval efforts and significantly increase our costs to recover or reproduce the data. To the extent that any disruption or security breach were to result in a loss of or damage to our data or applications, or inappropriate disclosure of personal, confidential or proprietary information, we could incur liability and the further development of LB-001 or any other product candidate could be delayed.

In the ordinary course of our business, we collect and store sensitive data, including intellectual property, clinical trial data, proprietary business information, personal data and personally identifiable information of our clinical trial subjects and employees, in our data centers and on our networks. The secure processing, maintenance and transmission of this information is critical to our operations. Despite our security measures and those of our third-party contractors, our information technology and infrastructure may be vulnerable to attacks by hackers or internal bad actors, or breached due to employee error, a technical vulnerability, malfeasance or other disruptions. Although, to our knowledge, we have not experienced any such material security breach to date, any such breach could compromise our networks and the information stored there could be accessed, publicly disclosed, lost or stolen. Any such access, disclosure or other loss of information could result in legal claims or proceedings, liability under laws that protect the privacy of personal information, significant regulatory penalties, and such an event could disrupt our operations, damage our reputation, and cause a loss of confidence in us and our ability to conduct clinical trials, which could adversely affect our reputation and delay our clinical development of our product candidates.

Interim “top-line” and preliminary data from our clinical trials that we announce or publish from time to time may change as more patient data become available and are subject to audit and verification procedures that could result in material changes in the final data.

From time to time, we may publish interim “top-line” or preliminary data from our clinical trials. Interim data from clinical trials that we may complete are subject to the risk that one or more of the clinical outcomes may materially change as patient enrollment continues and more patient data become available. Preliminary or “top-line” data also remain subject to audit and verification procedures that may result in the final data being materially different from the preliminary data we previously published. As a result, interim and preliminary data should be viewed with caution until the final data are available. Adverse differences between preliminary or interim data and final data could significantly harm our business prospects.

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Risks Related to Our Dependence on Third Parties

We currently contract with third parties for the manufacture and testing of materials. This reliance on third parties increases the risk that we will not have sufficient quantities of such materials, product candidates or any medicines that we may develop and commercialize, or that such supply will not be available to us at an acceptable cost, which could delay, prevent or impair our development or commercialization efforts.

We contract with third parties, including those with manufacturing facilities, to supply some of our discovery and preclinical research. We currently also rely on third-party manufacturers and other vendors for the manufacture and testing of our materials for clinical trials. We expect to continue to do so for commercial supply of any product candidates that we may develop and for which we or our collaborators obtain marketing approval. We do not have a long-term supply agreement with any of the third-party manufacturers, and we purchase our required supply on a purchase order basis.

We may be unable to establish any agreements with third-party manufacturers for clinical and commercial supply manufacturing, or to do so on acceptable terms. Even if we are able to establish agreements with third-party manufacturers, reliance on third-party manufacturers entails additional risks, including such any third-party manufacturer:

• experiencing staffing difficulties;

• undergoing changes in priorities or becoming financially distressed;

• forming relationships with other entities, some of which may be our competitors;

• experiencing unanticipated events that cause negative consequences in such third party's ability to fulfill its obligations to us, including due to the COVID-19 pandemic or natural disasters requiring delay or cessation of operations;

• experiencing business interruptions resulting from geo-political events, including war and terrorism, or natural disasters, including floods and fires;

• potential unauthorized disclosure or misappropriation of our intellectual property by CMOs, which may allow our potential competitors to access and exploit our proprietary technology and reduce our trade secret protection;

• having limited capacity for manufacturing slots;

• the possible breach of the manufacturing agreement by the third party;

• the possible termination or nonrenewal of the agreement by the third party at a time that is costly or inconvenient for us; and

• reliance on the third party for regulatory compliance, quality assurance, compliance with specifications, maintaining proper chain of custody, safety, and pharmacovigilance and related reporting.

Third-party manufacturers may not be able to comply with cGMP regulations or similar regulatory requirements in or outside the United States. Our failure, or the failure of our third-party manufacturers, to comply with applicable regulations could result in sanctions being imposed on us, including fines, injunctions, civil penalties, delays, suspension or withdrawal of approvals, license revocations, seizures or recalls of product candidates or medicines, operating restrictions, and criminal prosecutions, any of which could harm our business, financial condition, results of operations and prospects.

Any medicines that we may develop may compete with other product candidates and products for access to manufacturing facilities. There are a limited number of manufacturers that operate under cGMP regulations and that might be capable of manufacturing for us.

Any performance failure on the part of our existing or future manufacturers could delay clinical development or marketing approval. We do not currently have arrangements in place for redundant supply of complex biological raw materials, drug substance or drug product. If any one of our current contract manufacturers cannot perform as agreed, we may be required to replace that manufacturer. Although we believe that there are a few potential alternative manufacturers who could manufacture any product candidates we may develop, we may incur added costs and delays in identifying and qualifying any such replacement.

Our current and anticipated future dependence upon others for the manufacture of any product candidates we may develop or medicines may adversely affect our future profit margins and our ability to commercialize any medicines that receive marketing approval on a timely and competitive basis.

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We intend to rely on third parties to conduct, supervise and monitor our clinical trials. If those third parties do not successfully carry out their contractual duties, or if they perform in an unsatisfactory manner, it may harm our business.

We currently rely, and expect to continue to rely, on third parties to conduct some aspects of our research and preclinical testing. We currently rely, and expect to continue to rely, on CROs and other vendors, as well as clinical trial sites and investigators to ensure the proper and timely conduct of our clinical trials, and we expect to have limited influence over their actual performance. Any of these third parties may terminate their engagements with us at any time. If we need to enter into alternative arrangements, it would delay our product development activities.

We intend to rely upon CROs and other vendors to draw, distribute, store and test clinical samples, the data from which will be used to manage ongoing care of participants in our clinical studies and to monitor clinical safety and efficacy endpoints, and to store, manage and analyze clinical data for our programs. We also intend to rely on CROs and other vendors for the execution of future preclinical studies. Our reliance on CROs for clinical development activities limits our control over these activities, but we remain 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 the CROs does not relieve us of our regulatory responsibilities. We also expect to rely on other third parties to store and distribute drug supplies for our clinical trials. Any performance failure on the part of our CROs, distributors and other vendors could delay clinical development or marketing approval of any product candidates we may develop or commercialization of our medicines, producing additional losses and depriving us of potential product revenue.

We and our CROs and other vendors will be required to comply with the GLPs and GCPs, which are regulations and guidelines enforced by the FDA and are also required by the Competent Authorities of the Member States of the European Economic Area, or the EEA, and comparable foreign regulatory authorities in the form of International Conference on Harmonization guidelines for any of our product candidates that are in preclinical and clinical development. The regulatory authorities enforce GCPs through periodic inspections of trial sponsors, principal investigators and clinical trial sites. If we or our CROs fail to comply with GCPs, the clinical data generated in our clinical trials may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us to perform additional clinical trials before approving our marketing applications. We cannot be sure that upon inspection by a given regulatory authority, such regulatory authority will determine that any of our clinical trials comply with GCP regulations. In addition, our clinical trials must be conducted with product produced under cGMP regulations. Accordingly, if our CROs fail to comply with these regulations, we may be required to repeat clinical trials, which would delay the regulatory approval process. We also are required to register ongoing clinical trials and post the results of completed clinical trials on a government-sponsored database, clinicaltrials.gov, within certain timeframes. Failure to do so can result in fines, adverse publicity, and civil and criminal sanctions.

Our CROs and other vendors are not our employees, and we will not control whether or not they devote sufficient time and resources to our future clinical and preclinical programs. These CROs and other vendors may also have relationships with other commercial entities, including our competitors, for whom they may also be conducting clinical trials, or other product development activities which could harm our competitive position. We face the risk of potential unauthorized disclosure or misappropriation of our intellectual property by CROs and other vendors, which may reduce our trade secret protection and allow our potential competitors to access and exploit our proprietary technology. If our CROs and other vendors do not successfully carry out their contractual duties or obligations, fail to meet expected deadlines, or if the quality or accuracy of the clinical data they obtain is compromised due to the failure to adhere to our clinical protocols or regulatory requirements or for any other reasons, our clinical trials may be extended, delayed or terminated, and we may not be able to obtain regulatory approval for, or successfully commercialize any product candidate that we develop. As a result, our financial results and the commercial prospects for any product candidate that we develop would be harmed, our costs could increase, and our ability to generate revenues could be delayed.

If our relationship with any CROs or other vendors terminate, we may not be able to enter into arrangements with alternative CROs or other vendors or do so on commercially reasonable terms. Switching or adding additional CROs or other vendors involves substantial cost and requires management time and focus. In addition, there is a natural transition period when a new CRO commences work. As a result, delays occur, which can materially impact our ability to meet our desired clinical development timelines. Though we intend to carefully manage our relationships with our CROs and other vendors, there can be no assurance that we will not encounter challenges or delays in the future or that these delays or challenges will not harm on our business, financial condition, results of operations and prospects.

We have and may in the future enter into collaborations with third parties for the research, development and commercialization of certain of the product candidates or technologies we may develop. If any such collaborations are not successful, we may not be able to capitalize on the market potential of those product candidates and technologies.

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We may seek collaborative relationships for the development and commercialization of any product candidate or technology we may develop. For example, in January 2020, we announced a research collaboration with Takeda, in April 2021, we entered into an Exclusive Research Collaboration. License and Option Agreement with CANbridge, and in April 2021, we also entered into a research collaboration with Daiichi. Future collaborators could include large and mid-size pharmaceutical companies, regional and national pharmaceutical companies and biotechnology companies. Under any such arrangements with third parties, we will likely have limited control over the amount and timing of resources that our collaborators dedicate to the development or commercialization of any product candidates we may seek to develop with them. Our ability to generate revenues from these arrangements will depend partly on our collaborators’ abilities to successfully perform the functions assigned to them in these arrangements. We cannot predict the success of any collaboration that we enter into.

Failure to obtain such collaborative relationships could impair the potential for any product candidate or technology we may develop. We also may enter into collaborative relationships to provide funding to support our other research and development programs. The process of establishing and maintaining collaborative relationships is difficult, time-consuming and involves significant uncertainty, such as:

• a collaboration partner may have significant discretion in determining the efforts and resources that they will apply to these collaborations;

• a collaboration partner may shift its priorities and resources away from our product candidates or technologies due to a change in business strategies, or a merger, acquisition, sale or downsizing;

• a collaboration partner may delay preclinical studies or clinical trials, provide insufficient funding for a nonclinical program or clinical trial, stop a clinical trial, nonclinical study or abandon a product candidate or technology, repeat or conduct new preclinical studies or clinical trials, or require a new formulation of a product candidate or technology for testing;

• a collaboration partner may seek to renegotiate or terminate its relationship with us due to unsatisfactory clinical or nonclinical results, manufacturing issues, a change in business strategy, a change of control or other reasons;

• a collaboration partner may cease development in therapeutic areas that are the subject of our strategic collaboration;

• a collaboration partner may not devote sufficient capital or resources towards our product candidates or technologies;

• a collaboration partner may change the success criteria for a product candidate or technology thereby delaying or ceasing development;

• a collaboration partner may not properly obtain, maintain, enforce or defend our intellectual property or proprietary rights or may use our proprietary information in such a way as to invite litigation that could jeopardize or invalidate our proprietary information or expose us to potential litigation;

• a significant delay in initiation of certain development activities by a collaboration partner will also delay payment of milestones tied to such activities, thereby impacting our ability to fund our own activities;

• a collaboration partner could develop a product or technology that competes, either directly or indirectly, with our product candidate or technologies;

• a collaboration partner with commercialization obligations may not commit sufficient financial or human resources to the marketing, distribution or sale of a product;

• a collaboration partner with manufacturing responsibilities may encounter regulatory, resource or quality issues and be unable to meet demand requirements;

• collaborations involving a co-development relationship increases the risk to successful completion of a development program because it requires coordination between multiple sponsors on a number of topics, including regulatory and development strategy and safety reporting;

• a collaboration partner may terminate a strategic alliance and, if terminated, may result in a need for additional capital to pursue further development or commercialization of the applicable product candidates or technologies;

• a dispute may arise between us and a partner concerning the research, development or commercialization of a product candidate resulting in a delay in milestones, royalty payments or termination of an alliance and possibly resulting in costly litigation or arbitration which may divert management attention and resources;

• a partner may use our products or technology in such a way as to invite litigation from a third party;

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• we may lose certain valuable rights under circumstances identified in our collaborations, including if we undergo a change of control;

• collaboration agreements may not lead to development or commercialization of product candidates or technologies in the most efficient manner, or at all; and

• if a collaborator of ours were to be involved in a business combination, the continued pursuit and emphasis on our product development or commercialization program under such collaboration could be delayed, diminished or terminated.

If our collaborations do not result in the successful development and commercialization of products, or if one of our collaborators terminates its agreement with us, we may not receive any future research funding or milestone or royalty payments under the collaboration. If we do not receive the funding we expect under these agreements, our development of product candidates or technologies could be delayed and we may need additional resources to continue development. In addition, if one of our collaborators terminates its agreement with us, we may find it more difficult to find a suitable replacement collaborator or attract new collaborators, and our development programs may be delayed or the perception of us in the business and financial communities could be adversely affected. All of the risks relating to product development, regulatory approval, and commercialization described herein also apply to the activities of our collaborators.

We may in the future decide to collaborate with pharmaceutical and biotechnology companies for the development and potential commercialization of any product candidates or technologies we may develop. These relationships, or those like them, may require us to incur non-recurring and other charges, increase our near- and long-term expenditures, issue securities that dilute our existing stockholders or disrupt our management and business. In addition, we could face significant competition in seeking appropriate collaborators, and the negotiation process is time-consuming and complex. Our ability to reach a definitive collaboration agreement will depend, among other things, upon our assessment of the collaborator’s resources and expertise, the terms and conditions of the proposed collaboration and the proposed collaborator’s evaluation of several factors. If we license rights to any product candidates or technologies we or our collaborators may develop, we may not be able to realize the benefit of such transactions if we are unable to successfully integrate them with our existing operations and company culture.

If any collaborator fails to fulfill its responsibilities in a timely manner, or at all, our research, clinical development, manufacturing or commercialization efforts related to that collaboration could be delayed or terminated, or it may be necessary for us to assume responsibility for expenses or activities that would otherwise have been the responsibility of our collaborator. If we are unable to successfully transition terminated collaborative agreements, we may have to delay or discontinue further development of one or more of our product candidates or technologies, undertake development and commercialization activities at our own expense or find alternative sources of capital. Moreover, any collaborative partners we enter into agreements with in the future may shift their priorities and resources away from our product candidates or technologies or seek to renegotiate or terminate their relationships with us.

If conflicts arise between us and our collaborators or strategic partners, these parties may act in a manner adverse to us and could limit our ability to implement our strategies.

If conflicts arise between our collaborators or strategic partners, such as CANbridge, Daiichi, Stanford, NIH, CMRI, the University of Texas or Oregon Health & Science University, or OHSU, and us, the other party may act in a manner adverse to us and could limit our ability to implement our strategies. Some of our collaborators and strategic partners are conducting multiple product development efforts within each area that is the subject of the collaboration with us. Our collaborators or strategic partners, however, may develop, either alone or with others, products in related fields that are competitive with the products or potential products that are the subject of these collaborations. Competing products, either developed by the collaborators or strategic partners or to which the collaborators or strategic partners have rights, may result in the withdrawal of partner support for our product candidates or technologies.

Some of our collaborators or strategic partners could also become our competitors in the future. Our collaborators or strategic partners could develop competing products, preclude us from entering into collaborations with their competitors, fail to obtain timely regulatory approvals, terminate their agreements with us prematurely, or fail to devote sufficient resources to the development and commercialization of products. Any of these developments could harm our product development efforts.

Our collaborators or strategic partners may decide to adopt alternative technologies or may be unable to develop commercially viable products with our technology, which would negatively impact our revenues and our strategy to develop these products.

Our collaborators or strategic partners may adopt alternative technologies, which could decrease the marketability of our GeneRide or sAAVy technologies. Additionally, because our current or future collaborators or strategic partners are likely to be working on more than one development project, they could choose to shift their resources to projects other than those they are working on with us. If

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they do so, this would delay our ability to test our technology and would delay or terminate the development of potential products based on our GeneRide or sAAVy technologies. Further, our collaborators and strategic partners may elect not to develop products arising out of our collaborative and strategic partnering arrangements or to devote sufficient resources to the development, manufacturing, marketing or sale of these products. The failure to develop and commercialize a product candidate pursuant to our agreements with our current or future collaborators would prevent us from receiving future milestone and royalty payments which would negatively impact our revenues.

If we fail to comply with obligations in agreements under which we in-license or acquire development or commercialization rights to products, technology or data from third parties, including our agreements with Stanford, the University of Texas and NIH, we could lose such rights that are important to our business, and we may be unable to continue our development or commercialization programs as a result, which would be harmful to our business.

We are a party to agreements with the Board of Trustees of the Leland Stanford Junior University, or Stanford, and the Board of Regents of the University of Texas System, or the University of Texas, to license our core technology, and we are party to a license agreement with the NIH for development and commercialization rights to certain capsids including the transgene for LB-001. We are also party to an agreement with CMRI for development and commercialization rights to certain capsids including the first capsid produced from the sAAVy platform, sL65. We may enter into additional agreements with other parties in the future that impose diligence, development and commercialization timelines, milestone payments, royalties, insurance and other obligations on us.

In exchange for the rights granted to us pursuant to our agreements with Stanford, the University of Texas, the NIH and CMRI, we are obligated to make payments upon the achievement of certain milestone events and to pay annual maintenance fees and specified royalties. If we fail to comply with our obligations under these agreements or any future license agreements, our counterparties may have the right to terminate these agreements, in which event we might not be able to develop, manufacture or market any product candidate that is covered by these agreements, which 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 reinstated agreements with less favorable terms, or cause us to lose our rights under these agreements, including our rights to important intellectual property or technology.

Our business has been adversely affected by the ongoing coronavirus pandemic, and we expect it to continue to have a negative impact on our business.

In March 2020, the World Health Organization characterized the COVID-19 outbreak a “pandemic.” The pandemic and government measures taken in response have since had a significant impact, both direct and indirect, on businesses and commerce, as worker shortages have occurred; supply chains have been disrupted; facilities and production have been suspended; and demand for certain goods and services, such as medical services and supplies, has spiked, while demand for other goods and services, such as travel, has fallen. Although the FDA has approved certain therapies and vaccines, the willingness of individuals to get vaccinated and the overall efficacy of the vaccines once widely administered, especially as new strains of COVID-19 have been discovered, and the level of resistance these new strains have to the existing vaccines remains unknown.  

The Company is following federal, state and local requirements and guidelines with respect to COVID-19 and has allowed its employees to work on-premises in accordance with those requirements and guidelines.

Our research, development and manufacturing activities are dependent on our ability to continue our work on premises at our laboratory. We also rely on third parties, such as CROs and CMOs, located in areas that are affected by the COVID-19 pandemic for certain research, development and manufacturing activities. Many of these third parties also limited their staff from working on premises as part of their response to the COVID-19 pandemic. The COVID-19 pandemic may have a significant negative effect on our business and future results due to a variety of factors, including the health of our employees, our ability to maintain operations, the ability of our third-party vendors, suppliers and collaborators to continue operations, any further government and/or public actions taken in response to the pandemic and ultimately the length of the pandemic.  The Company is aware that certain of its third-party vendors are being affected by import/export and other restrictions due to COVID-19, which may have an impact on certain of the Company’s research, development and manufacturing activities. The full extent to which the COVID-19 pandemic, including the development of new variants, could directly or indirectly impact the Company’s business, results of operations and financial position will depend on future developments that are uncertain and cannot be accurately predicted.

Infections and deaths related to COVID-19, including as a result of new and more resistant strains, have significantly disrupted the United States’ healthcare and healthcare regulatory systems. Such disruptions have diverted healthcare resources away from regular activities at a number of institutions where clinical trials are normally conducted. Depending on the duration and severity of the pandemic, our efforts to engage with potential trial sites in start-up and other activities for our planned clinical trials or other studies

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may be adversely affected. In addition, other known and unknown factors caused by COVID-19 could materially delay other aspects of our clinical trials, including our ability to recruit and retain patients and principal investigators and site staff.

After a prolonged period of disruptions to inspection activities due to the COVID-19 pandemic, the FDA announced the resumption of routine domestic surveillance inspections on February 7, 2022. Depending on the course of the pandemic in various geographic areas, the FDA may not be able to maintain its planned restoration of standard operations and delays or setbacks are possible in the future. Additionally, regulatory authorities outside the United States may adopt similar restrictions or other policy measures in response to the COVID-19 pandemic and may experience delays in their regulatory activities.

Our activities will continue to require a significant expenditure of capital resources. The COVID-19 pandemic, including as a result of new and more resistant strains, as well as other pandemics or public health emergencies, could lead to disruptions of global financial markets and, if so, could impact our ability to access capital. An extended period of disruption in the economy and capital markets could significantly affect our ability to raise additional capital on a timely basis, which would significantly disrupt our programs and also require us to reevaluate our corporate strategy.

Risks Related to Our Intellectual Property

If we are unable to obtain and maintain sufficient patent protection for any product candidates and for our technologies, or if the scope of the patent protection obtained is not sufficiently broad, our competitors could develop and commercialize products and technology similar or identical to ours, and our ability to successfully commercialize our product candidates and our technologies may be adversely affected.

Our success depends in large part on our ability to obtain and maintain patent protection in the United States and other countries with respect to our platform technologies and any proprietary product candidates and technologies we develop. We seek to protect our proprietary position by in-licensing intellectual property relating to our platform technologies and filing patent applications in the United States and abroad related to our technologies and product candidates that are important to our business. If we or our licensors are unable to obtain or maintain patent protection with respect to our AAV capsid technology and genome editing platform technology and any proprietary products and technologies we develop, our business, financial condition, results of operations and prospects could be materially harmed. Additionally, if we do not adequately protect our intellectual property, our competitors may be able to erode or negate any competitive advantage we may have, which could harm our business and ability to achieve profitability.

The scope of patent protection in the United States and in other countries is uncertain. Pending and future patent applications may not result in issued patents that protect our business, in whole or in part, or that effectively prevent others from commercializing competitive products. Changes in either the patent laws or their interpretation in the United States and other countries may diminish our ability to protect our inventions, obtain, maintain, and enforce our intellectual property rights and, more generally, could affect the value of our intellectual property or narrow the scope of our owned and licensed patents. With respect to both in-licensed and owned intellectual property, we cannot predict whether the patent applications we and our licensors are currently pursuing will issue as patents in any particular jurisdiction or whether the claims of any issued patents will provide sufficient protection from competitors. In addition, the laws of foreign countries may not protect our rights to the same extent or in the same manner as the laws of the United States.

The patent prosecution process is expensive, time-consuming, and complex. We may not be able to file, prosecute, maintain, enforce, or license all necessary or desirable patent applications at a reasonable cost or in a timely manner. It is also possible that we, or any future partners, collaborators or licensees, may fail to identify patentable aspects of inventions made in the course of development and commercialization activities in time to obtain patent protection. Therefore, we may miss potential opportunities to strengthen our patent position. Additionally, although we enter into agreements containing non-disclosure and confidentiality obligations with parties who have access to confidential or patentable aspects of our research and development output, such as our employees, collaborators, contract manufacturers, consultants, and other third parties, any of these parties may breach the agreements and disclose such output before a patent application is filed, thereby jeopardizing our ability to seek patent protection.

It is possible that defects of form in the preparation or filing of our patents or patent applications may exist, or may arise in the future, for example with respect to proper priority claims, inventorship, claim scope or patent term adjustments. If we or our partners, collaborators, licensees, or licensors, whether current or future, fail to establish, maintain, or protect such patents and other intellectual property rights, such rights may be reduced or eliminated. If our partners, collaborators, licensees, or licensors are not fully cooperative or disagree with us as to the prosecution, maintenance, or enforcement of any patent rights, such patent rights could be compromised. If there are material defects in the form, preparation, prosecution, or enforcement of our patents or patent applications, such patents may be invalid and/or unenforceable, and such applications may never result in valid, enforceable patents. Any of these outcomes could impair our ability to prevent competition from third parties, which may have an adverse impact on our business.

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Pending patent applications cannot be enforced against third parties practicing the technology claimed in such applications unless and until a patent issues from such applications. Assuming the other requirements for patentability are met, currently, the first inventor to file a patent application is generally entitled to the patent. However, prior to March 16, 2013, in the United States, the first to invent was entitled to the patent. Publications of discoveries in the scientific literature often lag behind the actual discoveries, and patent applications in the United States and other jurisdictions are typically not published until 18 months after filing, or in some cases, not at all. Therefore, we cannot be certain that we were the first to make the inventions that will be claimed in our future patents or future patent applications, or that we will be the first to file for patent protection of such inventions. Similarly, we cannot be certain that parties from whom we do or may license or purchase patent rights were the first to make relevant claimed inventions, or were the first to file for patent protection for them. If third parties have filed patent applications on inventions claimed in our patents or applications on or before March 15, 2013, an interference proceeding in the United States can be initiated by such third parties to determine who was the first to invent any of the subject matter covered by the patent claims of our applications. If third parties have filed such applications after March 15, 2013, a derivation proceeding in the United States can be initiated by such third parties to determine whether our invention was derived from theirs.

The patent position of biotechnology and pharmaceutical companies generally is highly uncertain, involves complex legal and factual questions, and has been the subject of much litigation in recent years. No consistent policy regarding the breadth of claims allowed in biotechnology and pharmaceutical patents has emerged to date in the United States or in many foreign jurisdictions. 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 which protect our technology or product candidates or which effectively prevent others from commercializing competitive technologies and product candidates.

Moreover, the coverage claimed in a patent application can be significantly reduced before the patent is issued, and its scope can be reinterpreted after issuance. Even if patent applications we license or will file issue as patents, they may not issue in a form that will provide us with any meaningful protection, prevent competitors or other third parties from competing with us, or otherwise provide us with any competitive advantage. Any patents that we hold or in-license may be challenged by third parties, narrowed, circumvented invalidated, or held unenforceable, in whole or in part, which could limit our ability to stop others from using or commercializing similar or identical product candidates or technologies, or limit the duration of the patent protection of our product candidates or technologies. Consequently, we do not know whether any of our platform advances and product candidates will be protectable or remain protected by valid and enforceable patents. Our competitors or other third parties may be able to circumvent our patents by developing similar or alternative technologies or products in a non-infringing manner. Our competitors may also seek approval to market their own products similar to or otherwise competitive with our products. Alternatively, our competitors may seek to market biosimilar versions of any approved products by submitting abbreviated BLAs to the FDA in which they claim that patents owned or licensed by us are invalid, unenforceable, or not infringed. In these circumstances, we may need to defend or assert our patents, or both, including by filing lawsuits alleging patent infringement. In any of these types of proceedings, a court or other agency with jurisdiction may find our patents invalid or unenforceable, or that our competitors are competing in a non-infringing manner. Thus, even if we have valid and enforceable patents, these patents still may not provide protection against competing products or processes sufficient to achieve our business objectives.

Agreements through which we license patent rights may not give us control over patent prosecution or maintenance, so that we may not be able to control which claims or arguments are presented and may not be able to secure, maintain, or successfully enforce necessary or desirable patent protection from those patent rights. We have not had and do not have primary control over patent prosecution and maintenance for certain of the patents and patent applications we license, and therefore cannot guarantee that these patents and applications will be prosecuted in a manner consistent with the best interests of our business. We cannot be certain that patent prosecution and maintenance activities by our licensors have been or will be conducted in compliance with applicable laws and regulations or will result in valid and enforceable patents.

The issuance of a patent is not conclusive as to its inventorship, scope, validity, or enforceability and our patents may be challenged in the courts or patent offices in the United States and abroad. There is no assurance that all of the potentially relevant prior art relating to our patents and patent applications has been found. If such prior art exists, it may be used to invalidate a patent, or may prevent a patent from issuing from a pending patent application. We or our licensors may in the future become subject to a third-party pre-issuance submission of prior art to the United States Patent and Trademark Office, or the USPTO, or to other patent offices around the world. Alternately or additionally, we or our licensors may become involved in opposition, derivation, revocation, re-examination, post-grant and inter partes review, or interference proceedings and other similar proceedings in the United States or elsewhere, challenging our patent rights or the patent rights of others on which we rely to protect our business. For example, an anonymous third party filed an opposition in the European Patent Office, or the EPO, to European Patent No. 3 147 295 owned by Stanford. While we are not party to this opposition, this opposition relates to the LK03 capsid. We license certain intellectual property rights to the LK03 capsid from Stanford. An oral proceeding is currently scheduled for March 7, 2023. An adverse determination in any such submission, proceeding or litigation could reduce the scope of, or invalidate, our patent rights; limit the duration of the patent protection of our technology and products; allow third parties to commercialize our technology or products and compete directly with us, without

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payment to us; or result in our inability to manufacture or commercialize products without infringing third-party patent rights. In addition, if the breadth or strength of protection provided by our patents and patent applications is threatened, regardless of the outcome, it could dissuade companies from collaborating with us to license, develop or commercialize current or future product candidates. Moreover, we, or one of our licensors, may have to participate in interference proceedings declared by the USPTO to determine priority of invention or in post-grant challenge proceedings, such as oppositions in a foreign patent office, that challenge priority of invention or other features of patentability. Such challenges may result in loss of patent rights, loss of exclusivity, or in patent claims being narrowed, invalidated, or held unenforceable, 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 product candidates. Such proceedings also may result in substantial cost and require significant time from our scientists and management, even if the eventual outcome is favorable to us.

Pursuant to the terms of some of our license agreements with third parties, some of our third party licensors have the right, but not the obligation in certain circumstances to control enforcement of our licensed patents or defense of any claims asserting the invalidity of these patents. Even if we are permitted to pursue such enforcement or defense, we will require the cooperation of our licensors, and cannot guarantee that we would receive it and on what terms. We cannot be certain that our licensors will allocate sufficient resources or prioritize their or our enforcement of such patents or defense of such claims to protect our interests in the licensed patents. If we cannot obtain patent protection, or enforce existing or future patents against third parties, our competitive position and our financial condition could suffer.

Moreover, some of our in-licensed patents and patent applications are, and may in the future be, co-owned with third parties. Additionally, some of our future patent filings may be co-owned with third parties. If we are unable to obtain an exclusive license to any such third-party co-owners’ interests in such patents or patent applications, such co-owners may be able to license their rights to other third parties, including our competitors, and our competitors could market competing products and technology. In addition, we or our licensors may need the cooperation of any such co-owners of our owned and in-licensed patents in order to enforce such patents against third parties, and such cooperation may not be provided to us or our licensors. Any of the foregoing could harm our competitive position, business, financial conditions, results of operations and prospects.

Furthermore, our owned and in-licensed patents may be subject to a reservation of rights by one or more third parties. For example, the research resulting in certain of our owned and in-licensed patent rights and technology was funded in part by the U.S. government. As a result, the U.S. government has certain rights, including march-in rights, to such patent rights and technology. When new technologies are developed with government funding, the government generally obtains certain rights in any resulting patents, including a non-exclusive license authorizing the government to use the invention. For example, our licensors, including Stanford, have granted the U.S. government a non-exclusive, non-transferable, irrevocable, paid-up license to practice or have practiced for or on behalf of the United States, the inventions described in certain of our in-licensed patents and patent applications, including certain aspects of our in-licensed nuclease-free genome editing technology. If the government decides to exercise these rights, it is not required to engage us as its contractor in connection with doing so. These rights may permit the U.S. government to disclose our confidential information to third parties and to exercise march-in rights to use or allow third parties to use our licensed technology. The U.S. government can exercise its march-in rights if it determines that action is necessary because we fail to achieve practical application of the government-funded technology, because action is necessary to alleviate health or safety needs, to meet requirements of federal regulations, or to give preference to U.S. industry. In addition, our rights in such inventions may be subject to certain requirements to manufacture products embodying such inventions in the United States. Any exercise by the government of any of the foregoing rights could harm our competitive position, business, financial condition, results of operations, and prospects.

Our rights to develop and commercialize our technology and product candidates are subject, in part, to the terms and conditions of licenses granted to us by others.

We are heavily reliant upon licenses to certain patent rights and proprietary technology from third parties that are important or necessary to the development of our GeneRide platform technology and our sAAVy platform technology. These and other licenses may not provide exclusive rights to use such intellectual property and technology in all relevant fields of use and in all territories in which we may wish to develop or commercialize our technology and products in the future. As a result, we may not be able to prevent competitors from developing and commercializing competitive products in territories included in all of our licenses. For example, pursuant to our license agreements with Stanford and the NIH, the licensors may, under certain circumstances, grant a license to the patents that are the subject of such license agreements to a third party. Such third party would have full rights to the patent rights that are the subject of such licenses, which could impact our competitive position and enable a third party to commercialize products similar to our future product candidates and technology. In addition, our rights to our in-licensed patents and patent applications may be dependent, in part, on inter-institutional or other operating agreements between the joint owners of such in-licensed patents and patent applications. If one or more of such joint owners breaches such inter-institutional or operating agreements, our rights to such in-licensed patents and patent applications may be adversely affected, which could harm our competitive position, business, financial conditions, results of operations and prospects.

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In addition, we may not have the right to control the preparation, filing, prosecution, maintenance, enforcement, and defense of patents and patent applications covering the technology that we license from third parties. For example, pursuant to our intellectual property licenses for certain patent families from Stanford, our licensors retain control of preparation, filing, prosecution, maintenance, and enforcement and defense of their patents and patent applications. Therefore, we cannot be certain that these patents and patent applications will be prepared, filed, prosecuted, maintained, enforced, and defended in a manner consistent with the best interests of our business. If our licensors fail to prosecute, maintain, enforce, and defend such patents, or lose rights to those patents or patent applications, the rights we have licensed may be reduced or eliminated, and our right to develop and commercialize any of our products that are subject of such licensed rights could be adversely affected.

Our licensors may have relied on third party consultants or collaborators or on funds from third parties such that our licensors are not the sole and exclusive owners of the patents we in-licensed. If other third parties have ownership rights to our in-licensed patents, they may be able to license such patents to our competitors, and our competitors could market competing products and technology. This could adversely affect our competitive position, business, financial conditions, results of operations, and prospects.

If we fail to comply with our obligations in the agreements under which we license intellectual property rights from third parties or otherwise experience disruptions to our business relationships with our licensors, we could lose license rights that are important to our business.

We have entered into license agreements with third parties and may need to obtain additional licenses from our existing licensors and others to advance our research or allow commercialization of product candidates and technologies we may develop.

In our license agreements, and we expect in our future agreements, we have the right under specified conditions to bring, or permit our licensor(s) to bring, any actions against any third party for infringing on the patents we have exclusively licensed. Certain of our license agreements also require us to meet development thresholds and other obligations to maintain the license, including establishing a set timeline for developing and commercializing products. Disputes may arise regarding intellectual property subject to a licensing agreement, including:

• the scope of rights granted under the license agreement and other interpretation-related issues;

• the extent to which our technology and processes infringe on intellectual property of the licensor that is not subject to the licensing agreement;

• the sublicensing of patent and other rights under our collaborative development relationships;

• our diligence obligations under the license agreement and what activities satisfy those diligence obligations;

• the inventorship and ownership of inventions and know-how resulting from the joint creation or use of intellectual property by our licensors and us and our partners; and

• the priority of invention of patented technology.

If we fail to comply with our obligations under our license agreements, our licensors may have the right to terminate these license agreements, in which event we might not be able to market any product or technology that is covered by these agreements, or our licensors may convert the license to a non-exclusive license, which could adversely affect the value of the product candidate or technology being developed under the license agreement. Termination of these license agreements or reduction or elimination of our licensed rights may also result in our having to negotiate new or reinstated licenses with less favorable terms.

In spite of our best efforts, our licensors might conclude that we have materially breached our license agreements and might therefore terminate the license agreements, thereby removing our ability to develop and commercialize products and technology covered by these license agreements. If these in-licenses are terminated, or if the underlying patents fail to provide the intended exclusivity, competitors would have the freedom to seek regulatory approval of, and to market, products identical to ours. In addition, we may seek to obtain additional licenses from our licensors and, in connection with obtaining such licenses, we may agree to amend our existing licenses in a manner that may be more favorable to the licensors, including by agreeing to terms that could enable third parties (potentially including our competitors) to receive licenses to a portion of the intellectual property that is subject to our existing licenses. Any of these events could harm our competitive position, business, financial conditions, results of operations and prospects.

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Our intellectual property licenses with third parties may be subject to disagreements over contract interpretations, 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 currently 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, including the amount, if any, that may become due and payable to our licensors in connection with any sublicense income. Moreover, if disputes over intellectual property that we have licensed prevent or impair our ability to maintain our current licensing arrangements on commercially acceptable terms, we may be unable to successfully develop and commercialize the affected product candidates or technologies. If these events were to occur, they could harm our business, financial condition, results of operations and prospects.

We may not be successful in obtaining or maintaining necessary rights for current or future product candidates or technologies through acquisitions and in-licenses, which could delay or prevent us from commencing clinical trials and ultimately commercializing our current or future product candidates.

Because our programs may require the use of proprietary rights by third parties, the growth of our business likely will depend, in part, on our ability to acquire, in-license, or use these proprietary rights.

Many pharmaceutical companies, biotechnology companies, and academic institutions are competing with us in the fields of gene editing and gene therapy and filing patent applications potentially relevant to our business. In order to avoid infringing these third-party patents, or patents that issue from these third-party patent applications, we may find it necessary or prudent to obtain licenses from such third-party intellectual property holders. In addition, with respect to any patents we may co-own with third parties, we may require licenses to such co-owners’ interests in such patents.

We may be unable to acquire or in-license any compositions, methods of use, processes or other intellectual property rights from third parties that we identify as necessary for our technology and product candidates. The licensing or acquisition of third-party intellectual property rights is a competitive area, and more established companies may pursue strategies to license or acquire third-party intellectual property rights that we may consider attractive or necessary. These established companies may have a competitive advantage over us due to their size, capital resources and greater clinical development and commercialization capabilities. In addition, companies that perceive us to be a competitor may be unwilling to assign or license rights to us. We also may be unable to license or acquire third-party intellectual property rights on terms that would allow us to make an appropriate return on our investment or at all.

We sometimes collaborate with non-profit and academic institutions to accelerate our preclinical research or development under written agreements with these institutions. These institutions may provide us with an option to negotiate a license to any of the institution’s rights in technology resulting from the collaboration. Regardless of such option, we may be unable to negotiate a license within the specified timeframe or under terms that are acceptable to us or we may decide not to execute such option if we believe such license is not necessary to pursue our program. If we are unable or opt not to do so, the institution may offer the intellectual property rights to other parties, potentially blocking our ability to pursue our program.

It is possible that we may be unable to successfully obtain rights to required third-party intellectual property rights or maintain the existing intellectual property rights we have at a reasonable cost or on reasonable terms. In that event, we may be required to expend significant time and resources to redesign our technology, product candidates, or methods for manufacturing them or to develop or license replacement technology, all of which may not be feasible on a technical or commercial basis. If we are unable to do so, we may be unable to develop or commercialize the affected product candidate, which could harm our business, financial condition, results of operations, and prospects significantly.

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We may not be able to protect our intellectual property and proprietary rights throughout the world.

Filing, prosecuting, and defending patents in all countries throughout the world would be prohibitively expensive, and the laws of foreign countries may not protect our intellectual property rights to the same extent as the laws of the United States. In addition, our intellectual property license agreements may not always include worldwide rights. The requirements for patentability may differ in certain countries, particularly in developing countries; thus, even in countries where we do pursue patent protection, there can be no assurance that any patents will issue with claims that cover our products. Consequently, we may not be able to prevent third parties from practicing our inventions in all countries outside the United States, or from selling or importing products made using our inventions in and into the United States or other jurisdictions. Competitors may use our technologies in jurisdictions where we have not obtained patent protection to develop and market their own products and, further, may export otherwise infringing products to territories where we have patent protection or licenses but enforcement is not as strong as that in the United States or if our ability to enforce our patents to stop infringing activities is inadequate. These products may compete with our products, and our patents or other intellectual property rights may not be effective or sufficient to prevent them from competing.

Many companies have encountered significant problems in protecting and defending intellectual property rights in certain foreign jurisdictions. The legal systems of certain countries, particularly certain developing countries, do not favor the enforcement of patents, trade secrets, and other intellectual property protection, particularly those relating to biotechnology products, which could make it difficult for us to stop the infringement of our patents, misappropriation of our other intellectual property rights, or marketing of competing products in violation of our intellectual property and proprietary rights generally.

Many countries have compulsory licensing laws under which a patent owner may be compelled to grant licenses to third parties. In addition, many countries limit the enforceability of patents against government agencies or government contractors. In these countries, the patent owner may have limited remedies, which could materially diminish the value of such patent. If we or any of our licensors is forced to grant a license to third parties with respect to any patents relevant to our business, our competitive position may be impaired, and our business, financial condition, results of operations, and prospects may be adversely affected.

Agreements through which we license patent rights may not give us sufficient rights to permit us to pursue enforcement of our licensed patents or defense of any claims asserting the invalidity of these patents (or control of enforcement or defense) of such patent rights in all relevant jurisdictions as requirements may vary.

Proceedings to enforce our intellectual property and proprietary rights in foreign jurisdictions, whether or not successful, could result in substantial costs and divert our efforts, resources and attention from other aspects of our business. Moreover, such proceedings could put our patents at risk of being invalidated or interpreted narrowly and our patent applications at risk of not issuing and could provoke third parties to assert claims against us. We may not prevail in any lawsuits that we initiate, and the damages or other remedies awarded, if any, may not be commercially meaningful. Furthermore, while we intend to protect our intellectual property rights in major markets for our products, we cannot ensure that we will be able to initiate or maintain similar efforts in all jurisdictions in which we may wish to market our products. Accordingly, our efforts to enforce our intellectual property and proprietary rights around the world may be inadequate to obtain a significant commercial advantage from the intellectual property that we develop or license.

Obtaining and maintaining patent protection depends on compliance with various procedural, document submission, fee payment, and other requirements imposed by governmental patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these requirements.

Periodic maintenance fees, renewal fees, annuity fees, and various other government fees on patents and applications will be due to be paid to the USPTO and various government patent agencies outside of the United States over the lifetime of our licensed or owned patents and applications. In certain circumstances, we rely on our licensing partners to pay these fees due to U.S. and non-U.S. patent agencies. The USPTO and various non-U.S. government agencies require compliance with several procedural, documentary, fee payment, and other similar provisions during the patent application process and after a patent has issued. We are also dependent on our licensors to take the necessary action to comply with these requirements with respect to some of our licensed intellectual property. In some cases, an inadvertent lapse can be cured by payment of a late fee or by other means in accordance with the applicable rules. There are situations, however, in which non-compliance can result in abandonment or lapse of the patent or patent application, resulting in a partial or complete loss of patent rights in the relevant jurisdiction. In such an event, potential competitors might be able to enter the market with similar or identical products or technology, which could harm our business, financial condition, results of operations and prospects.

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Patent terms for our product candidates or technologies may be inadequate to protect our competitive position for an adequate amount of time.

Patents have a limited lifespan. In the United States, if all maintenance fees are timely paid, the natural expiration of a patent is generally 20 years from its earliest U.S. non-provisional filing date. Various extensions may be available, but the life of a patent, and the protection it affords, is limited. Even if patents covering our product candidates and technologies are obtained, once the patent life has expired, we may be open to competition from competitive products, including generics or biosimilars. Given the amount of time required for the development, testing and regulatory review of new product candidates or technologies, patents protecting such candidates might expire before or shortly after such candidates are commercialized. As a result, our intellectual property may not provide us with sufficient rights to exclude others from commercializing products similar or identical to ours.

We expect to seek extensions of patent terms in the United States and, if available, in other countries where we are prosecuting patents. Depending upon the timing, duration and specifics of any FDA marketing approval of any product candidates we may develop, one or more of our U.S. patents may be eligible for limited patent term extension under the Drug Price Competition and Patent Term Restoration Action of 1984, or the Hatch-Waxman Amendments. The Hatch-Waxman Amendments permit a patent extension term of up to five years beyond the normal expiration of the patent, which is limited to the approved indication (or any additional indications approved during the period of extension) as compensation for patent term lost during the FDA regulatory review process. A patent term extension cannot extend the remaining term of a patent beyond a total of 14 years from the date of product approval, only one patent may be extended, and only those claims covering the approved drug, a method for using it, or a method for manufacturing it may be extended. However, we may not be granted an extension because of, for example, failing to exercise due diligence during the testing phase or regulatory review process, failing to apply within applicable deadlines, failing to apply prior to expiration of relevant patents, or otherwise failing to satisfy applicable requirements. Moreover, the applicable time period or the scope of patent protection afforded could be less than we request. The applicable authorities, including the FDA and the USPTO in the United States, and any equivalent regulatory authority in other countries, may not agree with our assessment of whether such extensions are available, and may refuse to grant extensions to our patents, or may grant more limited extensions than we request. If we are unable to obtain patent term extension or term of any such extension is less than we request, our competitors may obtain approval of competing products following our patent expiration, and our business, financial condition, results of operations, and prospects could be materially harmed. Additionally or alternatively, our competitors may be able to take advantage of our investment in development and clinical trials by referencing our clinical and preclinical data and launch their product earlier than might otherwise be the case. If we are not able to obtain and enforce market exclusivity for our technologies or product candidates, development and commercialization of our product candidates may be adversely affected.

