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Collaborations and Other Arrangements
12 Months Ended
Dec. 31, 2024
Organization, Consolidation and Presentation of Financial Statements [Abstract]  
Collaborations and Other Arrangements

9. Collaborations and Other Arrangements

To accelerate the development and commercialization of gene editing products in multiple therapeutic areas, the Company has formed, and intends to seek other opportunities to form, strategic alliances with collaborators who can augment its leadership in CRISPR/Cas9 therapeutic development. As of December 31, 2024, the Company’s accounts receivable were related to its collaborations with Regeneron, AvenCell, SparingVision and ReCode, and the Company’s contract liabilities were related to its collaborations with Regeneron and SparingVision. As of December 31, 2023, the Company’s accounts receivable were related to its collaborations with Regeneron, SparingVision, AvenCell and Kyverna and the Company’s contract liabilities were related to its collaborations with Regeneron and SparingVision.

The following table presents changes in the Company’s accounts receivable and contract liabilities (in thousands):

 

 

Balance at
Beginning of
Period

 

 

Additions

 

 

Deductions

 

 

Balance at End
of Period

 

Year ended December 31, 2024

 

 

 

 

 

 

 

 

 

 

 

 

Accounts receivable

 

$

36,456

 

 

$

26,524

 

 

$

(54,463

)

 

$

8,517

 

Contract liabilities - deferred revenue

 

$

60,993

 

 

$

-

 

 

$

(22,076

)

 

$

38,917

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Balance at
Beginning of
Period

 

 

Additions

 

 

Deductions

 

 

Balance at End
of Period

 

Year ended December 31, 2023

 

 

 

 

 

 

 

 

 

 

 

 

Accounts receivable

 

$

3,768

 

 

$

51,421

 

 

$

(18,733

)

 

$

36,456

 

Contract liabilities - deferred revenue

 

$

63,771

 

 

$

40,312

 

 

$

(43,090

)

 

$

60,993

 

 

The Company recognized the following revenues as a result of changes in the contract liability balance (in thousands):

 

 

Year Ended December 31,

 

Revenue recognized in the period from:

 

2024

 

 

2023

 

 

2022

 

Amounts included in the contract liability at the beginning of the
      period

 

$

22,076

 

 

$

23,462

 

 

$

52,060

 

The Company has not incurred significant expenses to obtain collaboration agreements and costs to fulfill those contracts do not generate or enhance resources of the Company. As such, no costs to obtain or fulfill a contract have been capitalized in any period.

Regeneron Pharmaceuticals, Inc.

In April 2016, the Company entered into a license and collaboration agreement with Regeneron (as amended from time to time, the “2016 Regeneron Agreement”). The 2016 Regeneron Agreement has two principal components: i) a product development component under which the parties will research, develop and commercialize CRISPR/Cas-based therapeutic products primarily focused on genome editing in the liver, and ii) a technology collaboration component, pursuant to which the Company and Regeneron will engage in research-related activities aimed at discovering and developing novel technologies and improvements to CRISPR/Cas technology to enhance the Company’s genome editing platform. Under this agreement, the Company also may access the Regeneron Genetics Center and proprietary mouse models to be provided by Regeneron for a limited number of the Company’s liver programs. At the inception of the 2016 Regeneron Agreement, Regeneron selected the first of its 10 targets, transthyretin (“ATTR”) amyloidosis, which is subject to a co-development and co-promotion agreement between the Company and Regeneron (the “ATTR Co/Co”).

In connection with the 2016 Regeneron Agreement, the Company received a nonrefundable upfront payment of $75.0 million. In addition, on Regeneron programs that are not subject to Co/Co agreements, the Company may be eligible to earn, on a per-licensed target basis, (i) up to $25.0 million in development milestones, including for the dosing of the first patient in each of Phase I, Phase II and Phase III clinical trials, (ii) up to $110.0 million in regulatory milestones, including for the acceptance of a regulatory filing in the U.S., and for obtaining regulatory approval in the U.S. and in certain other identified countries, and (iii) up to $185.0 million in sales-based milestone payments. The Company is also eligible to earn royalties ranging from the high-single digits to low teens, in each case, on a per-product basis, which royalties are potentially subject to various reductions and offsets and incorporate the Company’s existing low- to mid-single-digit royalty obligations under a license agreement with Caribou. In connection with the 2016 Regeneron Agreement, Regeneron purchased $50.0 million of the Company’s common stock in a private placement under a stock purchase