In our industry, the majority of an innovative product’s commercial value is usually realized during the period in which it has market exclusivity. Market exclusivity is comprised of both patent and other intellectual property protection, as well as regulatory exclusivity.

In the United States and some other countries, when market exclusivity expires and generic/biosimilar versions of a product are approved and marketed, there usually are very substantial and rapid declines in the product’s sales. Accordingly, our success depends in part on our ability to obtain and maintain patents and other forms of intellectual property rights, including trademarks, trade secrets and in-licenses of intellectual property rights of others, for our product candidates and platform technologies, methods used to manufacture our product candidates, methods of patient stratification and methods for treating patients using our product candidates, as well as our ability to preserve our trade secrets, to prevent third parties from infringing upon our proprietary rights and to operate without infringing upon the proprietary rights of others. Certain research and development activities involved in pharmaceutical and biologics development are exempt from patent infringement in the United States and other jurisdictions, for example, in the United States by the provisions of 35 U.S.C. § 271(e)(1), or the Safe Harbor. However, in the United States and certain other jurisdictions, the Safe Harbor exemption is more limited after the sponsor obtains approval for a BLA. Therefore, the risk that a third party might allege patent infringement may increase as our products approach commercialization. We may not be able to apply for patents or obtain patent protection on certain aspects of our product candidates or our platform in a timely fashion or at all. Our existing issued and granted patents and any future patents we obtain may not be sufficiently broad to prevent others from using our technology or from developing competing products and technology. There is no guarantee that any of our pending patent applications will result in issued or granted patents, that any of our issued or granted patents will not later be found to be invalid or unenforceable, or that any issued or granted patents will include claims that are sufficiently broad to cover our product candidates, our platform technologies, or any methods relating to them, or to provide meaningful protection from our competitors.

Legal issues related to the patentability of biopharmaceuticals, and methods of their manufacture and use, are complex and uncertain in some countries. In some countries, applicants are not able to protect methods of treating human beings or medical treatment processes. Intellectual property protection varies throughout the world and is subject to change over time. Certain jurisdictions have enacted various rules and laws precluding issuance of patents encompassing any methods a doctor may practice on a human being or any other animal to treat a disease or condition. Thus, in some countries and jurisdictions, it may not be possible to patent some of our

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product candidates at all. In some countries and jurisdictions, only composition claims may be obtained, and only when those compositions are or contain compounds that are new and/or novel. Also, patents issued with composition claims (i.e., covering product candidates) cannot always be enforced to protect methods of using those compositions to treat or diagnose diseases or medical conditions. In such countries or jurisdictions, enforcement of patents to protect our product candidates, or their uses, may be difficult or impossible. Lack of patent protection in such cases may have a materially adverse effect on our business and financial condition.

Additionally, we expect that the product candidates we develop will be regulated as biologics in the United States and therefore they may be subject to competition from biosimilars approved through an abbreviated regulatory pathway. The Affordable Care Act includes a subtitle called the Biologics Price Competition and Innovation Act of 2009, or the BPCIA, which created an abbreviated approval pathway for biological products that are biosimilar to or interchangeable with an FDA-approved reference biological product. Under the BPCIA, an application for a biosimilar product may not be submitted to the FDA until four years following the date that the reference product was first approved by the FDA. In addition, the approval of a biosimilar product may not be made effective by the FDA until 12 years after the date on which the reference product was first approved.

During this 12-year period of exclusivity, another company may still market a competing version of the reference product if the FDA approves a BLA for the competing product containing the sponsor’s own preclinical data and data from adequate and well-controlled clinical trials to demonstrate the safety, purity and potency of the other company’s product. The BPCIA also created certain exclusivity periods for biosimilars approved as interchangeable products. At this juncture, it is unclear whether products deemed “interchangeable” by the FDA will, in fact, be readily substituted by pharmacies, which are governed by state pharmacy law. Since the passage of the BPCIA, many states have passed laws or amendments to laws, including laws governing pharmacy practices, which are state-regulated, to regulate the use of biosimilars. 

We believe that any of our product candidates approved as a biological product under a BLA should qualify for a 12-year period of regulatory exclusivity under the BPCIA. However, there is a risk that this exclusivity could be shortened due to congressional action or otherwise, or that the FDA will not consider our product candidates to be reference products for competing products, potentially creating the opportunity for generic competition sooner than anticipated. Other aspects of the BPCIA, some of which may impact the BPCIA exclusivity provisions, have also been the subject of recent litigation. Moreover, the extent to which a biosimilar, once approved, will be substituted for any one of our reference products in a way that is similar to traditional generic substitution for non-biological products is not yet clear, and will depend on a number of marketplace and regulatory factors that are still developing.

Furthermore, given the amount of time required for the development, testing and regulatory review of new product candidates, patents protecting such candidates, their manufacture or their use might expire before or shortly after those candidates receive regulatory approval and 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. We expect to seek extensions of patent terms where these are available upon regulatory approval in those countries where we are prosecuting patents. This includes in the United States under the BPCIA which permits a patent term extension of up to five years beyond the expiration of the patent. However, the applicable authorities, including the FDA in the United States, and any equivalent regulatory authority in other countries, may not agree with our assessment of whether such extensions are available, and may refuse to grant extensions to our patents, or may grant more limited extensions than we request. If this occurs, our competitors may take advantage of our investment in development and clinical trials by referencing our clinical and preclinical data and launch their product earlier than might otherwise be possible.

Changes to the patent law in the United States and other jurisdictions could diminish the value of patents in general, thereby impairing our ability to protect our products.

As is the case with other biopharmaceutical companies, our success is heavily dependent on intellectual property, particularly patents. Obtaining and enforcing patents in the biopharmaceutical industry involves both technological and legal complexity and is therefore costly, time consuming and inherently uncertain. Changes in either the patent laws or interpretation of the patent laws in the United States and other jurisdictions could increase the uncertainties and costs surrounding the prosecution of patent applications and the enforcement or defense of issued patents.

Patent reform legislation in the United States, including the Leahy-Smith America Invents Act, or the America Invents Act, could increase those uncertainties and costs.

Assuming that other requirements for patentability are met, prior to March 2013, in the United States, the first to invent the claimed invention was entitled to the patent, while outside the United States, the first to file a patent application was entitled to the patent. After March 2013, under the Leahy-Smith America Invents Act, or the America Invents Act, enacted in September 2011, the United States transitioned to a first inventor to file system in which, assuming that other requirements for patentability are met, the first inventor to file a patent application will be entitled to the patent on an invention regardless of whether a third party was the first to invent the claimed invention. The America Invents Act also includes a number of significant changes that affect the way patent

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applications will be prosecuted and also may affect patent litigation. These include allowing third party submission of prior art to the USPTO during patent prosecution and additional procedures to challenge the validity of a patent by USPTO administered post-grant proceedings, including post-grant review, inter partes review, and derivation proceedings. The America Invents Act, as well as any future patent reform, and implementation thereof could increase the uncertainties and costs surrounding the prosecution of our patent applications and the enforcement or defense of our issued patents, all of which could harm our business, financial condition, results of operations, and prospects.

In addition, the patent positions of companies in the development and commercialization of biologics and pharmaceuticals are particularly uncertain. U.S. Supreme Court rulings have narrowed the scope of patent protection available in certain circumstances and weakened the rights of patent owners in certain situations. This combination of events has created uncertainty with respect to the validity and enforceability of patents, once obtained. Depending on future actions by the U.S. Congress, the federal courts, the USPTO, and the relevant law-making bodies in other countries, the laws and regulations governing patents could change in unpredictable ways that could adversely affect our existing patent portfolio and our ability to protect and enforce our intellectual property in the future.

If we are unable to protect the confidentiality of our trade secrets, the value of our technology could be materially adversely affected, and our business and competitive position would be harmed.

In addition to seeking patents for some of our technology and product candidates, we also rely on trade secrets and confidentiality agreements to protect our unpatented know-how, technology, and other proprietary information and to maintain our competitive position. With respect to our GeneRide and sAAVy platforms, we consider trade secrets and know-how to be an important component of our intellectual property. Trade secrets and know-how can be difficult to protect. In particular, we anticipate that with respect to our GeneRide technology platform, these trade secrets and know-how will over time be disseminated within the industry through independent development, the publication of journal articles describing the methodology, and the movement of personnel from academic to industry scientific positions.

We seek to protect these trade secrets and other proprietary technology, in part, by entering into non-disclosure and confidentiality agreements with parties who have access to them, such as our employees, collaborators, contract manufacturers, consultants, and other third parties. We also enter into agreements containing confidentiality and invention or patent assignment obligations with our employees and certain consultants. We cannot guarantee that we have entered into such agreements with each party that may have or have had access to our trade secrets or proprietary technology and processes. Despite these efforts, any of these parties may breach the agreements and disclose our proprietary information, including our trade secrets, and we may not be able to obtain adequate remedies for such breaches. Enforcing a claim that a party illegally disclosed or misappropriated a trade secret is difficult, expensive, and time-consuming, and the outcome is unpredictable. Additionally, if the steps taken to maintain our trade secrets are deemed inadequate, we may have insufficient recourse against third parties for misappropriating the trade secret. In addition, 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 or other third party, we would have no right to prevent such third party, or those to whom they communicate such technology or information, from using that technology or information to compete with us. If any of our trade secrets were to be disclosed to or independently developed by a competitor or other third party, our business and competitive position could be materially and adversely harmed.

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

Competitors may infringe our intellectual property, such as our patents or trademarks, or the patents of our licensing partners. To counter infringement or unauthorized use, we may be required to file infringement claims. Additionally or alternatively, we may be required to defend against claims of infringement filed by third parties against us. In addition, our patents or the patents of our licensing partners may in the future become involved in inventorship, priority, or validity disputes. Filing infringement claims and countering and defending against claims regarding infringement or disputes of inventorship, priority, or validity can be expensive and time consuming and divert the time and attention of our management and scientific personnel.

If we or one of our licensors were to initiate legal proceedings against a third party, the defendant could counterclaim that such patent is invalid or unenforceable. In patent litigation in the United States, defendant counterclaims alleging invalidity or unenforceability are commonplace. Grounds for a validity challenge could be an alleged failure to meet any of several statutory requirements, including lack of novelty, obviousness, or non-enablement. Grounds for an unenforceability assertion could be an allegation that someone connected with prosecution of the patent withheld relevant information from the USPTO, or made a misleading statement, during prosecution. Third parties may in the future raise challenges to the validity of our patent claims before administrative bodies in the United States or abroad, even outside the context of litigation. Such mechanisms include re-examination, post-grant review, inter

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partes review, interference proceedings, derivation proceedings, and equivalent proceedings in foreign jurisdictions (e.g., opposition proceedings, such as the opposition in the EPO to European Patent No. 3 147 295 relating to LK03, described above).

The outcome following legal assertions of invalidity and unenforceability is unpredictable. With respect to the validity question, for example, we cannot be certain that there is no invalidating prior art, of which we or our licensing partners and the patent examiner were unaware during prosecution. If a third party were to prevail on a legal assertion of invalidity or unenforceability, we would lose at least part, and perhaps all, of the patent protection on our technology or platform, or any product candidates that we may develop. Such a loss of patent protection would have a material adverse impact on our business, financial condition, results of operations, and prospects.

In any patent infringement proceeding, there is a risk that a court may decide that a patent owned or in-licensed by us is invalid or unenforceable, in whole or in part, or may refuse to stop the other party from using the technology at issue on the grounds that our owned and in-licensed patents do not cover the technology in question. There is also a risk that, even if the validity of such patents is upheld, the court will construe the patent’s claims narrowly or decide that we do not have the right to stop the other party from using the invention at issue on the grounds that our patent claims do not cover the invention. An adverse outcome in any litigation or proceeding could put one or more of our owned or in-licensed patents at risk of being invalidated or interpreted narrowly and may curtail or preclude our ability to assert those patents against third parties and exclude third parties from making and selling similar or competitive products. Even if we establish infringement, the court may decide not to grant an injunction against further infringing activity and instead award only monetary damages, which may or may not be an adequate remedy.

Similarly, if we assert trademark infringement claims, a court may determine that the marks we have asserted are invalid or unenforceable, or that the party against whom we have asserted trademark infringement has superior rights to the marks in question. In this case, we could ultimately be forced to cease use of such trademarks.

Even if resolved in our favor, litigation or other legal proceedings relating to intellectual property claims may cause us to incur significant expenses and could distract our personnel from their normal responsibilities. The monetary cost of such litigation and the diversion of the attention of our management and scientific personnel could outweigh any benefit we receive as a result of the proceedings. Furthermore, because of the substantial amount of discovery required in connection with intellectual property litigation, there is a risk that some of our confidential information could be compromised by disclosure during this type of litigation. There could also be public announcements of the results of hearings, motions, or other interim proceedings or developments. If securities analysts or investors perceive these results to be negative, it could have a substantial adverse effect on the price of shares of our common stock. Such litigation or proceedings could substantially increase our operating losses and reduce the resources available for development activities or any future sales, marketing, or distribution activities. Moreover, there can be no assurance that we will have sufficient financial or other resources to conduct such litigation or proceedings adequately, which can last for years before they are concluded. Some of our competitors may be able to sustain the costs of such litigation or proceedings more effectively than we can because of their greater financial resources and more mature and developed intellectual property portfolios. Uncertainties resulting from the initiation and continuation of patent litigation or other proceedings could adversely affect our ability to compete in the marketplace.

The intellectual property landscape around genetic medicines is highly dynamic, and third parties may initiate legal proceedings alleging that we are infringing, misappropriating, or otherwise violating their intellectual property rights, the outcome of which would be uncertain and could adversely affect the success of our business.

Our commercial success depends, in part, upon our ability and the ability of our collaborators to develop, manufacture, market, and sell our product candidates and use our proprietary technologies without infringing, misappropriating, or otherwise violating the intellectual property and proprietary rights of third parties. However, the biotechnology and pharmaceutical industries are characterized by extensive litigation regarding patents and other intellectual property rights, and our research, development and commercialization activities may be subject to claims that we infringe or otherwise violate patents or other intellectual property rights owned or controlled by third parties. Moreover, due to the intense research and development that is taking place by several companies, including us and our competitors, in the fields of gene editing and gene therapy, the intellectual property landscape is in flux, and it may remain uncertain for the coming years. There may be significant intellectual property related litigation and proceedings relating to our owned and in-licensed, and other third party, intellectual property, and proprietary rights in the future. We may in the future become party to, or threatened with, adversarial proceedings or litigation regarding intellectual property rights with respect to our technology and any product candidates we may develop, including interference proceedings, post-grant review, inter partes review, and derivation proceedings before the USPTO and similar proceedings in foreign jurisdictions such as oppositions before the EPO, such as the opposition in the EPO to European Patent No. 3 147 295 relating to the LK03 capsid, described above. We cannot provide any assurances that third party patents do not exist which might be enforced against our current technology, manufacturing methods, product candidates, or future methods or products resulting in either an injunction prohibiting our manufacture or sales, or, with respect to our sales, an obligation on our part to pay royalties and/or other forms of compensation to third parties, which could be significant.

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Third parties may have U.S. and non-U.S. issued patents and pending patent applications relating to products and methods of use for the treatment of the disease indications for which we are developing our product candidates. There may be third-party patents or patent applications with claims to materials, methods of manufacture, or methods for treatment related to the use or manufacture of our technologies and product candidates. If we are not able to obtain or maintain a license on commercially reasonable terms to any third-party patents that cover our product candidates or activities, such third parties could potentially assert infringement claims against us, which could harm our business.

Because patent applications can take many years to issue, there may be currently pending patent applications which may later result in issued patents that our product candidates may be accused of infringing. In addition, third parties may obtain patents in the future and claim that use of our technologies infringes upon these patents. Accordingly, third parties may assert infringement claims against us based on existing or future intellectual property rights. Defense of these claims, regardless of their merit, would involve substantial litigation expense and would be a substantial diversion of employee resources from our business. Parties making claims against us may be able to sustain the costs of complex patent litigation more effectively than we can because they have substantially greater resources. Furthermore, because of the substantial amount of discovery required in connection with intellectual property litigation or administrative proceedings, there is a risk that some of our confidential information could be compromised by disclosure. In addition, any uncertainties resulting from the initiation and continuation of any litigation could have material adverse effect on our ability to raise additional funds or otherwise have a material adverse effect on our business, results of operations, financial condition and prospects. If any third-party patents or patent applications are found to cover our product candidates or their methods of use, we may not be free to manufacture or market our product candidates as planned without obtaining a license.

Even if we believe third-party intellectual property claims are without merit, there is no assurance that a court would find in our favor on questions of infringement, validity, enforceability, or priority. A court of competent jurisdiction could hold that these third-party patents are valid, enforceable, and infringed, which could materially and adversely affect our ability to commercialize any product candidates we may develop and any other product candidates or technologies covered by the asserted third party patents.

The outcome of intellectual property litigation is subject to uncertainties that cannot be adequately quantified in advance. The pharmaceutical and biotechnology industries have produced a significant number of patents, and it may not always be clear to industry participants, including us, which patents cover various types of products or methods of use. The coverage of patents is subject to interpretation by the courts, and the interpretation is not always uniform.

If we were sued for patent infringement, we would need to demonstrate that our product candidates, products or methods either do not infringe the patent claims of the relevant patent or that the asserted patent claims are invalid or unenforceable, and we may not be able to do this. Proving invalidity is difficult. For example, in the United States, proving invalidity requires a showing of clear and convincing evidence as to the invalidity of any such U.S. patent claim to overcome the presumption of validity enjoyed by issued patents. There is no assurance that a court of competent jurisdiction would invalidate the claims of any such U.S. patent. Even if we are successful in these proceedings, we may incur substantial costs and the time and attention of our management and scientific personnel could be diverted in pursuing these proceedings, which could significantly harm our business and operating results. In addition, we may not have sufficient resources to bring these actions to a successful conclusion.

If we are found to infringe a third party’s intellectual property rights, and we are unsuccessful in demonstrating that such patents are invalid or unenforceable, we could be required to obtain a license from such third party to continue developing, manufacturing, and marketing any product candidates we may develop and our technology. However, we may not be able to obtain any required license on commercially reasonable terms or at all. Even if we were able to obtain a license, it could be non-exclusive, thereby giving our competitors and other third parties access to the same technologies licensed to us, and it could require us to make substantial licensing and royalty payments. Alternatively or additionally, it could include terms that impede or destroy our ability to compete successfully in the commercial marketplace. We also could be forced, including by court order, to cease developing, manufacturing, and commercializing the infringing technology or product candidates. 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 or other intellectual property right. A finding of infringement could prevent us from commercializing our product candidates or force us to cease some of our business operations, which could harm our business. Claims that we have misappropriated the confidential information or trade secrets of third parties could have a similar adverse effect on our business, financial condition, results of operations, and prospects.

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We may be subject to claims that we, our employees, consultants, or advisors have wrongfully used or disclosed alleged trade secrets of their current or former employers or claims asserting ownership of what we regard as our own intellectual property.

Many of our employees and our licensors’ employees, consultants, and advisors are currently or were previously employed at universities or other biotechnology or pharmaceutical companies, some of which may be our competitors or potential competitors. Some of these individuals executed agreements containing proprietary rights, non-disclosure and non-competition obligations, or similar agreements, in connection with such current or previous employment. Although we try to ensure that our employees, consultants, and advisors do not use the proprietary information or know-how of others in their work for us, we may be subject to claims that we or these individuals have used or disclosed intellectual property, including trade secrets or other proprietary information, of any such third party. Litigation may be necessary to defend against these claims.

In addition, while it is our policy to require our employees and consultants who may be involved in the conception or development of intellectual property to execute agreements assigning such intellectual property to us, we may be unsuccessful in executing such an agreement with each party who, in fact, conceives or develops intellectual property that we regard as our own. 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.

If we fail in prosecuting or defending any such claims listed above, in addition to paying monetary damages, we may lose valuable intellectual property rights or personnel. Such intellectual property rights could be awarded to a third party, and we could be required to obtain a license from such third party to commercialize our technology or products. Such a license may not be available on commercially reasonable terms or at all. Even if we are successful in prosecuting or defending against such claims, litigation could result in substantial costs and be a distraction to our management and scientific personnel.

We may be subject to claims challenging the inventorship of our patents and other intellectual property.

We or our licensors may be subject to claims that former employees, collaborators or other third parties have an interest in our owned or in-licensed patents, trade secrets, or other intellectual property as an inventor or co-inventor. For example, we or our licensors may have inventorship disputes arise from conflicting obligations of employees, consultants or others who are involved in developing our product candidates. Litigation may be necessary to defend against these and other claims challenging inventorship or our or our licensors’ ownership of our owned or in-licensed patents, trade secrets or other intellectual property. If we or our licensors fail in defending any such claims, in addition to paying monetary damages, we may lose valuable intellectual property rights, such as exclusive ownership of, or right to use, intellectual property that is important to our product candidates. Even if we are successful in defending against such claims, litigation could result in substantial costs and be a distraction to management and other employees. Any of the foregoing could have a material adverse effect on our business, financial condition, results of operations and prospects.

Intellectual property rights do not necessarily address all potential threats.

The degree of future protection afforded by our intellectual property rights is uncertain because intellectual property rights have limitations and may not adequately protect our business or permit us to maintain our competitive advantage. For example:

• others may be able to make genetic medicine products that are similar to any product candidates we may develop or utilize similar genetic medicine technology but that are not covered by the claims of the patents that we license or may own in the future;

• others may be able to make or utilize genetic medicine technology that functions as a viable alternative to technology we may develop or technology covered by the claims of the patents that we license or may own in the future;

• we, or our license partners or current or future collaborators, might not have been the first to make the inventions covered by the issued patent or pending patent application that we license or may own in the future;

• we, or our license partners or current or future collaborators, might not have been the first to file patent applications covering certain of our or their inventions;

• others may independently develop similar or alternative technologies or duplicate any of our technologies without infringing our owned or licensed intellectual property rights;

• it is possible that our pending licensed patent applications or those that we may own in the future will not lead to issued patents;

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• issued patents that we hold rights to may be held invalid or unenforceable, including as a result of legal challenges by our competitors;

• our competitors might conduct research and development activities in countries where we do not have patent rights and then use the information learned from such activities to develop competitive products for sale in our major commercial markets;

• we may not develop additional proprietary technologies that are patentable;

• the patents of others may harm our business; and

• we may choose not to file a patent in order to maintain certain trade secrets or know-how, and a third party may subsequently file a patent covering such intellectual property.

Should any of these events occur, they could harm our business, financial condition, results of operations, and prospects.

Risks Related to Healthcare Laws and Other Legal Compliance Matters

Healthcare legislative reform measures and constraints on government budgets may harm our business and results of operations.

Third-party payors, whether domestic or foreign, or governmental or private, are developing increasingly sophisticated methods of controlling healthcare costs. In both the United States and certain foreign jurisdictions, there have been a number of legislative and regulatory changes to the health care system that could impact our ability to sell any products profitably. Within the United States, for example, the Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation Act of 2010, or the Affordable Care Act, significantly revised coverage and payment for health care services. The Affordable Care Act expanded healthcare coverage through Medicaid expansion and the implementation of the individual health insurance mandate and included a number of provisions relevant to coverage and payment of prescription drugs, such as subjecting biologic products to potential competition by lower-cost biosimilars; increasing minimum rebates under the Medicaid Drug Rebate Program and extending the Medicaid Drug Rebate program to prescription drugs covered by Medicaid managed care organizations; requiring manufacturers to provide discounts on Medicare Part D brand name prescription drugs sold to Medicare beneficiaries in the Medicare Part D coverage gap (i.e., the so-called “donut hole”); and subjecting manufacturers to new annual fees for certain branded prescription drugs.

We face uncertainties because there have been, and may be additional, federal legislative and administrative efforts to repeal, substantially modify or invalidate some or all of the provisions of the Affordable Care Act. For example, tax reform legislation was enacted at the end of 2017 that eliminated the tax penalty for individuals who do not maintain sufficient health insurance coverage beginning in 2019. As another example, legislation enacted in 2018 increased the discount on Medicare Part D drugs purchased by Medicare beneficiaries in the coverage gap in 2019. The Affordable Care Act has also been subject to judicial challenge. For example, in June 2021, the Supreme Court rejected a challenge to the constitutionality of the Affordable Care Act on the grounds that the states and individuals that brought the challenge did not have standing.

Beyond the Affordable Care Act, there have been ongoing reform efforts that affect pricing or payment for drug products or the healthcare industry more generally. For example, President Biden continues to push for reforms that would address the high cost of drugs. In response to an Executive Order from President Biden, the U.S. Secretary of Health and Human Services recently issued a comprehensive plan for addressing high drug prices that describes a number of legislative approaches and identifies administrative tools to address the high cost of drugs. Drug pricing reforms were included in major legislation supported by President Biden in 2021 (which ultimately stalled), and more recently, in the Inflation Reduction Act of 2022, which was signed into law in August 2022. President Biden also addressed drug pricing reform in his 2022 State of the Union address. Additional healthcare reform efforts have sought to address certain issues related to the COVID-19 pandemic, including an expansion of telehealth coverage under Medicare and accelerated or advanced Medicare payments to healthcare providers. Some of these changes have been and may continue to be subject to legal challenge.  For example, revisions to regulations under the federal anti-kickback statute would remove protection for traditional Medicare Part D discounts offered by pharmaceutical manufacturers to pharmacy benefit managers and health plans. Pursuant to court order, the removal was delayed and recent legislation imposed a moratorium on implementation of the rule until January 1, 2026. Adoption of new healthcare reform legislation at the federal or state level could affect demand for, or pricing of, our products or product candidates if approved for sale. We cannot predict, however, the ultimate content, timing or effect of any healthcare reform legislation or action, or its impact on us, and healthcare reform could increase compliance costs and may adversely affect our future business and financial results.

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There have been, and likely will continue to be, legislative and regulatory proposals at the foreign, federal and state levels directed at broadening the availability of healthcare and containing or lowering the cost of healthcare. We cannot predict the initiatives that may be adopted in the future. The continuing efforts of the government, insurance companies, managed care organizations and other payors of healthcare services to contain or reduce costs of healthcare and/or impose price controls may adversely affect:

• the demand for our drug product candidates, if we obtain regulatory approval;

• our ability to set a price that we believe is fair for our products;

• our ability to generate revenue and achieve or maintain profitability;

• the level of taxes that we are required to pay; and

• the availability of capital.

In addition, other legislative changes have been adopted that could have an adverse effect upon, and could prevent, our products’ or product candidates’ commercial success. More broadly, the Budget Control Act of 2011, as amended, or the Budget Control Act, includes provisions intended to reduce the federal deficit, including reductions in Medicare payments to providers through 2031 (except May 1, 2020 to March 31, 2022). Any significant spending reductions affecting Medicare, Medicaid or other publicly funded or subsidized health programs, or any significant taxes or fees imposed as part of any broader deficit reduction effort or legislative replacement to the Budget Control Act, or otherwise, could have an adverse impact on our anticipated product revenues.

While we have received orphan drug designation for LB-001 for the treatment of MMA, we may not be able to obtain orphan drug marketing exclusivity for LB-001 or enjoy the benefits associated with orphan drug designation.

In April 2019, the FDA granted orphan drug designation for LB-001 for the treatment of MMA, and in June 2021, the European Commission granted orphan drug designation to LB-001 for the treatment of MMA.

Under the U.S. Orphan Drug Act, the FDA may designate a drug as an orphan drug if it is a drug intended to treat a rare disease or condition, which is generally defined as a patient population of fewer than 200,000 individuals annually in the United States, or a patient population greater than 200,000 in the United States where there is no reasonable expectation that the cost of developing the drug will be recovered from sales in the United States. In the United States, orphan drug designation may entitle a party to financial incentives such as opportunities for grant funding towards clinical trial costs, tax advantages and user‑fee waivers.

In the European Union, orphan drug designation is intended to promote the development of drugs that are intended for the diagnosis, prevention or treatment of life‑threatening or chronically debilitating conditions affecting not more than 5 in 10,000 persons in the European Union and for which no satisfactory method of diagnosis, prevention, or treatment has been authorized (or the product would be a significant benefit to those affected). In addition, designation is granted for drugs intended for the diagnosis, prevention, or treatment of a life‑threatening, seriously debilitating or serious and chronic condition and when, without incentives, it is unlikely that sales of the drug in the European Union would be sufficient to justify the necessary investment in developing the drug. In the European Union, orphan drug designation entitles a party to financial incentives such as reduction of fees or fee waivers.

Generally, if a drug with an orphan drug designation subsequently receives the first marketing approval for the indication for which it has such designation, the drug may be entitled to a period of marketing exclusivity, which precludes the FDA or the EMA from approving another marketing application for the same drug and indication for that time period, except in limited circumstances.

Even if we obtain orphan drug exclusivity for a product candidate, that exclusivity may not effectively protect the product candidate from competition because different drugs can be approved for the same condition. Even after an orphan drug is approved, the FDA can subsequently approve a product candidate for the same condition if the FDA concludes that the later drug is clinically superior in that it is shown to be safer, more effective or makes a major contribution to patient care. In addition, a designated orphan drug may not receive orphan drug exclusivity if it is approved for a use that is broader than the indication for which it received orphan designation. Moreover, orphan drug exclusive marketing rights in the United States may be lost if the FDA later 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. Orphan drug designation neither shortens the development time or regulatory review time of a drug nor gives the drug any advantage in the regulatory review or approval process.

There is no guarantee that we will be able to enjoy the benefits associated with orphan drug designation. Further, we may seek orphan drug designation for our other product candidates in the future, however, there is no guarantee that we will obtain any such designation or be able to enjoy the benefits thereof.  

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Although we have received rare pediatric disease designation for LB-001 for the treatment of MMA, we may not be able to obtain such designation for other product candidates or enjoy the benefits associated with this designation.

Under the Rare Pediatric Disease Priority Review Voucher program, the FDA may award a rare pediatric disease priority voucher to a sponsor upon the approval of a BLA or New Drug Application, or NDA, for a product intended to treat or prevent a rare pediatric disease and that also satisfies other criteria. The voucher can then be used to obtain priority review for a subsequent BLA or NDA for a drug or biologic intended to treat any disease or condition, even if the drug or biologic would not otherwise quality for priority review. The voucher recipient also has the option of selling or transferring the voucher to another sponsor On December 27, 2020, Congress passed the Creating Hope Reauthorization Act, which extended the rare pediatric disease priority review voucher program, and after September 30, 2024, the FDA may only award a voucher for an approved rare pediatric disease product application if the sponsor has rare pediatric disease designation for the drug, and that designation was granted by September 30, 2024. After September 30, 2026, the FDA may not award any rare pediatric disease priority review vouchers.

Although rare pediatric disease designation has been granted for LB-001, there is no guarantee that the FDA will grant any future requests for rare pediatric disease designation for any of our product candidates. Moreover, even if we obtain rare pediatric disease designation, there is no guarantee that we will qualify for a rare pediatric disease priority review voucher upon FDA approval of the IND or BLA for a rare pediatric disease. Rare pediatric disease designation does not lead to faster development or regulatory review of the product or increase the likelihood that it will receive marketing approval.

Our current and future business operations will be subject to applicable healthcare regulatory laws, which could expose us to penalties and other sanctions.

We are or may become subject to healthcare regulation and enforcement by various government authorities.  These laws may constrain the business or financial arrangements and relationships through which we conduct our operations, including how we research, market, sell and distribute our product candidates, if approved. In the United States, biopharmaceutical companies and their products are subject to extensive regulation at the federal and state level, including laws intended to prevent fraud and abuse in the healthcare industry. These laws, some of which will apply only if and when we have an approved product, include:  

• the federal anti-kickback statute makes it illegal for any person or entity to knowingly and willfully, directly or indirectly, solicit, receive, offer, or pay any remuneration that is in exchange for or to induce the referral of business, including the purchase, order, lease of any good, facility, item or service for which payment may be made under a federal healthcare program, such as Medicare or Medicaid. The term “remuneration” has been broadly interpreted to include anything of value;

• federal false claims and false statement laws, including the federal civil False Claims Act, prohibits, among other things, any person or entity from knowingly presenting, or causing to be presented, for payment to, or approval by, federal programs, including Medicare and Medicaid, claims for items or services, including drugs, that are false or fraudulent;

• the Health Insurance Portability and Accountability Act of 1996, as amended, or HIPAA, which prohibits executing a scheme to defraud any healthcare benefit program or making false statements relating to healthcare matters and which also imposes certain requirements related to the privacy, security and transmission of individually identifiable health information on certain types of entities, which include many healthcare providers and health plans with which we interact;

• the FDCA, which among other things, strictly regulates drug product and medical device marketing, prohibits manufacturers from marketing such products prior to approval or for unapproved indications and regulates the distribution of samples;

• federal laws, including the Medicaid Drug Rebate Program, that require pharmaceutical manufacturers to report certain calculated product prices to the government or provide certain discounts or rebates to government authorities or private entities, often as a condition of reimbursement under government healthcare programs; federal consumer protection and unfair competition laws, which broadly regulate marketplace activities and activities that potentially harm consumers; and

• the so-called “federal sunshine” law, which requires pharmaceutical and medical device companies to monitor and report certain financial interactions with physicians, non-physician practitioners and teaching hospitals to the federal government for re-disclosure to the public.

Also, many state have similar laws and regulations, such as anti-kickback and false claims laws that may be broader in scope and may apply to claims reimbursed by private payors as well as government programs regardless of reimbursement. Additionally, we may be subject to state laws that require pharmaceutical companies to comply with the federal government’s and/or pharmaceutical industry’s voluntary compliance guidelines, impose specific restrictions on interactions between pharmaceutical companies and healthcare

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providers or require pharmaceutical companies to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures. Other state laws may require pharmaceutical companies to file reports relating to pricing and marketing information and state and local laws that require the registration of pharmaceutical sales representatives. Finally, there are state laws governing the privacy and security of health information, many of which differ from each other in significant ways and often are not preempted by HIPAA. Many of these laws and regulations also contain ambiguous requirements or require administrative guidance for implementation.  

Efforts to ensure that our business arrangements with third parties will comply with applicable healthcare laws and regulations will involve substantial costs. Given the breadth of the laws and regulations, limited guidance for certain laws and regulations and evolving government interpretations of the laws and regulations, governmental authorities may possibly conclude that our business practices may not comply with such laws. If our operations 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, exclusion from government funded healthcare programs, such as Medicare and Medicaid, and the curtailment or restructuring of our operations. Further, defending against any such actions can be costly, time-consuming and may require significant personnel resources. Therefore, even if we are successful in defending against any such actions that may be brought against us, our business may be impaired.

There are also similar healthcare laws and regulations in the European Union and other jurisdictions, including reporting requirements detailing interactions with and payments to healthcare providers. The provision of benefits or advantages to physicians to induce or encourage the prescription, recommendation, endorsement, purchase, supply, order, or use of medicinal products is prohibited in the European Union. Infringement of these laws could result in substantial fines and imprisonment. Payments made to physicians in certain European Union Member States must be publicly disclosed. These requirements are provided in the national laws, industry codes, or professional codes of conduct applicable in the European Union Member States. Failure to comply with these requirements could result in reputational risk, public reprimands, administrative penalties, fines or imprisonment.

We are subject to environmental, health and safety laws and regulations, and we may become exposed to liability and substantial expenses in connection with environmental compliance or remediation activities.

We and any CMOs and other third party vendors we engage are subject to numerous federal, state, and local environmental, health, and safety laws, regulations, and permitting requirements, including those governing laboratory procedures; the generation, handling, use, storage, treatment, and disposal of hazardous and regulated materials and wastes; the emission and discharge of hazardous materials into the ground, air, and water; and employee health and safety. Our operations involve the use of hazardous and flammable materials, including chemicals and biological and radioactive materials. Our operations also produce hazardous waste. We generally contract with third parties for the disposal of these materials and wastes. We cannot eliminate the risk of contamination or injury from these materials. In the event of contamination or injury resulting from our use of hazardous materials, we could be held liable for any resulting damages, and any liability could exceed our resources. Under certain environmental laws, we could be held responsible for costs relating to any contamination at our current or past facilities and at third-party facilities. We also could incur significant costs associated with civil or criminal fines and penalties.

Compliance with applicable environmental laws and regulations may be expensive, and current or future environmental laws and regulations may impair our research and product development efforts. In addition, we cannot entirely eliminate the risk of accidental injury or contamination from these materials or wastes. Although we maintain workers’ compensation insurance to cover us for costs and expenses we may incur due to injuries to our employees resulting from the use of hazardous materials, this insurance may not provide adequate coverage against potential liabilities. We do not carry specific biological or hazardous waste insurance coverage, and our property, casualty, and general liability insurance policies specifically exclude coverage for damages and fines arising from biological or hazardous waste exposure or contamination. Accordingly, in the event of contamination or injury, we could be held liable for damages or be penalized with fines in an amount exceeding our resources, and our clinical trials or regulatory approvals could be suspended, which could harm our business, financial condition, results of operations, and prospects.

In addition, we may incur substantial costs in order to comply with current or future environmental, health, and safety laws, regulations, and permitting requirements. These current or future laws, regulations, and permitting requirements may impair our research, development, or production efforts. Failure to comply with these laws, regulations, and permitting requirements also may result in substantial fines, penalties, or other sanctions or business disruption, which could harm our reputation, business, financial condition, results of operations, and prospects.

Any third-party contract manufacturers and suppliers we engage will also be subject to these and other environmental, health, and safety laws and regulations. Liabilities they incur pursuant to these laws and regulations could result in significant costs or an interruption in operations, which could harm our reputation, business, financial condition, results of operations and prospects.

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We are subject to stringent privacy laws, information security laws, regulations, policies and contractual obligations related to data privacy and security and changes in such laws, regulations, policies and contractual obligations could adversely affect our business.

We are subject to data privacy and protection laws and regulations that apply to the collection, transmission, storage and use of personally-identifying information, which among other things, impose certain requirements relating to the privacy, security and transmission of personal information, including comprehensive regulatory systems in the United States and the EU. The legislative and regulatory landscape for privacy and data protection continues to evolve in jurisdictions worldwide, and there has been an increasing focus on privacy and data protection issues with the potential to affect our business. Failure to comply with any of these laws and regulations could result in enforcement action against us, including fines, imprisonment of company officials and public censure, claims for damages by affected individuals, damage to our reputation and loss of goodwill, any of which could have a material adverse effect on our business, financial condition, results of operations or prospects.

There are numerous U.S. federal and state laws and regulations related to the privacy and security of personal information. For example, the HIPAA and its implementing regulations establish privacy and security standards that limit the use and disclosure of individually identifiable health information, or protected health information, and require the implementation of administrative, physical and technological safeguards to protect the privacy of protected health information and ensure the confidentiality, integrity and availability of electronic protected health information. While we have determined that we are neither a “covered entity” nor a “business associate” directly subject to HIPAA, many of the U.S. health care providers, including U.S. clinical trial sites, with which we interact are subject to HIPAA, and we have assumed contractual obligations related to protecting the privacy of personal information. Determining whether protected health information has been handled in compliance with applicable privacy standards and our contractual obligations can be complex and may be subject to changing interpretation. If we are unable to properly protect the privacy and security of protected health information, we could be found to have breached our contracts and we could face civil and criminal penalties. In addition, our operations have been affected by the California Consumer Privacy Act, or CCPA, which took effect on January 1, 2020. The CCPA gives California consumers (defined to include all California residents) certain rights, including the right to ask covered companies to disclose the types of personal information collected, the categories of sources from which such information was collected, the business purpose for collecting or selling the consumer’s personal information, the categories of third parties with whom a covered company shares personal information, and specific pieces of information collected by a covered company. The CCPA imposes several obligations on covered companies to provide notice to California consumers regarding their data processing activities. The CCPA also gives California consumers the right to ask covered companies to delete a consumer’s personal information and it places limitations on a covered company’s ability to sell personal information, including providing consumers a right to opt out of sales of their personal information. On November 3, 2020, California voters passed a ballot initiative approving the California Privacy Rights Act, or CPRA, which will significantly expand the CCPA to incorporate additional provisions, including a requirement that the use, retention, and sharing of personal information of California residents be reasonably necessary and proportionate to the purposes of collection or processing, granting additional protections for sensitive personal information, and requiring greater disclosures related to notice to residents regarding retention of information.  The CPRA will also expand personal information rights of California residents, including creating a right to opt out of sharing of personal information with third parties for advertising, expanding the lookback period for the right to know about personal information held by businesses, and expanding the right to erasure for information held by third parties.  Most CPRA provisions will take effect on January 1, 2023, though the obligations will apply to any personal information collected after January 1, 2022.  Other states are also enacting comprehensive privacy legislation, including Virginia, Utah and Colorado, each of which passed expansive privacy laws that take effect in 2023.

In addition, we may be subject to privacy and security laws in the various jurisdictions outside of the United States in which we operate, obtain or store personally identifiable information. The legislative and regulatory landscape for privacy and data protection continues to evolve, and there has been an increasing focus on privacy and data protection issues with the potential to affect our business. For example, the processing of personal data in the EEA is subject to the General Data Protection Regulation, or the GDPR, which took effect in May 2018. The GDPR increases obligations with respect to clinical trials conducted in the EEA, such as in relation to the provision of fair processing notices, responding to data subjects who exercise their rights and reporting certain data breaches to regulators and affected individuals. The GDPR also requires us to enter certain contractual arrangements with third parties that process GDPR-covered personal data on our behalf. The GDPR also increases the scrutiny applied to transfers of personal data from the EEA (including from clinical trial sites in the EEA) to countries that are considered by the European Commission to lack an adequate level of data protection, such as the United States. The July 2020 invalidation by the Court of Justice of the European Union of the EU-U.S. Privacy Shield framework, one of the mechanisms used to legitimize the transfer of personal data from the EEA to the United States, has led to increased scrutiny on data transfers from the EEA to the United States generally and may increase our costs of compliance with data privacy legislation. If our or our partners’ or service providers’ privacy or data security measures fail to comply with the GDPR requirements, we may be subject to litigation, regulatory investigations, enforcement notices requiring us to change the way we use personal data and/or fines of up to 20 million Euros or up to 4% of the total worldwide annual turnover of the preceding financial year, whichever is higher, as well as claims by affected individuals, negative publicity, reputational harm and a potential loss of business and goodwill.

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Data privacy remains an evolving landscape at both the domestic and international level, with new regulations coming into effect and continued legal challenges, and our ongoing efforts to comply with evolving laws and regulations may be costly and require ongoing modifications to our policies, procedures and systems. Our efforts to comply may also be unsuccessful. It is possible that these laws may be interpreted and applied in a manner that is inconsistent with our practices. Failure to comply with laws regarding data protection would expose us to risk of enforcement actions taken by data protection authorities in the EU and elsewhere and carries with it the potential for significant penalties if we are found to be non-compliant as well as private lawsuits by affected data subjects. Similarly, failure to comply with federal and state laws in the United States regarding privacy and security of personal information could expose us to penalties under such laws and private lawsuits brought by data subjects. Any such failure to comply with data protection and privacy laws could result in government-imposed fines or orders requiring that we change our practices, claims for damages by data subjects, regulatory investigations and enforcement action, litigation and significant costs for remediation, any of which could adversely affect our business. Even if we are not determined to have violated these laws, government investigations into these issues typically require the expenditure of significant resources and generate negative publicity, which could harm our business, financial condition, results of operations or prospects.

Risks Related to Employee Matters and Managing Growth

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

Our industry has experienced a high rate of turnover of management personnel in recent years. We are highly dependent on the development, regulatory, commercialization and business development expertise of Frederic Chereau, our President and Chief Executive Officer, as well as the other members of our management, scientific, clinical and technical teams. Although we have formal employment agreements with our executive officers, these agreements do not prevent them from terminating their employment with us at any time. We do not maintain “key person” insurance for any of our executives or other employees. The loss of the services of one or more of our executive officers or key employees could impede the achievement of our research, development and commercialization objectives, impact our ability to coordinate responsibilities and tasks, impact our corporate culture, and impact the retention of historical knowledge. Further, our Board of Directors and management succession planning efforts may not be effective, which could adversely impact our business.  

If we lose one or more of our executive officers or key employees, our ability to implement our business strategy successfully could be seriously harmed. Furthermore, replacing executive officers and key employees may be difficult and may take an extended period of time because of the limited number of individuals in our industry with the breadth of skills and experience required to develop, gain regulatory approval of and commercialize product candidates successfully. Competition to hire from this limited pool is intense, and we may be unable to hire, train, retain or motivate these additional key personnel on acceptable terms given the competition among numerous pharmaceutical and biotechnology companies for similar personnel, as well as from universities and research institutions. In addition, we rely on consultants and advisors, including scientific, technical, clinical and regulatory advisors, to assist us in formulating our research and development and commercialization strategy. Our consultants and advisors may be engaged by entities other than us and may have commitments under consulting or advisory contracts with other entities that may limit their availability to us. If we are unable to continue to attract and retain high quality personnel, our ability to develop and commercialize product candidates will be limited.

We may encounter difficulties in managing any growth, which could disrupt our operations.

We have experienced significant growth during periods in the past years. Our management has diverted, and may need to divert, a disproportionate amount of its attention away from our day-to-day activities to devote time to managing our growth. To manage any growth, we must continue to implement and improve our managerial, operational and financial systems, expand our facilities and continue to train additional qualified personnel. Due to our limited financial resources, we may not be able to effectively manage any expansion of our operations or train the additional qualified personnel. Our inability to effectively manage any expansion of our operations may result in weaknesses in our infrastructure, give rise to operational mistakes, loss of business opportunities, loss of employees and reduced productivity among remaining employees. Managing any growth could require significant capital expenditures and may divert financial resources from other projects, such as the development of additional product candidates and technologies. If our management is unable to effectively manage any growth, our expenses may increase more than expected, our potential ability to generate revenue could be reduced and we may not be able to implement our business strategy.

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Product liability lawsuits against us could cause us to incur substantial liabilities and could limit commercialization of any product candidates that we may develop.

We face an inherent risk of product liability exposure related to the testing of our product candidates in clinical trials and may face an even greater risk if we commercialize any products that we may develop. If we cannot successfully defend ourselves against claims that our product candidates caused injuries, we could incur substantial liabilities. Regardless of merit or eventual outcome, liability claims may result in:

• decreased demand for any product candidates or technologies that we may develop;

• loss of revenue;

• substantial monetary awards to trial participants or patients;

• significant time and costs to defend the related litigation;

• withdrawal of clinical trial participants;

• the inability to commercialize any product candidates or technologies that we may develop; and

• injury to our reputation and significant negative media attention.

Our insurance coverage may not be adequate to cover all liabilities that we may incur. We anticipate that we will need to increase our insurance coverage each time we commence a clinical trial and if we successfully commercialize any product candidate. 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.

We or the third parties upon whom we depend may be adversely affected by natural disasters or geo-political events and our business continuity and disaster recovery plans may not adequately protect us from a serious disaster.

Natural disasters and geo-political events (such as war or terrorism) could severely disrupt our operations or the operations of third parties upon whom we depend. If any such event occurred that prevented us from using all or a significant portion of our headquarters, that damaged critical infrastructure, such as our manufacturing facilities, or that otherwise disrupted operations, or similarly affected third parties whom we rely on, it may be difficult or, in certain cases, impossible for us to continue our business for a substantial period of time. The disaster recovery and business continuity plans we have in place may prove inadequate in the event of a serious disaster or similar event. We may incur substantial expenses as a result of the limited nature of our disaster recovery and business continuity plans, which could harm our business.

Risks Related to Commercialization

We face significant competition in an environment of rapid technological change, and there is a possibility that our competitors may achieve regulatory approval before us or develop therapies that are safer or more advanced or effective than ours, which may harm our financial condition and our ability to successfully market or commercialize any product candidates or technologies we may develop.

The development and commercialization of new genetic medicine products is highly competitive. Moreover, the gene editing field is characterized by rapidly changing technologies, significant competition and a strong emphasis on intellectual property. We will face competition with respect to any product candidates or technologies that we may seek to develop or commercialize in the future from major pharmaceutical companies, specialty pharmaceutical companies and biotechnology companies worldwide. 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.

Our focus is on the development of genetic medicines using our GeneRide and sAAVy technologies. If our current programs are approved for the indications we are pursuing or contemplate we may pursue, they may compete with other products currently under development, including gene editing and gene therapy products. There are a number of companies developing nuclease-based gene editing technologies using CRISPR/Cas9, TALENs, meganucleases, Mega-TALs and ZFNs, including bluebird bio, Caribou Biosciences, CRISPR Therapeutics, Editas Medicine, Intellia Therapeutics, Sangamo Therapeutics, Precision BioSciences and Graphite Bio. We may also compete with companies developing gene therapy products, including Homology Medicines, Astellas Pharma, Pfizer, bluebird bio, uniQure and Generation Bio. There are also companies pursuing base editing technologies, including Beam Therapeutics. Any products we may develop could also face competition from other products approved to treat the same disease based on other types of therapies, such as small molecule, antibody or protein therapies. There are several companies developing

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competing products that target MMA, the indication for which we are developing LB-001. These companies include Moderna Therapeutics with an mRNA-based approach, Selecta Biosciences using an AAV gene therapy, and HemoShear Therapeutics using a small molecule. If any of our competitors obtains regulatory approval for a treatment for MMA, it could negatively affect our ability to successfully commercialize LB-001, if approved. There are several companies developing competing products that target CN, the indication for which we conducted research activities for LB-301, including Genethon, Selecta Biosciences, Moderna Therapeutics, and the Institute for Life Changing Medicines. If any of our competitors obtain regulatory approval for a treatment for CN, it could negatively affect our ability to successfully commercialize LB-301. Additionally, other companies may have or be developing competing products that target HT1, the indication for which we are developing LB-401. If any of our competitors obtain regulatory approval for a treatment for HT1, it could negatively affect our ability to successfully commercialize LB-401.

Many of our current or potential competitors, either alone or with their collaboration partners, have significantly greater financial resources and expertise in research and development, manufacturing, preclinical testing, conducting clinical trials and marketing approved products than we do. Mergers and acquisitions in the pharmaceutical, biotechnology and gene therapy industries may result in even more resources being concentrated among a smaller number of our competitors. Smaller or early-stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large and established companies. These competitors also compete with us in recruiting and retaining qualified scientific and management personnel and establishing clinical trial sites and patient registration for clinical trials, as well as in acquiring technologies complementary to, or necessary for, our programs. Our commercial opportunity could be reduced or eliminated if our competitors develop and commercialize products that are safer or more effective, have fewer or less severe side effects, are more convenient, or are less expensive than any products that we may develop. Our competitors also may obtain FDA or other regulatory approval for their products more rapidly than we may obtain approval for ours, which could result in our competitors establishing a strong market position before we are able to enter the market. Additionally, technologies developed by our competitors may render our potential product candidates or technologies uneconomic or obsolete, and we may not be successful in marketing any product candidates or technologies we may develop against competitors.

In addition, as a result of the expiration or successful challenge of our patent rights, we could face more litigation with respect to the validity and/or scope of patents relating to our competitors’ products. The availability of our competitors’ products could limit the demand, and the price we are able to charge, for any products that we may develop and commercialize.

The successful commercialization of our product candidates will depend in part on the extent to which third-party payors, including government healthcare programs as well as private health insurance, establish favorable coverage and reimbursement for our product candidates, if approved.

The availability and adequacy of coverage and reimbursement by third-party payors, including governmental healthcare programs such as Medicare and Medicaid, as well as private health insurance, will likely be essential for most patients to be able to afford our product candidates, assuming regulatory approval. Our initial target patient populations are relatively small and costs associated with the development and commercialization of our genomic medicine product candidates are substantial. We therefore expect the cost of a single administration of our products to be significant if and when approved. Our ability to achieve acceptable levels of coverage and reimbursement for such products and services required to administer such products by third-party payors will likely have a significant impact on our ability to successfully commercialize our product candidates. We cannot be sure that coverage and reimbursement in the United States, the European Union or elsewhere will be available for our product candidates or any product that we may develop, and any reimbursement that may become available may be decreased or eliminated in the future.

There is significant uncertainty related to third party payor coverage and reimbursement of newly-approved products. The regulations that govern marketing approvals, pricing, and reimbursement for new medicines vary widely from country to country. Obtaining coverage and reimbursement for a product from third-party payors is a time-consuming and costly process that could require us to provide scientific and clinical support for the use of our product candidates to each payor separately, with no assurance that coverage and adequate reimbursement will be applied consistently or obtained in the first instance. We may not be able to provide data sufficient to gain acceptance with respect to coverage and reimbursement. We therefore cannot predict at this time what third-party payors will decide with respect to the coverage and reimbursement for our product candidates. Even if we obtain coverage and adequate reimbursement for our product candidates with respect to a particular third-party payor, there is no certainty that coverage will be maintained or reimbursement rates will not change.

Within the United States, no uniform policy for coverage and reimbursement for products exists among third-party payors. Coverage and reimbursement for products can differ significantly from payor to payor and coverage and reimbursement by one payor does not guarantee coverage and reimbursement by another payor. Third-party payors increasingly are limiting coverage and utilization of pharmaceutical products and challenging prices charged for pharmaceutical products and services. Assuming we obtain coverage for a product by a third-party payor, the third-party payor may implement utilization management controls, such as requiring pre-approval before our product will be covered for a particular patient, which may limit access to our product. In addition, the reimbursement rates may not be adequate or may require co-payments that patients find unacceptably high. Net prices for our products may be reduced by

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mandatory discounts or rebates that we are required to provide to certain government healthcare programs or private payors or by discounts we negotiate with third party payors. In addition, given the novel nature of our product candidates, we may need to develop new reimbursement models in order to realize adequate value. Even if we are able to develop such models, third-party payors may not be able or willing to adopt such new models, and patients may be unable to afford that portion of the cost that such models may require them to bear. If coverage is limited, access to our products is subject to utilization management controls or reimbursement is inadequate, we may not be able to successfully commercialize our product candidates, and may not be able to obtain a satisfactory financial return on our product candidates.

Outside the United States, international operations are generally subject to extensive governmental price controls and other market regulations, and we believe the increasing emphasis on cost-containment initiatives in Europe and other countries have and will continue to put pressure on the pricing and usage of our product candidates. In many countries, the prices of medical products are subject to varying price control mechanisms as part of national health systems. Other countries allow companies to fix their own prices for medical products, but monitor and control company profits. Additional foreign price controls or other changes in pricing regulation could restrict the amount that we are able to charge for our product candidates. Accordingly, in markets outside the United States, the reimbursement for our product candidates may be reduced compared with the United States and may be insufficient to generate commercially-reasonable revenue and profits. Furthermore, some countries require approval of the sale price of a medicine before it can be marketed. In many countries, the pricing review period begins after marketing or product licensing approval is granted. In some foreign markets, prescription pharmaceutical pricing remains subject to continuing governmental control even after initial approval is granted. As a result, we might obtain marketing approval for a medicine in a particular country, but then be subject to price regulations that delay our commercial launch of the medicine, possibly for lengthy time periods, and negatively impact the revenues we are able to generate from the sale of the medicine in that country. Adverse pricing limitations may hinder our ability to recoup our investment in one or more product candidates, even if any product candidates we may develop obtain marketing approval.

If the market opportunities for any product candidates we may develop are smaller than we believe they are, our revenues, if any, may be adversely affected, and our business may suffer. Because the target patient populations for many of the product candidates we may develop are small, we must be able to successfully identify patients and achieve a significant market share to achieve and maintain profitability and growth.

We currently focus our research and product development on treatments for rare pediatric diseases. Our projections 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 product candidates we may develop, are based on estimates. These estimates may prove to be incorrect and new studies may change the estimated incidence or prevalence of these diseases. The number of patients in the United States, Europe and elsewhere may turn out to be lower than expected, and patients may not be amenable to treatment with our products, or may become increasingly difficult to identify or gain access to, all of which could harm our business, financial condition, results of operations, and prospects.

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

If LB-001 or any other product candidate we develop is approved for commercialization, it may be marketed in certain jurisdictions outside the United States. We expect that we will be subject to additional risks related to international pharmaceutical operations, including:

• economic weakness, including inflation, or political instability in particular non-U.S. economies and markets;

• different regulatory requirements for drug and biologic approvals and rules governing drug and biologic commercialization in foreign countries;

• reduced protection for intellectual property rights;

• difficulties in compliance with non-U.S. laws and regulations;

• changes in non-U.S. regulations and customs, tariffs and trade barriers;

• changes in non-U.S. currency exchange rates and currency controls;

• changes in a specific country’s or region’s political or economic environment;

• trade protection measures, import or export licensing requirements or other restrictive actions by U.S. or non-U.S. governments;

• negative consequences from changes in tax laws;

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• foreign reimbursement, pricing and insurance regimes;

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

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

• difficulties associated with staffing and managing international operations, including differing labor relations;

• potential noncompliance with the U.S. Foreign Corrupt Practices Act of 1977, as amended, or the FCPA, the U.K. Bribery Act 2010 and similar anti-bribery and anticorruption laws in other jurisdictions;

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

• business interruptions resulting from geo-political events, including war and terrorism, or natural disasters including floods and fires.

We have no prior experience in these areas. In addition, there are complex regulatory, tax, labor and other legal requirements imposed by both the European Union, many of the individual countries in Europe and other countries outside of Europe with which we will need to comply. Many U.S.-based biotechnology companies have found the process of marketing their own products outside the United States to be very challenging.

We are subject to U.S. and certain foreign export and import controls, sanctions, embargoes, anti-corruption laws, and anti-money laundering laws and regulations. Compliance with these legal standards could impair our ability to compete in domestic and international markets. We can face criminal liability and other serious consequences for violations, which can harm our business.

We are subject to export control and import laws and regulations, including the U.S. Export Administration Regulations, U.S. Customs regulations, various economic and trade sanctions regulations administered by the U.S. Treasury Department’s Office of Foreign Assets Controls, the FCPA, the U.S. domestic bribery statute contained in 18 U.S.C. §201, the U.S. Travel Act, the USA PATRIOT Act, and other state and national anti-bribery and anti-money laundering laws in the countries in which we conduct activities. Anti-corruption laws are interpreted broadly and prohibit companies and their employees, agents, contractors, and other collaborators from authorizing, promising, offering, or providing, directly or indirectly, improper payments or anything else of value to recipients in the public or private sector. We may engage third parties to sell our products sell our products outside the United States, to conduct clinical trials, and/or to obtain necessary permits, licenses, patent registrations, and other regulatory approvals. We have direct or indirect interactions with officials and employees of government agencies or government-affiliated hospitals, universities, and other organizations. We can be held liable for the corrupt or other illegal activities of our employees, agents, contractors, and other collaborators, even if we do not explicitly authorize or have actual knowledge of such activities. Any violations of the laws and regulations described above may result in substantial civil and criminal fines and penalties, imprisonment, the loss of export or import privileges, debarment, tax reassessments, breach of contract and fraud litigation, reputational harm, and other consequences.

Risks Related to Our Common Stock and Indebtedness

The market price of our common stock may be volatile and fluctuate substantially, which could result in substantial losses for purchasers of our common stock.

Our stock price may be volatile. The stock market in general, and the market for pharmaceutical and biopharmaceutical companies in particular, has experienced extreme price and volume fluctuations that have often been unrelated or disproportionate to changes in the operating performance of companies whose stock is experiencing those price and volume limitations. Broad market and industry factors may seriously affect the market price of our common stock, regardless of our actual performance. As a result of this volatility, our stockholders may not be able to sell their shares of common stock at or above the price at which they purchased them. The market price for our common stock may be influenced by many factors, including:

• results of or developments with respect to our preclinical studies,clinical trials or regulatory approvals or those with respect to product candidates of our competitors, or announcements about new research programs or product candidates of ours or our competitors;

• the success of competitive products or technologies;

• actual or expected changes in our growth rate relative to our competitors;

• developments related to our collaborations;

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• regulatory actions with respect to any product candidate we may develop or our competitors’ products and product candidates;

• regulatory or legal developments in the United States and other countries;

• development of new product candidates or technologies that may address our markets and make our product candidates or technologies less attractive;

• failure or discontinuation of any of our product development and research programs;

• changes in physician, hospital or healthcare provider practices that may make our product candidates or technologies less useful;

• announcements by us, our partners or our competitors of significant acquisitions, strategic partnerships, joint ventures, collaborations or capital commitments;

• 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, technologies or clinical development programs;

• failure to meet or exceed financial estimates and projections of the investment community or that we provide to the public;

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

• actual or expected changes in estimates as to financial results, development timelines or recommendations by securities analysts; variations in our financial results, development timelines, or recommendations by securities analysts, or those of companies that are perceived to be similar to us;

• announcement or expectation of additional financing efforts;

• sales of our common stock by us, our insiders, or other stockholders;

• limited public float;

• expiration of market stand-off or lock-up agreement;

• changes in the structure of healthcare payment systems;

• market conditions in the pharmaceutical and biotechnology sectors;

• developments or changing views regarding the use of genomic medicines, including those that involve genome editing;

• general economic, industry and market conditions; and

• the other factors described in this “Risk Factors” section and others beyond our control.

The terms of our Loan and Security Agreement place restrictions on our operating and financial flexibility.

In July 2019, we entered into the Loan Agreement, under which term loans in an aggregate principal amount of $20.0 million were made available to us in two tranches, subject to certain terms and conditions. As of September 30, 2022, we had drawn down the $10.0 million first tranche. In the second quarter of 2021, we met the conditions to initiate drawdown of the $10.0 million second tranche but did not exercise our right to do so and the option to draw down the second tranche of the Term Loans expired. See Note 5 to our financial statements found elsewhere in this Quarterly Report on Form 10-Q. The Loan Agreement contains representations and warranties, affirmative and negative covenants applicable to us and our subsidiaries and events of default, as more fully described in the Loan Agreement. The affirmative covenants include, among others, covenants requiring us and our subsidiaries to maintain our legal existence and material governmental approvals, deliver certain financial reports, maintain insurance coverage and maintain certain cash balances in controlled accounts. The negative covenants include, among others, changes in business, management, ownership or business locations, mergers or acquisitions, indebtedness, encumbrances, maintenance of collateral accounts, distributions, investments, transactions with affiliates and subordinated debt.

The Loan Agreement also includes events of default, the occurrence and continuation of which provide Oxford Finance LLC, as collateral agent, with the right to exercise remedies against us and the collateral securing the loans under the Loan Agreement, including foreclosure against our properties securing the Loan Agreement, including our cash, potentially requiring us to renegotiate our agreement on terms less favorable to us or to immediately cease operations. These events of default include, among other things, the nonpayment of principal, interest, and other amounts when due, breach of of covenants, a material adverse change event, cross-defaults on material indebtedness, misrepresentations, material judgments, bankruptcy and delisting.

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Further, if we are liquidated, the Lenders’ right to repayment would be senior to the rights of the holders of our common stock to receive any proceeds from the liquidation. The lenders could declare a default upon the occurrence of any event that they interpret as a material adverse change as defined under the Loan Agreement, thereby requiring us to repay the loan immediately or to attempt to reverse the declaration of default through negotiation or litigation. Any declaration by the lenders of an event of default could significantly harm our business and prospects and could cause the price of our common stock to decline.

Our principal stockholders and management own a significant percentage of our stock and are able to exercise significant influence over matters subject to stockholder approval.

As of September 30, 2022, our executive officers and directors, combined with our stockholders who reported owning more than 5% of our common stock in SEC filings, together with their respective affiliates, beneficially owned a significant percentage of our outstanding common stock, including shares subject to outstanding options that are exercisable within 60 days after such date. Accordingly, these stockholders are able to exert a significant degree of influence over our management and affairs and over matters requiring stockholder approval, including the election of our Board of Directors and approval of significant corporate transactions. This concentration of ownership could have the effect of entrenching our management or Board of Directors, delaying or preventing a change in control or otherwise discouraging a potential acquirer from attempting to obtain control of us, which in turn could have a material and adverse effect on the fair market value of our common stock.

We are an “emerging growth company” and a “smaller reporting company,” and the reduced disclosure requirements applicable to emerging growth companies and smaller reporting companies may make our common stock less attractive to investors.

We are an “emerging growth company,” as defined in the Jumpstart Our Business Startups Act of 2012, or the JOBS Act, and may remain an emerging growth company until December 31, 2023. For so long as we remain an emerging growth company, we are permitted and plan to rely on exemptions from certain disclosure requirements that are applicable to other public companies that are not emerging growth companies. However, if certain events occur prior to December 31, 2023, including if we become a “large accelerated filer,” our annual gross revenues exceed $1.235 billion or we issue more than $1.0 billion of non-convertible debt in any three-year period, we will cease to be an emerging growth company prior to December 31, 2023. For so long as we remain an emerging growth company, we are permitted and intend to rely on exemptions from certain disclosure requirements that are applicable to other public companies that are not emerging growth companies. These exemptions include:

• not being required to comply with the auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act of 2002, or SOX Section 404, in the assessment of our internal control over financial reporting;

• not being required to comply with any requirement that may be adopted by the Public Company Accounting Oversight Board regarding mandatory audit firm rotation or a supplement to the auditor’s report providing additional information about the audit and the financial statements;

• reduced disclosure obligations regarding executive compensation; and

• exemptions from the requirements of holding a nonbinding advisory vote on executive compensation and golden parachute payments.

We cannot predict whether investors will find our common stock less attractive if we rely on these exemptions. If some investors find our common stock less attractive as a result, there may be a less active trading market for our common stock and our stock price may be reduced or more volatile. Under the JOBS Act, emerging growth companies can also delay adopting new or revised accounting standards until such time as those standards apply to private companies. We have irrevocably elected to “opt out” of this exemption from new or revised accounting standards and, therefore, will be subject to the same new or revised accounting standards as other public companies that are not emerging growth companies. As a result, changes in rules of U.S. GAAP or their interpretation, the adoption of new guidance, or the application of existing guidance to changes in our business could significantly affect our financial position and results of operations.

Further, even after we no longer qualify as an emerging growth company, we may still qualify as a “smaller reporting company,” which would allow us to take advantage of many of the same exemptions from disclosure requirements, including reduced disclosure obligations regarding executive compensation in our periodic reports and proxy statements. In addition, if we are a smaller reporting company, we would not be required to comply with the auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act, or Section 404. These exemptions and reduced disclosures in our SEC filings due to our status as a smaller reporting company may make it harder for investors to analyze our results of operations and financial prospects. Investors may find our common stock less attractive because we may rely on these exemptions. If some investors find our common stock less attractive as a result, there may be a less active trading market for our common stock and our stock prices may be more volatile.

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Provisions in our restated certificate of incorporation and restated by-laws and under Delaware law could make an acquisition of our company, which may be beneficial to our stockholders, more difficult and may prevent attempts by our stockholders to replace or remove our current management.

Provisions in our restated certificate of incorporation and our restated by-laws may discourage, delay or prevent a merger, acquisition or other change in control of our company that stockholders may consider favorable, including transactions in which our stockholders might otherwise receive a premium for their shares. These provisions could also limit the price that investors might be willing to pay in the future for shares of our common stock, thereby depressing the market price of our common stock. In addition, because our Board of Directors is responsible for appointing the members of our management team, these provisions may frustrate or prevent any attempts by our stockholders to replace or remove our current management by making it more difficult for stockholders to replace members of our Board of Directors. Among other things, our restated certificate of incorporation and our restated by-laws include provisions that:

• authorize “blank check” preferred stock, which could be issued by our Board of Directors without stockholder approval and may contain voting, liquidation, dividend and other rights superior to our common stock;

• create a classified Board of Directors whose members serve staggered three-year terms;

• specify that special meetings of our stockholders can be called only by our Board of Directors;

• prohibit stockholder action by written consent;

• establish an advance notice procedure for stockholder approvals to be brought before an annual meeting of our stockholders, including proposed nominations of persons for election to our Board of Directors;

• provide that vacancies on our Board of Directors may be filled only by a majority of directors then in office, even though less than a quorum;

• provide that our directors may be removed only for cause;

• specify that no stockholder is permitted to cumulate votes at any election of directors;

• expressly authorized our Board of Directors to modify, alter or repeal our amended and restated by-laws; and

• require supermajority votes of the holders of our common stock to amend specified provisions of our amended and restated certificate of incorporation and amended and restated by-laws.

Moreover, because we are incorporated in Delaware, we are governed by the provisions of Section 203 of the DGCL, which prohibits a person who owns in excess of 15% of our outstanding voting stock from merging or combining with us for a period of three years after the date of the transaction in which the person acquired in excess of 15% of our outstanding voting stock, unless the merger or combination is approved in a prescribed manner.

The existence of the foregoing provisions and anti-takeover measures could limit the price that investors might be willing to pay in the future for shares of our common stock. They could also deter potential acquirers of our company, thereby reducing the likelihood that our stockholders could receive a premium for their common stock in an acquisition.

Our ability to use net operating loss carryforwards and certain other tax attributes may be subject to certain limitations.

We have incurred substantial losses during our history and anticipate that we will continue to incur significant losses for the foreseeable future; thus, we do not know whether or when we will generate the U.S. taxable income necessary to utilize our net operating loss carryforwards, or NOLs. To the extent that we continue to generate taxable losses, unused losses will carry forward to offset a portion of future taxable income, if any, until such unused losses expire, if ever. Under Sections 382 and 383 of the Internal Revenue Code of 1986, as amended, if a corporation undergoes an “ownership change”, generally defined as a greater than 50 percentage point change (by value) in its equity ownership by certain stockholders over a three-year period, the corporation’s ability to use its pre-change NOLs and other pre-change tax attributes (such as research and development tax credits) to offset its post-change income or taxes may be limited. We may have experienced ownership changes in the past and may experience ownership changes in the future as a result of subsequent shifts in our stock ownership (some of which shifts are outside our control). As a result, if we earn net taxable income, our ability to use our pre-change NOLs to offset such taxable income could be subject to limitations. Similar provisions of state tax law may also apply. As a result, even if we achieve profitability, we may be unable to use a material portion of our NOLs and other tax attributes.

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Because we do not anticipate paying any cash dividends on our common stock in the foreseeable future, capital appreciation, if any, would be the sole source of gain for our stockholders.

We have never declared or paid any cash dividends on our common stock. We currently anticipate that we will retain future earnings for the development, operation and expansion of our business and do not anticipate declaring or paying any cash dividends for the foreseeable future. In addition, any future credit facility may contain terms prohibiting or limiting the amount of dividends that may be declared or paid on our common stock. As a result, capital appreciation, if any, of our common stock would be the sole source of gain on an investment in our common stock for the foreseeable future.

Our certificate of incorporation and bylaws designate the state or federal courts in the State of Delaware and the federal district courts of the United States, respectively, as the exclusive forum for certain types of actions and proceedings that may be initiated by our stockholders, which could limit our stockholders’ ability to obtain a favorable judicial forum for disputes with us or our directors, officers or employees.

Our certificate of incorporation provides that, unless our Board of Directors otherwise determines, the state courts in the State of Delaware or, if no state court located within the State of Delaware has jurisdiction, the federal court for the District of Delaware, will be the sole and exclusive forum for any derivative action or proceeding brought on our behalf, any action asserting a claim of breach of a fiduciary duty owed by any of our directors or officers to our company or our stockholders, any action asserting a claim against us or any of our directors or officers arising pursuant to any provision of the DGCL or our certificate of incorporation or by-laws, or any action asserting a claim against us or any of our directors or officers governed by the internal affairs doctrine. Additionally, our bylaws provide that the federal district courts of the United States will be the exclusive forum for resolving any complaint asserting a cause of action arising under the Securities Act. These exclusive forum provisions may limit the ability of our stockholders to bring a claim in a judicial forum that such stockholders find favorable for disputes with us or our directors or officers, which may discourage such lawsuits against us and our directors and officers.

We could be subject to securities class action litigation.

In the past, securities class action litigation has often been brought against a company following a decline in the market price of its securities. This risk is especially relevant for us because biopharmaceutical companies have experienced significant stock price volatility in recent years. If we face such litigation, it could result in substantial costs and a diversion of management’s attention and resources, which could harm our business.

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Item 6. Exhibits.

EXHIBIT 2.1	—	Agreement and Plan of Merger, dated October 3, 2022, by and among LogicBio Therapeutics, Inc., Alexion Pharmaceuticals, Inc. and Camelot Merger Sub, Inc. (incorporated by reference to Exhibit 2.1 to the Current Report on Form 8-K of LogicBio Therapeutics, Inc. filed with the SEC on October 3, 2022)
EXHIBIT 3.1	—	Fourth Amended and Restated Certificate of Incorporation (incorporated by reference to Exhibit 3.1 to the Company’s Current Report on Form 8-K, File No. 001-38707, filed on October 29, 2018).
EXHIBIT 3.2	—	Amended and Restated Bylaws (incorporated by reference to Exhibit 3.2 to the Company’s Current Report on Form 8-K, File No. 001-38707, filed on October 29, 2018).
EXHIBIT 10.1	—	Consulting Agreement by and between Cecilia Jones and LogicBio Therapeutics, Inc., effective as of September 23, 2022 (incorporated by reference to Exhibit 10.1 to the Company’s Current Report on Form 8-K, filed on September 23, 2022).
EXHIBIT 10.2+*	—	Amended and Restated Exclusive Research Collaboration, License and Option Agreement, dated as of October 2, 2022, between LogicBio Therapeutics, Inc. and CANbridge Care Pharma Hong Kong Limited
EXHIBIT 10.3	—	Tender and Support Agreement, dated as of October 3, 2022, by and among Alexion Pharmaceuticals, Inc., Camelot Merger Sub, Inc., OrbiMed Israel Partners II, L.P., OrbiMed Private Investments VI, L.P., OrbiMed Genesis Master Fund, L.P. and The Biotech Growth Trust PLC. (incorporated by reference to Exhibit 99.1 to the Current Report on Form 8-K of LogicBio Therapeutics, Inc. filed with the SEC on October 3, 2022)
EXHIBIT 10.4	—	Tender and Support Agreement, dated as of October 3, 2022, by and among Alexion Pharmaceuticals, Inc., Camelot Merger Sub, Inc. and BioDiscovery 5 (incorporated by reference to Exhibit 99.2 to the Current Report on Form 8-K of LogicBio Therapeutics, Inc. filed with the SEC on October 3, 2022).
EXHIBIT 31.1*	—	Rule 13a—14(a) / 15d—14(a) Certifications — Chief Executive Officer.
EXHIBIT 31.2*	—	Rule 13a—14(a) / 15d—14(a) Certifications — Chief Financial Officer.
EXHIBIT 32.1**	—	Section 1350 Certifications.
EXHIBIT 101.INS*	—	Inline XBRL Instance Document. The instance document does not appear in the Interactive Data File because its XBRL tags are embedded within the Inline XBRL document.
EXHIBIT 101.SCH*	—	Inline XBRL Taxonomy Extension Schema Document.
EXHIBIT 101.CAL*	—	Inline XBRL Taxonomy Extension Calculation Linkbase Document.
EXHIBIT 101.DEF*	—	Inline XBRL Taxonomy Extension Definition Linkbase Document.
EXHIBIT 101.LAB*	—	Inline XBRL Taxonomy Extension Label Linkbase Document.
EXHIBIT 101.PRE*	—	Inline XBRL Taxonomy Extension Presentation Linkbase Document.
EXHIBIT 104*	—	Cover Page Interactive Data File (embedded within the Inline XBRL document)

* Filed herewith.

** Furnished herewith.

+ Portions of this exhibit have been omitted pursuant to Item 601(b)(10)(iv) of Regulation S-K.

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SIGNATURES

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

	LogicBio Therapeutics, Inc.
Dated: November 14, 2022	By:	/s/ Frederic Chereau
		Frederic Chereau
		President and Chief Executive Officer
Dated: November 14, 2022	By:	/s/ Josh Blacher
		Josh Blacher
		Interim Chief Financial Officer

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EX-10.2

Exhibit 10.2

CERTAIN CONFIDENTIAL INFORMATION CONTAINED IN THIS DOCUMENT, MARKED BY [***], HAS BEEN OMITTED BECAUSE IT IS NOT MATERIAL AND IS THE TYPE OF INFORMATION THAT LOGICBIO THERAPEUTICS, INC. TREATS AS PRIVATE OR CONFIDENTIAL

AMENDED AND RESTATED

EXCLUSIVE RESEARCH COLLABORATION, LICENSE AND OPTION AGREEMENT

BETWEEN

LOGICBIO THERAPEUTICS, INC.

AND

CANBRIDGE CARE PHARMA HONG KONG LIMITED

Dated October 2, 2022

i

Schedules and Exhibits

Schedule 1.69Existing In-License Agreements

Schedule 1.114LogicBio Licensed Patent Rights

Schedule 2.2.1Permitted Subcontractors

Schedule 7.2Disclosure Schedule

Schedule 7.2.15Manufacturing Agreements

Exhibit ATechnology Transfer Requirements

Exhibit BPompe and Fabry Research Plan

ii

AMENDED AND RESTATED

EXCLUSIVE RESEARCH COLLABORATION, LICENSE AND OPTION AGREEMENT

This Amended and Restated Exclusive Research Collaboration, License and Option Agreement (this “Agreement”) is entered into as of October 2, 2022 (the “Restatement Date”) by and between CANbridge Care Pharma Hong Kong Limited, a Hong Kong limited liability company, having its registered address at FLAT/RM A 12/F, ZJ300, 300 Lockhart Road, Wan Chai, Hong Kong (“CANbridge”) and LogicBio Therapeutics, Inc., a Delaware corporation having its principal place of business at 65 Hayden Ave., Floor 2, Lexington, MA 02421, United States (“LogicBio”).  CANbridge and LogicBio are sometimes referred to herein individually as a “Party” and collectively as the “Parties.”

RECITALS

WHEREAS, the Parties entered into that certain Exclusive Research Collaboration, License and Option Agreement dated April 26, 2021 (such date is the “Effective Date” and such agreement is the “Original Agreement”); and

WHEREAS, the Parties now desire to amend and restate the Original Agreement on the terms set forth herein.

NOW, THEREFORE, the Parties hereto agree as follows:

Article 1
DEFINITIONS

1.1 “Accounting Standards” means United States generally accepted accounting principles or International Financial Reporting Standards, as applicable, which principles or standards are currently used at the relevant time and consistently applied by the applicable Party.

1.2 “Acquirer” has the meaning given in Section 2.6.2 (Effect of Change of Control on Exclusivity).

1.3 “Acquisition Transaction” has the meaning set forth in Section 2.6.3 (Acquisition of Competing Product).

1.4 “Adverse Event” means any untoward medical occurrence in a human clinical study subject or in a patient who is administered a product, whether or not considered related to such product, including any undesirable sign (including abnormal laboratory findings of clinical concern), symptom, or disease associated with the use of a product.

1.5 “Affiliate” means any Person directly or indirectly controlled by, controlling, or under common control with, a Party, but only for so long as such control continues.  For purposes of this definition, “control” (including, with correlative meanings, “controlled by,” “controlling,” and “under common control with”) will be presumed to exist with respect to a Person in the event of the possession, direct or indirect, of (a) the power to direct or cause the direction of the management and policies of such Person (whether through ownership of securities, by contract, including without limitation, variable interest entity contractual arrangements or otherwise), or (b) more than 50% of the voting securities or other comparable equity interests.  The Parties acknowledge that in the case of certain entities organized under the laws of certain countries outside of the United States, the maximum percentage ownership permitted by law for a foreign investor may be less than 50%, and that in such case, such lower percentage will be substituted in the preceding sentence, provided

thatsuch foreign investor has the power to direct or cause the direction of the management and policies of such Person.  Neither of the Parties will be deemed to be an “Affiliate” of the other solely as a result of their entering into this Agreement.

1.6 “Agreement Payments” has the meaning set forth in Section 6.8 (Income Tax Withholding).

1.7 “Applicable Law” means applicable laws, statutes, rules, regulations, and other pronouncements having the effect of law of any Governmental Authority that may be in effect from time to time, including disclosure obligations required by any securities exchange or securities commission having authority over a Party and any applicable rules, regulations, guidances, or other requirements of any Regulatory Authority that may be in effect from time to time.

1.8 “Applicant” has the meaning set forth in Section 9.8.2 (Access to Confidential Information).

1.9 “Applicant Response” has the meaning set forth in Section 9.8.3(b) (Disclosure of Applicant’s Response).

1.10 “Assigned Regulatory Submissions” has the meaning set forth in Section 4.1.3(a) (Transfer of Other Assigned Regulatory Submissions).

1.11 “Bankruptcy Filing” has the meaning set forth in Section 11.3 (Termination for Insolvency).

1.12 “Bayh-Dole Act” means the Patent and Trademark Law Amendments Act of 1980, as amended, codified at 35 U.S.C. §§ 200-212, as well as any regulations promulgated pursuant thereto, including 37 C.F.R. Part 401, and any successor statutes or regulations.

1.13 “Biosimilar Application” has the meaning set forth in Section 9.8.1 (Response to Biosimilar Applicants; Notice).

1.14 “Biosimilar Product” means, with respect to a Product, in a country, any gene therapy product or genetic medicine product, as applicable, that is a generic, biosimilar, or interchangeable product with respect to such product sold by a Third Party [***] and that (a) is subject to a license for administration to humans under Section 351(a) or 351(k) of the PHSA and (i) contains an active ingredient that is the same as the active ingredient of such product or (ii) is “biosimilar” (as defined in Section 351(i)(2) of the PHSA) or “interchangeable” (as defined in Section 351(i)(3) of the PHSA) to such product (or otherwise bioequivalent, biosimilar, interchangeable, or the like, in each case, to such product under analogous laws for gene therapy products or genetic medicine products), (b) has been licensed as a similar biological medicinal product by EMA pursuant to Directive 2001/83/EC, as may be amended, or any subsequent or superseding law, statute or regulation, or (c) has otherwise received Regulatory Approval as a generic, biosimilar, bioequivalent, or interchangeable product from another applicable Regulatory Authority in such country, including by referencing Regulatory Approvals (or data therein) of such product, or, in each case, (a), (b), or (c), an analogous law, statute, or regulation for gene therapy products or genetic medicine products.

1.15 “BPCIA” has the meaning set forth in Section 9.8.1 (Response to Biosimilar Applicants; Notice).

1.16 “Breaching Party” has the meaning set forth in Section 11.2.1 (Material Breach and Cure Period).

1.17 “Business Day” means any day other than a Saturday, Sunday, or bank or other public holiday in Boston, Massachusetts, the People’s Republic of China or Hong Kong.

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1.18 “Calendar Quarter” means the respective periods of three consecutive calendar months ending on March 31^st, June 30^th, September 30^th, or December 31^st in any Calendar Year; provided, however, that the first Calendar Quarter of the Term will extend from the Effective Date to the end of the first complete Calendar Quarter thereafter and the last Calendar Quarter of the Term will end on the effective date of termination or expiration of the Agreement.

1.19 “Calendar Year” means any calendar year beginning on January 1^st and ending on December 31st, provided, however, that the first Calendar Year of the Term will begin on the Effective Date and end on December 31 and the last Calendar Year of the Term will end on the effective date of termination or expiration of the Agreement.

1.20 “CANbridge” has the meaning set forth in the Preamble.

1.21 “CANbridge Background Improvement Know-How” means any Know-How that (a) is Created by or on behalf of either Party or its Affiliates in connection with the performance of activities under this Agreement, (b) constitutes an improvement to CANbridge Background Technology disclosed to LogicBio or its Affiliates in performance of this Agreement and (c) is not LogicBio Background Improvement Technology, in each case to the extent such Know-How relates specifically to a Target or a Product (including any gene therapy construct encapsulated in the Product).

1.22 “CANbridge Background Improvement Patent Right” means any Patent Right that claims CANbridge Background Improvement Know-How.

1.23 “CANbridge Background Improvement Technology” means collectively, (a) the CANbridge Background Improvement Know-How and (b) CANbridge Background Improvement Patent Rights.

1.24 “CANbridge Background Know-How” means all Know-How, other than Joint Collaboration Know-How and CANbridge Collaboration Know-How, that CANbridge or any of its Affiliates Controls as of the Effective Date or that comes into the Control of CANbridge or any of its Affiliates during the Term and that is disclosed to LogicBio or its Affiliate under this Agreement.

1.25 “CANbridge Background Patent Right” means any Patent Right, other than any Joint Collaboration Patent Right or CANbridge Collaboration Patent Right, that CANbridge or any of its Affiliates Controls as of the Effective Date or that comes into the Control of CANbridge or any of its Affiliates during the Term and that is disclosed to LogicBio or its Affiliate under this Agreement.

1.26 “CANbridge Background Technology” means (a) the CANbridge Background Know-How and (b) CANbridge Background Patent Rights.

1.27 “CANbridge Collaboration Know-How” has the meaning set forth in Section 9.1.2(a) (CANbridge Collaboration Technology).

1.28 “CANbridge Collaboration Patent Rights” has the meaning set forth in Section 9.1.2(a) (CANbridge Collaboration Technology).

1.29 “CANbridge Collaboration Technology” means (a) the CANbridge Collaboration Know-How and (b) CANbridge Collaboration Patent Rights.

3

1.30 “CANbridge Field” means the treatment, diagnosis or prevention of any and all indications in humans.

1.31 “CANbridge House Marks” means CANbridge’s and its Affiliates’ trade names, corporate names and corporate logos.

1.32 “CANbridge Indemnitee” has the meaning set forth in Section 10.1 (Indemnification by LogicBio).

1.33 “CANbridge Licensed Know-How” means all Know-How that (a) is Controlled by CANbridge or any of its Affiliates as of the Effective Date or during the Term, (b) is not generally known, and (c) is [***] to perform LogicBio’s obligations under this Agreement.

1.34 “CANbridge Licensed Patent Right” means any Patent Right that (a) is Controlled by CANbridge or any of its Affiliates as of the Effective Date or during the Term and (b) claims CANbridge Licensed Know-How or is otherwise [***] to perform LogicBio’s obligations under this Agreement.

1.35 “CANbridge Licensed Technology” means collectively, (a) the CANbridge Licensed Know-How and (b) CANbridge Licensed Patent Rights.

1.36 “CANbridge Product Technology” means all Patent Rights and Know-How that (a) are Controlled by CANbridge or its Affiliates on the date of termination of this Agreement and (b) are [***] prior to the date of termination to Exploit any Product in the Territory in the CANbridge Field.

1.37 “CANbridge Response” has the meaning set forth in Section 9.8.3(c) (Preparation of CANbridge Response).

1.38 “CANbridge Share” has the meaning set forth in Section 2.3.4 (New In-License Agreement Upfront / Milestones).

1.39 “Change of Control” means, with respect to a Party, that: (a) any Third Party acquires directly or indirectly the beneficial ownership of any voting security of such Party, or if the percentage ownership of such Third Party in the voting securities of such Party is increased through stock redemption, cancellation, or other recapitalization, and immediately after such acquisition or increase such Third Party is, directly or indirectly, the beneficial owner of voting securities representing more than 50% of the total voting power of all of the then-outstanding voting securities of such Party; (b) a merger, consolidation, recapitalization, or reorganization of such Party is consummated, other than any such transaction that would result in shareholders or equity holders of such Party immediately prior to such transaction owning more than 50% of the outstanding voting securities of the surviving entity (or its parent entity) immediately following such transaction; (c) the shareholders or equity holders of such Party approve a plan of complete liquidation of such Party, or an agreement for the sale or disposition by such Party of all or substantially all of such Party’s assets, other than pursuant to the transaction described above or to an Affiliate; or (d) the sale or transfer to a Third Party of all or substantially all of such Party’s consolidated assets taken as a whole.  Notwithstanding the foregoing, any transaction or series of transactions effected for the purpose of financing the operations of the applicable Party or one or more of its applicable Affiliates (such as a public offering of equity securities to investors) will not be deemed a “Change of Control” for purposes of this Agreement.

1.40 “Claim” has the meaning set forth in Section 10.1 (Indemnification by LogicBio).

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1.41 “Clinical Trial” means any clinical trial in humans that is designed to generate data in support or maintenance of an IND or MAA, or other similar marketing application, including any Phase I Clinical Trial, Phase I/II Clinical Trial, Phase II Clinical Trial, Phase III Clinical Trial, Pivotal Clinical Trial, or any post-approval clinical trial in humans.

1.42 “CMO” means a Third Party contract manufacturing organization.

1.43 “CMRI” means Children’s Medical Research Institute, an Australian corporation.

1.44 “CMRI Agreement” means the License Agreement by and between LogicBio Australia and CMRI, dated as of [***], as the same may be amended from time to time in accordance with this Agreement.

1.45 “Co-Owner” has the meaning set forth in the CMRI Agreement.

1.46 “Combination Product” means a product (a) that is sold in the form of a combination that contains or comprises a Product together with one or more other therapeutically active pharmaceutical agents (whether coformulated or copackaged or otherwise sold for a single price), (b) that contains or comprises a Product and is sold for a single invoice price together with any (i) companion diagnostic related to a Product, or (ii) product, process, service, or therapy other than a Product (such additional therapeutically active pharmaceutical agent, companion diagnostic, or product, process, service or therapy and each of (a) and (b)(i) or (b)(ii), an “Other Component”), or (c) that contains or comprises a Product and is defined as a “combination product” by the FDA pursuant to 21 C.F.R. §3.2(e) or its foreign equivalent.

1.47 “Commercialization” means any and all activities directed to the marketing, promotion, distribution, offering for sale, sale, having sold, importing, having imported, exporting, having exported, or other commercialization of a pharmaceutical or biologic product, but excluding activities directed to Manufacturing, Development, or Medical Affairs. “Commercialize,” “Commercializing,” and “Commercialized” will be construed accordingly.

1.48 “Commercially Reasonable Efforts” means, with respect to the efforts to be expended, or considerations to be undertaken, by a Party or its Affiliate with respect to any objective or activity to be undertaken hereunder, [***].

1.49 “Competing Infringement” has the meaning set forth in Section 9.5.1 (Patent Enforcement; Notice).

1.50 “Competing Product” means any [***], other than a Product, that [***], [***] and [***].

1.51 “Confidential Information” means, with respect to a Party subject to Section 8.1.2 (Confidential Information), all Know-How or other information, including proprietary information and materials (whether or not patentable) regarding or embodying such Party’s technology, products, business information, or objectives, that is communicated by or on behalf of such Party (the “Disclosing Party” with respect to such information) to the other Party (the “Receiving Party” with respect to such information) or its permitted recipients, including information disclosed by such Party prior to the Effective Date pursuant to the Confidentiality Agreement, provided that LogicBio Background Improvement Know-How will be the Confidential Information of LogicBio regardless of which Party first disclosed any such LogicBio Background Improvement Know-How to the other Party.

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1.52 “Confidentiality Agreement” means that certain Confidentiality Agreement, dated as of [***], by and between CANbridge Pharmaceuticals Inc. and LogicBio Therapeutics Inc.

1.53 “Control” or “Controlled” means, with respect to a Party, the possession by a Party or its Affiliates (whether by ownership, license, or otherwise, other than pursuant to this Agreement) of, (a) with respect to any tangible Know-How or Materials, the legal authority or right to physical possession of such tangible Know-How or Materials, with the right to provide such tangible Know-How or Materials to the other Party on the terms set forth herein, (b) with respect to Patent Rights, intangible Know-How, or other intellectual property, the legal authority or right to grant a license, sublicense, access, or right to use (as applicable) to the other Party under such Patent Rights, intangible Know-How, or other intellectual property on the terms set forth herein, or (c) with respect to a product or component thereof, the legal authority or right to grant a license, sublicense, access, or right to use (as applicable) to the other Party under Patent Rights that Cover or proprietary Know-How that is incorporated in or embodies such product or component on the terms set forth herein, in each case ((a), (b), and (c)), without breaching or otherwise violating the terms of any arrangement or agreement with a Third Party in existence as of the time such Party or its Affiliates would first be required hereunder to grant the other Party such access, right to use, license, or sublicense; provided that, [***]. Notwithstanding anything in this Agreement to the contrary, a Party and its Affiliates will be deemed to not Control any of the foregoing (a)-(c) that are owned or controlled by a Third Party described in the definition of “Change of Control,” or such Third Party’s Affiliates (other than an Affiliate of such Party prior to the Change of Control) (each, an “Independent Affiliate”), (a) prior to the closing of such Change of Control, except to the extent that any such Patent Rights or Know-How were developed by such Third Party prior to such Change of Control using or incorporating such Party’s or its pre-existing Affiliate’s Know-How or Patent Rights, or (b) after such Change of Control to the extent that such Patent Rights or Know-How are developed or conceived by such Third Party or its Affiliates (other than such Party) after such Change of Control without using or incorporating such Party’s or its pre-existing Affiliate’s Know-How or Patent Rights and are not developed or conceived by personnel who were employees or consultants of such Party or its pre-existing Affiliates.

1.54 “Cover,” “Covering” or “Covered” means, with respect to a particular subject matter at issue and a relevant Patent Right or individual claim in such Patent Right, as applicable, that the manufacture, use, sale, offer for sale, or importation of such subject matter would infringe such Patent Right or, as to a pending claim included in such Patent Right, the making, using, keeping, selling, offering for sale or importation of such compound or product would infringe such Patent Right if such pending claim were to issue in an issued patent without modification.

1.55 “Created” means created, made, developed, invented, generated, conceived, or reduced to practice. “Create” and “Creating” have their correlative meanings.

1.56 “Cure Period” has the meaning set forth in Section 11.2.1 (Material Breach and Cure Period).

1.57 “Damages” means damages of any kind or nature (including claims for an account of profits), past, present, or future, arising from any claims (as such term is defined in Section 2.7 (Release)) based on acts or omissions occurring on or before the Restatement Date in any jurisdiction in the world or available under any state, provincial, federal, or international law, or the law of any country (or any other act, action, administrative rule or procedure, legislation or regulation of any kind or description), including any actual, general, specific, direct, indirect, commercial, economic, consequential, incidental, special, punitive, exemplary, or treble damages, that can be obtained directly, indirectly, or by way of contribution or indemnity, under any theory of liability whatsoever, including but not limited to, any liability that is contributory, strict, contractual or tortious in

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character, whether at law or in equity. The term “Damages” will also include loss of revenue, loss of expected profits or expected savings, extradition of infringer’s profits, fines, monetary penalties, court costs, interest, pre-judgment and post-judgment interest, attorney’s fees, expert fees, and any other related costs or expenses. “Damages” will specifically include damages for unknown claims (as such term is defined in Section 2.7 (Release)) that are based on acts or omissions occurring on or before the Restatement Date that are unknown to the claiming party as of the Restatement Date.

1.58 “Debarred” means, with respect to an individual or entity, that such individual or entity has been debarred or suspended under 21 U.S.C. §335(a) or (b), the subject of a conviction described in Section 306 of the FD&C Act, excluded from a federal or governmental health care program, debarred from federal contracting, convicted of or pled nolo contendere to any felony, or to any federal or state legal violation (including misdemeanors) relating to prescription drug products or fraud, the subject to OFAC sanctions or on the OFAC list of specially designated nationals, or the subject of any similar sanction of any Governmental Authority in the Territory.

1.59 “Development” means all internal and external research, development, and regulatory activities related to pharmaceutical or biologic products, including (a) research, toxicology testing and studies, non-clinical and preclinical testing, studies, and other activities, and Clinical Trials, and (b) preparation, submission, review, and development of data or information for the purpose of submission to a Regulatory Authority to obtain authorization to conduct Clinical Trials and to obtain, support, or maintain Regulatory Approval of a pharmaceutical or biologic product and interacting with Regulatory Authorities following receipt of Regulatory Approval in the applicable country or region for such pharmaceutical or biologic product regarding the foregoing, but excluding activities directed to Manufacturing, Medical Affairs, or Commercialization.  Development will include development and regulatory activities for additional forms, formulations, or indications for a pharmaceutical or biologic product after receipt of Regulatory Approval of such product (including label expansion), including Clinical Trials initiated following receipt of Regulatory Approval or any Clinical Trial to be conducted after receipt of Regulatory Approval that was mandated by the applicable Regulatory Authority as a condition of such Regulatory Approval with respect to an approved formulation or indication (such as post-marketing studies, observational studies, in each case, if required by any Regulatory Authority in any region in the Territory to support or maintain Regulatory Approval for a pharmaceutical or biologic product in such region). “Develop,” “Developing,” and “Developed” will be construed accordingly.

1.60 “Development Milestone Event” has the meaning set forth in Section 6.3.1 (Development Milestones).

1.61 “Development Milestone Payment” has the meaning set forth in Section 6.3.1 (Development Milestones).

1.62 “Directed to” means, with respect to a given gene therapy product and a given Target, that such gene therapy product contains a nucleic acid sequence that encodes such Target, a homolog or ortholog of such Target, or a functional portion of such Target, homolog or ortholog.

1.63 “Disclosing Party” has the meaning set forth in Section 1.51 (Confidential Information).

1.64 “Dispute” has the meaning set forth in Section 12.1 ([***] Dispute Resolution Mechanism).

1.65 “Divest” means, with respect to a Competing Product, the sale, exclusive license or other transfer by the applicable Party and its Affiliates of all of their development and commercialization rights with respect to such Competing Product to a Third Party without the retention or reservation of any

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development or commercialization obligation, interest or participation rights (other than solely an economic interest or the right to enforce customary terms contained in the relevant agreements effectuating such transaction).

1.66 “Dollar” means the U.S. dollar, and “$” will be interpreted accordingly.

1.67 “Effective Date” has the meaning set forth in the preamble.

1.68 “EMA” means the European Medicines Agency or any successor agency or authority having substantially the same function.

1.69 “Existing In-License Agreement” means any agreement set forth in Schedule 1.69 (Existing In-License Agreements), as updated from time to time.

1.70 “Exploit” means to make, have made, import, export, distribute, use, have used, keep, sell, have sold, or offer for sale, including to research, Develop, Manufacture, have Manufactured, perform or have performed Medical Affairs, Commercialize, register, modify, enhance, improve, or otherwise exploit. “Exploitation” and “Exploiting” will be construed accordingly.

1.71 “FD&C Act” means the Federal Food, Drug and Cosmetic Act, as the same may be amended or supplemented from time to time.

1.72 “FDA” means the U.S. Food and Drug Administration, or any successor agency thereto.

1.73 “First Commercial Sale” means, on a country-by-country and product-by-product basis, the first sale of a given Product under this Agreement by CANbridge, its Affiliates, or its Sublicensees to an end user or prescriber for use, consumption, or resale of such product in a country in the Territory in the CANbridge Field where Regulatory Approval and (in the case of countries outside the U.S.) any necessary Pricing and Reimbursement Approval of the product has been obtained and where the sale results in a recordable Net Sale. “First Commercial Sale” will not include: [***].

1.74 “Force Majeure” means any event beyond the reasonable control of the affected Party, including embargoes; war or acts of war, including terrorism, insurrections, riots, or civil unrest; strikes, lockouts, or other labor disturbances; pandemics, epidemics, fire, floods, earthquakes, or other acts of nature; or acts, omissions, or delays in acting by any Governmental Authority (including the refusal of the competent Governmental Authorities to issue required Regulatory Approvals due to reasons other than the affected Party’s negligence or willful misconduct or any other cause within the reasonable control of the affected Party); and failure of plant or machinery (provided, in each case, that such event or failure could not have been prevented by the exercise of skill, diligence, and prudence that would be reasonably and ordinarily expected from a skilled and experienced person engaged in the same type of undertaking under the same or similar circumstances). The Parties agree that the effects of the COVID-19 pandemic that is ongoing as of the Effective Date (including related government orders) may be invoked as a Force Majeure for the purposes of this Agreement even though the pandemic is ongoing as of the Effective Date and those effects may be reasonably foreseeable (but are not known for certain) as of the Effective Date.

1.75 “FTE” means a qualified full time person, or more than one person working the equivalent of a full-time person, where “full time” is based upon a total of [***] hours per Calendar Year of work carried out by one or more duly qualified employees of a Party or its Affiliates.

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1.76 “FTE Costs” means, for any period, the [***] in such period.  FTEs will be pro‑rated on a daily basis if necessary.

1.77 “FTE Rate” means [***] per annum, subject, in each case, to [***].

1.78 “Good Laboratory Practices,” “GLP,” or “cGLP” means the then-current standards, practices and procedures promulgated or endorsed by the FDA as set forth in 21 C.F.R. Part 58 (or any successor statute or regulation), including related regulatory requirements imposed by the FDA and comparable regulatory standards, practices and procedures promulgated by the EMA, PMDA, or other Regulatory Authority applicable to the Territory, as they may be updated from time to time, including applicable guidelines promulgated under the ICH.

1.79 “Good Manufacturing Practices,” “GMP,” or “cGMP” means the then-current and phase appropriate good manufacturing practices required by the FDA, as set forth in the FD&C Act, as amended, and the regulations promulgated thereunder, for the Manufacture and testing of pharmaceutical materials, and comparable Applicable Law related to the manufacture and testing of pharmaceutical materials in jurisdictions outside the United States. “Good Manufacturing Practices,” “GMP,” or “cGMP” also means the quality guidelines promulgated by the International Conference on Harmonization (ICH), including the ICH Q7A, titled “Q7A Good Manufacturing Practice Guidance for Active Pharmaceutical Ingredients” and the policies promulgated thereunder, in each case, as they may be updated from time to time.

1.80 “Governmental Authority” means any court, tribunal, arbitrator, agency, commission, department, ministry, official, authority, or other instrumentality of any nation, state, county, city, or other political subdivision thereof or of any multinational governmental body.

1.81 “Greater China” means People’s Republic of China, Hong Kong, Macau and Taiwan.

1.82 “Immediate Patent Infringement Action” has the meaning set forth in Section 9.8.3(d) (Negotiation; LogicBio Rights).

1.83 “In-License Agreements” means, collectively, the Existing In-License Agreements and the New In-License Agreements.

1.84 “IND” means (a) an Investigational New Drug application as defined in the FD&C Act, as amended, and applicable regulations promulgated thereunder by the FDA, (b) a Clinical Trial authorization application for a product filed with a Regulatory Authority in any other regulatory jurisdiction outside the U.S., the filing of which (in the case of (a) or (b)) is necessary to commence or conduct clinical testing of a pharmaceutical or biologic product in humans in such jurisdiction, or (c) documentation issued by a Regulatory Authority that permits the conduct of clinical testing of a pharmaceutical or biologic product in humans in such jurisdiction.

1.85 “IND Effective Date” means, with respect to a Product, the earlier of (a) the date that is [***] following the filing of the first IND for such Product in the U.S. in the CANbridge Field, ifthe filing Party or its Affiliates has not received any notice of a clinical hold (including any complete or partial clinical hold) or any other administrative delay from the FDA during such [***] period; provided that, if the filing party or its Affiliate does receive a notice of such a clinical hold (including any complete or partial clinical hold) or there is such other administrative delay, then the applicable date under this clause (a) for such Product will be the date on which the FDA lifts such clinical hold or such other administrative delay is otherwise resolved and the FDA first allows such Product to be administered to a human pursuant to such IND, or (b) the date on which such

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Product is first permitted by the applicable Regulatory Authority in a Major European Market to be administered to a human in the CANbridge Field pursuant to an IND in accordance with Applicable Law.

1.86 “Indemnifying Party” has the meaning set forth in Section 10.3.1 (Indemnification Procedure; Notice).

1.87 “Indemnitee” has the meaning set forth in Section 10.3.1 (Indemnification Procedure; Notice).

1.88 “Independent Affiliate” has the meaning set forth in Section 1.53 (Control).

1.89 “Infringed Patent List” has the meaning set forth in Section 9.8.3(d) (Negotiation; LogicBio Rights).

1.90 “Infringement Action” has the meaning set forth in Section 9.5.2(a) ([***]).

1.91 “Initial Patent List” has the meaning set forth in Section 9.8.3(a) (Preparation of Initial Patent List).

1.92 “Initiating Party” has the meaning set forth in Section 9.5.2(f) (Infringement Actions for Infringements by Third Parties; Procedures).

1.93 “Initiation” means, with respect to any Clinical Trial, [***].

1.94 “Intellectual Property” means all Patent Rights, rights to Inventions, copyrights, design rights, trademarks, trade secrets, Know-How, materials, and all other intellectual property rights (whether registered or unregistered) and all applications and rights to apply for any of the foregoing anywhere in the world.

1.95 “Internal Revenue Code” means the United States Internal Revenue Code of 1986, as amended.

1.96 “Invention” means any process, method, utility, formulation, composition of matter, article of manufacture, material, creation, discovery or finding, or any improvement thereof, that is conceived, reduced to practice, or otherwise invented, whether patentable or not.

1.97 “Joint Collaboration Know-How” has the meaning set forth in Section 9.1.2(c) (Joint Collaboration Technology).

1.98 “Joint Collaboration Patent Right” has the meaning set forth in Section 9.1.2(c) (Joint Collaboration Technology).

1.99 “Joint Collaboration Technology” means (a) the Joint Collaboration Know-How and (b) Joint Collaboration Patent Rights.

1.100 “Know-How” means any (a) proprietary scientific or business information or materials, including records, improvements, modifications, techniques, assays, designs, protocols, formulas, data (including physical data, chemical data, toxicology data, animal data, raw data, clinical data, and analytical and quality control data), dosage regimens, control assays, product specifications, marketing, pricing and distribution costs, Inventions, algorithms, technology, forecasts, profiles, strategies, plans, results in any form whatsoever, know-how, and trade secrets (in each case,

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whether or not patentable, copyrightable, or otherwise protectable), and (b) any information embodied in chemical or biological materials or physical embodiments of any of the foregoing.

1.101 “LogicBio” has the meaning set forth in the Preamble.

1.102 “LogicBio Background Improvement Know-How” means any Know-How that (a) is Created by or on behalf of either Party or its Affiliates in connection with the performance of activities under this Agreement and (b) constitutes an improvement to LogicBio Background Technology disclosed to CANbridge or its Affiliate in performance of this Agreement, in each case to the extent such Know-How does not relate specifically to a Target or a Product (including any gene therapy construct encapsulated in the Product).

1.103 “LogicBio Background Improvement Patent Right” means any Patent Right that claims LogicBio Background Improvement Know-How.

1.104 “LogicBio Background Improvement Technology” means collectively, (a) the LogicBio Background Improvement Know-How and (b) LogicBio Background Improvement Patent Rights.

1.105 “LogicBio Background Know-How” means all Know-How, other than Joint Collaboration Know-How and LogicBio Collaboration Know-How, that LogicBio or any of its Affiliates Controls as of the Effective Date or that comes into the Control of LogicBio or any of its Affiliates during the Term.

1.106 “LogicBio Background Patent Right” means any Patent Right, other than any Joint Collaboration Patent Right or LogicBio Collaboration Patent Right, that LogicBio or any of its Affiliates Controls as of the Effective Date or that comes into the Control of LogicBio or any of its Affiliates during the Term.

1.107 “LogicBio Background Technology” means (a) the LogicBio Background Know-How and (b) LogicBio Background Patent Rights.

1.108 “LogicBio Collaboration Know-How” has the meaning set forth in Section 9.1.2(b) (LogicBio Collaboration Technology).

1.109 “LogicBio Collaboration Patent Rights” has the meaning set forth in Section 9.1.2(b) (LogicBio Collaboration Technology).

1.110 “LogicBio Collaboration Technology” means (a) the LogicBio Collaboration Know-How and (b) LogicBio Collaboration Patent Rights.

1.111 “LogicBio House Marks” means LogicBio’s and its Affiliates’ trade names, corporate names and corporate logos.

1.112 “LogicBio Indemnitee” has the meaning set forth in Section 10.2 (Indemnification by CANbridge).

1.113 “LogicBioLicensed Know-How” means all Know-How that (a) is Controlled by LogicBio or any of its Affiliates as of the Effective Date or during the Term, (b) is not generally known, and (c) is [***] to Exploit any Product in the CANbridge Field, including, to the extent described in clauses (b) and (c), LogicBio’s interest in Joint Collaboration Know-How.

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1.114 “LogicBioLicensed Patent Right” means any Patent Right that (a) is Controlled by LogicBio or any of its Affiliates as of the Effective Date or during the Term and (b) (i) claims LogicBio Licensed Know-How or (ii) is otherwise [***] to Exploit any Product in the CANbridge Field, including, to the extent described in clauses (a) and (b), LogicBio’s interest in Joint Collaboration Patent Rights.  The LogicBio Licensed Patent Rights as of the Effective Date are set forth on Schedule 1.114(LogicBio Licensed Patent Rights).

1.115 “LogicBio Product-Specific Licensed Patent Right” means any LogicBio Licensed Patent Right that includes claims that specifically recite the composition of matter, formulation or method of use of a Product or that pertain solely to a Target.

1.116 “LogicBio Technology” means collectively, (a) the LogicBio Licensed Know-How and (b) LogicBio Licensed Patent Rights.

1.117 “LogicBio’s Knowledge” means the actual knowledge [***], of the following: [***].

1.118 “Losses” has the meaning set forth in Section 10.1 (Indemnification by LogicBio).

1.119 “MAA” means any biologics license application or other marketing authorization application, in each case, filed with the applicable Regulatory Authority in a country or other regulatory jurisdiction, which application is required to commercially market or sell a pharmaceutical or biologic product in such country or jurisdiction (and any amendments thereto), including all Biologics License Applications (BLA) or equivalent submitted to the FDA in the United States in accordance with the PHSA or any analogous application or submission with any Regulatory Authority outside of the United States.

1.120 “Major European Markets” means the [***].

1.121 “Major Market Country” means the [***].

1.122 “Manufacture” means activities directed to manufacturing, manufacturing process development, processing, formulating, packaging, labeling, filling, finishing, assembly, quality assurance, quality control, testing, and release, shipping, or storage of any pharmaceutical or biologic product (or any components or process steps involving any product or any companion diagnostic), placebo, or comparator agent, as the case may be, including process development, qualification, and validation, scale-up, pre-clinical, clinical, and commercial manufacture and analytic development, product characterization, and stability testing, but excluding activities directed to Development, Commercialization, or Medical Affairs. “Manufacturing” will be construed accordingly.

1.123 “Materials” has the meaning set forth in Section 3.1.2 (Transfer of Materials).

1.124 “Medical Affairs” means activities conducted by a Party’s medical affairs departments (or, if a Party does not have a medical affairs department, the equivalent function thereof), including communications with key opinion leaders, medical education, symposia, advisory boards (to the extent related to medical affairs or clinical guidance), activities performed in connection with patient registries, other medical programs and communications, including educational grants, research grants (including conducting investigator-initiated studies), and charitable donations to the extent related to medical affairs and not to other activities that involve the promotion, marketing, sale, or other Commercialization of the Products and are not conducted by a Party’s medical affairs (or equivalent) departments.

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1.125 “Mono Product” has the meaning set forth in Section 1.126 (Net Sales).

1.126 “Net Sales” means the gross amounts invoiced by CANbridge or its Affiliates or Sublicensees (other than Third Party Distributors) (each, a “Selling Party”) under this Agreement to Third Parties (including to Third Party Distributors), for the sale, supply or other disposition of any Product, less the following deductions actually taken, paid, accrued, allowed, included, or allocated with respect to such sales of such product, and in accordance with the applicable Accounting Standards:

[***]

Notwithstanding the foregoing, [***].

In the case of any Combination Product sold in a given country in the Territory, Net Sales for the purpose of determining royalties and Sales Milestone Events of the Combination Product in such country will be calculated by [***].

If, on a country-by-country basis, the applicable Mono Product is sold separately in a country, but all of the Other Components in the Combination Product are not sold separately in such country, then Net Sales for the purpose of determining royalties and Sales Milestone Events of the Combination Product for such country will be calculated by [***].

If, on a country-by-country basis, the Product, included in the Combination Product is not sold separately as a Mono Product in such country, but all of the Other Components included in the Combination Product are sold separately in such country, then Net Sales for the purpose of determining royalties and Sales Milestone Events of the Combination Product for such country will be calculated by [***].

If neither the applicable Mono Product nor the Other Components in a Combination Product are sold separately in a given country, then [***].  

1.127 “New In-License Agreement” has the meaning set forth in Section 2.3.2 (New In-License Agreements).

1.128 “New License Agreement” has the meaning set forth in Section 2.2.3 (Survival of Sublicenses).

1.129 “Non-Breaching Party” has the meaning set forth in Section 11.2.1 (Material Breach and Cure Period).

1.130 “Original Agreement” has the meaning set forth in the Recitals.

1.131 “Other Component(s)” has the meaning set forth in Section 1.126 (Net Sales).

1.132 “Out-of-Pocket Costs” means, with respect to certain activities hereunder, [***].

1.133 “Patent Challenge” has the meaning set forth in Section 11.5 (Termination for Patent Challenge).

1.134 “Patent Rights” means any and all (a) patents, (b) patent applications, including all provisional and non-provisional applications, patent cooperation treaty (PCT) applications, substitutions, continuations, continuations-in-part, divisions and renewals, and all patent rights granted thereon, (c) all patents-of-addition, reissues, re-examinations and extensions or restorations by existing or future extension or restoration mechanisms, including supplementary protection certificates, patent

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term extensions, and equivalents thereof, (d) inventor’s certificates, letters patent, or (e) any other substantially equivalent form of government issued right, excluding any rights provided by Regulatory Exclusivity, substantially similar to any of the foregoing described in subsections (a) through (d) above, anywhere in the world.

1.135 “Patent Term Extension” has the meaning set forth in Section 9.9 (Patent Term Extensions).

1.136 “Per Product Annual Net Sales” has the meaning set forth in Section 6.3 (Royalties).

1.137 “Person” means any individual, firm, corporation, partnership, limited liability company, trust, business trust, joint venture, Governmental Authority, association or other entity.

1.138 “Pharmacovigilance Agreement” has the meaning set forth in Section 4.1.5 (Pharmacovigilance).

1.139 “Phase I Clinical Trial” means a Clinical Trial (or any arm thereof) of a pharmaceutical or biologic product with the endpoint of determining initial tolerance, safety, metabolism, pharmacokinetic or pharmacodynamic information in single dose, single ascending dose, multiple dose, or multiple ascending dose regimens, that satisfies the requirements of U.S. federal regulation 21 C.F.R. § 312.21(a) and its successor regulation or equivalents in other jurisdictions.

1.140 “Phase I/II Clinical Trial” means a Clinical Trial (or any arm thereof) of a pharmaceutical or biologic product with the endpoint of determining initial tolerance, safety, metabolism, pharmacokinetic or pharmacodynamic information in single dose, single ascending dose, multiple dose, or multiple ascending dose regimens, and evaluating its effectiveness for a particular indication or indications in one or more specified doses or its short term tolerance and safety, as well as its pharmacokinetic and pharmacodynamic information in patients with the indications under study, that is prospectively designed to generate sufficient data (if successful) to commence a Pivotal Clinical Trial for such product, and that satisfies the requirements of U.S. federal regulation 21 C.F.R. §§ 312.21(a) and (b) and its successor regulation or equivalents in other jurisdictions.

1.141 “Phase II Clinical Trial” means a Clinical Trial (or any arm thereof) of a pharmaceutical or biologic product with the endpoint of evaluating its effectiveness for a particular indication or indications in one or more specified doses or its short term tolerance and safety, as well as its pharmacokinetic and pharmacodynamic information in patients with the indications under study, that is prospectively designed to generate sufficient data (if successful) to commence a Phase III Clinical Trial for such product, and that satisfies the requirements of U.S. federal regulation 21 C.F.R. § 312.21(b) and its successor regulation or equivalents in other jurisdictions.

1.142 “Phase III Clinical Trial” means a Clinical Trial (or any arm thereof) of a pharmaceutical or biologic product on a sufficient number of patients, which trial the FDA permits to be conducted under an open IND and is designed to: (a) establish that the pharmaceutical or biologic product is safe and efficacious for its intended use; (b) define warnings, precautions and adverse reactions that are associated with the pharmaceutical or biologic product in the dosage range to be prescribed; and (c) support an MAA by a Regulatory Authority for the pharmaceutical or biologic product, and that satisfies the requirements of U.S. federal regulation 21 C.F.R. § 312.21(c) and its successor regulation or equivalents in other jurisdictions.

1.143 “PHSA” has the meaning set forth in Section 9.8.1 (Response to Biosimilar Applicants; Notice).

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1.144 “Pivotal Clinical Trial” means any (a) Phase III Clinical Trial, or (b) other Clinical Trial (or any arm thereof) of a pharmaceutical or biologic product on a sufficient number of patients, the results of which, together with prior data and information concerning such product, are intended to be or otherwise are sufficient, without any additional Clinical Trial to meet the evidentiary standard for demonstrating the safety, purity, efficacy, and potency of such active substance of such product established by a Regulatory Authority in any particular jurisdiction and that is intended to support, or otherwise supports, the filing of an MAA by a Regulatory Authority in such jurisdiction (including any bridging study). Notwithstanding any provision to the contrary set forth in this Agreement,treatment of patients as part of an expanded access program, compassionate sales or use program (including named patient program or single patient program), or an indigent program, in each case, will not be included in determining whether or not a Clinical Trial is a Pivotal Clinical Trial or whether a patient has been dosed thereunder.

1.145 “PMDA” means Japan’s Pharmaceuticals and Medical Devices Agency and any successor agency or authority having substantially the same function.

1.146 “Pompe and Fabry Research Plan” means the research plan attached as Exhibit B to this Agreement, as amended from time to time in accordance with the terms of this Agreement.

1.147 “Pricing and Reimbursement Approval” means the approvals, agreements, determinations, or governmental decisions establishing (a) a price for the applicable Product, that can be legally charged to consumers and (b) the level of reimbursement for the applicable Product, that will be reimbursed by Governmental Authorities, in each case ((a) and (b)) if required in a given jurisdiction or country in connection with Commercialization of such Product, in such jurisdiction or country.

1.148 “Product” means any gene therapy product resulting from the conduct of activities under the Research Plan or the Pompe and Fabry Research Plan, [***] which is Directed to a Target, [***].

1.149 “Proposed Biosimilar Product” has the meaning set forth in Section 9.8.1 (Response to Biosimilar Applicants; Notice).

1.150 “Proposed CANbridge Response” has the meaning set forth in Section 9.8.3(c) (Preparation of CANbridge Response).

1.151 “Proposed Initial Patent List” has the meaning set forth in Section 9.8.3(a) (Preparation of Initial Patent List).

1.152 “Prosecuting Party” means, with respect to any Patent Right, the Party that is responsible for the Prosecution of such Patent Right pursuant to Section 9.4 (Patent Prosecution).

1.153 “Prosecution” means the filing, preparation, prosecution (including any interferences, reissue proceedings, reexaminations, oppositions and similar proceedings), post-grant reviews, requests for patent term adjustments, and maintenance of Patent Rights. For clarity, Prosecution excludes any applications or requests for Patent Term Extension. When used as a verb, “Prosecute” means to engage in Prosecution.

1.154 “Publishing Party” has the meaning set forth in Section 8.9.2 (Publication Rights).

1.155 “Receiving Party” has the meaning set forth in Section 1.51 (Confidential Information).

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1.156 “Redacted Agreement” has the meaning set forth in Section 8.5 (Confidential Treatment).

1.157 “Regulatory Approval” means, with respect to a particular country or other regulatory jurisdiction, any approval of an MAA, or other approval, product, or establishment license, registration, or authorization of any Regulatory Authority necessary for the Development, Manufacture, Commercialization, or other Exploitation of a pharmaceutical or biologic product in such country or other regulatory jurisdiction, excluding, in each case, Pricing and Reimbursement Approval.

1.158 “Regulatory Authority”means any applicable Governmental Authority with jurisdiction or authority over the Development, Manufacture, Commercialization, or other Exploitation (including Regulatory Approval, or Pricing and Reimbursement Approval) of pharmaceutical or biologic products in a particular country or other regulatory jurisdiction, and any corresponding national or regional regulatory authorities.

1.159 “Regulatory Exclusivity” means any exclusive marketing rights or data exclusivity rights conferred by any Regulatory Authority with respect to a Product in a country or jurisdiction in the Territory, other than a Patent Right, that prohibits a Person from (a) relying on safety or efficacy data generated by or on behalf of a Party with respect to such product in an application for Regulatory Approval of a Biosimilar Product, or (b) Commercializing a product or a Biosimilar Product, including orphan drug exclusivity or rights similar thereto in other countries or regulatory jurisdictions.

1.160 “Regulatory Submissions” means any filing, application, or submission with any Regulatory Authority in support of the Development, Manufacture, Commercialization, or other Exploitation of a pharmaceutical or biologic product (including to obtain, support, or maintain Regulatory Approval from that Regulatory Authority), and all correspondence or communication with or from the relevant Regulatory Authority, as well as minutes of any material meetings, telephone conferences, or discussions with the relevant Regulatory Authority. Regulatory Submissions include all INDs, MAAs, and other applications for Regulatory Approval and their equivalents.

1.161 “Research Plan” means the Research Plan (as defined in the Original Agreement) as in effect as of the Restatement Date.

1.162 “Restatement Date” has the meaning set forth in the Preamble.

1.163 “Royalty Period” has the meaning set forth in Section 6.4.2 (Royalty Period).

1.164 “Sales Milestone Event” has the meaning set forth in Section 6.3.2 (Sales Milestones).

1.165 “Sales Milestone Payment” has the meaning set forth in Section 6.3.2 (Sales Milestones).

1.166 “Selling Party” has the meaning set forth in Section 1.126 (Net Sales).

1.167 “Subcontractor” means a Third Party contractor engaged by a Party to perform certain obligations or exercise certain rights of such Party under this Agreement on a fee-for-service basis (including contract research organizations, Third Party Distributors, or CMOs), excluding all Sublicensees.

1.168 “Sublicensees” means any Third Party to whom CANbridge or any of its Affiliates grants a sublicense of its rights hereunder to Exploit Products, excluding all Subcontractors.

1.169 “Target” means each of (a) [***], and (b) [***].

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1.170 “Taxes” has the meaning set forth in Section 6.8 (Income Tax Withholding).

1.171 “Term” has the meaning set forth in Section 11.1 (Term).

1.172 “Terminated Target” means any Target with respect to which the Agreement is terminated. If the Agreement is terminated in its entirety, then all Targets will be Terminated Targets.

1.173 “Territory” means worldwide.

1.174 “Third Party” means any Person other than CANbridge or LogicBio or their respective Affiliates.

1.175 “Third Party Distributor” means, with respect to a country, any Third Party that purchases its requirements for Products, in such country from CANbridge or its Affiliates or Sublicensees and is appointed as a distributor to distribute, market, and resell such product in such country, even if such Third Party is granted ancillary rights to Develop, package, or obtain Regulatory Approval of such product in order to distribute, market, or sell such product in such country.

1.176 “Third Party MSA” has the meaning set forth in Section 3.1.4 (Certain Agreements).

1.177 “Third Party Patent Challenge” has the meaning set forth in Section 9.7.2 (Parties’ Patent Rights).

1.178 “Trademark” means all trademarks, service marks, trade names, brand names, sub-brand names, trade dress rights, product configuration rights, certification marks, collective marks, logos, taglines, slogans, designs or business symbols and all words, names, symbols, colors, shapes, designations or any combination thereof that function as an identifier of source or origin or quality, whether or not registered, and all statutory and common law rights therein, and all registrations and applications therefor, together with all goodwill associated with, or symbolized by, any of the foregoing.

1.179 “[***] Manufacturing System” means LogicBio’s proprietary manufacturing process described in [***], and [***] and [***].For the avoidance of doubt, “[***] Manufacturing System” will not include any technology described in [***] other than the LogicBio proprietary manufacturing processes described therein.

1.180 “U.S.” or “United States” means the United States of America, its territories and possessions, including Puerto Rico.

1.181 “Valid Claim” means, [***], (a) a claim of any issued and unexpired patent [***] whose validity, enforceability, or patentability has not been terminated by any of the following: [***], or (b) a claim within a patent application [***].

Article 2
LICENSE GRANTS; AMENDMENT AND RESTATEMENT

2.1 License to CANbridge.  Subject to the terms and conditions of this Agreement, LogicBio hereby grants to CANbridge a worldwide, royalty-bearing license, with the right to sublicense through multiple tiers (subject to the provisions of Section 2.2 (Sublicensing by CANbridge; Responsibility)), under LogicBio’s interest in the LogicBio Technology to Exploit the Products in the CANbridge Field, which license (a) is, notwithstanding any provision to the contrary in this Agreement, non-exclusive to the extent relating to the [***] Manufacturing System and (b)

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otherwise is exclusive (even as to LogicBio, except to the extent [***] for LogicBio to perform (or have performed) the activities allocated to LogicBio under this Agreement).

2.2 Sublicensing and Subcontracting by CANbridge; Responsibility.

2.2.1 Sublicensing Rights; Subcontracting.  Subject to Section 2.3 (Existing In-License Agreements), Section 2.6.5 (Restrictions Relating to [***] Manufacturing System) and this Section 2.2 (Sublicensing by CANbridge; Responsibility), and without limiting Section 13.1 (Performance through Affiliates) CANbridge may grant sublicenses under Section 2.1 (License to CANbridge) through multiple tiers to any of its Affiliates or to any Third Party, and may otherwise subcontract the performance of its obligations and the exercise of its rights under this Agreement to any Affiliate or to any Third Party, provided that any such sublicense granted by CANbridge, its Affiliate or its Third Party Sublicensee and any such subcontracting arrangement will be consistent with the terms of this Agreement and will include invention assignment, confidentiality, non-disclosure, non-use and intellectual property provisions at least as restrictive or protective of the Parties as those set forth in this Agreement, except that, in the case of subcontractors, the term of any such confidentiality agreement shall be customary for the nature of the subcontractor. CANbridge will remain responsible for each such Affiliate’s or Third Party Sublicensee’s or subcontractor’s compliance with all obligations under this Agreement applicable to such Affiliate or Third Party Sublicensee or Third Party subcontractor and the grant of any sublicense or subcontract to any Affiliate or any Third Party will not relieve CANbridge of any of its obligations hereunder; provided that any subcontractor’s or Sublicensee’s actions under this Agreement will be attributable to CANbridge in determining whether CANbridge has used Commercially Reasonable Efforts under this Agreement. Notwithstanding the foregoing or anything to the contrary in this Agreement,  neither CANbridge nor its Affiliates will (a) grant sublicenses under the rights granted to CANbridge under this Agreement to any Third Party Sublicensee to conduct activities [***] or (b) subcontract the performance of its obligations or exercise of its rights under this Agreement to a Third Party conducting activities [***], in each case ((a) or (b)) without the prior written consent of LogicBio (not to be unreasonably withheld); provided, however, that LogicBio’s consent is hereby deemed given for CANbridge to subcontract obligations hereunder to any of the entities listed on Schedule 2.2.1.

2.2.2 Third Party Sublicensees.  CANbridge will provide LogicBio with written notice of any sublicense granted by CANbridge under Section 2.1 (License to CANbridge) to any Third Party no later than [***] after the effective date thereof (including the identity of the Third Party Sublicensee and the region in which such rights have been sublicensed and a general description of the rights granted). CANbridge or its applicable Affiliate will provide LogicBio with a true and complete copy of each Third Party sublicense agreement, if and as applicable, provided that CANbridge may redact any confidential or proprietary information contained therein that is not necessary for determining compliance with the terms of this Agreement.

2.2.3 Survival of Sublicenses.  Upon termination of this Agreement for any reason, upon the request of any Sublicensee not then in breach of its sublicense agreement or the terms of this Agreement applicable to such Sublicensee, LogicBio hereby automatically grants to each such Sublicensee a direct license on the same terms as this Agreement, taking into account any difference in license scope, territory, and duration of sublicense grant (each a “New License Agreement”).  Within [***] of said

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termination (or such longer time period as is mutually agreed by LogicBio and such Sublicensee), LogicBio will enter into a confirmatory New License Agreement between LogicBio to such Sublicensee. Under any such New License Agreement between LogicBio and such former Sublicensee, such Sublicensee will be required to pay to LogicBio the same amounts in consideration for such direct grant as LogicBio would have otherwise received from CANbridge pursuant to this Agreement on account of such Sublicensee’s Exploitation of the Products had this Agreement not been terminated. Under such New License Agreement, the Parties agree that LogicBio will not be bound by any grant of rights broader than, and will not be required to perform any obligation other than those rights and obligations contained in, this Agreement and all applicable rights of LogicBio set forth in this Agreement will be included in such New License Agreement.

2.3 In-License Agreements.  

2.3.1 Existing In-License Agreements.  Notwithstanding any provision to the contrary in this Agreement, CANbridge acknowledges and agrees that the rights and licenses granted to CANbridge pursuant to Section 2.1 (Licenses to CANbridge) are subject to the applicable terms of each Existing In-License Agreement with respect to the LogicBio Technology, that is being sublicensed thereunder, to the extent such applicable terms have been disclosed to CANbridge or its Affiliates as of the Restatement Date.  Any payment obligations arising under the Existing In-License Agreements as a result of the research, Development and Commercialization of a Product by CANbridge, its Affiliates and Sublicensees under this Agreement will be paid solely by LogicBio. Attached as Schedule 1.69 are all Existing In-License Agreements as amended as of the Restatement Date.

2.3.2 New In-License Agreements.  Certain LogicBio Technology may be acquired by or licensed to LogicBio during the Term pursuant to an agreement with a Third Party executed after the Restatement Date (each, a “New In-License Agreement”).  For any New In-License Agreement pursuant to which LogicBio or its Affiliates would obtain rights to any Know-How or Patent Right that would, if solely-owned and internally developed by LogicBio, be included in the LogicBio Technology hereunder, LogicBio will [***].

2.3.3 New In-License Agreement Royalties.  CANbridge shall [***] royalties arising under the New In-License Agreement(s) as a result of Net Sales by CANbridge, its Affiliates or Sublicensees of Products. [***].

2.3.4 New In-License Agreement Upfront/Milestones.  Any up-front fees or milestone payments arising under any New In-License Agreement shall be [***].

2.3.5 Opt-Out.  Notwithstanding the foregoing provisions of this Section 2.3 (In-License Agreements), CANbridge may elect [***] and, thereafter, CANbridge shall have no obligation to [***] under such New In-License Agreement with respect to such Products, other than any such payments that had accrued prior to such election.

2.4 Licenses to LogicBio.

2.4.1 Collaboration License.  Subject to the terms and conditions of this Agreement, CANbridge hereby grants to LogicBio a royalty-free, non-exclusive license, with the

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right to sublicense solely in accordance with the provisions of Section 2.4.2 (Subcontracting by LogicBio), under the CANbridge Licensed Technology for the purpose of performing the activities allocated to LogicBio under the Pompe and Fabry Research Plan, in each case in accordance with the terms of this Agreement.

2.4.2 Subcontracting by LogicBio.

(a) LogicBiomay enter into subcontracts with Affiliates or Third Parties acting by or for the benefit of LogicBio with respect to the performance of LogicBio’s obligations under this Agreement solely as provided in this Section 2.4.2 (Sublicensing and Subcontracting by LogicBio).

(b) Each subcontract entered into by LogicBio pursuant to this Section 2.4.2 (Subcontracting by LogicBio) will be consistent with the terms of this Agreement and will include invention assignment, confidentiality, nondisclosure, non-use and intellectual property provisions at least as restrictive or protective of the Parties as those set forth in this Agreement, except that the term of any such confidentiality agreement shall be customary for the nature of the sublicensee or subcontractor. No grant of any subcontract to a Third Party or an Affiliate will relieve LogicBio of its obligations hereunder. LogicBio will provide CANbridge with written notice of any such subcontract entered into by LogicBio with a Third Party no later than [***] after the effective date thereof and will provide CANbridge with a true and complete copy of each subcontract agreement, except that LogicBio may redact any confidential or proprietary information contained therein that is not necessary for CANbridge to determine compliance with this Agreement.

2.5 No Implied Licenses.  Except as expressly provided in this Agreement, neither Party will be deemed to have granted the other Party any license or other right with respect to any Intellectual Property of such Party.

2.6 Exclusivity and Restrictions.

2.6.1 Exclusivity Covenant.  Subject to Section 2.6.2 (Effect of Change of Control on Exclusivity) and Section 2.6.3 (Acquisition of Competing Product), during the Term neither Party nor its Affiliates will, directly or indirectly, (a) Exploit a Competing Product in the Territory or (b) license, sell, assign or otherwise provide rights to, or jointly develop with, a Third Party to enable such Third Party to Exploit a Competing Product in the Territory.

2.6.2 Effect of Change of Control on Exclusivity.  Notwithstanding the provisions of Section 2.6.1 (Exclusivity Covenant), if a Party undergoes a Change of Control during the Term and, as of immediately prior to or following the closing of such Change of Control, any Person that becomes an Independent Affiliate of such Party upon such Change of Control or any of such Person’s Affiliates existing immediately prior to such Change of Control or following such Change of Control other than the applicable Party  or Affiliates of such Party existing prior to such Change of Control (collectively, the “Acquirer”) is Exploiting any Competing Product, then the applicable Party will not be in breach of Section 2.6.1 (Exclusivity Covenant) as a result of such activities with respect to any such Competing Product (provided that, with respect to Competing Products that arise after such Change of Control, the Acquirer does not access or use

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any intellectual property Controlled by the applicable Party in the conduct of activities related to such Competing Product), and the applicable Party or the Acquirer, as applicable, will, (a) adopt reasonable procedures to segregate all Exploitation of the Competing Product from Exploitation of Products under this Agreement, and conduct any activities under this Agreement separately from all activities relating to the Competing Product, including through the maintenance of separate lab notebooks and records; and (b) establish reasonable firewall protections and safeguards designed to ensure the activities of its personnel under this Agreement are segregated from all activities relating to the Competing Product (except that management personnel may review and evaluate plans and information regarding the Exploitation of products under this Agreement as well as the Competing Product in connection with portfolio decision-making).

2.6.3 Acquisition of Competing Product. Notwithstanding the provisions of Section 2.6.1 (Exclusivity Covenant), if a Party or any of its Affiliates acquires rights to Exploit a Competing Product as the result of a merger, acquisition or combination with or of a Third Party other than a Change of Control (each, an “Acquisition Transaction”), such Party will, within [***] after the closing of such Acquisition Transaction, notify the other Party in writing of such acquisition and either:

(a) notify the other Party in writing that such Party or its Affiliate will Divest its rights to such Competing Product, in which case, within [***] after the closing of the Acquisition Transaction, such Party or its Affiliate will Divest such Competing Product; or

(b) notify the other Party in writing that it is ceasing all such Exploitation with respect to the Competing Product, in which case, within [***] after the other Party’s receipt of such notice, such Party and its Affiliates will cease all such activities.

Prior to the time of divestiture pursuant to Section 2.6.3(a) or prior to the termination of activities pursuant to Section 2.6.3(b), as applicable, the relevant Party and its Affiliates will (a) adopt reasonable procedures to segregate all Exploitation of the Competing Product from Exploitation of Products under this Agreement, and conduct any activities under this Agreement separately from all activities relating to the Competing Product, including through the maintenance of separate lab notebooks and records; and (b) establish reasonable firewall protections and safeguards designed to ensure the activities of its personnel under this Agreement are segregated from all activities relating to the Competing Product (except that management personnel may review and evaluate plans and information regarding the Exploitation of products under this Agreement as well as the Competing Product in connection with portfolio decision-making).

2.6.4 Non-Solicitation. During the Term, neither Party, nor any Affiliate thereof, will, or will assist any Third Party to, [***].

2.6.5 Restrictions Relating to [***] Manufacturing System. Notwithstanding any provision to the contrary in this Agreement, the license granted to CANbridge under the LogicBio Technology related to the [***] Manufacturing System does not include [***].

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2.7 Release. Each Party, on behalf of itself and each of its Affiliates and their respective officers, directors, inventors, managers, members, employees, contractors, agents, experts, consultants, and successors-in-interest hereby releases the other Party and any Affiliate, successor, or assign thereof, and each of their respective officers, directors, managers, members, employees, experts, consultants, subcontractors and attorneys from any and all claims or Damages of any kind or nature, at law, in equity, or otherwise, known or unknown, suspected or unsuspected, disclosed or undisclosed, relating to, based upon, or arising out of the conduct (or lack of conduct) of any activity under this Agreement prior to the Restatement Date. For the purpose of this Section 2.7 (Release), “claims” means any claims, counterclaims, cross-claims, defenses, allegations, demands, debts, dues, liabilities, requests for declaratory relief, proceedings, actions, or causes of action of any kind and of whatsoever nature or character, arising in any jurisdiction in the world (regardless of whether existing in the past or present, whether known or unknown, or whether accrued, actual, contingent, latent or otherwise) made or brought for the purpose of recovering any damages (including Damages) or royalties or obtaining any equitable relief or any other relief of any kind and any and all claims for reimbursement of legal fees, costs and disbursements.

Article 3
TECHNOLOGY TRANSFER and completion of research

3.1 Technology Transfer and Completion of Research.  

3.1.1 Technology Transfer Requirements. LogicBio will conduct a technology transfer to CANbridge in accordance with the terms set forth in the Technology Transfer Requirements.  The purpose of the Technology Transfer Requirements will be to ensure the complete and timely transfer of the LogicBio Licensed Know-How to CANbridge so that CANbridge is able to use such LogicBio Licensed Know-How for all purposes licensed to CANbridge hereunder. The Parties will [***]. Each Party shall bear its own costs and expenses in the performance of the Technology Transfer Requirements.

3.1.2 Transfer of Materials.  LogicBio will provide CANbridge with certain biological materials or chemical compounds as provided in the Technology Transfer Requirements (collectively, “Materials”).  The Materials will be used only in the performance of activities conducted in accordance with this Agreement and Applicable Law.  CANbridge will use the Materials with prudence and appropriate caution in any experimental work as not all of their characteristics may be known. Concurrently with the delivery of such Materials, LogicBio will provide CANbridge with the most current material safety data sheet for the Materials upon transfer of any Materials. LogicBio will notify CANbridge if any Materials supplied by LogicBio are “Licensed Materials” under the CMRI Agreement, and, without limiting the foregoing, CANbridge’s use of any Materials of LogicBio that are “Licensed Materials” under the CMRI Agreement will be subject to the additional terms and restrictions set forth in the CMRI Agreement with respect thereto. Except as expressly set forth in this Agreement, including the Technology Transfer Requirements, THE MATERIALS ARE PROVIDED “AS IS” AND WITHOUT ANY REPRESENTATION OR WARRANTY, EXPRESS OR IMPLIED, INCLUDING ANY IMPLIED WARRANTY OF MERCHANTABILITY OR OF FITNESS FOR ANY PARTICULAR PURPOSE OR ANY WARRANTY THAT THE USE OF THE MATERIALS WILL NOT INFRINGE OR VIOLATE ANY PATENT OR OTHER PROPRIETARY RIGHTS OF ANY THIRD PARTY.

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3.1.3 Conduct of Research Activities.  LogicBio will conduct the activities allocated to LogicBio under the Pompe and Fabry Research Plan in accordance with the terms thereof and within the timelines set forth therein.

3.1.4 Certain Agreements.  The Technology Transfer Requirements includes a list of key statements of work between LogicBio and Third Party service providers that relate to the activities under this Agreement.  LogicBio agrees to [***] to (a) [***] and (b) [***].  The Parties acknowledge that [***].  For the avoidance of doubt, except [***], LogicBio shall have no obligation to [***].

3.1.5 Limitation on Technology Transfer. Notwithstanding any provision to the contrary in this Agreement, including in the Technology Transfer Requirements, LogicBio will not have any obligation under this Agreement to [***].

3.1.6 Technology Transfer Cooperation. LogicBio will make its relevant personnel reasonably available to CANbridge to respond to CANbridge’s reasonable queries in connection with such technology transfer. Such availability will continue until [***] for questions associated with reports provided to CANbridge on or before [***] and until [***] for questions associated with reports provided to CANbridge on or before [***]. Following such period, LogicBio will not have any obligation under this Agreement to [***].

Article 4REGULATORY MATTERS

4.1 Regulatory Matters for Products.

4.1.1 Responsibility. Except as otherwise provided in this Agreement, CANbridge will be [***].  LogicBio will, and will cause its Affiliates to, [***].

4.1.2 Ownership of Regulatory Submissions.  As between the Parties, all Regulatory Submissions relating to the Products in the Territory within the CANbridge Field will be [***].

4.1.3 Transfer of Regulatory Submissions and Data.

(a) Transfer of Regulatory Submissions. On a Target-by-Target basis, LogicBio has or will deliver and assign to CANbridge (or a mutually agreed upon Third Party) copies (in electronic or other format) of those Regulatory Submissions, if any, Controlled by LogicBio or its Affiliates at such time that relate to the Development, Commercialization or Manufacture of Products with respect to such Target (the “Assigned Regulatory Submissions”).

(b) Disclosure of Pre-Clinical Data. On a Target-by-Target basis, contemporaneously with the assignment of Regulatory Submissions provided for in Section 4.1.3(a) (Transfer of Regulatory Submissions), LogicBio has or will provide to CANbridge (or a mutually agreed upon Third Party) separate copies (in electronic or other format) of the study reports from all studies conducted under the Research Plan that are Controlled by LogicBio and that relate to the Products

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Directed to such Target (to the extent not previously provided to CANbridge or a mutually agreed upon Third Party).

(c) Cooperation. Subject to the terms and conditions of this Agreement, on a Target-by-Target basis, upon the request of CANbridge, LogicBio will execute and deliver, or will cause to be executed and delivered, to CANbridge (or a mutually agreed upon Third Party) such endorsements, assignments, and other documents as may be reasonably necessary to assign, convey, transfer, and deliver to CANbridge, all of LogicBio’s rights, title, and interests in and to the applicable Assigned Regulatory Submissions, including submitting to the applicable Regulatory Authority a letter or other necessary documentation (with copy to CANbridge) notifying such Regulatory Authority of the transfer of ownership of the relevant INDs assigned to CANbridge pursuant to Section 4.1.3(a) (Transfer of Regulatory Submissions).

4.1.4 Safety Database.  CANbridge will own the global safety database with respect to all Products. The Parties will cooperate with regard to the reporting and handling of safety information involving any Product in accordance with Applicable Laws and other regulations on pharmacovigilance and clinical safety.

4.1.5 Pharmacovigilance. Not later than the date on which the first IND with respect to a Product is submitted to a Regulatory Authority, the Parties will discuss and enter into, as appropriate, a pharmacovigilance agreement on reasonable and customary terms that will provide, among other things, guidelines and responsibilities for (a) the receipt, investigation, recording, review, communication, reporting, and exchange between the Parties of Adverse Event reports and other safety information relating to the Products, (b) appropriate reconciliation procedures to ensure adequate and compliant exchange of safety data related to the Products, and (c) contact with Regulatory Authorities with respect to the foregoing, in each case ((a)-(c)), in accordance with Applicable Law (each, a “Pharmacovigilance Agreement”). The Pharmacovigilance Agreement will contain terms no less stringent than those required by ICH Guidelines or other applicable guidelines in order to allow the Parties to meet the applicable regulatory and legal requirements regarding the management of safety data under Applicable Law throughout the Territory.

4.1.6 Recalls and Voluntary Withdrawals.  CANbridge will use [***] to notify LogicBio promptly following its determination that any event, incident, or circumstance has occurred that may result in the need for a recall, market suspension, or market withdrawal of a Product in the Territory.  CANbridge will have the [***]. For all recalls, market suspensions, or market withdrawals undertaken pursuant to this Section 4.1.6 (Recalls and Voluntary Withdrawals), CANbridge will be [***].

4.1.7 Right of Reference.  Each Party hereby grants to the other Party and its designees a right of reference under all Regulatory Submissions Controlled by said Party or its Affiliates for each Product that pertain solely to the safety of the Product, as reasonably determined by the grantor Party, in the case of LogicBio as the grantee Party, for purposes of obtaining and maintaining approvals for products other than the Products or to Develop products other than the Products anywhere in the world and, in the case of CANbridge as the grantee Party, for purposes of obtaining and maintaining approvals for the Products or to Develop the Products anywhere in the world. Each Party will, and will ensure that its Affiliates will, take actions reasonably necessary to

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effect such grant of right of reference to the other Party (or its designee), including by providing the other Party with sufficient rights to grant the right of reference described in this Section 4.1.7(Right of Reference) and making such filings as may be required by Regulatory Authorities to record such grant.

Article 5
CLINICAL DEVELOPMENT AND COMMERCIALIZATION

5.1 Development and Commercialization of Products.

5.1.1 Clinical Development and Medical Affairs.  Except with respect to activities that may be allocated to LogicBio under the Pompe and Fabry Research Plan or the Technology Transfer Requirements, CANbridge will have [***] with respect to, the Development of, and the performance of all Medical Affairs with respect to, all Products in the CANbridge Field.  

5.1.2 Commercialization.

(a) Activities.  Subject to the terms of this Agreement, CANbridge will have [***], the Commercialization of all Products.

(b) Trademarks.  CANbridge will own all Trademarks used in connection with the Exploitation of the Products in the CANbridge Field in the Territory, and have [***], for all matters relating to the selection and use of such trademarks, including the selection, filing, prosecution, maintenance, defense, and enforcement thereof. Throughout the Term and thereafter, LogicBio will not adopt or use, register or attempt to register in the Territory any Trademark, domain name, or similar commercial symbol that includes, or is confusingly similar to, CANbridge’s trademarks used in connection with any Products.

5.1.3 Diligence Obligations. CANbridge, itself or through its Affiliates, Sublicensees, or subcontractors, will use Commercially Reasonable Efforts to Develop and seek Regulatory Approval for one Product directed to each Target in each of [***].

PAYMENTS AND ROYALTIES

6.1 Upfront Payment.  Within [***] after the Effective Date, [***], CANbridge will pay LogicBio a one‑time, non‑refundable, non‑creditable upfront payment of Ten Million Dollars ($10,000,000). LogicBio hereby acknowledges the full and timely payment of such amount by CANbridge.

6.2 Payment of Research Costs. The Parties agree and acknowledge that certain research payments were made by CANbridge to LogicBio prior to the Restatement Date and LogicBio will have no obligation to [***]. The Parties further agree that [***].

6.3 Milestone Payments.

6.3.1 Development Milestones for Products.  On a Target-by-Target basis, within [***] after the first achievement of each milestone event set forth in this Section 6.3.1 (Development Milestones for Products) (each, a “Development Milestone Event”)

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for the first Product Directed to a Target by or on behalf of CANbridge, any of its Affiliates or any Sublicensee, CANbridge will notify LogicBio in writing of the achievement of such Development Milestone Event and, within [***] of CANbridge’s receipt of an invoice for such payment, CANbridge will make a non‑refundable and non‑creditable milestone payment to LogicBio in the amount set forth in this Section 6.3.1(Development Milestones for Products) corresponding to such Development Milestone Event (each, a “Development Milestone Payment”). Each Development Milestone Payment will be payable [***].

TABLE 6.3.1 – Development Milestones for Products
Development Milestone Event	Development Milestone Payment
[***]	[***]
[***]	[***]
[***]	[***]
[***]	[***]
[***]	[***]

[***].

6.3.2 Sales Milestones for Products. Within [***] after the first achievement of each milestone event set forth in this Section 6.3.2 (Sales Milestones for Products) (each, a “Sales Milestone Event”) for each Product by or on behalf of CANbridge, any of its Affiliates or any Sublicensee, CANbridge will notify LogicBio in writing of the achievement of such Sales Milestone Event and, within [***] of CANbridge’s receipt of an invoice for such payment, CANbridge will make a non‑refundable and non‑creditable milestone payment to LogicBio in the amount set forth in this Section 6.3.2 (Sales Milestones for Products) corresponding to such Sales Milestone Event (each, a “Sales Milestone Payment”). [***].

TABLE 6.3.2 – Sales Milestones for Products
Sales Milestone Event	Sales Milestone Payment
[***]	[***]
[***]	[***]
[***]	[***]

6.4 Royalties for Products.

6.4.1 Royalty Payments.  On a Product‑by‑Product and country‑by‑country basis, during the Royalty Period of a Product in a country in the Territory, CANbridge will pay to LogicBio tiered royalties on the Net Sales of such Product in the CANbridge Field during each Calendar Year at the following rates:

TABLE 6.4.1 – Royalty Payments
Net Sales of Product in the CANbridge Field in the Territory During the Relevant Calendar Year (“Per Product Annual Net Sales”)	[***]

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Global Net Sales of the Product in the CANbridge Field in a Calendar Year of up to $[***]	[***]
The portion of global Net Sales of the Product in the CANbridge Field in a Calendar Year which is in excess of $[***] but less than or equal to $[***]	[***]
The portion of global Net Sales of the Product in the CANbridge Field in a Calendar Year which is in excess of $[***] but less than or equal to $[***]	[***]
The portion of global Net Sales of the Product in the CANbridge Field in a Calendar Year which is in excess of $[***]	[***]

Each marginal royalty rate set forth in Table 6.4.1 (Royalty Payments) above will apply only to that portion of Per Product Annual Net Sales that falls within the indicated range.

6.4.2 Royalty Period.  [***]

6.4.3 Royalty Reports; Payments.

(a) Flash Reports. No later than [***] after the end of each Calendar Quarter during which any Royalties are owed, CANbridge will submit to LogicBio a written flash report of CANbridge’s reasonable, good faith estimate of (i) Net Sales of Products sold, in the currency for which such Products were sold, by or on behalf of CANbridge and its Affiliates and Sublicensees during such Calendar Quarter, and (ii) the royalties payable on such Net Sales.

(b) Royalty Reports.  No later than [***] after the end of each Calendar Quarter during which any Royalties are owed, CANbridge will submit to LogicBio a written report of Net Sales of Products sold, in the currency for which such Products were sold, by or on behalf of CANbridge and its Affiliates and Sublicensees during such Calendar Quarter, and the royalties payable on such Net Sales [***] paid hereunder.

(c) Royalty Payments.  Royalties will be payable on a Calendar Quarter basis and CANbridge will make any such payments within [***] after the end of the Calendar Quarter during which the applicable Net Sales of Products occurred.

6.4.4 Payment Adjustments.

(a) Expiration of Valid Claims and Regulatory Exclusivity.  On a Product-by-Product and country-by-country basis, if during the Royalty Period for a Product in a given country, there is no Valid Claim within the LogicBio Technology Covering the manufacture, use or sale of such Product in such country and no Regulatory Exclusivity with respect to such Product in such country, then commencing in the [***] after the date on which this Section 6.4.4(a) (Expiration of Valid Claims) applies and for the remainder of the Royalty Period for such Product in such country during which there remains

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no Valid Claim within the LogicBio Technology Covering the manufacture, use or sale of such Product in such country and no Regulatory Exclusivity with respect to such Product in such country, the royalty rates set forth in Table 6.4.1(Royalty Payments) with respect to such Product in such country will be reduced by [***] for the purposes of determining the royalties payable under Section 6.4.1(Royalty Payments), subject to Section 6.4.4(d)(Maximum Payment Adjustments).

(b) Biosimilar Competition. If during any Calendar Quarter during the Royalty Period for a Product in a given country, [***]and such Product sold in such Calendar Quarter in such country (as determined by data obtained from a mutually agreed upon Third Party source), then the percentage royalty payable on Net Sales of such Product in such country will be reduced by [***] for such Calendar Quarter.

(c) Third Party Payments.  If CANbridge makes a royalty payment under any agreement with a Third Party pursuant to which CANbridge obtains a license or other rights under a Patent Right(s) owned or controlled by such Third Party (whether by acquisition or license) that is necessary to Exploit one or more Products in the CANbridge Field, then CANbridge may offset against the royalties due to LogicBio for such Products an amount equal to [***] of the royalties paid to such Third Party under such agreement in connection with the Exploitation of Products, in all cases, subject to Section 6.4.4(d) (Maximum Payment Adjustments).

(d) Maximum Payment Adjustments. In no event will the royalty rate applicable to any Product in a given Calendar Quarter be less than [***] as a result of the aggregate reductions permitted pursuant to Section 6.4.4(a) (Expiration of Valid Claims), Section 6.4.4(b) (Biosimilar Competition) and Section 6.4.4(c) (Third Party Payments).

6.5 Payment Method.  All payments to be made between the Parties under this Agreement will be made in Dollars and may be paid by wire transfer in immediately available funds to a bank account designated by the receiving Party.

6.6 Currency Exchange.  The rate of exchange to be used in computing the amount of currency equivalent in U.S. Dollars owed to a Party under this Agreement will be the monthly average exchange rate between each currency of origin and U.S. Dollars as reported by [***] or an equivalent resource as agreed by the Parties.

6.7 Late Payments.  If a Party does not receive payment of any sum due to it on or before the due date, simple interest will thereafter accrue on the sum due to such Party until the date of payment at the per annum rate equal to [***].

6.8 Income Tax Withholding. Except as otherwise provided in this Section 6.8 (Income Tax Withholding), each Party will pay all income and other taxes (including interest) imposed on or measured with respect to its own income accruing to it under this Agreement (“Taxes”). [***], provided, however, [***]. [***].

6.9 Foreign-Derived Deduction Eligible Income Reporting.  CANbridge will obtain and deliver to LogicBio, reasonably promptly following LogicBio’s request to provide, information as reasonably

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requested by LogicBio to meet any documentation requirements imposed by regulations issued under Section 250 of the Internal Revenue Code for the treatment of an appropriate portion of such amounts as “foreign-derived deduction eligible income” within the meaning of Section 250 of the Internal Revenue Code and the regulations thereunder.

6.10 Value Added Tax.  It is understood and agreed between the Parties that any payments made by any Party under this Agreement are [***] any value added tax or similar tax imposed upon such payments. Where such tax is properly chargeable in respect of any supply of goods or services made under this Agreement, [***].

6.11 Record Retention; Audits. Each Party will maintain and will cause its Affiliates and all Sublicensees to maintain, complete and accurate records in sufficient detail to permit the other Party to confirm the accuracy of the calculation of payments under this Agreement. Upon prior notice of at least [***], but not more than [***], such records will be available during regular business hours for a period of [***] from the end of the Calendar Year to which they pertain for examination at the expense of the requesting Party by an independent certified public accountant selected by the requesting Party  (or, if LogicBio is the requesting Party, CMRI) and reasonably acceptable to the other Party, for the sole purpose of verifying the accuracy of the financial reports and correctness of the payments furnished by the other Party pursuant to this Agreement. Any such auditor will not disclose the other Party’s Confidential Information, except to the extent such disclosure is necessary to verify the accuracy of the financial reports furnished by the other Party or the amount of payments due by the other Party under this Agreement. The accountant’s report will be disclosed simultaneously to both Parties, and such report will be the Confidential Information of each Party (with both Parties being deemed the Receiving Party with respect thereto) and subject to the terms Article 8 (Confidentiality); provided that, if LogicBio is the requesting Party, the Parties may disclose each such report to CMRI under the obligations of confidentiality and non-use set forth in the CMRI Agreement. Any amounts shown to be owed but unpaid will be paid within [***] after the date of the accountant’s report.  Any amounts shown to have been overpaid will be refunded within [***] after the date of the accountant’s report. The requesting Party will [***] of such audit unless such audit discloses an underpayment by the other Party of more than the greater of (a) [***] of the amount due and (b) [***]. The audit rights in this Section 6.11 (Record Retention; Audits) will survive the Term for [***] following the effective date of any termination or expiration of this Agreement.

Article 7
REPRESENTATIONS, WARRANTIES, AND COVENANTS

7.1 Mutual Representations and Warranties of the Parties.  Each Party represents and warrants to the other Party as of the Effective Date and the Restatement Date that:

7.1.1 Organization.  It is a corporation duly organized, validly existing, and in good standing under the laws of the jurisdiction of its organization, and has all requisite power and authority, corporate or otherwise, to execute, deliver, and perform this Agreement.

7.1.2 Binding Agreement.  This Agreement is a legal and valid obligation binding upon such Party and enforceable in accordance with its terms, subject to the effects of bankruptcy, insolvency, or other laws of general application affecting the enforcement of creditor rights, judicial principles affecting the availability of specific performance, and general principles of equity (whether enforceability is considered a proceeding at law or equity).

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7.1.3 Authorization. The execution, delivery, and performance of this Agreement by such Party has been duly authorized by all necessary corporate action and does not conflict with any agreement, instrument, or understanding, oral or written, to which it is a party or by which it is bound, nor violate any Applicable Law or any order, writ, judgment, injunction, decree, determination, or award of any court or governmental body, or administrative or other agency presently in effect applicable to such Party.

7.1.4 No Further Approval.  It is not aware of any government authorization, consent, approval, license, exemption, or filing or registration with any court or governmental department, commission, board, bureau, agency or instrumentality, domestic or foreign, under any Applicable Law, currently in effect, necessary for, or in connection with, the transactions contemplated by this Agreement or any other agreement or instrument executed in connection herewith, or for the performance by it of its obligations under this Agreement and such other agreements (except for Regulatory Approvals, Pricing and Reimbursement Approvals, and similar authorizations from Governmental Authorities necessary for the Exploitation of Products as contemplated hereunder).

7.1.5 No Inconsistent Obligations.  It is not under any obligation, contractual or otherwise, to any Person that conflicts with or is inconsistent in any material respect with the terms of this Agreement, or that would impede the diligent and complete fulfillment of its obligations hereunder.

7.1.6 No Debarment.  Neither it nor any of its respective Affiliates has been Debarred by the FDA, is not subject to any similar sanction of other Governmental Authorities in the Territory, and, to its knowledge, neither it nor any of its respective Affiliates has used or engaged, in any capacity, in connection with this Agreement or any ancillary agreements (if any), any Person who either has been Debarred by such a Regulatory Authority.

7.2 Additional Representations and Warranties of LogicBio.  LogicBio represents and warrants to CANbridge as of the Effective Date (subject to Schedule 9.2 (Disclosure Schedule) of the Original Agreement) and as of the Restatement Date (except as otherwise set forth in Schedule 7.2 (Disclosure Schedule)):

7.2.1 it has the full right, power, and authority to grant all of the licenses and rights granted to CANbridge under this Agreement;

7.2.2 LogicBio’s rights, title, and interests to all the LogicBio Technology are free of any lien, encumbrance, charge, security interest, mortgage, or liability;

7.2.3 (a) Schedule 1.114 (LogicBio Licensed Patent Rights) sets forth a complete and accurate list of all Patent Rights that are owned or otherwise Controlled by LogicBio or any of its Affiliates and that claim any LogicBio Licensed Know-How, other than any such Patent Right that is a provisional patent application that claims any LogicBio Licensed Know-How that is [***] for the Manufacture of Products (the “LogicBio Licensed Patent Rights Exception”); (b) LogicBio owns or otherwise Controls all Patent Rights listed on Schedule 1.114 (LogicBio Licensed Patent Rights); and (c) except as otherwise noted on Schedule 1.114 (LogicBio Licensed Patent Rights), LogicBio either exclusively owns all rights, title, and interests in and to such Patent Rights, or where LogicBio does not exclusively own any such Patent Right, Schedule 1.114 (LogicBio Licensed Patent Rights) identifies the Third Party that, to LogicBio’s

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Knowledge, Controls such Patent Rights and the agreement pursuant to which LogicBio Controls such Patent Right;

7.2.4 (a) all LogicBio Licensed Patent Rights that are owned by LogicBio, are being diligently prosecuted in the respective patent offices in accordance with Applicable Law; (b) to LogicBio’s knowledge, the inventorship of the LogicBio Licensed Patent Rights is properly identified on each issued patent or patent application in the LogicBio Licensed Patent Rights; and (c) all fees required to be paid by LogicBio in any jurisdiction in order to maintain the LogicBio Licensed Patent Rights have been timely paid and the LogicBio Licensed Patent Rights are subsisting, and to LogicBio’s Knowledge, valid and enforceable;

7.2.5 there is no pending litigation, or [***], litigation that has been threatened against LogicBio in writing, that alleges, or any written communication received by LogicBio alleging, that LogicBio’s practice of the LogicBio Technology prior to the Effective Date has infringed, misappropriated, or otherwise violated the Intellectual Property of any Third Party;

7.2.6 there are no claims, judgments, or settlements against or pending with respect to the LogicBio Technology or amounts with respect thereto, owed by LogicBio or any of its Affiliates, and LogicBio has not received written notice threatening any such claims, judgments, or settlements;

7.2.7 [***], no Third Party has challenged the ownership, scope, duration, validity, enforceability, priority, or right to use any LogicBio Licensed Patent Rights (including, by way of example, through the institution of or written threat of institution of interference, inter partes review, reexamination, protest, opposition, nullity, or similar invalidity proceeding before the United States Patent and Trademark Office or any foreign patent authority or court);

7.2.8 [***], no Third Party is infringing, misappropriating, or otherwise violating, or threatening to infringe, misappropriate, or otherwise violate the LogicBio Technology;

7.2.9 LogicBio has not previously assigned, transferred, conveyed, or granted any license or other rights under the LogicBio Technology;

7.2.10 to the extent permissible under Applicable Law, all employees, agents, advisors, consultants, contractors or other representatives of LogicBio or its Affiliates performing activities under this Agreement are and will be under an obligation to assign all rights, title, and interests in and to their Inventions and other Know-How, whether or not patentable, and Intellectual Property therein, to LogicBio or its Affiliates as the sole owner thereof, and CANbridge will have no obligation to contribute to any remuneration of any inventor employed or previously employed by LogicBio or any of its Affiliates in respect of any such Inventions and other Know-How and Intellectual Property therein that are so assigned to LogicBio or its Affiliate(s);

7.2.11 Other than the Existing In-License Agreements, there are no Third Party agreements pursuant to which LogicBio Controls any of the LogicBio Technology;

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7.2.12 no written notice of default or termination has been received or given under any agreement pursuant to which LogicBio Controls any LogicBio Technology;

7.2.13 LogicBio and its Affiliates have taken [***] consistent with industry practices to protect the secrecy, confidentiality, and value of all LogicBio Licensed Know-How that constitutes trade secrets under Applicable Law (including requiring all employees, consultants, and independent contractors to execute binding and enforceable agreements requiring all such employees, consultants, and independent contractors to maintain the confidentiality of such LogicBio Licensed Know-How);

7.2.14 except as expressly set forth on Schedule 7.2.14, the LogicBio Technology has not been created pursuant to, and is not subject to, any funding agreement with any Governmental Authority or any Third Party, and is not subject to the requirements of the Bayh-Dole Act or any similar provision of any Applicable Law; LogicBio and its Affiliates have complied [***] with all reporting requirements under Applicable Law with respect to those Inventions and Patent Rights identified on Schedule 7.2.14;

7.2.15 LogicBio has provided to CANbridge a complete list of all agreements relating to the Manufacture or supply of the Products and components thereof that are in effect (excluding any agreements for the purchase of ordinary course components or materials acquired by purchase order or without a supply agreement), which complete list appears on Schedule 7.2.15; there are no exclusivity provisions or any other restrictions in any agreement between LogicBio or its Affiliates, on the one hand, and any Third Party relating to the Manufacture or supply of the Products and components thereof to LogicBio, on the other hand, that would limit CANbridge’s ability to Manufacture, or have the Products and components thereof Manufactured;

7.2.16 all works of authorship and all other materials subject to copyright protection included in LogicBio Licensed Know-How are original and were either created by employees of LogicBio or its Affiliates within the scope of their employment or are otherwise works made for hire, or all right, title, and interest in and to such materials have been legally and fully assigned and transferred to LogicBio or such Affiliate, and all rights in all Inventions and discoveries, developed or invented by any employee or independent contractor of LogicBio or such Affiliate during the course of their employment (or other retention) by LogicBio or such Affiliate, and included in LogicBio Licensed Know-How, or that are the subject of one or more LogicBio Licensed Patent Rights, have been assigned in writing to LogicBio or its Affiliate; and

7.2.17 None of the LogicBio Licensed Patent Rights Exceptions are specifically related to the Targets or the Products.

7.3 Covenants of LogicBio.

7.3.1 LogicBio covenants to CANbridge that LogicBio will not, and will cause its Affiliates not to license, sell, assign, or otherwise transfer to any Person any LogicBio Technology (or agree to do any of the foregoing).

7.3.2 LogicBio will (a) maintain Control of all LogicBio Licensed Know-How; (b) not breach or be in default under any In-License Agreements in a manner that would give rise to a right of termination under any such agreement; and (c) not terminate or amend any In-License Agreements in a manner that adversely affects CANbridge’s rights

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under this Agreement with respect to the Products or Targets, without CANbridge’s prior written consent.  If LogicBio receives notice of any alleged [***] breach under any In-License Agreement, then LogicBio will promptly, but in no event less than [***] thereafter, provide written notice thereof to CANbridge and grant CANbridge the right (but not the obligation) to cure any such alleged breach.

7.4 Mutual Covenants.

7.4.1 Neither Party nor its Affiliates will use or engage, in any capacity, in connection with this Agreement or any ancillary agreements (if any), any Person who either has been Debarred by a Regulatory Authority.  Each Party will inform the other Party in writing promptly if it or any Person engaged by it or any of its Affiliates who is performing services under this Agreement or any ancillary agreements (if any) is Debarred, or if any action, suit, claim, investigation, or legal or administrative proceeding is pending or, to such Party’s knowledge (or LogicBio’s Knowledge in the case of LogicBio), is threatened, relating to the debarment or conviction of such Party, any of its Affiliates or any such Person performing services hereunder or thereunder.

7.4.2 Each Party and its Affiliates will comply [***] with all Applicable Laws (including all anti-bribery laws and export control laws) in the Exploitation of the Products and performance of its obligations and exercise of its rights under this Agreement.

7.4.3 Neither Party will [***] transfer to the other Party any goods, software, technology, or services that are (a) controlled at a level other than EAR99, or for reasons other than anti-terrorism, under the U.S. Export Administration Regulations; (b) controlled under the U.S. International Traffic in Arms Regulations; (c) specifically identified as an E.U. Dual Use Item; or (d) on an applicable export control list of a jurisdiction within the Territory.

7.5 Transparency Reporting.  Each Party will be responsible for tracking and reporting transfers of value initiated and controlled by its and its Affiliates’ employees, contractors, and agents pursuant to the requirements of the transparency or marketing reporting laws of any Governmental Authority in the Territory, including Section 6002 of the ACA, commonly referred to as the “Sunshine Act.”

7.6 DISCLAIMER OF WARRANTIES.  EXCEPT AS OTHERWISE EXPRESSLY SET FORTH HEREIN, NEITHER PARTY MAKES ANY REPRESENTATION OR EXTENDS ANY WARRANTIES OF ANY KIND, EITHER EXPRESS OR IMPLIED, INCLUDING ANY IMPLIED WARRANTIES OF TITLE, NON-INFRINGEMENT, MERCHANTABILITY, OR FITNESS FOR A PARTICULAR PURPOSE. IN PARTICULAR, NEITHER PARTY MAKES ANY REPRESENTATION NOR EXTENDS ANY WARRANTY THAT THE DEVELOPMENT CANDIDATES OR PRODUCTS WILL BE SUCCESSFULLY DEVELOPED OR COMMERCIALIZED HEREUNDER.

7.7 LIMITATION OF LIABILITY.  EXCEPT FOR DAMAGES RESULTING FROM BREACHES OF [***] AND WITHOUT LIMITING EITHER PARTY’S OBLIGATIONS IN RESPECT OF [***], IN NO EVENT WILL EITHER PARTY HAVE ANY CLAIMS AGAINST OR LIABILITY TO THE OTHER PARTY WITH RESPECT TO ANY INDIRECT, PUNITIVE, SPECIAL, INCIDENTAL, OR CONSEQUENTIAL DAMAGES, OR ANY CLAIMS FOR LOST PROFITS OR REVENUES, ARISING UNDER OR IN CONNECTION WITH THIS AGREEMENT UNDER ANY THEORY OF LIABILITY, EVEN IF SUCH PARTY HAS BEEN INFORMED OR SHOULD HAVE KNOWN OF THE POSSIBILITY OF SUCH DAMAGES.

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Article 8
CONFIDENTIALITY

8.1 Confidential Information.  It is understood and agreed by the Parties that:

8.1.1 The terms and conditions, but not the existence, of this Agreement (and, for clarity, the Original Agreement) will be considered Confidential Information of both Parties, with each Party being deemed a Receiving Party with respect thereto, and kept confidential by each of the Parties in accordance with this Article 8 (Confidentiality).

8.1.2 The Know-How and Patent Rights Controlled by a Party and all reports, information, and data provided by a Party to the other Party or its Affiliates or representatives hereunder will be considered such owning, Controlling or providing Party’s Confidential Information, as applicable, except that, (a) in addition to any information that is expressly designated herein as the Confidential Information of both Parties, any findings in respect of any audit or inspection performed under this Agreement will also be the Confidential Information of both Parties, unless expressly identified as the Confidential Information of a single Party hereunder, and (b) any Know-How or Patent Rights assigned by one Party to the other pursuant to the terms of this Agreement will be considered Confidential Information of the Party to whom such Know-How or Patent Rights are assigned.

8.2 Non-Disclosure and Non-Use Obligation.  Except as otherwise expressly set forth herein, the Receiving Party will, during the Term and for a period of [***] thereafter, keep the Confidential Information of the Disclosing Party confidential using at least the same degree of care with which the Receiving Party holds its own Confidential Information [***] and will not (a) disclose such Confidential Information to any Person without the prior written approval of the Disclosing Party, except, solely to exercise its rights or perform its obligations under this Agreement, to its employees, Affiliates, Sublicensees, and Subcontractors, consultants, or agents who have a need to know such Confidential Information, all of whom will be similarly bound by confidentiality, non-disclosure, and non-use provisions at least as restrictive or protective of the Parties as those set forth in this Agreement and for whom the Disclosing Party will be responsible, or (b) use such Confidential Information for any purpose other than for the purposes contemplated by this Agreement. The Receiving Party will use [***] to cause the foregoing Persons to comply with the restrictions on use and disclosure set forth in this Section 8.2 (Non-Disclosure and Non-Use Obligation) and will be responsible for ensuring that such Persons maintain the Disclosing Party’s Confidential Information in accordance with this Article 8 (Confidentiality). Each Party will promptly notify the other Party of any misuse or unauthorized disclosure of the other Party’s Confidential Information.

8.3 Exemptions.  Information of a Disclosing Party will not be Confidential Information of such Disclosing Party to the extent that the Receiving Party can demonstrate through competent evidence that such information: (a) is already in the possession of the Receiving Party at the time of its receipt from the Disclosing Party and not through a prior disclosure by or on behalf of the Disclosing Party, (b) is generally available to the public before its receipt from the Disclosing Party, (c) became generally available to the public or otherwise part of the public domain after its disclosure by the Disclosing Party and other than through any act or omission of the Receiving Party or any of its Affiliates or disclosees in breach of this Agreement, including pursuant to Section 8.9.2 (Publication Rights), (d) is subsequently disclosed to the Receiving Party or any of its Affiliates without obligation of confidentiality by a Third Party who may rightfully do so and is not under a conflicting obligation of confidentiality to the Disclosing Party, or (e) is developed independently by employees, Subcontractors, consultants or agents of the Receiving Party or any of its Affiliates

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without use of or reliance upon the Disclosing Party’s Confidential Information. No combination of features or disclosures will be deemed to fall within the foregoing exclusions merely because individual features are published or available to the general public or in the rightful possession of the Receiving Party unless the combination itself and principle of operation are published or available to the general public or in the rightful possession of the Receiving Party.

8.4 Permitted Disclosures.  In addition to the exceptions contained in Section 8.2 (Non-Disclosure and Non-Use Obligation) and Section 8.3 (Exemptions), the Receiving Party may disclose Confidential Information of the Disclosing Party to the extent (and solely to the extent) that such disclosure is reasonably necessary in the following instances:

8.4.1 (a) the Prosecution of Patent Rights as contemplated by this Agreement; or (b) Regulatory Submissions and other filings with Governmental Authorities (including Regulatory Authorities), as necessary for the Exploitation of a Product;

8.4.2 disclosure of the existence and applicable terms of this Agreement, the status and results of Exploitation of one or more Products to actual or bona fide potential investors, acquirors, Sublicensees, lenders, and other financial or commercial partners, and their respective attorneys, accountants, banks, investors, and advisors, solely for the purpose of evaluating or carrying out an actual or potential investment, acquisition, sublicense, debt transaction, or collaboration; provided that, in each such case, (a) such Persons are bound by obligations of confidentiality, non-disclosure, and non-use provisions at least as restrictive or protective of the Parties as those set forth in this Agreement or otherwise customary for such type and scope of disclosure, (b) that any such disclosure is limited to the maximum extent practicable for the particular context in which it is being disclosed, and (c) notwithstanding the foregoing (a) and (b), that the term of such confidentiality obligation must be consistent with industry standards, but in all cases at least [***];

8.4.3 if required by Applicable Law, including as may be required in connection with any filings made with, or by the disclosure policies of a securities exchange (as set forth in additional detail in Section 8.5 (Confidential Treatment)); provided that the Party seeking to disclose the Confidential Information of the other Party: (a) use all reasonable efforts to inform the other Party prior to making any such disclosures and cooperate with the other Party in seeking a protective order or other appropriate remedy (including redaction); and (b) whenever possible, request confidential treatment of such information in accordance with Section 8.5 (Confidential Treatment);

8.4.4 to prosecute or defend litigation so long as there is [***] prior written notice given by the Receiving Party before filing, and to enforce Patent Rights in connection with the Receiving Party’s rights and obligations pursuant to this Agreement; and

8.4.5 to allow the Receiving Party to exercise its rights and perform its obligations hereunder, provided that such disclosure is covered by terms of confidentiality and non-use at least as restrictive as those set forth herein.

If and whenever any Confidential Information is disclosed in accordance with this Section 8.4 (Permitted Disclosures), such disclosure will not cause any such information to cease to be Confidential Information except to the extent that such disclosure results in a public disclosure of such information (other than by breach of this Agreement).

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8.5 Confidential Treatment.  Notwithstanding any provision to the contrary set forth in this Agreement, if a Party is required to make a disclosure of the other Party’s Confidential Information pursuant to Section 8.4.3and Section 8.4.4, then it will, to the extent not prohibited by Applicable Law or judicial or administrative process, except where impracticable, give reasonable advance notice to the other Party of such proposed disclosure and use reasonable efforts to secure confidential treatment of such information and will only disclose that portion of Confidential Information that is legally required to be disclosed as advised by its legal counsel.  In any event, each Party agrees to take all reasonable action to avoid disclosure of Confidential Information of the other Party hereunder.  In addition, the Parties acknowledge that either or both Parties may be obligated to file a copy of this Agreement (or portions of this Agreement or an abstract of the terms of this Agreement) with the SEC or other Governmental Authorities.  Each Party will be entitled to make such a required filing, provided that it initially files a redacted copy of this Agreement (or portions of this Agreement or an abstract of the terms of this Agreement) (“Redacted Agreement”) and requests confidential treatment of the terms redacted from this Agreement for a reasonable period of time.  In the event of any such filing, each Party will (a) permit the other Party to review and comment upon such request for confidential treatment [***] reasonably in advance of its submission to the SEC or such other Governmental Authorities, and (b) [***] the other Party’s comments thereon to the extent consistent with the then-current legal requirements governing redaction of information from material agreements that must be publicly filed in the applicable country.  Each Party will be responsible for its own legal and other external costs in connection with any such filing, registration, or notification.

8.6 Relationship to Confidentiality Agreement.  This Agreement supersedes the Confidentiality Agreement; provided, however, that all “Confidential Information” disclosed or received by the Parties and their Affiliates thereunder will be deemed the Confidential Information of the originally disclosing Party hereunder and will be subject to the terms and conditions of this Agreement.

8.7 Equitable Relief.  Given the nature of the Confidential Information and the competitive damage that could result to a Party upon unauthorized disclosure, use, or transfer of its Confidential Information to any Third Party, the Parties agree that monetary damages may not be a sufficient remedy for any breach of this Article 8 (Confidentiality).  In addition to all other remedies, a Party will be entitled to seek specific performance and injunctive and other equitable relief as a remedy for any breach or threatened breach of this Article 8 (Confidentiality).

8.8 Use of Name and Logo. Subject to Section 8.9.1 (Announcement), neither LogicBio nor CANbridge will use the other Party’s or its Affiliates’ (or, if the other Party is LogicBio, CMRI’s or any Co-Owner’s) name or logo in any label, press release, or product advertising, or for any other promotional purpose, without first obtaining the other Party’s written consent.

8.9 Publications.

8.9.1 Announcements. Except as may be expressly permitted under Section 8.4 (Permitted Disclosures), neither Party will make any public announcement regarding this Agreement without the prior written approval of the other Party, except for either Party’s references to the other as the licensor or licensee (as applicable) or a collaboration partner under this Agreement. Each Party will be permitted to issue press releases indicating the payment of amounts due hereunder, in each case upon the prior written consent of the other Party (consent not to be unreasonably withheld, conditioned or delayed).  After the issuance of permitted public disclosure by a Party, either Party may make subsequent public disclosures reiterating such information without having to obtain the other Party’s prior consent and approval so long as the

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information in such other public announcement remains true, correct, and the most current information with respect to the subject matters set forth therein.  For clarity, nothing in this Agreement will prevent CANbridge from making any scientific publication or public announcement concerning CANbridge’s Development, Manufacture or Commercialization activities with respect to any Product under this Agreement.

8.9.2 Publication Rights.  CANbridge may, in its sole discretion, publish or present results of activities conducted by or on behalf of CANbridge with respect to a Product, provided, however, that LogicBio will have the right to review all such proposed publications and presentations prior to submission of such publication or prior to presentation, as applicable, in accordance with the procedures set forth in this Section 8.9.2 (Publication Rights).  If CANbridge intends to make any such publication or presentation with respect to any Product, then CANbridge will first provide LogicBio with a copy of the applicable proposed abstract, manuscript, or presentation no less than [***] prior to its intended submission for publication or presentation. LogicBio will respond in writing promptly and in no event later than [***] after receipt of the proposed material with (i) any concerns regarding any the disclosure of any information or subject matter that, [***], would present issues as to patentability of any invention that, as between the Parties, would be owned by LogicBio or (ii) any request for the removal of any of LogicBio’s Confidential Information. In the event of any concern raised regarding protection of Intellectual Property rights of LogicBio, CANbridge will not submit such publication or make such presentation that contains such information for [***] after the date on which CANbridge provided the copy of the proposed publication or presentation to LogicBio. Subject to Section 10.4 (Permitted Disclosures), CANbridge will remove any Confidential Information of LogicBio for which LogicBio requests such removal from any such proposed publication or presentation. For clarity, LogicBio may, in its sole discretion, publish any LogicBio Technology, excluding the Joint Collaboration Know-How.

8.9.3 Clinical Trial Transparency.  Both Parties agree to collaborate to maintain compliance with all Applicable Law related to Clinical Trial transparency, as well as any industry guidelines/codes of conduct, or other obligations that may apply to either the sponsor of any Clinical Trial or the owner of any Regulatory Approval, as related to any Product. The Parties will cooperate to maintain Clinical Trial transparency consistent with each sponsor’s Clinical Trial registration, summary result, and data sharing transparency policies and will support disclosure of Confidential Information as needed based on the needs of the sponsors of the study or the Regulatory Approval holder with respect to any Product.

Article 9
INTELLECTUAL PROPERTY

9.1 Ownership.

9.1.1 Background Technology.  CANbridge will own and retain all of its rights, title and interests in and to the CANbridge Background Technology, and LogicBio will own and retain all of its rights, title and interests in and to the LogicBio Background Technology.

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9.1.2 Collaboration Technology.

(a) CANbridge Collaboration Technology.  CANbridge will own all rights, title and interests in and to (a) (i) all Know-How, other than LogicBio Background Improvement Know-How, that is Created solely by or on behalf of CANbridge or its Affiliates in connection with the performance of CANbridge’s activities under this Agreement and (ii) all CANbridge Background Improvement Know-How, regardless of inventorship ((i) and (ii), collectively, the “CANbridge Collaboration Know-How”) and (b) all Patent Rights that claim CANbridge Collaboration Know-How, including all CANbridge Background Improvement Patent Rights (the “CANbridge Collaboration Patent Rights”).

(b) LogicBio Collaboration Technology.LogicBio will own all rights, title and interests in and to (a) (i) all Know-How, other than CANbridge Background Improvement Know-How, that is Created solely by or on behalf of LogicBio or its Affiliates in connection with the performance of LogicBio activities under this Agreement and (ii) all LogicBio Background Improvement Know-How, regardless of inventorship ((i) and (ii), collectively, the “LogicBio Collaboration Know-How”) and (b) all Patent Rights that claim LogicBio Collaboration Know-How, including all LogicBio Background Improvement Patent Rights (the “LogicBio Collaboration Patent Rights”).

(c) Joint Collaboration Technology.  The Parties will jointly own all rights, title and interests in and to (a) all Know-How, other than LogicBio Background Improvement Know-How and CANbridge Background Improvement Know-How, that is Created jointly by or on behalf of CANbridge or its Affiliates, on the one hand, and LogicBio or its Affiliates, on the other hand, in connection with the performance of activities under this Agreement (the “Joint Collaboration Know-How”) and (b) all Patent Rights that claim Joint Collaboration Know-How (the “Joint Collaboration Patent Rights”).

9.1.3 Inventorship. All determinations of inventorship under this Agreement will be made in accordance with U.S. patent law.

9.1.4 Disclosure.  Each Party will promptly disclose to the other Party all Inventions within the CANbridge Collaboration Know-How, LogicBio Collaboration Know-How, and Joint Collaboration Know-How that it Creates, whether solely or jointly with others (in any event, prior to the filing of any patent application with respect to such Inventions), including all invention disclosures or other similar documents submitted to such Party by its or its Affiliates’ employees, agents, or independent contractors relating thereto.  Each Party will also promptly respond to reasonable requests from the other Party for additional information relating thereto.

9.2 Assignments.

9.2.1 Assignment by CANbridge.  CANbridge will and hereby does assign to LogicBio all of CANbridge’s rights, title, and interests in and to LogicBio Background Improvement Technology, and LogicBio hereby accepts such assignment.  Without limiting Section 9.2.2 (CANbridge Covenants in Support of Assignment), if CANbridge is unable to assign any LogicBio Background Improvement Technology, then CANbridge hereby grants to LogicBio a royalty-free, fully paid-up, worldwide,

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exclusive (even as to CANbridge, subject to the terms and conditions of this Agreement), perpetual, irrevocable license (with the right to grant sublicenses through multiple tiers) under such LogicBio Background Improvement Technology for any and all purposes, subject to the terms and conditions of this Agreement. For clarity, CANbridge will not file any Patent Rights claiming any Invention within the LogicBio Background Improvement Technology.

9.2.2 CANbridge Covenants in Support of Assignment.  CANbridge will take (and cause its Affiliates and sublicensees (including Sublicensees, as applicable), and their respective employees, agents, and contractors to take) such further actions reasonably requested by LogicBio to evidence the assignments set forth in this Section 9.2.1 (Assignment by CANbridge) and to assist LogicBio in obtaining Patent Rights and other Intellectual Property protection for Inventions within the LogicBio Background Improvement Technology, including executing further assignments, consents, releases, and other commercially reasonable documentation and providing good faith testimony by affidavit, declaration, in-person, or other proper means in support of any effort by LogicBio to establish, perfect, defend, or enforce its rights in the LogicBio Background Improvement Technology through prosecution of governmental filings, regulatory proceedings, litigation, and other means. CANbridge will obligate its employees, Affiliates, sublicensees (including Sublicensees), and subcontractors to assign all Inventions and other Intellectual Property Created in connection with any activities performed in connection with this Agreement, prior to the performance of such activities, to CANbridge (or directly to LogicBio, as applicable) to ensure that CANbridge can comply with its obligations under Section 9.2.1 (Assignment by CANbridge), and CANbridge will promptly obtain such assignment. Without limitation, CANbridge will cooperate with LogicBio if LogicBio applies for U.S. or foreign patent protection for Inventions within the LogicBio Background Improvement Technology and will obtain the cooperation of the individual inventors of any such Inventions or Know-How.

9.2.3 Assignment by LogicBio.  LogicBio will and hereby does assign to CANbridge all of LogicBio’s rights, title, and interests in and to CANbridge Background Improvement Technology, and CANbridge hereby accepts such assignment.  Without limiting Section 9.2.4 (LogicBio Covenants in Support of Assignment), if LogicBio is unable to assign any CANbridge Background Improvement Technology, then LogicBio hereby grants to CANbridge a royalty-free, fully paid-up, worldwide, exclusive (even as to LogicBio, subject to the terms and conditions of this Agreement), perpetual, irrevocable license (with the right to grant sublicenses through multiple tiers) under such CANbridge Background Improvement Technology for any and all purposes, subject to the terms and conditions of this Agreement. For clarity, LogicBio will not file any Patent Rights claiming any Invention within the CANbridge Background Improvement Technology.

9.2.4 LogicBio Covenants in Support of Assignment.  LogicBio will take (and cause its Affiliates and sublicensees (including Sublicensees, as applicable), and their respective employees, agents, and contractors to take) such further actions reasonably requested by CANbridge to evidence the assignments set forth in Section 9.2.3 (Assignment by LogicBio) and to assist CANbridge in obtaining Patent Rights and other Intellectual Property protection for Inventions within the CANbridge Background Improvement Technology, including executing further assignments, consents, releases, and other commercially reasonable documentation and providing good faith testimony by

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affidavit, declaration, in-person, or other proper means in support of any effort by CANbridge to establish, perfect, defend, or enforce its rights in the CANbridge Background Improvement Technology through prosecution of governmental filings, regulatory proceedings, litigation, and other means. LogicBio will obligate its employees, Affiliates, sublicensees (including Sublicensees), and subcontractors to assign all Inventions and other Intellectual Property Created in connection with any activities performed in connection with this Agreement, prior to the performance of such activities, to LogicBio (or directly to CANbridge, as applicable) to ensure that LogicBio can comply with its obligations under Section 9.2.3(Assignment by LogicBio), and LogicBio will promptly obtain such assignment. Without limitation, LogicBio will cooperate with CANbridge if CANbridge applies for U.S. or foreign patent protection for Inventions within the CANbridge Background Improvement Technology and will obtain the cooperation of the individual inventors of any such Inventions or Know-How.

9.3 Joint Collaboration Technology.  Subject to the terms and conditions set forth in this Agreement, including the licenses granted in Section 2.1 (License to CANbridge), the Parties will jointly own all Joint Collaboration Technology, and each Party is entitled to practice the Joint Collaboration Technology for all purposes on a worldwide basis and to license such Joint Collaboration Technology through multiple tiers without consent of the other Party (where consent is required by Applicable Law, such consent is deemed hereby granted) and without a duty of accounting to the other Party.  Each Party will grant and hereby does grant to the other Party all further permissions, consents, and waivers with respect to, and all licenses under, the Joint Collaboration Technology, throughout the world, necessary to provide the other Party with full rights of use and Exploitation of the Joint Collaboration Technology.  Without limitation, each Party will cooperate with the other Party if the Parties determine to apply for U.S. or foreign patent protection for any Joint Collaboration Technology and will obtain the cooperation of the individual inventors of any such Joint Collaboration Technology.

9.4 Patent Prosecution.

9.4.1 Patent Coordinators. Each Party will appoint a patent coordinator reasonably acceptable to the other Party (each, a “Patent Coordinator”) to serve as such Party's primary liaison with the other Party on matters relating to the Prosecution and enforcement of LogicBio Licensed Patent Rights and Joint Collaboration Patent Rights. The Patent Coordinators will meet in person or by means of telephone or video conference at least once each Calendar Quarter during the Agreement Term. Each Party may replace its Patent Coordinator at any time by providing notice in writing to the other Party.

9.4.2 Prosecution of LogicBio Licensed Patent Rights.  

(a) As between the Parties, CANbridge will have the [***] and LogicBio will have the [***]. The Parties will [***] in Prosecuting LogicBio Licensed Patent Rights, and [***].

(b) If a Prosecuting Party decides to abandon or allow to lapse, or otherwise determines not to Prosecute a LogicBio Licensed Patent Right in any country or region in the Territory (including failing to timely respond to a patent office communication without timely filing a continuation or divisional application to preserve such Patent Rights), then the Prosecuting Party will provide reasonable

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prior written notice to the other Party of such intention (which notice the Prosecuting Party will provide to the other Party, to the extent possible, no later than [***] prior to the next deadline for any action that must be taken to avoid abandonment with respect to any such Patent Right), so as to provide the other Party a reasonable amount of time to meet any applicable deadline to establish or preserve such Patent Rights in such country or region. In such case, upon written notice to the Prosecuting Party, the other Party will have the right (but not the obligation) to assume responsibility for continuing Prosecution of such Patent Right in such country or region in [***] at the other Party’s sole expense.  [***].

(c) If CANbridge has the right to control the Prosecution of any LogicBio Licensed Patent Right pursuant to this Section 9.4.2 (Prosecution of LogicBio Licensed Patent Rights), then LogicBio will promptly deliver to CANbridge copies of all necessary files related to such Patent Rights with respect to which responsibility has been transferred and will take all actions and execute all documents reasonably necessary for CANbridge to assume such Prosecution, which files and documents will be the Confidential Information of LogicBio hereunder.

(d) The Parties will [***]; provided that, unless LogicBio otherwise agrees, LogicBio will have the [***] and will, to the extent permitted by Applicable Law, make [***].  The Parties will use good faith efforts to agree on such actions, with the goal of maximizing the value of the Parties’ respective patent portfolios.

9.4.3 Prosecution of Joint Collaboration Patent Rights.  Notwithstanding Section 9.4.2 (Prosecution of LogicBio Licensed Patent Rights), as between the Parties, CANbridge will have [***]. CANbridge will [***] relating to its Prosecution of such Joint Collaboration Patent Rights and will consider in good faith the comments, requests, and suggestions of LogicBio with respect to such Prosecution of such Joint Collaboration Patent Rights.  LogicBio will have the [***] any Joint Collaboration Patent Rights that do not Cover a Product. LogicBio will [***] relating to its Prosecution of such Joint Collaboration Patent Rights and will [***]. The Parties will [***]. If a Party decides to abandon or allow to lapse, or otherwise determines not to Prosecute a Joint Collaboration Patent Right in any country or region in the Territory (including failing to timely respond to a patent office communication without timely filing a continuation or divisional application to preserve such Patent Rights), then such Party will [***] of such intention ([***] prior to the next deadline for any action that must be taken to avoid abandonment with respect to any such Patent Right), so as to provide such other Party a reasonable amount of time to meet any applicable deadline to establish or preserve such Patent Rights in such country or region. In such case, upon written notice [***] to assume responsibility for continuing Prosecution of such Patent Right in such country or region [***] and such Party will promptly deliver to such other Party copies of all necessary files related to such Patent Rights with respect to which responsibility has been transferred and will take all actions and execute all documents reasonably necessary for such other Party to assume such Prosecution.

[***].

9.4.4 Prosecution of LogicBio Background Patent Rights and LogicBio Collaboration Patent Rights Other than LogicBio Licensed Patent Rights.  As between the Parties, LogicBio will have [***] the (a) LogicBio Background Patent Rights and (b) LogicBio

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Collaboration Patent Rights (in each case ((a) and (b)), other than the LogicBio Licensed Patent Rights) [***].

9.4.5 Prosecution of CANbridge Background Patent Rights and CANbridge Collaboration Patent Rights.  As between the Parties, CANbridge will have [***] the CANbridge Background Patent Rights and the CANbridge Collaboration Patent Rights [***].

9.4.6 Cooperation and Coordination.  The non-Prosecuting Party will (a) obtain and deliver to the Prosecuting Party any necessary documents for the Prosecuting Party to exercise its rights to Prosecute all Patent Rights pursuant to Section 9.4 (Patent Prosecution), as applicable, (b) render all signatures that will be necessary in connection with all such patent filings, and (c) assist the Prosecuting Party in all other reasonable ways that are necessary for the issuance of those Patent Rights for which such Prosecuting Party is responsible, as well as for the preparation, Prosecution of such Patent Rights.

9.5 Enforcement of LogicBio Licensed Technology.

9.5.1 Notice.  During the Term, the Parties will promptly notify each other in writing if either Party becomes aware of any suspected, threatened, or actual infringement or misappropriation by any Third Party of any LogicBio Technology arising from the making, using, offering to sell, selling, or importing of a product in the CANbridge Field in the Territory that would be competitive with a Product (a “Competing Infringement”).  Each Party will provide any available evidence of such Competing Infringement with such notification.

9.5.2 Infringement Actions for Infringements by Third Parties.

(a) [***]. Subject to this Section 9.5.2 (Infringement Actions for Infringements by Third Parties), as between the Parties, during the Term, CANbridge will have [***] with respect to any LogicBio Product-Specific Licensed Patent Right. [***].

(b) [***].  CANbridge will have a period of [***] after its receipt or delivery of notice under Section 9.5.1 (Notice) [***] described in Section 9.5.2(a) ([***]) against any Competing Infringement (or to settle or otherwise secure the abatement of such Competing Infringement).  [***].

(c) [***].  Except as provided in Section 9.5.2(a) ([***]), LogicBio has [***].

(d) [***].  Subject to [***] to Section 9.5.2(a) ([***]), LogicBio will have a period of [***] after its receipt or delivery of notice under Section 9.5.1 (Notice) [***].

(e) CANbridge Enforcement of LogicBio Patent Rights.  If CANbridge has the right to enforce LogicBio Licensed Patent Rights pursuant to Section 9.5.2(a) ([***]) or Section 9.5.2(d) ([***]), then the Parties will discuss in good faith and determine the strategy for enforcing such claims included in such LogicBio Licensed Patent Rights.  CANbridge will not take any action with respect to such enforcement that is inconsistent with the strategy agreed to by the Parties. LogicBio will be entitled to separate representation in such matter by counsel of its own choice and at its own expense.  CANbridge acknowledges and agrees that

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the LogicBio Licensed Patent Rights may be licensed to or from Third Parties who have rights with respect to the enforcement of such LogicBio Licensed Patent Rights and that CANbridge’s rights to conduct any enforcement activities with respect to the LogicBio Licensed Patents Rights are subject in all cases to such rights of any such Third Party(ies). LogicBio may consult with such Third Party licensors prior to making any decisions with respect to enforcement activities under this Section 9.5.2(Infringement Actions for Infringements by Third Parties).  CANbridge will provide LogicBio with drafts of all material papers to be filed with the court reasonably in advance of their being filed, so that LogicBio (and LogicBio’s licensors, as applicable) can comment and provide input with respect to such draft filings, and CANbridge will consider such comments in good faith.  If such action is brought by CANbridge pursuant to Section 9.5.2(a)([***]), or Section 9.5.2(d)([***]) then CANbridge will discuss such action with LogicBio and [***].

(f) Procedures.  Without limiting the foregoing, if the Party having the right to initiate an Infringement Action under this Section 9.5.2 (Infringement Actions against Infringements by Third Parties) (the “Initiating Party”) desires to initiate such Infringement Action but may not do so due to Applicable Law or regulation (even as the assignee or exclusive licensee of such infringed Patent Right), then such Initiating Party may require that the other Party join as a named party in such action at the Initiating Party’s sole cost and expense.  The Initiating Party will take the lead in the control and conduct of any such Infringement Action under Section 9.5.2 (Infringement Actions for Infringements by Third Parties) and will keep the other Party reasonably informed of any such Infringement Action, and the other Party will reasonably assist the Initiating Party in any such Infringement Action under Section 9.5.2 (Infringement Actions for Infringements by Third Parties) at the Initiating Party’s expense.  In no event may the Initiating Party settle any such Infringement Action in a manner that would limit the rights of the other Party or impose any obligation on the other Party, in each case, without the other Party’s prior written consent, which consent will not be unreasonably withheld, conditioned, or delayed.

(g) Recoveries.  Any amount recovered in any Infringement Action under Section 9.5.2 (Infringement Actions for Infringements by Third Parties), including any amount recovered in any settlement of such Infringement Action, will be shared as follows:

(i) [***]; then

(ii) [***].

9.6 Infringement of Third Party Rights.

9.6.1 Notice.  If any Product becomes the subject of a Third Party’s claim or assertion of infringement of a Patent Right within the Territory, the Party first having notice of the claim or assertion will promptly notify the other Party.

9.6.2 Defense.  Except as otherwise provided in Article 10 (Indemnification; Insurance), CANbridge will have [***], to defend any such Third Party claim or assertion of infringement of a Patent Right, [***].  [***]. The non-defending Party will reasonably

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cooperate with the Party conducting the defense of the claim or assertion, including if required to conduct such defense, furnishing a power of attorney.

9.6.3 Settlement; Licenses.  Except as otherwise provided in Article 10 (Indemnification; Insurance), neither Party will enter into any settlement of any claim described in this Section 9.6 (Infringement of Third Party Rights) that affects the other Party’s rights or interests without such other Party’s written consent, such consent not to be unreasonably withheld, conditioned, or delayed.  Each Party will have the right to decline to defend or to tender the defense of any claim described in this Section 9.6 (Infringement of Third Party Rights) upon reasonable written notice to the other Party, including if the other Party fails to agree to a settlement that the declining Party proposes.  Except as otherwise provided in Article 10 (Indemnification; Insurance), any settlement or license fees incurred by [***] under this Section 9.6.3 (Settlement; Licenses) will be allocated in accordance with the principle set forth in 6.4.4(c) (Third Party Payments), to the extent that the Intellectual Property that is the subject of such settlement license Covers the manufacture, use or sale of a Product in the relevant country for which such rights are licensed thereunder.

9.7 Patent Oppositions and Other Proceedings.

9.7.1 Third-Party Patent Rights Covering Products.  If [***] desires to bring an opposition, action for declaratory judgment, nullity action, interference, declaration for non-infringement, reexamination, inter partes reviews, post-grant reviews, or other attack upon the validity, title, or enforceability of a Patent Right owned or controlled by a Third Party and having one or more claims that Cover a Product (except insofar as such action is a counterclaim to or defense of, or accompanies a defense of, a Third Party’s claim or assertion of infringement under Section 9.6 (Infringement of Third Party Rights), in which case the provisions of Section 9.6 (Infringement of Third Party Rights) will govern), [***] will so notify [***], and the Parties will promptly confer to determine whether to bring such action, the strategy to be employed in connection with any such action, or the manner in which to settle such action.  CANbridge will have [***]. The Party not bringing an action under this Section 9.7.1 (Third-Party Patent Rights Covering Products) will be entitled to separate representation in such proceeding by counsel of its own choice and at its own expense and will cooperate fully with the Party bringing such action.  Any awards or amounts received in bringing any such action will be first allocated to reimburse the initiating Party’s expenses in such action, and any remaining amounts will be allocated between the Parties as provided in Section 9.5.2(g) (Recoveries).  [***].

9.7.2 Parties’ Patent Rights.  If any LogicBio Licensed Patent Right becomes the subject of any proceeding commenced by a Third Party within the Territory in connection with an opposition, reexamination request, action for declaratory judgment, nullity action, interference, inter partes reviews, post-grant reviews or other attack upon the validity, title or enforceability thereof (a “Third Party Patent Challenge”) (except insofar as such action is a counterclaim to or defense of, or accompanies a defense of, an action for infringement against a Third Party under Section 9.5 (Enforcement of LogicBio Licensed Technology) in which case the provisions of such Section will govern), then [***] responsible for Prosecuting such Patent Right as set forth in Section 9.4 (Patent Prosecution) hereof, will control such defense at its own expense. The controlling Party will permit the non-controlling Party to participate in the proceeding to the extent permissible under Applicable Law, and to be represented by its own counsel in such

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proceeding, [***].  If either Party decides that it does not wish to defend against such action, then the other Party will have a backup right to assume defense of such Third Party action [***].

9.8 Response to Biosimilar Applicants.

9.8.1 Notice.  In the event that any Party (a) receives a copy of an application submitted to the FDA under subsection (k) of Section 351 of the Public Health Services Act (the “PHSA,” and such application, a “Biosimilar Application”) for which a Product is a “reference product,” whether or not such notice or copy is provided under any Applicable Law applicable to the approval or Manufacture of any biosimilar or interchangeable biological product (including under the Biologics Price Competition and Innovation Act (“BPCIA”), United States Patient Protection and Affordable Care Act or implementing FDA regulations and guidance) (a “Proposed Biosimilar Product”) or (b) otherwise becomes aware that such a Biosimilar Application has been filed (such as in an instance described in Section 351(l)(9)(C) of the PHSA), then such Party will promptly provide the other Party with written notice thereof. The terms “reference product” and “biosimilar or interchangeable biological product” will have the respective meanings given to those terms in the BPCIA.

9.8.2 Access to Confidential Information.  Upon written request from [***] and to the extent permitted by Applicable Law, [***] will provide [***] with confidential access to the Biosimilar Application and such other information that describes the process used to Manufacture the Proposed Biosimilar Product, in each case, to the extent provided to [***] by the Third Party that submitted the Biosimilar Application (the “Applicant”); provided, however, that prior to receiving the Biosimilar Application and such Confidential Information, [***] will provide notice to [***] confirming its agreement to be subject to the confidentiality provisions in Section 351(l)(1)(B)(iii) of the PHSA.

9.8.3 Proposed Patent List.

(a) Preparation of Initial Patent List.  As soon as practicable after the date of receipt by [***] of a copy of a Biosimilar Application and related Manufacturing information and [***] receipt of the notice contemplated by Section 9.8.1 (Response to Biosimilar Applicants; Notice), or within such other timeframe as the Parties may agree, each Party will prepare a proposed list (the “Proposed Initial Patent List”) of the LogicBio Licensed Patent Rights, or, in the case of CANbridge, other Patent Rights Controlled by [***], that such Party reasonably believes would be infringed by the Manufacture or sale of the Proposed Biosimilar Product and will exchange such Proposed Initial Patent List with the other Party. [***]. The Parties will exchange such Proposed Initial Patent List no later than [***] after the date of receipt by [***] of a copy of the Biosimilar Application and related Manufacturing information, but in any event at least [***] prior to sending the Initial Patent List to the Applicant. As soon as practicable following the date of exchange by the Parties of the Proposed Initial Patent List, [***].  Not later than [***] following [***] receipt of the Biosimilar Application and related Manufacturing information under Section 351(l)(2) of the PHSA, CANbridge will provide the Applicant with a copy of the Initial Patent List.

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(b) Disclosure of Applicant’s Response.  Provided that [***] has agreed to comply with the confidentiality provisions in Section 351(l)(1)(B)(iii) of the PHSA and to the extent permitted by Applicable Law, CANbridge will [***] (the “Applicant Response”).

(c) Preparation of [***] Response.  As soon as practicable following the date of receipt by [***] of the Applicant Response, or within such other timeframe as the Parties may agree, [***].

(d) Negotiation; [***] Rights.  After [***] provides the Applicant with a copy of the [***] Response, [***]. Without limiting the foregoing, within [***] following the exchange of such lists by [***] and the Applicant pursuant to Section 351(l)(5)(B)(i) of the PHSA, [***] will, to the extent permitted by Applicable Law, notify [***] of any [***] included on the combined list(s) referenced in Section 351(l)(6)(A) or (B) of the PHSA that will be the subject of an Immediate Patent Infringement Action.

9.8.4 Supplements to Initial Patent List.  Each Party will inform the other Party within [***] days of receipt of an issue notification of any U.S. Patent Right within the LogicBio Licensed Patent Rights for which such Party is controlling Prosecution and that is issued after such Party has provided the Initial Patent List to the Applicant, and will provide the other Party with a copy of the allowed claims in such U.S. Patent Right. [***].

9.9 CREATE Act. Notwithstanding anything to the contrary in this Article 9, neither Party will have the right to make an election under the CREATE Act when exercising its rights under this Article 9 without the prior written consent of the other Party, which will not be unreasonably withheld, conditioned or delayed. With respect to any such permitted election, the Parties will use reasonable efforts to cooperate and coordinate their activities with respect to any submissions, filings or other activities in support thereof. The Parties acknowledge and agree that this Agreement is a “joint research agreement” as defined in the CREATE Act. Notwithstanding the foregoing, the other Party’s consent under this Section 9.9 will not be required in connection with an obviousness-type double patenting rejection in any patent application claiming a CANbridge Collaboration Patent Rights, LogicBio Collaboration Patent Rights and Joint Collaboration Patent Rights, or uses thereof.

9.10 Patent Term Extensions.  [***] requests for patent term extensions, supplementary protection certificates, or equivalents thereto in any country in the Territory, in each case where applicable to a Product (hereinafter “Patent Term Extensions”); [***]. [***] costs and expenses relating to the Patent Term Extensions [***]. [***] will provide support, assistance, and all necessary documents, in [***] for the purpose of supporting, filing, obtaining, and maintaining Patent Term Extensions.

9.11 Third Party Rights; Existing Obligations.  Notwithstanding any provision to the contrary in this Agreement, each Party’s rights under this Article 9 (Intellectual Property) with respect to the Prosecution and enforcement of any LogicBio Licensed Patent Right is subject to the rights of certain Third Parties to the extent set forth in any Existing In-License Agreement.

Article 10
INDEMNIFICATION; INSURANCE

10.1 Indemnification by LogicBio. LogicBio hereby agrees to defend, indemnify, and hold harmless CANbridge and its Affiliates and each of their respective directors, officers, employees, agents,

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and representatives (each, a “CANbridge Indemnitee”) from and against any and all claims, suits, actions, demands, liabilities, expenses, or losses, including reasonable legal expenses and attorneys’ fees (collectively, the “Losses”), to which any CANbridge Indemnitee may become subject to as a result of any claim, demand, action, or other proceeding by any Third Party (each, a “Claim”) to the extent such Losses arise directly or indirectly out of:

10.1.1 the breach by LogicBio of any of its representations, warranties, agreements, or covenants under this Agreement; or

10.1.2 the [***] of LogicBio or its Affiliate or its licensee (other than CANbridge or its Affiliate or Sublicensee), or any officer, director, employee, agent, or representative thereof.

Except, with respect to each of Section 10.1.1 through Section 10.1.2, to the extent such Losses arise from any action for which CANbridge has an indemnification obligation to LogicBio Indemnitee under Section 10.2.1 through Section 10.2.3.

10.2 Indemnification by CANbridge.  CANbridge hereby agrees to defend, indemnify, and hold harmless LogicBio and its Affiliates, and each of their respective directors, officers, employees, agents and representatives and CMRI, its Affiliates (as defined in the CMRI Agreement), the Co-Owners and their Personnel (as defined in the CMRI Agreement) (each, a “LogicBio Indemnitee”) from and against any and all Losses to which any LogicBio Indemnitee may become subject as a result of any Claim to the extent such Losses arise directly or indirectly out of:

10.2.1 the breach by CANbridge of any of its representations, warranties, agreements, or covenants under this Agreement;

10.2.2 any claims of any nature arising out of the Exploitation of any Product by or on behalf of CANbridge, its Affiliates or Sublicensees (other than by any LogicBio Indemnitee); or

10.2.3 the [***] of CANbridge or its Affiliate or Sublicensee, or any officer, director, employee, agent, or representative thereof.

Except, with respect to each of Section 10.2.1 through Section 10.2.3, to the extent such Losses arise from any action for which LogicBio has an indemnification obligation to CANbridge under Section 10.1.1 through Section 10.1.2.

10.3 Indemnification Procedure.

10.3.1 Notice.  Promptly after a LogicBio Indemnitee or a CANbridge Indemnitee (each, an “Indemnitee”) receives notice of a pending or threatened Claim, such Indemnitee will give written notice of the Claim to the Party from whom the Indemnitee is entitled to receive indemnification pursuant to Section 10.1 (Indemnification by LogicBio) or Section 10.2 (Indemnification by CANbridge), as applicable (the “Indemnifying Party”).  An Indemnitee’s delay in providing or failure to provide such notice will not relieve the Indemnifying Party of its indemnification obligations, except to the extent it can demonstrate prejudice due to the delay or lack of notice.

10.3.2 Defense.  Upon receipt of notice under Section 10.3.1 (Indemnification Procedure; Notice) from the Indemnitee, the Indemnifying Party will have the duty to either

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compromise or defend, at its own expense and by counsel (reasonably satisfactory to Indemnitee), such Claim. The Indemnifying Party will promptly (and in any event not more than [***]  after receipt of the Indemnitee’s original notice) notify the Indemnitee in writing that it acknowledges its obligation (which acknowledgement will not be deemed or construed as an admission of liability, either under this Article 10(Indemnification) or otherwise) to indemnify the Indemnitee with respect to the Claim pursuant to this Section 10.3(Indemnification Procedures) and of its intention to either compromise or defend such Claim.  Once the Indemnifying Party gives such notice to the Indemnitee, the Indemnifying Party is not liable to the Indemnitee for the fees of other counsel or any other expenses subsequently incurred by the Indemnitee in connection with such defense, other than the Indemnitee’s reasonable expenses of investigation and cooperation.  However, the Indemnitee will have the right to employ separate counsel and to control the defense of a Claim at its own expense.

10.3.3 Cooperation.  The Indemnitee will cooperate fully with the Indemnifying Party and its legal representatives in the investigation and defense of any Claim.  The Indemnifying Party will keep the Indemnitee informed on a reasonable and timely basis as to the status of such Claim (to the extent the Indemnitee is not participating in the defense of such Claim) and conduct the defense of such Claim in a prudent manner.

10.3.4 Settlement.  If an Indemnifying Party assumes the defense of a Claim, then no compromise or settlement of such Claim may be effected by the Indemnifying Party without the Indemnitee’s written consent (such consent not to be unreasonably withheld, conditioned, or delayed).  Notwithstanding any provision to the contrary set forth in this Agreement, the Indemnitee’s consent will not be required of a settlement where: (a) there is no finding or admission of any violation of law or any violation of the rights of any Person and no effect on any other claims that may be made against the Indemnitee; (b) the sole relief provided is monetary damages that are paid in full by the Indemnifying Party; and (c) the Indemnitee’s rights under this Agreement are not adversely affected.  If the Indemnifying Party fails to assume defense of a Claim within a reasonable time, then the Indemnitee may settle such Claim on such terms as it deems appropriate with the consent of the Indemnifying Party (such consent not to be unreasonably withheld, conditioned, or delayed), and the Indemnifying Party will be obligated to indemnify the Indemnitee for such settlement as provided in this Article 10 (Indemnification).

10.4 Insurance.  Each Party will, at its own expense, procure and maintain during the Term and for a period of [***] thereafter, insurance policies, including product liability insurance when applicable, adequate to cover its obligations hereunder and that are consistent with normal business practices of prudent companies similarly situated. Without limiting the foregoing, the types of insurance and minimum limits with respect to CANbridge will include at least the following:

(a) Prior to commencing any use in humans of Products, a comprehensive commercial general liability and products liability policy to cover all sums which it may become legally liable to pay as compensation consequent upon:

(i) death of, or bodily injury (including disease or illness) to, any person; and

(ii) loss of, or damage to, property,

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happening anywhere in the Territory arising out of or in connection with this Agreement. The limit of liability provided by this policy for each and every event will not be less than $[***].

(b) [***] by CANbridge or its Affiliates in connection with the Products (and CANbridge will ensure that [***].

(c) Prior to commencing any use in humans of Products, or use in humans of techniques within the LogicBio Technology, no fault clinical trial insurance with a limit of liability of not less than $[***] for each and every event.

(d) Any other insurance required by Applicable Law.

Such insurance will not be construed to create a limit of a Party’s liability with respect to its indemnification obligations under this Article 10 (Indemnification).  Each Party will provide the other Party with written evidence of such insurance or self-insurance upon request. Each Party will provide the other Party with prompt written notice of cancellation, non-renewal, or material change in such insurance or self-insurance that could materially adversely affect the rights of such other Party hereunder and will provide such notice within [***] after any such cancellation, non‑renewal, or material change.

Article 11
TERM AND TERMINATION

11.1 Term.  This Agreement will commence upon the Effective Date and, if not otherwise terminated earlier pursuant to this Article 11 (Term and Termination), will continue, on a Product-by-Product and country-by-country basis, in full force and effect until the expiration of the Royalty Period applicable to such Product and such country and will expire in its entirety upon the expiration of the final Royalty Period (such period from the Effective Date until the expiration of this Agreement with respect to all Products, the “Term”).

11.2 Termination for Material Breach.

11.2.1 Material Breach and Cure Period. Subject to Section 11.2.2 (Disputes Regarding Material Breach), either Party (the “Non-Breaching Party”) may terminate this Agreement in the event the other Party (the “Breaching Party”) has materially breached this Agreement, and such material breach has not been cured within [***] after receipt of written notice of such breach by the Breaching Party from the Non-Breaching Party (or within [***] in the case of any payment breach) (such [***]- or [***]-day period, the “Cure Period”).  If a breach relates: (i) solely to Products Directed to a single Target, any such termination shall be limited solely to rights pertaining to such Target and (ii) to matters beyond the scope of the foregoing clause (i), then any such termination shall pertain to the Agreement in its entirety.  The written notice describing the alleged material breach will provide sufficient detail to put the Breaching Party on notice of such material breach. Any termination pursuant to this Section 11.2.1 (Material Breach and Cure Period) will become effective at the end of the Cure Period, unless the Breaching Party has cured any such material breach prior to the expiration of such Cure Period, or, if such material breach is not curable prior to the expiration of the applicable, then such Cure Period will be extended so long as the Breaching Party has (a) provided to the Non-Breaching Party a written plan that is

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reasonably calculated to effect a cure of such material breach, and (b) the Breaching Party commits to and diligently carries out such plan as provided to the Non-Breaching Party, provided that, in no event will the Cure Period be extended to more than a total of [***].

11.2.2 Disputes Regarding Material Breach.  If the Parties reasonably and in good faith disagree as to the scope or existence  of a material breach, then the Breaching Party that disputes whether there has been a material breach may contest the allegation in accordance with Article 12 (Dispute Resolution), and the applicable Cure Period will toll upon the initiation of such dispute resolution procedures.  If, as a result of such dispute resolution process, it is finally determined pursuant to Article 12 (Dispute Resolution) that the Breaching Party committed a material breach of this Agreement, then the applicable Cure Period will resume and unless such alleged breach was cured during the pendency of such Cure Period (once resumed), this Agreement will terminate (in whole or in part, as applicable) effective as of the expiration of such Cure Period.  This Agreement will remain in full force and effect during the pendency of any such dispute resolution proceeding and all Cure Periods.  Any such dispute resolution proceeding will not suspend any obligations of either Party hereunder and each Party will use reasonable efforts to mitigate any damages. Any payments that are made by one Party to the other Party pursuant to this Agreement pending resolution of the Dispute will be promptly refunded if it is determined pursuant to Article 12 (Dispute Resolution) that such payments are to be refunded by one Party to the other Party.  If, as a result of such dispute resolution proceeding, it is determined that the Breaching Party did not commit such material breach (or such material breach was cured in accordance with this Section 11.2 (Termination for Material Breach)), then no termination of this Agreement will be effective, and this Agreement will continue in full force and effect.

11.3 Termination for Insolvency.  To the extent permitted by Applicable Law, either Party may terminate this Agreement upon providing written notice to the other Party on or after the time that such other Party files or institutes a bankruptcy, reorganization, liquidation, or receivership proceeding or upon the appointment of a receiver or trustee over all or substantially all property, or upon an assignment of a substantial portion of the assets for the benefit of creditors (a “Bankruptcy Filing”), by the other Party; provided, however, that in the case of any involuntary bankruptcy proceeding, such right to terminate will only become effective if the applicable Party consents to the involuntary bankruptcy or such proceeding is not dismissed within [***] after the filing thereof. In the event of any termination pursuant to this Section 11.3 (Termination for Insolvency):

11.3.1 All rights and licenses now or hereafter granted by a Party to the other Party under or pursuant to this Agreement are, for all purposes of Section 365(n) of Title 11 of the United States Bankruptcy Code, licenses of rights to “intellectual property” as defined in the U.S. Bankruptcy Code.  Upon a Bankruptcy Filing by a Party, such Party agrees that the non-bankrupt Party, as licensee of such rights under this Agreement, will retain and may fully exercise all of its rights and elections under the U.S. Bankruptcy Code.  Each Party acknowledges and agrees that “embodiments” of intellectual property rights within the meaning of Section 365(n) include laboratory notebooks, cell lines, product samples, and inventory, research studies and data, all Regulatory Approvals (and all applications for Regulatory Approval) and rights of reference therein, in each case that are within the LogicBio Technology (in the event that LogicBio is the bankrupt Party) or within the CANbridge Licensed Technology (in the event that CANbridge is the bankrupt Party). If (A) a case under the U.S. Bankruptcy Code is commenced by or

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against a Party, (B) this Agreement is rejected as provided in the U.S. Bankruptcy Code, and (C) the non-bankrupt Party elects to retain its rights hereunder as provided in Section 365(n) of the U.S. Bankruptcy Code, the bankrupt Party (in any capacity, including debtor-in-possession) and its successors and assigns (including a trustee) will:

(a) provide the non-bankrupt Party with a complete duplicate of (or complete access to, as appropriate) all such intellectual property rights (including all embodiments thereof) held by the bankrupt Party and such successors and assigns, or otherwise available to them, immediately upon the non-bankrupt Party’s written request.  Whenever the bankrupt Party or any of its successors or assigns provides to the non-bankrupt Party any of the intellectual property rights licensed hereunder (or any embodiment thereof) pursuant to this Section11.3.1(a)(Termination for Insolvency), the non-bankrupt Party will have the right to perform the bankrupt Party’s obligations hereunder with respect to such intellectual property rights, but neither such provision nor such performance by the non-bankrupt Party will release the bankrupt Party from liability resulting from rejection of the license or the failure to perform such obligations; and

(b) not interfere with the non-bankrupt Party’s rights under this Agreement, or any agreement supplemental hereto, to such intellectual property rights (including such embodiments), including any right to obtain such intellectual property rights (or such embodiments) from another entity, to the extent provided in Section 365(n) of the U.S. Bankruptcy Code.

11.4 Termination for Convenience.  CANbridge will be entitled to terminate this Agreement in its entirety, at its sole discretion, at any time upon [***] prior written notice to LogicBio thereof.

11.5 Termination for Patent Challenge.  If CANbridge or any of its Affiliates or Sublicensees, directly or indirectly through any Third Party, commences any interference or opposition proceeding with respect to, challenges the validity or enforceability of, or opposes any extension of or the grant of a supplementary protection certificate with respect to, any LogicBio Licensed Patent Right (a “Patent Challenge”), then LogicBio will have the right to terminate this Agreement on [***] written notice to CANbridge; such termination of such license to be effective immediately following such notice period; provided that if CANbridge or its Affiliate or Sublicensee withdraws (or causes to be withdrawn) such Patent Challenge within [***] after being requested to do so by LogicBio in writing (which termination notice will be deemed a request), then LogicBio will have no right to terminate this Agreement pursuant to this Section 11.5 (Termination for Patent Challenge). For clarity, LogicBio may not terminate this Agreement pursuant to this Section 11.5 (Termination for Patent Challenge) if CANbridge or its Affiliate or Sublicensee is required by legal process to be joined as a party in any Patent Challenge by a Third Party. In addition, notwithstanding the foregoing, LogicBio will have no right to terminate this Agreement pursuant to this Section 11.5 (Termination for Patent Challenge) with respect to: (i) any affirmative defense or other validity, enforceability, or non-infringement challenge, whether in the same action or in any other agency or forum of competent jurisdiction, advanced by CANbridge, or any of its Affiliates or Sublicensees in response to any claim or action brought in the first instance by, or on behalf of, LogicBio or any Third Party, or (ii) any Patent Challenge that is commenced by a Sublicensee, provided that CANbridge demands that such Sublicensee withdraw such Patent Challenge promptly after CANbridge becomes aware of such Patent Challenge and terminates the sublicense agreement with the applicable Sublicensee if such Sublicensee does not withdraw such Patent Challenge within [***] after receipt of notice from CANbridge.

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11.6 Termination for Cessation of Development or Commercialization. On a Target-by-Target basis, if (i) CANbridge and its Affiliates and their respective Sublicensees do not conduct any [***] Development or Commercialization activities with respect to a Target in [***] for a continuous period of longer than [***], then LogicBio may, in its sole discretion, terminate this Agreement with respect to such Target upon [***] prior written notice to CANbridge.  Notwithstanding any provision to the contrary set forth in this Agreement, the foregoing [***] period will automatically be tolled for any period that such inactivity is due to (a) a decision to stop or delay further Development or Commercialization, as applicable, due to a safety concern or (b) any event of Force Majeure.  For clarity, material activities undertaken to resolve a clinical hold imposed by an applicable Regulatory Authority or an inability of CANbridge to obtain supply of Products that are Manufactured by CANbridge or on behalf of CANbridge by a Third Party will be considered material Development or Commercialization activities.

11.7 Effects of Termination.

11.7.1 Effects of Termination Generally.  Upon termination of this Agreement in its entirety or with respect to a Target:

(a) Wind-Down.  Promptly following receipt by the applicable Party of a notice of termination pursuant to this Article 11 (Term and Termination), the Parties will begin to wind-down their respective activities under this Agreement with respect to the Terminated Targets.  The Parties will coordinate the wind-down of each Party’s efforts under this Agreement, and, if the applicable committees have disbanded, then the Parties will establish an appropriate committee to coordinate such wind-down.

(b) Termination of Rights and Licenses.  Without limiting the effect that such termination will have on any provisions of this Agreement, other than as expressly set forth herein, including those provisions that this Agreement expressly provides will survive such termination and subject to Section 2.2.3 (Survival of Sublicenses), all rights and licenses granted herein to either Party will terminate with respect to the Terminated Targets; provided that such licenses will continue as necessary for the Parties to complete the orderly wind-down of their activities under this Agreement in accordance with Applicable Law and as otherwise required in accordance with Section 11.7.1(a) (Wind-Down).

(c) Termination of Payment Obligations.  All payment obligations hereunder with respect to the Terminated Targets will terminate, other than those that are accrued and unpaid as of the effective date of such termination.

(d) Sell-Off Right.  During the [***] period following the effective date of termination, CANbridge will have the right to sell or otherwise dispose of the then-existing inventory of Products with respect to the Terminated Targets on hand at the time of such termination or in the process of Manufacturing, in accordance with the terms of this Agreement.

(e) Return of Confidential Information.  Upon the expiration or termination of this Agreement, (i) the Receiving Party will return to the Disclosing Party or, as directed by the Disclosing Party, destroy (and certify such destruction in writing) all Confidential Information of the Disclosing Party (or if the Agreement is terminated with respect to one or more Targets but not in its entirety, all Confidential Information of the Disclosing Party that solely relates to the Terminated Targets), that is in the Receiving Party’s possession or control and (ii) CANbridge will ensure that any contact manufacturer engaged by CANbridge returns to LogicBio or, as directed by LogicBio, destroys (and certifies such destruction in writing) all Confidential Information of LogicBio (or if the Agreement is terminated with respect to one or more Targets but not in its entirety, all

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such Confidential Information of LogicBio that solely relates to the Terminated Targets), provided, however, that in each case ((i) and (ii)), copies may be retained and stored by the Receiving Party solely for the purpose of determining its obligations under this Agreement, subject to the non-disclosure and non-use obligation under Article 8(Confidentiality).  In addition, the Receiving Party will not be required to return or destroy Confidential Information contained in any computer system back-up records made in the ordinary course of business; provided that such Confidential Information may not be accessed without the Disclosing Party’s prior written consent or as required by Applicable Law.

(f) Transfer of Prosecution Rights. Upon the expiration or termination of this Agreement, CANbridge will promptly cooperate and assist in transitioning to LogicBio the prosecution and maintenance of all Patent Rights that CANbridge is prosecuting or maintaining pursuant to Section 9.4 (Patent Prosecution) as of the effective date of termination, including by executing documents in a timely manner as may be reasonably necessary to allow LogicBio to continue such prosecution and maintenance.

11.7.2 Grantback License; Product Reversion.  In addition to those general effects set forth in Section 11.7.1 (Effects of Termination Generally), the following terms of this Section 11.7.2 (Grantback License; Product Reversion) will apply (A) with respect to a Terminated Target, solely if LogicBio terminates this Agreement with respect to such Terminated Target under Section 11.2 (Termination for Material Breach), Section 11.3 (Termination for Insolvency), Section 11.5 (Termination for Patent Challenge) or Section 11.6 (Termination for Cessation of Development or Commercialization), if requested by LogicBio, CANbridge will negotiate in good faith towards an agreement pursuant to which: (a) CANbridge would grant a license under CANbridge Product Technology and Product Trademarks, (b) CANbridge would assign or otherwise provide to LogicBio the benefit of Clinical Trial data, Regulatory Submissions, and Regulatory Approvals applicable to any Products with respect to the Terminated Targets, (c) CANbridge would sell to LogicBio any or all of the inventory of the Products with respect to the Terminated Targets.

11.8 Remedies.  Notwithstanding any provision to the contrary set forth in this Agreement, except as otherwise set forth in this Agreement, termination or expiration of this Agreement will not relieve the Parties of any liability or obligation that accrued hereunder prior to the effective date of such termination or expiration, nor prejudice either Party’s right to obtain performance of any obligation.  Each Party will be free, pursuant to Article 12 (Dispute Resolution), to seek, without restriction as to the number of times it may seek, damages, expenses, and remedies that may be available to it under Applicable Law or in equity and will be entitled to offset the amount of any damages and expenses obtained against the other Party in a final determination under Section 12.3 (Litigation), against any amounts otherwise due to such other Party under this Agreement.

11.9 Election to Continue in Lieu of Termination.  Notwithstanding the foregoing, if CANbridge has the right to terminate this Agreement pursuant to Section 11.2 (Termination for Material Breach)

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based on an uncured breach by LogicBio, CANbridge may elect, in lieu of such termination and in addition to any other remedies available to CANbridge at law or in equity, to maintain this Agreement in [***].

11.10 Survival. In the event of termination of this Agreement, in addition to the provisions of this Agreement that continue in effect in accordance with their terms, the following provisions of this Agreement will survive:  Article 1 (Definitions), Section 2.2.3 (Survival of Sublicenses),Section 2.5 (No Implied Licenses), Article 6 (Payments and Royalties) (solely to the extent applicable to any payments due and payable under this Agreement as of the effective date of termination or due to the sell off of existing inventory pursuant to Section 11.7.1(d) (Sell-Off Right)), Section 6.11 (Record Retention; Audits) (until [***] following the effective date of termination), Section 7.6 (Disclaimer of Warranties), Section 7.7 (Limitation of Liability), Article 8 (Confidentiality) (until the end of the confidentiality term set forth in Section 8.2 (Non-Disclosure and Non-Use Obligation)), Section 9.1 (Ownership), Section 11.1 (Term), Section 11.7 (Effects of Termination), Section 11.9 (Survival), Article 12 (Dispute Resolution) and Article 13 (Miscellaneous).

Article 12
DISPUTE RESOLUTION

12.1 [***] Dispute Resolution Mechanism.  The Parties agree that, except as expressly set forth in this Agreement, the procedures set forth in this Article 12 (Dispute Resolution) will be the exclusive mechanism for resolving any dispute, controversy, or claim between the Parties arising out of or relating to this Agreement (whether based on contract, tort or otherwise) (each, a “Dispute,” and collectively, the “Disputes”) that is not resolved through [***] between the Parties pursuant to [***].

12.2 Resolution by Executive Officers.  Except as otherwise provided in this Section 12.2 (Resolution by Executive Officers) or as provided in Section 12.4 (Preliminary Injunctions), in the event of any Dispute regarding the construction or interpretation of this Agreement, or the rights, duties, or liabilities of either Party hereunder, the Parties will first attempt in [***] to resolve such Dispute by negotiation and consultation between themselves.  In the event that such Dispute is not resolved on an informal basis within [***], either Party may, by written notice to the other Party, refer the Dispute to the Executive Officer of the other Party for attempted resolution by [***] within [***] after such notice is received.  Each Party may, in its sole discretion, seek resolution of any and all Disputes that are not resolved under this Section 12.2 (Resolution by Executive Officers) in accordance with Section 12.3 (Litigation).

12.3 Litigation.  Any unresolved Dispute that was subject to Section 12.2 (Resolution by Executive Officers), will be brought exclusively in a court of competent jurisdiction, federal or state, located in Boston, Massachusetts, and in no other jurisdiction.  Each Party hereby irrevocably consents to personal jurisdiction and venue in, and irrevocably agrees to service of process issued or authorized by any such court in any such action or proceeding.  The Parties hereby irrevocably waive any objection which they may now have or hereafter have to the laying of venue in the federal or state courts of Massachusetts in any such action or proceeding, and hereby irrevocably waive and agree not to plead or claim in any such court that any such action or proceeding brought in any such court has been brought in an inconvenient forum. The Parties hereby agree that any final judgment rendered by any such federal or state court of Massachusetts in any action or proceeding involving any Dispute, from which no appeal can be or is taken, may be enforced by the prevailing Party in any court of competent jurisdiction.

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12.4 Preliminary Injunctions.  Notwithstanding any provision to the contrary set forth in this Agreement, in the event of an actual or threatened breach of a Party’s obligations under this Agreement, a Party may seek a temporary restraining order or a preliminary injunction from any court of competent jurisdiction in order to prevent immediate and irreparable injury, loss, or damage on a provisional basis.

12.5 Patent and Trademark Disputes.  Notwithstanding any provision to the contrary set forth in this Agreement, any and all issues regarding the scope, construction, validity, and enforceability of any Patent Rights or trademark relating to a Product that is the subject of this Agreement will be determined in a court or other tribunal, as the case may be, of competent jurisdiction under the applicable patent or trademark laws of the country in which such Patent Rights or trademark rights were granted or arose.

12.6 Confidentiality.  Any and all activities conducted under this Article 12 (Dispute Resolution), including any and all proceedings and decisions hereunder, will be deemed Confidential Information of each of the Parties, and will be subject to Article 8 (Confidentiality), to the extent applicable in accordance with Applicable Law.

Article 13
MISCELLANEOUS

13.1 Performance through Affiliates.  Each Party may discharge any obligations and exercise any rights hereunder through delegation of its obligations or rights to any of its Affiliates.  Each Party hereby guarantees the performance by its Affiliates of such Party’s obligations under this Agreement and will cause its Affiliates to comply with the provisions of this Agreement in connection with such performance.

13.2 Notices.  All notices and other communications given or made pursuant hereto will be in writing (including by electronic mail) and will be deemed to have been duly given (a) on the date delivered, if delivered personally, or on the next Business Day after being sent by reputable overnight courier (with delivery tracking provided, signature required, and delivery prepaid), in each case, to the Parties at the following addresses, or (b) on the date sent if sent by email to the email address provided below and if confirmed by confirmatory return email to the email address specified below (or at such other address, or email address for a Party as will be specified by notice given in accordance with this Section 13.2 (Notices)).

If to CANbridge:

CANbridge Care Pharma Hong Kong Limited

Room 402, Huawen International Plaza

999 West Zhongshan Road, Changning District

Shanghai, 200051, P.R. China

Attention: [***]

Email:[***]

with a copy (which will not constitute notice) to:

Goodwin Procter LLP
100 Northern Avenue
Boston, MA 02210, U.S.A.
Attention: [***]
Email: [***]

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If to LogicBio:

LogicBio Therapeutics, Inc.
65 Hayden Ave., Floor 2,
Lexington, MA 02421, U.S.A.

Attention: [***]
Email:[***]

with a copy (which will not constitute notice) to:

Ropes & Gray LLP
800 Boylston Street
Boston, MA 02199, U.S.A.
Attention: [***]
Email: [***]

13.3 Force Majeure.  Both Parties will be excused from the performance of their obligations under this Agreement to the extent that such performance is prevented or delayed by Force Majeure and the nonperforming Party promptly provides notice of the prevention or delay to the other Party.  Such excuse will be continued so long as the condition constituting Force Majeure continues and the nonperforming Party takes reasonable efforts to remove the condition. Notwithstanding the foregoing, a Party will not be excused from making payments owed hereunder because of a Force Majeure affecting such Party. If a Force Majeure persists for more than [***], then the Parties will discuss in [***] the modification of the Parties’ obligations under this Agreement in order to mitigate the delays caused by such Force Majeure.

13.4 Assignment.  Neither Party may assign or transfer this Agreement or any rights or obligations hereunder without the prior written consent of the other, except that a Party may make such an assignment without the other Party’s consent to (a) Affiliates pursuant to Section 13.1 (Performance by Affiliates), provided that such Party will remain responsible for such Affiliate’s conduct and compliance with its obligations under this Agreement, or (b) a successor to all or substantially all of the business of such Party to which this Agreement relates, whether in a merger, sale of stock, acquisition, or similar transaction or series of related transactions.  Any permitted assignment will be binding on the successors of the assigning Party.  Any assignment or attempted assignment by either Party in violation of the terms of this Section 13.4 (Assignment) will be null, void, and of no legal effect.

13.5 Severability.  If any one or more of the provisions of this Agreement is held to be invalid or unenforceable by any court or tribunal of competent jurisdiction from which no appeal can be or is taken, then the provision will be considered severed from this Agreement and will not serve to invalidate any remaining provisions hereof.  The Parties will make a good faith effort to replace any invalid or unenforceable provision with a valid and enforceable one such that the objectives contemplated by the Parties when entering this Agreement may be realized.

13.6 English Language.  This Agreement will be written and executed in, and all other communications under or in connection with this Agreement, will be in the English language.  Any translation into any other language will not be an official version thereof, and in the event of any conflict in interpretation between the English version and such translation, the English version will control.

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13.7 Waiver and Non-Exclusion of Remedies.  Any term or condition of this Agreement may be waived at any time by the Party that is entitled to the benefit thereof, but no such waiver will be effective unless set forth in a written instrument duly executed by or on behalf of the Party waiving such term or condition.  The waiver by either Party of any right hereunder or of the failure to perform or of a breach by the other Party will not be deemed a waiver of any other right hereunder or of any other breach or failure by such other Party whether of a similar nature or otherwise.  The rights and remedies provided herein are cumulative and do not exclude any other right or remedy provided by Applicable Law or otherwise available except as expressly set forth herein.

13.8 Further Assurance.  Each Party will duly execute and deliver, or cause to be duly executed and delivered, such further instruments and do and cause to be done such further acts and things, including the filing of such assignments, agreements, documents, and instruments, as may be necessary or as the other Party may reasonably request in connection with this Agreement or to carry out more effectively the provisions and purposes hereof.

13.9 Relationship of the Parties.  It is expressly agreed that LogicBio, on the one hand, and CANbridge, on the other hand, will be independent contractors and that the relationship between the two Parties will not constitute a partnership, joint venture or agency.  Neither LogicBio nor CANbridge will have the authority to make any statements, representations or commitments of any kind, or to take any action that will be binding on the other, without the prior written consent of the other Party to do so.  All Persons employed by a Party will be employees of that Party and not of the other Party and all expenses and obligations incurred by reason of such employment will be for the account and expense of such Party.

13.10 Construction.  Except where the context otherwise requires, wherever used, the singular will include the plural, the plural will include the singular, and the use of any gender will be applicable to all genders.  Whenever this Agreement refers to a number of days without using a term otherwise defined herein, such number refers to calendar days.  The captions of this Agreement are for the convenience of reference only and in no way define, describe, extend, or limit the scope or intent of this Agreement or the intent of any provision contained in this Agreement. The terms “including,” “include,” “includes,” or “for example” will not limit the generality of any description preceding such term and as used herein will have the same meaning as “including, but not limited to” or “including, without limitation.”  The word “shall” will be construed to have the same meaning and effect as the word “will.”  References to any specific law, rule or regulation, or article, Section or other division thereof, will be deemed to include the then-current amendments thereto or any replacement or successor law, rule or regulation thereof. The term “or” will be interpreted in the inclusive sense commonly associated with the term “and/or.”  Any reference herein to any person or entity will be construed to include the person’s or entity’s successors and assigns.  The language of this Agreement will be deemed to be the language mutually chosen by the Parties and no rule of strict construction will be applied against either Party hereto.  Each Party represents that it has been represented by legal counsel in connection with this Agreement and acknowledges that it has participated in the drafting hereof.  In interpreting and applying the terms and provisions of this Agreement, the Parties agree that no presumption will apply against the Party which drafted such terms and provisions.

13.11 Governing Law. This Agreement was prepared in the English language, which language will govern the interpretation of, and any dispute regarding, the terms of this Agreement.  This Agreement and all disputes arising out of or related to this Agreement or any breach hereof will be governed by and construed under the laws of the State of New York, without giving effect to any choice of law principles that would require the application of the laws of a different state or jurisdiction.  Notwithstanding any other provision in this Agreement, the Parties expressly reject

57

the application to this Agreement, all transactions and activities contemplated hereby, and all Disputes of (a) the United Nations Convention on Contracts for the International Sale Of Goods, and (b) the 1974 Convention on the Limitation Period in the International Sale of Goods, as amended by that certain Protocol, concluded at Vienna, Austria on April 11, 1980.

13.12 Entire Agreement and Amendment.  This Agreement, including the Exhibits and Schedules hereto, sets forth the complete, final and exclusive agreement and all the covenants, promises, agreements, warranties, representations, conditions and understandings between the Parties hereto with respect to the subject matter hereof and supersedes all prior and contemporaneous agreements and understandings between the Parties with respect to the subject matter hereof, including, from and after the Restatement Date, the Original Agreement.  There are no covenants, promises, agreements, warranties, representations, conditions or understandings, either oral or written, between the Parties other than as are set forth herein and therein.  No subsequent alteration, amendment, change or addition to this Agreement will be binding upon the Parties unless reduced to a writing explicitly stating the Parties’ intent to amend this Agreement that is signed by an authorized officer of each Party.  In the event of any inconsistency between the body of this Agreement and either any Exhibits or Schedules to this Agreement or any subsequent agreements ancillary to this Agreement, unless otherwise express stated to the contrary in such Exhibit, Schedule or ancillary agreement, the terms contained in this Agreement will control.

13.13 Counterparts.  This Agreement may be executed in two or more counterparts, each of which will be deemed an original, but all of which together will constitute one and the same instrument.  This Agreement may be executed by facsimile, .pdf, or other electronically transmitted signatures and such signatures will be deemed to bind each Party as if they were the original signatures.

[Signature Page Follows]

58

IN WITNESS WHEREOF, the Parties have caused this Agreement to be executed by their respective duly authorized officers as of the Restatement Date.

CANbridge Care Pharma Hong Kong Limited
By:	/s/ James Xue
	(Signature)
Name:	James Xue
Title:	Director
Signing Time:	10/03/2022

LogicBio Therapeutics, Inc.
By:	/s/ Frederic Chereau
	(Signature)
Name:	Frederic Chereau
Title:	President and Chief Executive Officer
Signing Time:	10/02/2022

EX-31.1

Exhibit 31.1

Certification of Principal Executive Officer pursuant to Exchange Act Rules 13a-14(a)

and 15d-14(a), as adopted pursuant to Section 302 of Sarbanes-Oxley Act of 2002

I, Frederic Chereau, certify that:

1. I have reviewed this Quarterly Report on Form 10-Q of LogicBio Therapeutics, Inc.;

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; and

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; and

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.

Date: November 14, 2022	By:	/s/ Frederic Chereau
		Frederic Chereau
		President and Chief Executive Officer (Principal Executive Officer)

EX-31.2

Exhibit 31.2

Certification of Principal Financial Officer pursuant to Exchange Act Rules 13a-14(a)

and 15d-14(a), as adopted pursuant to Section 302 of Sarbanes-Oxley Act of 2002

I, Josh Blacher, certify that:

1. I have reviewed this Quarterly Report on Form 10-Q of LogicBio Therapeutics, Inc.;

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; and

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; and

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.

Date: November 14, 2022	By:	/s/ Josh Blacher
		Josh Blacher
		Chief Financial Officer (Principal Financial Officer)

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 the Quarterly Report on Form 10-Q of LogicBio Therapeutics, Inc. (the “Company”) for the quarter ended September 30, 2022 as filed with the Securities and Exchange Commission on the date hereof (the “Report”), each of the undersigned officers of the company, hereby certifies, pursuant to Section 1350 of Chapter 63 of Title 18, United States Code, that to the best of his or her knowledge:

(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.

Dated: November 14, 2022	/s/ Frederic Chereau
	Frederic Chereau President and Chief Executive Officer
Dated: November 14, 2022	/s/ Josh Blacher
	Josh Blacher Chief Financial Officer

XML

Document and Entity Information - shares	9 Months Ended
	Sep. 30, 2022	Nov. 08, 2022
Cover [Abstract]
Document Type	10-Q
Amendment Flag	false
Document Period End Date	Sep. 30, 2022
Document Fiscal Period Focus	Q3
Document Fiscal Year Focus	2022
Entity Registrant Name	LogicBio Therapeutics, Inc.
Entity Central Index Key	0001664106
Trading Symbol	LOGC
Current Fiscal Year End Date	--12-31
Entity Filer Category	Non-accelerated Filer
Entity Small Business	true
Entity Emerging Growth Company	true
Entity Ex Transition Period	true
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Shell Company	false
Entity File Number	001-38707
Entity Tax Identification Number	47-1514975
Entity Address, Address Line One	65 Hayden Avenue
Entity Address, Address Line Two	2nd Floor
Entity Address, City or Town	Lexington
Entity Address, State or Province	MA
Entity Address, Postal Zip Code	02421
City Area Code	617
Local Phone Number	245-0399
Entity Common Stock, Shares Outstanding		32,962,733
Security Exchange Name	NASDAQ
Title of 12(b) Security	Common Stock, par value $0.0001 per share
Document Quarterly Report	true
Document Transition Report	false
Entity Incorporation, State or Country Code	DE

XML

Condensed Consolidated Balance Sheets - USD ($) $ in Thousands	Sep. 30, 2022	Dec. 31, 2021
CURRENT ASSETS:
Cash and cash equivalents	$ 30,780	$ 53,480
Accounts receivable		30
Prepaid expenses and other current assets	1,996	2,156
Total current assets	32,776	55,666
Property and equipment, net	1,503	1,911
Restricted cash	622	622
Operating lease right-of-use asset	3,700	4,571
TOTAL ASSETS	38,601	62,770
CURRENT LIABILITIES:
Accounts payable	1,111	718
Accrued expenses and other current liabilities	2,321	3,704
Operating lease liabilities	1,335	1,227
Current portion of long-term debt	3,306	3,295
Current portion of deferred revenue	8,360	10,639
Total current liabilities	16,433	19,583
Long-term debt, net of issuance costs and discount	2,626	5,006
Operating lease liabilities, net of current portion	2,711	3,725
Deferred revenue, net of current portion	1,092	3,729
Total liabilities	22,862	32,043
COMMITMENTS AND CONTINGENCIES (Note 11)
STOCKHOLDERS’ EQUITY:
Preferred stock, par value of $0.0001 per share; 25,000,000 shares authorized; no shares issued and outstanding as of September 30, 2022 and December 31, 2021
Common stock, par value of $0.0001 per share; 175,000,000 shares authorized; 32,962,733 and 32,952,306 shares issued and outstanding as of September 30, 2022 and December 31, 2021, respectively	3	3
Additional paid-in capital	173,237	170,744
Accumulated deficit	(157,501)	(140,020)
Total stockholders’ equity	15,739	30,727
TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY	$ 38,601	$ 62,770

XML

Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares	Sep. 30, 2022	Dec. 31, 2021
Statement Of Financial Position [Abstract]
Common stock, par value	$ 0.0001	$ 0.0001
Common stock, shares authorized	175,000,000	175,000,000
Common stock, shares issued	32,962,733	32,952,306
Common stock, shares outstanding	32,962,733	32,952,306
Preferred stock, par value	$ 0.0001	$ 0.0001
Preferred stock, shares authorized	25,000,000	25,000,000
Preferred stock, shares issued	0	0
Preferred stock, shares outstanding	0	0

XML

Condensed Consolidated Statements of Operations - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021
REVENUE
Collaboration and service revenue	$ 2,717	$ 2,120	$ 8,732	$ 3,383
OPERATING EXPENSES
Research and development	5,051	7,806	15,524	21,482
General and administrative	3,410	4,257	10,293	12,081
Total operating expenses	8,461	12,063	25,817	33,563
LOSS FROM OPERATIONS	(5,744)	(9,943)	(17,085)	(30,180)
OTHER INCOME (EXPENSE):
Interest income	147	3	197	13
Interest expense	(176)	(270)	(591)	(824)
Other (expense) income, net	(2)	(3)	(2)	(3)
Total other expense, net	(31)	(270)	(396)	(814)
Loss before income taxes	(5,775)	(10,213)	(17,481)	(30,994)
Income tax benefit		28		28
Net loss	$ (5,775)	$ (10,185)	$ (17,481)	$ (30,966)
Net loss per share—basic	$ (0.18)	$ (0.31)	$ (0.53)	$ (0.96)
Net loss per share—diluted	$ (0.18)	$ (0.31)	$ (0.53)	$ (0.96)
Weighted-average common stock outstanding—basic	32,962,733	32,443,960	32,962,221	32,181,912
Weighted-average common stock outstanding—diluted	32,962,733	32,443,960	32,962,221	32,181,912

XML

Condensed Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021
Statement Of Income And Comprehensive Income [Abstract]
Net loss	$ (5,775)	$ (10,185)	$ (17,481)	$ (30,966)
Other comprehensive income:
Comprehensive loss	$ (5,775)	$ (10,185)	$ (17,481)	$ (30,966)

XML

Condensed Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands	Total	At The Market Offering [Member]	Common Stock [Member]	Common Stock [Member] At The Market Offering [Member]	Additional Paid-in Capital [Member]	Additional Paid-in Capital [Member] At The Market Offering [Member]	Accumulated Deficit [Member]
Beginning Balance at Dec. 31, 2020	$ 61,427		$ 3		$ 161,415		$ (99,991)
Beginning Balance, Shares at Dec. 31, 2020			31,775,748
Vesting of restricted stock, Shares			31,372
Issuance of common stock related to at-the-market offerings, net of issuance costs		$ 2,091				$ 2,091
Issuance of common stock, Shares				251,086
Stock-based compensation expense	989				989
Net loss	(10,282)						(10,282)
Ending Balance at Mar. 31, 2021	54,225		$ 3		164,495		(110,273)
Ending Balance, Shares at Mar. 31, 2021			32,058,206
Beginning Balance at Dec. 31, 2020	61,427		$ 3		161,415		(99,991)
Beginning Balance, Shares at Dec. 31, 2020			31,775,748
Net loss	(30,966)
Ending Balance at Sep. 30, 2021	38,764		$ 3		169,718		(130,957)
Ending Balance, Shares at Sep. 30, 2021			32,945,616
Beginning Balance at Mar. 31, 2021	54,225		$ 3		164,495		(110,273)
Beginning Balance, Shares at Mar. 31, 2021			32,058,206
Vesting of restricted stock, Shares			84,384
Exercise of options	52				52
Exercise of options, Shares			70,620
Issuance of common stock related to at-the-market offerings, net of issuance costs		45				45
Issuance of common stock, Shares				9,156
Stock-based compensation expense	997				997
Net loss	(10,499)						(10,499)
Ending Balance at Jun. 30, 2021	44,820		$ 3		165,589		(120,772)
Ending Balance, Shares at Jun. 30, 2021			32,222,366
Vesting of restricted stock, Shares			11,489
Exercise of options	35				35
Exercise of options, Shares			49,926
Issuance of common stock related to at-the-market offerings, net of issuance costs		$ 2,962				$ 2,962
Issuance of common stock, Shares				661,835
Stock-based compensation expense	1,132				1,132
Net loss	(10,185)						(10,185)
Ending Balance at Sep. 30, 2021	38,764		$ 3		169,718		(130,957)
Ending Balance, Shares at Sep. 30, 2021			32,945,616
Beginning Balance at Dec. 31, 2021	30,727		$ 3		170,744		(140,020)
Beginning Balance, Shares at Dec. 31, 2021			32,952,306
Vesting of restricted stock, Shares			10,427
Stock-based compensation expense	846				846
Net loss	(6,662)						(6,662)
Ending Balance at Mar. 31, 2022	24,911		$ 3		171,590		(146,682)
Ending Balance, Shares at Mar. 31, 2022			32,962,733
Beginning Balance at Dec. 31, 2021	30,727		$ 3		170,744		(140,020)
Beginning Balance, Shares at Dec. 31, 2021			32,952,306
Net loss	(17,481)
Ending Balance at Sep. 30, 2022	15,739		$ 3		173,237		(157,501)
Ending Balance, Shares at Sep. 30, 2022			32,962,733
Beginning Balance at Mar. 31, 2022	24,911		$ 3		171,590		(146,682)
Beginning Balance, Shares at Mar. 31, 2022			32,962,733
Stock-based compensation expense	849				849
Net loss	(5,044)						(5,044)
Ending Balance at Jun. 30, 2022	20,716		$ 3		172,439		(151,726)
Ending Balance, Shares at Jun. 30, 2022			32,962,733
Stock-based compensation expense	798				798
Net loss	(5,775)						(5,775)
Ending Balance at Sep. 30, 2022	$ 15,739		$ 3		$ 173,237		$ (157,501)
Ending Balance, Shares at Sep. 30, 2022			32,962,733

XML

Condensed Consolidated Statements of Stockholders' Equity (Parenthetical) - USD ($) $ in Thousands	3 Months Ended
	Sep. 30, 2021	Jun. 30, 2021	Mar. 31, 2021
Issuance costs	$ 92	$ 1	$ 65

XML

Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021
CASH FLOWS FROM OPERATING ACTIVITIES:
Net loss	$ (17,481)	$ (30,966)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation expense	508	451
Stock-based compensation expense	2,493	3,118
Non-cash interest expense	131	173
Non-cash lease expense	836	811
Changes in operating assets and liabilities:
Prepaid expenses and other current assets	160	1,220
Accounts receivable	30	144
Accounts payable	393	456
Accrued expenses and other current liabilities	(2,254)	26
Deferred revenue	(4,916)	10,733
Net cash used in operating activities	(20,100)	(13,834)
CASH FLOWS FROM INVESTING ACTIVITIES:
Purchases of property and equipment	(100)	(734)
Net cash used in investing activities	(100)	(734)
CASH FLOWS FROM FINANCING ACTIVITIES:
Proceeds from exercise of stock options		87
Net proceeds from at-the-market common stock issuances		5,098
Principal repayments on term loan	(2,500)	(1,111)
Net cash (used in) provided by financing activities	(2,500)	4,074
NET DECREASE IN CASH, CASH EQUIVALENTS AND RESTRICTED CASH	(22,700)	(10,494)
Cash, cash equivalents and restricted cash at beginning of year	54,102	70,697
Cash, cash equivalents and restricted cash at end of period	31,402	60,203
RECONCILIATION OF CASH, CASH EQUIVALENTS AND RESTRICTED CASH
Cash and cash equivalents	30,780	59,581
Long-term restricted cash	622	622
Total cash, cash equivalents and restricted cash	31,402	60,203
SUPPLEMENTAL DISCLOSURE OF CASH FLOW INFORMATION:
Cash paid for interest	$ 460	651
SUPPLEMENTAL DISCLOSURE OF NON-CASH ACTIVITIES:
Property and equipment purchases in accrued expenses and accounts payable		$ 48

XML

NATURE OF BUSINESS AND BASIS OF PRESENTATION

9 Months Ended

Sep. 30, 2022

Accounting Policies [Abstract]

NATURE OF BUSINESS AND BASIS OF PRESENTATION

1. NATURE OF BUSINESS AND BASIS OF PRESENTATION

Business Overview

LogicBio^® Therapeutics, Inc. (“LogicBio” or the “Company”) was incorporated in 2014 as a Delaware corporation. Its principal office is in Lexington, Massachusetts. LogicBio is a clinical-stage genetic medicine company pioneering genome editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood. The Company's genome editing platform, GeneRide^®, is a new approach to precise gene insertion harnessing a cell's natural deoxyribonucleic acid (“DNA”) repair process potentially leading to durable therapeutic protein expression levels. The Company's gene delivery platform, sAAVy™, is an adeno-associated virus (“AAV”) capsid engineering platform designed to optimize gene delivery for treatments in a broad range of indications and tissues. The Company’s proprietary manufacturing process, mAAVRx^TM, is a novel process aimed at improving production yields for viral vector manufacturing.

The Company’s lead product candidate, LB-001, is a single-administration, genome editing therapy developed using its GeneRide technology, currently in Phase 1/2 development for the treatment of methylmalonic acidemia (“MMA”) in pediatric patients. MMA is a rare and life-threatening genetic disorder affecting approximately 1 in 50,000 newborns in the United States that often results in developmental delays and other long-term complications and a high rate of hospitalizations.

In April 2021, the Company entered into an Exclusive Research Collaboration, License and Option Agreement (the “Original CANbridge Agreement”) with CANbridge Care Pharma Hong Kong Limited (“CANbridge”), pursuant to which LogicBio granted CANbridge (a) an exclusive worldwide license to certain of the Company’s intellectual property rights, including those relating to AAV sL65 (“sL65”), the first capsid produced from the sAAVy platform, to develop, manufacture and commercialize gene therapy candidates for the treatment of Fabry and Pompe diseases, (b) an option to obtain an exclusive worldwide license to certain of the Company’s intellectual property rights, including those relating to sL65, to develop and commercialize gene therapy candidates for the treatment of two additional indications, and (c) an exclusive option to obtain an exclusive license to develop and commercialize LB-001 for the treatment of MMA in China, Taiwan, Hong Kong and Macau. The Original CANbridge Agreement was amended and restated on October 3, 2022. For the description of the amended and restated agreement, see Note 13 in the accompanying notes to our unaudited consolidated financial statements. Also in April 2021, the Company announced a research collaboration with Daiichi Sankyo Company, Limited (“Daiichi”) for the development of treatments for two indications based on GeneRide. In addition, the Company entered into a research collaboration with Takeda Pharmaceutical Company Limited (“Takeda”) in January 2020 to develop LB-301, an investigational therapy leveraging GeneRide, for the treatment of Crigler-Najjar syndrome (“CN”), a rare pediatric disease. The work under the research plan was completed in 2021.  

Since its inception, the Company has devoted the majority of its efforts to research and development, including its preclinical and clinical development activities and its manufacturing and process development activities, raising capital, and providing general and administrative support for these operations. The Company is subject to a number of risks similar to those of other companies conducting high-risk, early-stage research and development. Principal among these risks are clinical and regulatory risks associated with drug development, dependency on key individuals and intellectual property, competition from other products and companies, and the technical risks associated with the successful research, development and clinical manufacturing of its product candidates. The Company’s success is dependent upon its ability to continue to raise additional capital in order to fund ongoing research and development, meet its obligations and, ultimately, obtain regulatory approval of its product candidates, successfully commercialize its products and technologies, and if approved, generate revenue and attain profitable operations.

Proposed Acquisition by Alexion Pharmaceuticals, Inc.

On October 3, 2022, the Company entered into an Agreement and Plan of Merger (as it may be amended from time to time, the “Merger Agreement”), by and among the Company, Alexion Pharmaceuticals, Inc., a Delaware corporation (“Alexion” or “Parent”), and Camelot Merger Sub, Inc., a Delaware corporation and a wholly-owned subsidiary of Parent (“Merger Sub”). Pursuant to the Merger Agreement, on October 18, 2022, Merger Sub commenced a tender offer to purchase, subject to the satisfaction or waiver of certain conditions set forth in the Merger Agreement, any and all of the issued and outstanding shares of common stock, par value $0.0001 per share, of the Company (the “Shares”), at a price of $2.07 per Share, to the seller in cash, without interest, less any applicable withholding taxes, upon the terms and subject to the conditions set forth in the Offer to Purchase, dated October 18, 2022 (the “Offer to Purchase,” and together with the Letter of Transmittal and other related materials, as may be amended, which together constitute the “Offer”).

On the same date as the consummation of the Offer and subject to the satisfaction or waiver of certain conditions set forth in the Merger Agreement, Merger Sub will be merged with and into the Company (the “Merger”) without a vote of the Company’s stockholders to adopt the Merger Agreement and consummate the Merger, in accordance with Section 251(h) of the of the Delaware General Corporation Law (the “DGCL”), with the Company continuing as the surviving corporation and as a wholly-owned subsidiary of Parent. The Offer is subject to certain important conditions; see “Item 1A. Risk Factors” for more information.

COVID-19 Impact

The Company continues to closely monitor the COVID-19 pandemic in order to promote the safety of its personnel and to continue advancing its research and development activities. The Company is following federal, state and local requirements and guidelines with respect to the COVID-19 pandemic and has allowed its employees to work on-premises in accordance with those requirements and guidelines.

The COVID-19 pandemic did not have a material impact on the Company’s results of operations, cash flow and financial position as of and for the quarter ended September 30, 2022. However, the Company is aware that certain of its third-party vendors are being affected by import/export and other restrictions due to COVID-19, which may have an impact on certain of the Company’s research, development and manufacturing activities. The full extent to which the COVID-19 pandemic, including the development of new variants, could directly or indirectly impact the Company’s business, results of operations and financial position will depend on future developments that are uncertain and cannot be accurately predicted.

We cannot predict the impact of the progression of the COVID-19 pandemic on future results due to a variety of factors, including the health of our and our third-party vendors’, suppliers’ and collaborators’ employees, our ability to maintain operations, the ability of our third-party vendors, suppliers and collaborators to continue operations, any further government and/or public actions taken in response to the pandemic and ultimately the length of the pandemic. We plan to continue to closely monitor the COVID-19 pandemic in order to ensure the safety of our personnel and to continue advancing our research and development activities.

Liquidity and Capital Resources

The Company has had recurring losses since inception and incurred a loss of $17,481 during the nine months ended September 30, 2022. Net cash used in operations for the nine months ended September 30, 2022 was $20,100. The Company expects to continue to generate operating losses and use cash in operations for the foreseeable future. As of September 30, 2022, the Company had cash and cash equivalents of $30,780 which management believes will be sufficient to fund its operating expenses and capital expenditure requirements into the second quarter of 2023, however due to the uncertainties described below, there is substantial doubt about the Company’s ability to continue as a going concern. On a quarterly basis, the Company is required to conduct an accounting analysis under ASC 205-40, Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern, (“ASC 205-40”). The result of the Company’s ASC 205-40 analysis is that there is substantial doubt about the Company’s ability to continue as a going concern through the next twelve months from the date of issuance of these unaudited condensed consolidated financial statements.

The Company will require substantial additional capital to fund its research and development, including its preclinical and clinical development activities and its manufacturing and process development activities, and ongoing operating expenses.  In connection with the proposed Merger with Alexion, following a consummation of the Offer and subject to the satisfaction or waiver of certain conditions set forth in the Merger Agreement, the Company would continue as a wholly-owned subsidiary of Alexion. Without taking into account the proposed Merger with Alexion, management’s plans to mitigate the conditions that raise substantial doubt on its ability to fund ongoing R&D and other operating activities include raising additional capital through equity or debt financings, payments from its collaborators, strategic transactions, or a combination of those approaches. These plans may also include the possible elimination or deferral of certain research programs or operating expenses unless and until additional capital is received. There can be no assurance that the Company will be successful in raising additional capital or that such capital, if available, will be on terms that are acceptable to the Company, its Board of Directors, or that the Company will be successful in deferring certain operating expenses.

The accompanying unaudited interim condensed consolidated financial statements have been prepared on a going concern basis, which contemplates the realization of assets and satisfaction of liabilities in the ordinary course of business. The unaudited condensed consolidated financial statements do not include any adjustments relating to the recoverability and classification of recorded asset amounts or the amounts and classification of liabilities that might result from the outcome of this uncertainty.

Basis of Presentation

The accompanying unaudited condensed consolidated financial statements have been prepared by the Company in conformity with accounting principles generally accepted in the United States of America (“U.S. GAAP”) and pursuant to the rules and regulations of the U.S. Securities and Exchange Commission (“SEC”) for interim financial statements. Certain information and footnote disclosures normally included in financial statements prepared in accordance with U.S. GAAP have been condensed or omitted pursuant to such rules and regulations. However, the Company believes that the disclosures are adequate to make the information presented not misleading. These unaudited condensed consolidated financial statements should be read in conjunction with the Company’s audited consolidated financial statements and the notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2021, filed with the SEC on March 4, 2022.

The unaudited interim condensed consolidated financial statements have been prepared on the same basis as the audited consolidated financial statements. In the opinion of management, the accompanying unaudited interim condensed consolidated financial statements contain all adjustments that are necessary for a fair statement of the Company’s financial position as of September 30, 2022, consolidated results of operations for the three and nine months ended September 30, 2022 and 2021 and cash flows for the nine months ended September 30, 2022 and 2021. Such adjustments are of a normal and recurring nature. The results of operations for the three and nine months ended September 30, 2022 are not necessarily indicative of the results of operations that may be expected for the year ending December 31, 2022.

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SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES

9 Months Ended

Sep. 30, 2022

Accounting Policies [Abstract]

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES

2. SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES

Significant Accounting Policies

The Company’s significant accounting policies are disclosed in the audited consolidated financial statements and the notes thereto, which are included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2021, filed with the SEC on March 4, 2022. Since the date of those financial statements, there have been no material changes to its significant accounting policies.

Recently Issued Accounting Pronouncements

From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board (“FASB”) or other standard setting bodies that are adopted by the Company as of the specified effective date. The Company believes that the impact of recently issued standards that are not yet effective will not have a material impact on the Company’s consolidated financial statements upon adoption.

In March 2020, the FASB issued Accounting Standards Update (“ASU”) 2020-04, Facilitation of the Effects of Reference Rate Reform on Financial Reporting (Topic 848). This ASU provides optional expedients and exceptions for applying U.S. GAAP to transactions affected by reference rate (e.g., LIBOR) reform if certain criteria are met, for a limited period of time to ease the potential burden in accounting for (or recognizing the effects of) reference rate reform on financial reporting. The ASU is effective now through December 31, 2022. The Company will evaluate transactions or contract modifications, including any related to its July 2019 loan and security agreement that uses LIBOR as a reference rate, occurring as a result of reference rate reform and determine whether to apply the optional guidance on an ongoing basis. The ASU is currently not expected to have a material impact on the Company’s consolidated financial statements and related disclosures.

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FAIR VALUE MEASUREMENTS

9 Months Ended

Sep. 30, 2022

Fair Value Disclosures [Abstract]

FAIR VALUE MEASUREMENTS

3. FAIR VALUE MEASUREMENTS

The following tables present information about the Company’s financial assets measured at fair value on a recurring basis and indicate the level of the fair value hierarchy utilized to determine such fair values:

Description	September 30, 2022		Quoted Prices in Active Markets for Identical Assets (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Other Observable Inputs (Level 3)
Assets
Money market funds and other cash equivalents	$	29,834	$	29,834	$	—	$	—
Total financial assets	$	29,834	$	29,834	$	—	$	—

Description	December 31, 2021		Quoted Prices in Active Markets for Identical Assets (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Other Observable Inputs (Level 3)
Assets
Money market funds and other cash equivalents	$	52,866	$	52,866	$	—	$	—
Total financial assets	$	52,866	$	52,866	$	—	$	—

When developing fair value estimates, the Company maximizes the use of observable inputs and minimizes the use of unobservable inputs. When available, the Company uses quoted market prices to measure fair value. The valuation technique used to measure fair value for the Company’s financial assets is a market approach, using prices and other relevant information generated by market transactions involving identical or comparable assets. If market prices are not available, the fair value measurement is based on models that use primarily market-based parameters including yield curves, volatilities, credit ratings and currency rates. In certain cases where market rate assumptions are not available, the Company is required to make judgments about assumptions market participants would use to estimate the fair value of a financial instrument.

The Company did not have any transfers of assets between levels of the fair value measurement hierarchy during the nine months ended September 30, 2022.

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ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES

9 Months Ended

Sep. 30, 2022

Payables And Accruals [Abstract]

ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES

4. ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES

Accrued expenses and other current liabilities at September 30, 2022 and December 31, 2021 consisted of the following:

	September 30, 2022		December 31, 2021
Accrued compensation and benefits	$	1,648	$	2,289
Accrued professional services		665		1,341
Other		8		74
Total accrued expenses and other current liabilities	$	2,321	$	3,704

Accrued compensation and benefits consists primarily of accrued bonuses. Accrued professional services consists primarily of consulting services, legal services and services provided by contract research organizations (“CRO”) and contract manufacturing organizations (“CMO”).

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DEBT

9 Months Ended

Sep. 30, 2022

Debt Disclosure [Abstract]

DEBT

5. DEBT

On July 2, 2019 (the “Closing Date”), the Company entered into a loan and security agreement (the “Loan Agreement”), for term loans with Oxford Finance LLC (“Oxford”) and Horizon Technology Finance Corporation (“Horizon,” and, together with Oxford, the “Lenders”). The Loan Agreement allowed the Company to borrow up to $20,000 issuable in two equal tranches (the “Term Loans”). On the Closing Date, the first tranche of $10,000 was drawn down by the Company. In September 2020 and March 2021, the Company entered into amendments to the Loan Agreement, each of which extended the availability of the second $10,000 tranche subject to certain conditions. In the second quarter of 2021, the Company met the conditions necessary to initiate the drawdown of the second tranche but did not exercise its right to do so, and the option to draw down the second tranche of the Term Loans expired unused.

The outstanding loan balance will accrue interest at the greater of (i) the rate of the one-month U.S. LIBOR rate plus 6.25% and (ii) 8.75%. The Loan Agreement provides for an interest only period until July 1, 2021, followed by thirty-six equal monthly payments of principal and interest continuing through June 1, 2024 (the “Maturity Date”). The Company has the option to prepay the outstanding balance prior to maturity, subject to a prepayment fee ranging from 1.0% to 3.0% depending upon when the prepayment occurs. Upon repayment of the Term Loans, the Company is required to make a final payment to the Lenders equal to 4.5% of the original principal amount of the Term Loans funded which will be accrued by charges to interest expense over the term of the loans using the effective interest method.

In conjunction with the Loan Agreement, the Company issued warrants to the Lenders to purchase 15,686 shares of common stock (“Warrants”) at a per share exercise price of $12.75, a maximum contractual term of 10 years and exercisable immediately. The fair value of the Warrants was accounted for as a debt discount and calculated to be approximately $136 using the Black-Scholes method. The Company determined the Warrants met the criteria for equity classification, and, as such, the fair value of the Warrants is recorded as additional paid-in capital on the condensed consolidated balance sheets. Finally, the Company incurred costs related to the issuance of the Term Loans of approximately $150. Both the debt discount and issuance costs will be accreted to Long-term debt, net of issuance costs and discount by charges to interest expense over the term of the loan using the effective interest method.

The Loan Agreement contains customary representations, warranties and covenants and also includes customary events of default. Events of default include, among other things, the non-payment of principal, interest, and other amounts when due, breach of covenants, a material adverse change event, cross-default on material indebtedness, misrepresentations, material judgments, bankruptcy and delisting. Upon the occurrence of an event of default, a default interest rate of an additional 5.0% per annum may be applied to the outstanding loan balances, and the Lenders may declare all outstanding obligations immediately due and payable. Borrowings under the Loan Agreement are collateralized by substantially all the Company’s assets, other than its intellectual property, which include maintaining certain cash balances in controlled accounts.

Interest expense related to the Loan Agreement was $176 and $591 for the three and nine months ended September 30, 2022, respectively. Interest expense related to the Loan Agreement was $270 and $824 for the three and nine months ended September 30, 2021, respectively. The effective rate on the Loan Agreement, including the amortization of the debt discount and issuance costs, and accretion of the final payment, was 11.34% at September 30, 2022. The components of the long-term debt balance are as follows:

	September 30, 2022			December 31, 2021
Notes payable, gross	$	5,556		$	8,055
Less: Unamortized debt discount and    issuance costs		(46	)		(93	)
Accretion of final payment fee		422			339
Carrying value of notes payable		5,932			8,301
Less: Current portion of long-term debt		(3,306	)		(3,295	)
Long-term debt, net of issuance costs and    discount	$	2,626		$	5,006

As of September 30, 2022, the estimated future principal payments due were as follows:

	As of September 30, 2022
2022	$	833
2023		3,333
2024		1,389
Thereafter		—
Total principal payments	$	5,555

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STOCK-BASED COMPENSATION

9 Months Ended

Sep. 30, 2022

Disclosure Of Compensation Related Costs Sharebased Payments [Abstract]

STOCK-BASED COMPENSATION

6. STOCK-BASED COMPENSATION

Equity Incentive Plans

In December 2014, the Company adopted the LogicBio Therapeutics, Inc. 2014 Equity Incentive Plan, as amended (the “2014 Plan”), for the issuance of stock options and other stock-based awards. Subsequently, in October 2018, the Company’s 2018 Equity Incentive Plan (the “2018 Plan”) was adopted, superseding the 2014 Plan; no further awards have been, or will be, made under the 2014 Plan. The 2018 Plan was established to provide equity-based ownership opportunities for employees and directors, as well as outside consultants and advisors. Any awards granted under the 2014 Plan prior to the adoption of the 2018 Plan remained outstanding in accordance with their respective terms.

Under the 2018 Plan, there is an annual increase on January 1 of each year from 2019 until 2028, of the lesser of (i) 4% of the number of shares of common stock outstanding on December 31 of the prior year or (ii) a number of shares of common stock as determined by the Company’s Board of Directors (“Board”). Accordingly, on January 1, 2022, the number of shares available for future grant under the 2018 Plan increased by 1,318,271 shares. At September 30, 2022, there were 997,841 shares available for future grant under the 2018 Plan.

The 2018 Plan is administered by the Compensation Committee of the Board, except with respect to such matters that are not delegated to the Compensation Committee by the Board (collectively, the “Administrator”). The exercise prices, vesting and certain other restrictions are determined at the discretion of the Administrator, except that the exercise price per share of stock options may not be less than 100% of the fair market value of the common stock on the date of grant. The term of stock options awarded under the 2018 Plan may not exceed 10 years from the grant date. Stock options, shares of restricted stock and restricted stock units (“RSUs”) granted to employees, officers, members of the Board, advisors, and consultants of the Company typically vest over one to four years.  

Stock Options

During the nine months ended September 30, 2022 and 2021, the Company granted options with time-based vesting to purchase 1,672,250   and 2,097,256 shares of common stock, respectively, with a weighted-average grant date fair value per share of $0.44 and $4.13, respectively. The Company recorded stock-based compensation expense for options granted of $2,491 and $2,812 during the nine months ended September 30, 2022 and 2021, respectively. As of September 30, 2022, there were 5,390,188 outstanding options, of which 2,931,063 were unvested, and $6,019 of unrecognized stock-based compensation expense to be recognized over a weighted-average period of 2.3 years.

Restricted Stock

The Company has granted shares of restricted stock with time-based and performance-based vesting conditions from time to time, and subsequent grants may be made. The Company did not grant any restricted stock during the nine months ended September 30, 2022 or 2021. During the nine months ended September 30, 2022, no expense was recorded for restricted stock previously granted. During the nine months ended September 30, 2021, the Company recorded stock-based compensation expense of $132 related to restricted stock previously granted. As of September 30, 2022, there were no unvested shares of restricted stock outstanding.

Restricted Stock Units

The Company has granted RSUs with time-based vesting conditions from time to time. Each RSU represents the right to receive one share of the Company’s common stock upon vesting. The fair value is calculated based upon the Company’s closing stock price on the date of grant, and the stock-based compensation expense is recognized over the vesting period. The Company did not grant any RSUs during the nine months ended September 30, 2022. The Company granted 5,939 RSUs during the nine months ended September 30, 2021. The Company recorded stock-based compensation for RSUs of $2 and $174 during the nine months ended September 30, 2022 and 2021, respectively. As of September 30, 2022, there were no unvested RSUs outstanding.

Stock-Based Compensation Expense

Total stock-based compensation expense recorded as research and development and general and administrative expenses, respectively, for employees, directors and non-employees for the nine months ended September 30, 2022 and 2021 is as follows:

	Nine Months Ended September 30,
	2022		2021
Research and development	$	872	$	1,051
General and administrative		1,621		2,067
Total stock-based compensation expense	$	2,493	$	3,118

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STOCKHOLDERS EQUITY

9 Months Ended

Sep. 30, 2022

Stockholders Equity [Abstract]

STOCKHOLDERS EQUITY

7. STOCKHOLDERS’ EQUITY

Open Market Sale Agreement

On November 15, 2019, the Company entered into an Open Market Sale Agreement (the “Open Market Sale Agreement”) with Jefferies LLC, as agent (“Jefferies”), and filed a related prospectus supplement, pursuant to which the Company may issue and sell shares of its common stock at the then current market prices having an aggregate offering price of up to $50,000 (the “Open Market Shares”) from time to time through Jefferies (the “Open Market Offering”).

Under the Open Market Sale Agreement, Jefferies may sell the Open Market Shares by methods deemed to be an “at the market offering” as defined in Rule 415(a)(4) promulgated under the Securities Exchange Act of 1934, as amended. The Company may sell the Open Market Shares in amounts and at times to be determined by the Company from time to time subject to the terms and conditions of the Open Market Sale Agreement, but it has no obligation to sell any of the Open Market Shares in the Open Market Offering.

The Company or Jefferies may suspend or terminate the offering of Open Market Shares upon notice to the other party and subject to other conditions. The Company has agreed to pay Jefferies commissions for its services in acting as agent in the sale of the Open Market Shares in the amount of up to 3.0% of gross proceeds from the sale of the Open Market Shares pursuant to the Open Market Sale Agreement. The Company has also agreed to provide Jefferies with customary indemnification and contribution rights.

During the nine months ended September 30, 2022, the Company did not issue any Open Market Shares. During the nine months ended September 30, 2021, the Company issued 922,077 Open Market Shares at a weighted-average price of $5.70 per share, resulting in net proceeds to the Company of $5,098. At September 30, 2022, the Company had $41,253 in aggregate gross offering amount available under the Open Market Sale Agreement.

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REVENUE

9 Months Ended

Sep. 30, 2022

Revenue From Contract With Customer [Abstract]

REVENUE

8. REVENUE

Service Revenue

Takeda Agreement

In January 2020, the Company entered into a Research Collaboration and Option Agreement with Takeda (“Takeda Agreement”), which expired by its own terms in the year ended December 31, 2021.

The Company assessed the Takeda Agreement in accordance with ASC 606 and concluded that it represented a contract with a customer and was within the scope of ASC 606. The promised goods and services represented one combined performance obligation and the entire transaction price was allocated to that single combined performance obligation. In addition, the Company concluded that the exclusive option to obtain an exclusive license to LB-301 granted to Takeda under the Takeda Agreement (the “License Option”) did not provide any discounts or other rights. Terms related to an exclusive license negotiated after the exercise of the License Option would be part of a separate contract and reflect applicable standalone selling prices. As such, the Company concluded the License Option was not considered to be a material right.

Under the Takeda Agreement, Takeda was obligated to reimburse the Company for the costs incurred under the research plan. Costs incurred were billed by the Company to Takeda from time to time. The Company elected to recognize revenue under the "right to invoice" practical expedient based on the Company's right to invoice Takeda at an amount that approximated the value transferred to the customer and the performance completed to date. During 2022, there was no service revenue recognized under the Takeda Agreement. During the three and nine months ended September 30, 2021, the Company recognized $43 and $556, respectively, in service revenue under the Takeda Agreement.

Daiichi Sankyo Agreement

In April 2021, the Company entered into a Research Collaboration and Exclusive Option Agreement with Daiichi (the “Daiichi Agreement”) for the development of gene therapy candidates for two indications based on the GeneRide platform (each, a “Daiichi Candidate”). Under the terms of the Daiichi Agreement, Daiichi will fund all research and development activities related to the development of the Daiichi Candidates under a mutually agreed research plan (the “Daiichi Research Plan”). The Daiichi Agreement also provides Daiichi with an exclusive, non-binding option for each Daiichi Candidate to negotiate in good faith for a certain period of time to enter into a license agreement with respect to each such Daiichi Candidate (the “Daiichi License Options”).

The Company assessed the Daiichi Agreement in accordance with ASC 606 and concluded that it represents a contract with a customer and is within the scope of ASC 606. The Company concluded that its conduct of research services under the Daiichi Research Plan, which includes a research data package, participation in various joint oversight committees, a research license and a materials transfer, represents a single combined performance obligation. The Company determined the transaction price totaled $2,000, which included an upfront payment of $1,000 and an additional $1,000 prepayment of the first-year research and development fees. The entire transaction price will be allocated to the single combined performance obligation, which is transferred over the expected term of the conduct of the research services. Terms related to exclusive licenses negotiated after the exercise of the Daiichi License Options will be part of a separate contract and reflect applicable standalone selling prices. As such, the Company concluded the Daiichi License Options are not considered to be a material right.

The upfront payment of $2,000 was recorded as deferred revenue and is being recognized as revenue over time in conjunction with the Company’s conduct of research services as the research services are the primary component of the combined performance obligation. Revenue associated with the upfront payment and ongoing research services will be recognized using an input-based measurement of actual costs incurred as a percentage of the estimated total costs expected to be incurred over the expected term of conduct of the research services. The Company believes this input-based method to recognize revenue best reflects the transfer of value to Daiichi. During the three and nine months ended September 30, 2022, the Company recognized $382 and $1,124, respectively, as service revenue under the Daiichi Agreement. During the three and nine months ended September 30, 2021, the Company recognized $392

and $572, respectively, as service revenue under the Daiichi Agreement. As of September 30, 2022, there was $959 in deferred revenue related to the Daiichi Agreement, all of which is classified as current deferred revenue.  

Collaboration Revenue

CANbridge Agreement

In April 2021, the Company entered into an Exclusive Research Collaboration, License and Option Agreement with CANbridge (the “Original CANbridge Agreement”). The Original CANbridge Agreement was amended and restated on October 3, 2022.  For the description of the amended and restated agreement, see Note 13 in the accompanying notes to our unaudited consolidated financial statements.

Under the terms of the Original CANbridge Agreement, the Company granted CANbridge (a) an exclusive worldwide license to certain of the Company’s intellectual property rights, including those relating to sL65, to develop, manufacture and commercialize gene therapy candidates for the treatment of Fabry and Pompe diseases (the “Fabry and Pompe License”), (b) an option to obtain an exclusive worldwide license to certain of the Company’s intellectual property rights, including those relating to sL65, to develop and commercialize gene therapy candidates for the treatment of two additional indications (the “Candidate Options”) and (c) an exclusive option to obtain an exclusive license to develop and commercialize LB-001 for the treatment of MMA (the “LB-001 Option”) in China, Taiwan, Hong Kong and Macau (“Greater China”). Pursuant to the Original CANbridge Agreement, LogicBio and CANbridge collaborated in the development of the gene therapy candidates referenced in (a) above for the treatment of Fabry and Pompe diseases plus, upon CANbridge’s exercise of the applicable Candidate Options, two additional indications under a mutually agreed research plan (the “CANbridge Research Plan”).

Under the Original CANbridge Agreement, the Company received an upfront, non-refundable and non-creditable payment of $10,000 from CANbridge a portion of which was paid to a third party under certain of the Company’s in-licensing obligations. In addition, CANbridge was obligated to reimburse the Company for research and development costs incurred by the Company for activities related to the development of the gene therapy candidates for Fabry and Pompe diseases under the CANbridge Research Plan.

In addition, under the Original CANbridge Agreement, the Company was eligible to receive up to $542,000 in aggregate from CANbridge contingent on the achievement of specified clinical, regulatory and sales milestones relating to the named gene therapy candidates for Fabry and Pompe diseases, the additional indications for which CANbridge exercises the Candidate Options, and the payment of any option exercise fees. The Company was also eligible to receive up to $49,000 in aggregate clinical, regulatory and sales milestones for LB-001, subject to the exercise of the LB-001 Option, and the payment of the LB-001 Option exercise fee. CANbridge was obligated to pay to the Company royalties based on an escalating tiered, mid- to high-single digit percentage of the annual worldwide net sales for each non-LB-001 indication pursued. In addition, CANbridge was obligated to pay to the Company royalties based on an escalating tiered, high-single digit to mid-double digit percentage of the annual Greater China net sales for LB-001 for the treatment of MMA, subject to the exercise of the LB-001 Option.

The Company applied ASC Topic 808, Collaborative Arrangements (“ASC 808”) and determined that the Original CANbridge Agreement was within the scope of ASC 808. Furthermore, the Company determined that certain aspects of the Original CANbridge Agreement represented a vendor-customer relationship as CANbridge represents a customer for certain activities. As such, the Company applied the relevant guidance from ASC 606 to evaluate the appropriate accounting for the vendor-customer aspects of the Original CANbridge Agreement. In accordance with ASC 606, the Company identified its performance obligation as a grant of a license to CANbridge for certain of its intellectual property rights, including those relating to sL65, and its conduct of research services under the CANbridge Research Plan, which included participation in various joint oversight committees and a technology transfer. The Company determined that its grant of a license to CANbridge to certain of its intellectual property subject to certain conditions was not distinct as it did not have stand-alone value to CANbridge apart from the services to be performed by the Company pursuant to the Original CANbridge Agreement. A third party would not be able to provide research and development services due to the specific nature of the intellectual property and knowledge required to perform the services, and CANbridge could not benefit from the license without the corresponding services. The Company also concluded that the LB-001 Option and Candidate Options were not provided to CANbridge at a significant discount. The terms of the options, including the upfront exercise fee and applicable milestone payments, reflected applicable standalone selling prices at the time of the Original CANbridge Agreement. As such, the Company concluded that none of the options were considered to be material rights and, as such, were not performance obligations.  

Accordingly, the Company determined that its grant of a license to CANbridge and its performance of research and development services under the CANbridge Research Plan should be accounted for as one combined performance obligation, and that the combined performance obligation is transferred over the expected term of the conduct of the research and development services. 

In accordance with ASC 606, the Company determined that the initial transaction price under the Original CANbridge Agreement was $10,878, consisting of the upfront, non-refundable and non-creditable payment of $10,000 and an upfront payment of estimated quarterly research costs $878. The upfront payment of $10,878 was initially recorded as deferred revenue and, along with payments related to the Company’s performance of research services under the CANbridge Research Plan, will be recognized as revenue using an input-based measurement of actual costs incurred as a percentage of the estimated total costs expected to be incurred over the expected term of conduct of the research services. The Company believes this input-based method to recognize revenue best reflects the transfer of value to CANbridge. The Company recorded the initial $878 prepayment of the quarterly research and development fees as deferred revenue, and such fees will be recognized as revenue as the research services are delivered.

The Company also assessed the effects of variable elements including the likelihood of receiving (i) various clinical, regulatory and commercial milestone payments, and (ii) royalties on net sales of any product candidate. Based on its assessment, the Company concluded that, based on the likelihood of these uncertain events occurring, there was not a significant variable element included in the transaction price. Accordingly, the Company has not assigned a transaction price to these variable elements given the substantial uncertainty related to their achievement and has not assigned a transaction price to any CANbridge milestone or royalties.

During the three and nine months ended September 30, 2022, the Company recognized collaboration revenue of $2,335 and $7,608, respectively, under the Original CANbridge Agreement. During the three and nine months ended September 30, 2021, the Company recognized collaboration revenue of $1,685 and $2,255, respectively, under the Original CANbridge Agreement. As of September 30, 2022, aggregate deferred revenue related to the Original CANbridge Agreement was $8,493 of which $7,401 was classified as current. Both the current and non-current deferred revenue amounts will be recognized during the expected term of the conduct of research and development services. As a direct result of the Company’s entry into the Original CANbridge Agreement, the Company incurred $775 in sublicense fees to certain of its existing licensors which was expensed to research and development expense during the second quarter of 2021.

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INCOME TAXES

9 Months Ended

Sep. 30, 2022

Income Tax Disclosure [Abstract]

INCOME TAXES

9. INCOME TAXES

For the nine months ended September 30, 2022 and the year ended December 31, 2021, the Company maintained a full valuation allowance on federal and state deferred tax assets since management does not forecast the Company to be in a taxable position in the near future.

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LOSS PER SHARE

9 Months Ended

Sep. 30, 2022

Earnings Per Share [Abstract]

LOSS PER SHARE

10. LOSS PER SHARE

Basic loss per share is computed by dividing net loss by the weighted-average shares of common stock outstanding, without consideration to common stock equivalents:

	Three Months Ended September 30,						Nine Months Ended September 30,
	2022			2021			2022			2021
Numerator:
Net loss	$	(5,775	)	$	(10,185	)	$	(17,481	)	$	(30,966	)
Denominator:
Weighted-average common stock outstanding		32,962,733			32,443,960			32,962,221			32,181,912
Net loss per share — basic and diluted	$	(0.18	)	$	(0.31	)	$	(0.53	)	$	(0.96	)

The Company’s potentially dilutive shares, which include any outstanding stock options, warrants and unvested restricted stock, are considered to be common stock equivalents and are only included in the calculation of diluted net loss when their effect is dilutive.

The Company excluded the following potential common stock equivalents from the computation of diluted net loss per share attributable to common stockholders because including them would have had an anti-dilutive effect for the three and nine months ended September 30, 2022 and 2021.

	September 30, 2022		September 30, 2021
Unvested restricted common stock		—		11,153
Unvested restricted stock units		—		5,939
Options to purchase common stock		5,390,188		4,508,640
Loan warrants		15,686		15,686

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COMMITMENTS AND CONTINGENCIES

9 Months Ended

Sep. 30, 2022

Commitments And Contingencies Disclosure [Abstract]

COMMITMENTS AND CONTINGENCIES

11. COMMITMENTS AND CONTINGENCIES

Operating Lease

The Company has historically entered into lease arrangements for its facilities and certain equipment. As of September 30, 2022, the Company had one operating lease with required future minimum payments related to its headquarters in Lexington, MA.

In November 2019, the Company entered into a lease agreement for office, laboratory and vivarium space located at 65 Hayden Avenue, Lexington, Massachusetts (“65 Hayden Ave Lease”) to replace the Company’s prior headquarters located at 99 Erie Street Cambridge, Massachusetts. Under the terms of the 65 Hayden Ave Lease, the Company leases approximately 23,901 square feet of space and initially paid an annual base rent of approximately $1,494, subject to scheduled annual increases, plus certain operating expenses and taxes. The Company took possession of the space on April 1, 2020 (“Lease Commencement Date”) and the lease will continue through July 1, 2025 (“Lease Termination Date”). The Company has an option to extend the lease for a single additional term of 5 years. Upon execution of the 65 Hayden Ave Lease, the Company executed a $622 cash-collateralized letter of credit. Lease payments are due in monthly installments through the Lease Termination Date.

At the Lease Commencement Date, the Company performed a lease assessment under the guidance prescribed in ASC Topic 842, Leases (“ASC 842”), and concluded that the 65 Hayden Ave Lease was an operating lease. As such, the Company recorded an operating lease right-of-use asset and corresponding operating lease liability on the consolidated balance sheets of $6,428 which reflected the net present value of future payments under the lease. The discount rate used to calculate the net present value of future payments was the Company’s incremental borrowing rate at the Lease Commencement Date, which was 7.6%.

The following table contains a summary of the lease costs recognized under ASC 842 and other information pertaining to the Company’s operating leases for the three and nine months ended September 30, 2022 and 2021:

	Three Months Ended September 30,				Nine Months Ended September 30,
	2022		2021		2022		2021
Operating leases
Lease cost
Operating lease cost	$	378	$	378	$	1,134	$	1,134
Variable lease cost		231		208		669		639
Total lease cost	$	609	$	586	$	1,803	$	1,773
Other year-to-date lease information
Operating cash flows used    for operating leases					$	1,166	$	385
Operating lease liabilities    arising from obtaining    right-of-use assets					$	—	$	—

The following table contains a summary of the lease liabilities recognized on the Company’s unaudited condensed consolidated balance sheet as of September 30, 2022 and on the Company’s audited consolidated balance sheet as of December 31, 2021:

	As of September 30, 2022			As of December 31, 2021
Other operating lease information
Operating lease liabilities — short-term	$	1,335		$	1,227
Operating lease liabilities — long-term	$	2,711		$	3,725
Weighted-average remaining lease term	2.8 years			3.5 years
Weighted-average discount rate		7.60	%		7.60	%

The variable lease costs for the three and nine months ended September 30, 2022 and 2021 include common area maintenance and other operating charges. As the Company’s leases do not provide an implicit rate, the Company utilized its incremental borrowing rate based on what it would normally pay to borrow on a collateralized basis over a similar term for an amount equal to the lease payments at the commencement date in determining the present value of lease payments.

Future minimum lease payments under the Company’s operating lease as of September 30, 2022 and December 31, 2021, were as follows:

	As of September 30, 2022			As of December 31, 2021
Maturity of lease liabilities
2022	$	396		$	1,562
2023		1,609			1,609
2024		1,656			1,656
2025		841			841
Thereafter		—			—
Total lease payments		4,502			5,668
Less: imputed interest		(456	)		(716	)
Total operating lease liabilities	$	4,046		$	4,952

Legal Proceedings

Since the announcement of the Merger Agreement, three complaints have been filed, captioned: Bushansky v. LogicBio Therapeutics, Inc. et al., No. 1:22-CV-09145 (S.D.N.Y.) (the “Bushansky Complaint”), Wilhelm v. LogicBio Therapeutics, Inc. et al., No. 1:22-CV-09186 (S.D.N.Y.) (the “Wilhelm Complaint”), and Carlisle v. LogicBio Therapeutics, Inc. et al., No. 1:22-CV-01418 (D. Del.) (the “Carlisle Complaint”) (collectively, the “Complaints”).  Each of the Complaints names the Company and its directors as defendants.  The Company has also received eight stockholder demand letters, including one draft complaint.  These are the “Vugman Demand Letter,” the “Way Demand Letter,” the “Wheeler Demand Letter,” the “Barbeau Demand Letter,” the “Redfield Demand Letter,” the “Ortega Demand Letter,” the “Browning Demand Letter,” and the “Smith Demand Letter” (collectively, the “Demand Letters”).  The Barbeau Demand Letter contained a draft complaint, captioned Barbeau v. LogicBio Therapeutics, Inc. et al.  Moreover, the Company received a stockholder demand letter seeking access to the Company’s books and records pursuant to Delaware Code Title 8, Section 220 (the “Section 220 Demand”).

The Complaints allege that the Company and its directors violated federal securities laws by failing to disclose material information in the Solicitation/Recommendation Statement on Schedule 14D-9 filed by the Company with the SEC on October 18, 2022 (together with the exhibits thereto, the “Schedule 14D-9”).  The Complaints generally seek, among other things, to enjoin the Company from proceeding with closing the Offer, the Merger and the other transactions contemplated by the Merger Agreement (the “Transactions”) unless and until it discloses and disseminates the alleged material information or, in the event the Transactions are consummated, a rescission of the Transactions and a recovery of the rescissory damages.  In addition, the Complaints seek an award of plaintiffs’ costs, including reasonable allowance for attorneys’ fees and costs.  The Carlisle Complaint also seeks a declaration that the defendants violated federal securities laws. The Demand Letters generally seek that certain allegedly omitted information in the Schedule 14D-9 be disclosed. The Section 220 Demand seeks access to the Company’s books and records relevant to the Transactions and the Company’s board members. The Section 220 Demand cites substantially similar allegations to those in the Complaints and Demand Letters as the basis for seeking to inspect the information requested in the Section 220 Demand.

The Company believes that the allegations in the Complaints, Demand Letters and Section 220 Demand are without merit and intends to defend against them vigorously.

At this time, no assessment can be made as to the likely outcome of the Complaints, Demand Letters or Section 220 Demand or whether the outcome will be material to the Company. The Company has not recorded an expense related to the outcome of the foregoing matters because it is not yet possible to determine if a potential loss is probable nor reasonably estimable.

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RELATED PARTIES

9 Months Ended

Sep. 30, 2022

Related Party Transactions [Abstract]

RELATED PARTIES

12. RELATED PARTIES

The Company is party to a consulting service agreement with Mark Kay, who is a co-founder of the Company and a member of the Board. Under the terms of this agreement, the Company has agreed to pay an annual fee of $68 for research and development consulting services. For each of the three- and nine- months ended September 30, 2022 and 2021, the Company recorded research and development expense of $17 and $51, respectively, related to consulting services received from Mark Kay.

In addition, as a result of his participation on the Scientific Advisory Board in June 2021, Mark Kay earned $5 and received a non-qualified stock option to purchase 5,000 shares of the Company’s common stock with a fair value of $13, which are being expensed over a two-year vesting period. Expenses related to Mark Kay’s participation on the Scientific Advisory Board are recorded in research and development expense.

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SUBSEQUENT EVENTS

9 Months Ended

Sep. 30, 2022

Subsequent Events [Abstract]

SUBSEQUENT EVENTS

13. SUBSEQUENT EVENTS

Proposed Acquisition by Alexion Pharmaceuticals, Inc.

The description of the Merger Agreement with Alexion in Note 1 in the accompanying notes to our unaudited consolidated financial statements is incorporated by reference hereto.

A&R CANbridge Agreement

On October 3, 2022, the Company and CANbridge entered into the Amended and Restated Exclusive Research Collaboration, License and Option Agreement (the “A&R CANbridge Agreement”), dated as of October 2, 2022, which amends the Original CANbridge Agreement.

Under the A&R CANbridge Agreement, the Candidate Options and the LB-001 Option were removed as mutually agreed by both parties. Further, in connection with the Fabry and Pompe License, the A&R CANbridge Agreement grants CANbridge a non-exclusive license to certain Company intellectual property rights related to manufacturing for the Fabry and Pompe programs. Additionally, under the A&R CANbridge Agreement, the CANbridge Research Plan was amended to reflect that the full transfer of the technology pertaining to the Fabry and Pompe programs to CANbridge was effective as of October 31, 2022. Under the A&R CANbridge Agreement, CANbridge does not have an obligation to pay or reimburse additional research expenses.

Under the A&R CANbridge Agreement, the Company will be eligible to receive clinical, regulatory and commercial milestone payments of up to $224.5 million in the aggregate (assuming one product for each indication). In accordance with the removal of the two additional Candidate Options and LB-001 Option, milestones related thereto were removed in the A&R CANbridge Agreement. Under the A&R CANbridge Agreement, the tiered royalty rates payable to the Company will range from low single digit to mid-single digit rates. Under the A&R CANbridge Agreement, LogicBio and CANbridge will negotiate a product reversion agreement in good faith in the event of a termination of the A&R CANbridge Agreement under certain circumstances.

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SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Policies)

9 Months Ended

Sep. 30, 2022

Accounting Policies [Abstract]

Significant Accounting Policies

Significant Accounting Policies

The Company’s significant accounting policies are disclosed in the audited consolidated financial statements and the notes thereto, which are included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2021, filed with the SEC on March 4, 2022. Since the date of those financial statements, there have been no material changes to its significant accounting policies.

Recently Issued Accounting Pronouncements

Recently Issued Accounting Pronouncements

From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board (“FASB”) or other standard setting bodies that are adopted by the Company as of the specified effective date. The Company believes that the impact of recently issued standards that are not yet effective will not have a material impact on the Company’s consolidated financial statements upon adoption.

In March 2020, the FASB issued Accounting Standards Update (“ASU”) 2020-04, Facilitation of the Effects of Reference Rate Reform on Financial Reporting (Topic 848). This ASU provides optional expedients and exceptions for applying U.S. GAAP to transactions affected by reference rate (e.g., LIBOR) reform if certain criteria are met, for a limited period of time to ease the potential burden in accounting for (or recognizing the effects of) reference rate reform on financial reporting. The ASU is effective now through December 31, 2022. The Company will evaluate transactions or contract modifications, including any related to its July 2019 loan and security agreement that uses LIBOR as a reference rate, occurring as a result of reference rate reform and determine whether to apply the optional guidance on an ongoing basis. The ASU is currently not expected to have a material impact on the Company’s consolidated financial statements and related disclosures.

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FAIR VALUE MEASUREMENTS (Tables)

9 Months Ended

Sep. 30, 2022

Fair Value Disclosures [Abstract]

Financial Assets Measured at Fair Value on Recurring Basis

The following tables present information about the Company’s financial assets measured at fair value on a recurring basis and indicate the level of the fair value hierarchy utilized to determine such fair values:

Description	September 30, 2022		Quoted Prices in Active Markets for Identical Assets (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Other Observable Inputs (Level 3)
Assets
Money market funds and other cash equivalents	$	29,834	$	29,834	$	—	$	—
Total financial assets	$	29,834	$	29,834	$	—	$	—

Description	December 31, 2021		Quoted Prices in Active Markets for Identical Assets (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Other Observable Inputs (Level 3)
Assets
Money market funds and other cash equivalents	$	52,866	$	52,866	$	—	$	—
Total financial assets	$	52,866	$	52,866	$	—	$	—

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ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES (Tables)

9 Months Ended

Sep. 30, 2022

Payables And Accruals [Abstract]

Schedule of Accrued Expenses and Other Current Liabilities

Accrued expenses and other current liabilities at September 30, 2022 and December 31, 2021 consisted of the following:

	September 30, 2022		December 31, 2021
Accrued compensation and benefits	$	1,648	$	2,289
Accrued professional services		665		1,341
Other		8		74
Total accrued expenses and other current liabilities	$	2,321	$	3,704

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DEBT (Tables)

9 Months Ended

Sep. 30, 2022

Debt Disclosure [Abstract]

Schedule of Long-term Debt

The components of the long-term debt balance are as follows:

	September 30, 2022			December 31, 2021
Notes payable, gross	$	5,556		$	8,055
Less: Unamortized debt discount and    issuance costs		(46	)		(93	)
Accretion of final payment fee		422			339
Carrying value of notes payable		5,932			8,301
Less: Current portion of long-term debt		(3,306	)		(3,295	)
Long-term debt, net of issuance costs and    discount	$	2,626		$	5,006

Schedule of Estimated Future Principal Payments

As of September 30, 2022, the estimated future principal payments due were as follows:

	As of September 30, 2022
2022	$	833
2023		3,333
2024		1,389
Thereafter		—
Total principal payments	$	5,555

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STOCK-BASED COMPENSATION (Tables)

9 Months Ended

Sep. 30, 2022

Disclosure Of Compensation Related Costs Sharebased Payments [Abstract]

Schedule of Stock-Based Compensation Expense

Total stock-based compensation expense recorded as research and development and general and administrative expenses, respectively, for employees, directors and non-employees for the nine months ended September 30, 2022 and 2021 is as follows:

	Nine Months Ended September 30,
	2022		2021
Research and development	$	872	$	1,051
General and administrative		1,621		2,067
Total stock-based compensation expense	$	2,493	$	3,118

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LOSS PER SHARE (Tables)

9 Months Ended

Sep. 30, 2022

Earnings Per Share [Abstract]

Computation of Basic and Diluted Net Loss Per Share

Basic loss per share is computed by dividing net loss by the weighted-average shares of common stock outstanding, without consideration to common stock equivalents:

	Three Months Ended September 30,						Nine Months Ended September 30,
	2022			2021			2022			2021
Numerator:
Net loss	$	(5,775	)	$	(10,185	)	$	(17,481	)	$	(30,966	)
Denominator:
Weighted-average common stock outstanding		32,962,733			32,443,960			32,962,221			32,181,912
Net loss per share — basic and diluted	$	(0.18	)	$	(0.31	)	$	(0.53	)	$	(0.96	)

Computation of Potentially Anti-Dilutive Securities

The Company excluded the following potential common stock equivalents from the computation of diluted net loss per share attributable to common stockholders because including them would have had an anti-dilutive effect for the three and nine months ended September 30, 2022 and 2021.

	September 30, 2022		September 30, 2021
Unvested restricted common stock		—		11,153
Unvested restricted stock units		—		5,939
Options to purchase common stock		5,390,188		4,508,640
Loan warrants		15,686		15,686

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COMMITMENTS AND CONTINGENCIES (Tables)

9 Months Ended

Sep. 30, 2022

Leases [Abstract]

Summary of Lease Costs

The following table contains a summary of the lease costs recognized under ASC 842 and other information pertaining to the Company’s operating leases for the three and nine months ended September 30, 2022 and 2021:

	Three Months Ended September 30,				Nine Months Ended September 30,
	2022		2021		2022		2021
Operating leases
Lease cost
Operating lease cost	$	378	$	378	$	1,134	$	1,134
Variable lease cost		231		208		669		639
Total lease cost	$	609	$	586	$	1,803	$	1,773
Other year-to-date lease information
Operating cash flows used    for operating leases					$	1,166	$	385
Operating lease liabilities    arising from obtaining    right-of-use assets					$	—	$	—

The following table contains a summary of the lease liabilities recognized on the Company’s unaudited condensed consolidated balance sheet as of September 30, 2022 and on the Company’s audited consolidated balance sheet as of December 31, 2021:

	As of September 30, 2022			As of December 31, 2021
Other operating lease information
Operating lease liabilities — short-term	$	1,335		$	1,227
Operating lease liabilities — long-term	$	2,711		$	3,725
Weighted-average remaining lease term	2.8 years			3.5 years
Weighted-average discount rate		7.60	%		7.60	%

Schedule of Future Minimum Rental Payments for Operating Leases

Future minimum lease payments under the Company’s operating lease as of September 30, 2022 and December 31, 2021, were as follows:

	As of September 30, 2022			As of December 31, 2021
Maturity of lease liabilities
2022	$	396		$	1,562
2023		1,609			1,609
2024		1,656			1,656
2025		841			841
Thereafter		—			—
Total lease payments		4,502			5,668
Less: imputed interest		(456	)		(716	)
Total operating lease liabilities	$	4,046		$	4,952

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Nature of Business and Basis of Presentation - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended						9 Months Ended
	Sep. 30, 2022	Jun. 30, 2022	Mar. 31, 2022	Sep. 30, 2021	Jun. 30, 2021	Mar. 31, 2021	Sep. 30, 2022	Sep. 30, 2021	Oct. 18, 2022	Dec. 31, 2021
Schedule Of Nature Of Business And Basis Of Presentation [Line Items]
Common stock, par value	$ 0.0001						$ 0.0001			$ 0.0001
Net loss	$ 5,775	$ 5,044	$ 6,662	$ 10,185	$ 10,499	$ 10,282	$ 17,481	$ 30,966
Net cash used in operating activities							20,100	13,834
Cash and cash equivalents	$ 30,780			$ 59,581			$ 30,780	$ 59,581		$ 53,480
Merger Agreement [Member] | Subsequent Event [Member]
Schedule Of Nature Of Business And Basis Of Presentation [Line Items]
Common stock, par value									$ 0.0001
Sale of stock, price per share									$ 2.07

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Fair Value Measurements - Financial Assets and Liabilities Measured at Fair Value on Recurring Basis (Detail) - Fair Value, Measurements, Recurring [Member] - USD ($) $ in Thousands	Sep. 30, 2022	Dec. 31, 2021
Fair Value Assets And Liabilities Measured On Recurring Basis [Line Items]
Total financial assets	$ 29,834	$ 52,866
Fair Value, Inputs, Level 1 [Member]
Fair Value Assets And Liabilities Measured On Recurring Basis [Line Items]
Total financial assets	29,834	52,866
Money Market Funds And Other Cash Equivalents [Member]
Fair Value Assets And Liabilities Measured On Recurring Basis [Line Items]
Total financial assets	29,834	52,866
Money Market Funds And Other Cash Equivalents [Member] | Fair Value, Inputs, Level 1 [Member]
Fair Value Assets And Liabilities Measured On Recurring Basis [Line Items]
Total financial assets	$ 29,834	$ 52,866

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Fair Value Measurements - Additional Information (Detail)	9 Months Ended
	Sep. 30, 2022 USD ($)
Fair Value, Inputs, Level 1 [Member]
Fair Value Assets And Liabilities Measured On Recurring Basis [Line Items]
Transfers between fair value measure levels	$ 0
Fair Value, Inputs, Level 2 [Member]
Fair Value Assets And Liabilities Measured On Recurring Basis [Line Items]
Transfers between fair value measure levels	0
Fair Value, Inputs, Level 3 [Member]
Fair Value Assets And Liabilities Measured On Recurring Basis [Line Items]
Transfers between fair value measure levels	$ 0

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Accrued Expenses and Other Current Liabilities - Schedule of Accrued Expenses and Other Current Liabilities (Detail) - USD ($) $ in Thousands	Sep. 30, 2022	Dec. 31, 2021
Payables And Accruals [Abstract]
Accrued compensation and benefits	$ 1,648	$ 2,289
Accrued professional services	665	1,341
Other	8	74
Total accrued expenses and other current liabilities	$ 2,321	$ 3,704

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Debt - Additional Information (Detail) - Oxford Finance LLC And Horizon Technology Finance Corporation [Member]		3 Months Ended		9 Months Ended
	Jul. 02, 2019 USD ($) $ / shares shares	Sep. 30, 2022 USD ($)	Sep. 30, 2021 USD ($)	Sep. 30, 2022 USD ($) Payment	Sep. 30, 2021 USD ($)	Mar. 31, 2021 USD ($)	Sep. 30, 2020 USD ($)
Term Loan [Member]
Debt Instrument [Line Items]
Maximum borrowing capacity	$ 20,000,000
Proceeds from term loan	$ 10,000,000
Extended borrowing capacity						$ 10,000,000	$ 10,000,000
Interest rate description				The outstanding loan balance will accrue interest at the greater of (i) the rate of the one-month U.S. LIBOR rate plus 6.25% and (ii) 8.75%. The Loan Agreement provides for an interest only period until July 1, 2021, followed by thirty-six equal monthly payments of principal and interest continuing through June 1, 2024 (the “Maturity Date”).
Number of monthly payments | Payment				36
Debt instruments maturity date				Jun. 01, 2024
Debt instrument, effective rate	4.50%	11.34%		11.34%
Debt instrument, interest rate, stated percentage	8.75%
Term loan event of default description				Events of default include, among other things, the non-payment of principal, interest, and other amounts when due, breach of covenants, a material adverse change event, cross-default on material indebtedness, misrepresentations, material judgments, bankruptcy and delisting.
Term loan default interest rate	5.00%
Interest expense		$ 176,000	$ 270,000	$ 591,000	$ 824,000
Term Loan [Member] | Minimum [Member]
Debt Instrument [Line Items]
Debt instrument, percentage of pre payment fees	1.00%
Term Loan [Member] | Maximum [Member]
Debt Instrument [Line Items]
Debt instrument, percentage of pre payment fees	3.00%
Term Loan [Member] | LIBOR [Member]
Debt Instrument [Line Items]
Debt instrument, basis spread on variable rate	6.25%
Term A Loan [Member]
Debt Instrument [Line Items]
Debt Issuance Costs	$ 150,000
Number of securities called by warrants | shares	15,686
Exercise price of warrants | $ / shares	$ 12.75
Warrants maximum contractual term	10 years
Warrant, fair value	$ 136,000

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Debt - Schedule of Long-term Debt (Detail) - USD ($) $ in Thousands	Sep. 30, 2022	Dec. 31, 2021
Debt Disclosure [Abstract]
Notes payable, gross	$ 5,556	$ 8,055
Less: Unamortized debt discount and issuance costs	(46)	(93)
Accretion of final payment fee	422	339
Carrying value of notes payable	5,932	8,301
Less: Current portion of long-term debt	(3,306)	(3,295)
Long-term debt, net of issuance costs and discount	$ 2,626	$ 5,006

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Debt - Schedule of Estimated Future Principal Payments (Detail) $ in Thousands	Sep. 30, 2022 USD ($)
Debt Disclosure [Abstract]
2022	$ 833
2023	3,333
2024	1,389
Total principal payments	$ 5,555

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Stock-Based Compensation - Additional Information (Detail) - USD ($)	9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021	Jan. 01, 2022
2018 Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Percentage of shares issued on common stock outstanding	4.00%
Number of shares available for future grant	997,841		1,318,271
Stock option expiration period	10 years
2018 Plan | Maximum [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Exercise price per share of stock options as percentage of fair market value of common stock	100.00%
Plans
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock- based compensation expense	$ 2,493,000	$ 3,118,000
Plans | Options to Purchase Common Stock [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Shares, Granted	1,672,250	2,097,256
Stock- based compensation expense	$ 2,491,000	$ 2,812,000
Weighted-average grant-date fair values of options granted	$ 0.44	$ 4.13
Unrecognized compensation cost, recognized cost	2 years 3 months 18 days
Unrecognized compensation cost	$ 6,019,000
Option outstanding	5,390,188
Plans | Unvested Stock Options [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Option outstanding	2,931,063
Plans | Restricted Stock
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock- based compensation expense	$ 0	$ 132,000
Restricted stock, granted	0	0
Stock options outstanding, unvested	0
Plans | Restricted Stock Unit [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock- based compensation expense	$ 2,000	$ 174,000
Restricted stock, granted	0	5,939
Stock options outstanding, unvested	0
Plans | Maximum [Member] | Non Statutory Stock Option [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock option vesting period	4 years
Plans | Minimum [Member] | Non Statutory Stock Option [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock option vesting period	1 year

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Stock-Based Compensation - Schedule of Stock-Based Compensation Expense (Detail) - Plans - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	$ 2,493	$ 3,118
Research and Development Expense [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	872	1,051
General and Administrative Expense [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	$ 1,621	$ 2,067

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Stockholders' Equity - Additional Information (Detail) - Open Market Sale Agreement [Member] - USD ($)		9 Months Ended
	Nov. 15, 2019	Sep. 30, 2022	Sep. 30, 2021
Stockholders' Equity
Issuance of common stock, Shares		0	922,077
Weighted average price per share of stock issued			$ 5.70
Proceeds from issuance of common stock			$ 5,098,000
Common stock, aggregate offering price		$ 41,253,000
Jefferies LLC [Member]
Stockholders' Equity
Common stock, aggregate offering price	$ 50,000,000
Jefferies LLC [Member] | Maximum [Member]
Stockholders' Equity
Percentage of commissions and fees on selling shares	3.00%

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Revenue - Additional Information (Detail) - USD ($)	1 Months Ended	3 Months Ended			9 Months Ended
	Apr. 30, 2021	Sep. 30, 2022	Sep. 30, 2021	Jun. 30, 2021	Sep. 30, 2022	Sep. 30, 2021	Dec. 31, 2021
Deferred Revenue Arrangement [Line Items]
Revenue		$ 2,717,000	$ 2,120,000		$ 8,732,000	$ 3,383,000
Research and development fees		5,051,000	7,806,000		15,524,000	21,482,000
Current portion of deferred revenue		8,360,000			8,360,000		$ 10,639,000
Collaborative Arrangement [Member] | CANbridge [Member]
Deferred Revenue Arrangement [Line Items]
Deferred revenue	$ 10,878,000	8,493,000			8,493,000
Upfront Payment	10,000,000
Research and development expense	878,000
Research and development fees				$ 775,000	878,000
Deferred revenue recognized		2,335,000	1,685,000		7,608,000	2,255,000
Current portion of deferred revenue		7,401,000			7,401,000
Daiichi [Member]
Deferred Revenue Arrangement [Line Items]
Deferred revenue	2,000,000	959,000			959,000
Service [Member] | Daiichi [Member]
Deferred Revenue Arrangement [Line Items]
Revenue		$ 382,000	392,000		1,124,000	572,000
Upfront Payment					1,000,000
Research and development expense					1,000,000
Service [Member] | Takeda [Member]
Deferred Revenue Arrangement [Line Items]
Revenue			$ 43,000		$ 0	$ 556,000
Clinical Regulatory And Sales Relating To Gene Therapy Candidates For Fabry Disease And Pompe Diseases [Member] | Collaborative Arrangement [Member] | CANbridge [Member]
Deferred Revenue Arrangement [Line Items]
Milestone payment	542,000,000
Clinical, Regulatory and Sales For LB-001 [Member] | Collaborative Arrangement [Member] | CANbridge [Member]
Deferred Revenue Arrangement [Line Items]
Milestone payment	$ 49,000,000

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Loss Per Share - Computation of Basic and Diluted Net Loss Per Share (Detail) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended						9 Months Ended
	Sep. 30, 2022	Jun. 30, 2022	Mar. 31, 2022	Sep. 30, 2021	Jun. 30, 2021	Mar. 31, 2021	Sep. 30, 2022	Sep. 30, 2021
Numerator:
Net loss	$ (5,775)	$ (5,044)	$ (6,662)	$ (10,185)	$ (10,499)	$ (10,282)	$ (17,481)	$ (30,966)
Denominator:
Weighted-average common stock outstanding—basic	32,962,733			32,443,960			32,962,221	32,181,912
Weighted-average common stock outstanding—diluted	32,962,733			32,443,960			32,962,221	32,181,912
Net loss per share—basic	$ (0.18)			$ (0.31)			$ (0.53)	$ (0.96)
Net loss per share—diluted	$ (0.18)			$ (0.31)			$ (0.53)	$ (0.96)

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Loss Per Share - Computation of Potentially Anti-Dilutive Securities (Detail) - shares	3 Months Ended		9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021
Unvested Restricted Common Stock [Member]
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Potential dilutive securities excluded from computation of diluted net loss per common share		11,153		11,153
Unvested Restricted Stock Units [Member]
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Potential dilutive securities excluded from computation of diluted net loss per common share		5,939		5,939
Options to Purchase Common Stock [Member]
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Potential dilutive securities excluded from computation of diluted net loss per common share	5,390,188	4,508,640	5,390,188	4,508,640
Loan Warrants [Member]
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Potential dilutive securities excluded from computation of diluted net loss per common share	15,686	15,686	15,686	15,686

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Commitments and Contingencies - Additional Information (Detail) $ in Thousands	9 Months Ended
	Sep. 30, 2022 USD ($) ft² OperatingLease
Number of operating leases | OperatingLease	1
Massachusetts
Operating lease expiration date	Jul. 01, 2025
Operating lease commencement date	Apr. 01, 2020
Leased square feet | ft²	23,901
Payments for base rent	$ 1,494
Lease extend term	5 years
Letters of credit outstanding, amount	$ 622
Right-of-use asset obtained in exchange for operating lease liability	$ 6,428
Discount rate	7.60%

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Commitments and Contingencies - Summary of Lease Costs (Detail) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021	Dec. 31, 2021
Leases [Abstract]
Operating lease cost	$ 378	$ 378	$ 1,134	$ 1,134
Variable lease cost	231	208	669	639
Total lease cost	609	$ 586	1,803	1,773
Operating cash flows used for operating leases			1,166	$ 385
Operating lease liabilities — short-term	1,335		1,335		$ 1,227
Operating lease liabilities — long-term	$ 2,711		$ 2,711		$ 3,725
Weighted-average remaining lease term	2 years 9 months 18 days		2 years 9 months 18 days		3 years 6 months
Weighted-average discount rate	7.60%		7.60%		7.60%

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Commitments and Contingencies - Summary of Future Minimum Lease Payments (Detail) - USD ($) $ in Thousands	Sep. 30, 2022	Dec. 31, 2021
Leases [Abstract]
2022	$ 396	$ 1,562
2023	1,609	1,609
2024	1,656	1,656
2025	841	841
Thereafter	0	0
Total lease payments	4,502	5,668
Less: imputed interest	(456)	(716)
Total operating lease liabilities	$ 4,046	$ 4,952

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Related Parties - Additional Information (Detail) - Consulting Agreements [Member] - USD ($) $ in Thousands	3 Months Ended		6 Months Ended	9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021	Jun. 30, 2021	Sep. 30, 2022	Sep. 30, 2021
Related Party Transaction [Line Items]
Consulting agreement annual fee				$ 68
Consulting fee	$ 17	$ 17		$ 51	$ 51
Scientific Advisory Board [Member]
Related Party Transaction [Line Items]
Consulting fee			$ 5
Common Stock [Member] | Scientific Advisory Board [Member]
Related Party Transaction [Line Items]
Shares, Granted			5,000
Total Fair value			$ 13
Vesting period			2 years

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Subsequent Events - Additional Information (Detail) $ in Millions	Oct. 03, 2022 USD ($)
A&R CANbridge Agreement | Subsequent Event [Member]
Subsequent Event [Line Items]
Maximum eligible milestone payments to be received	$ 224.5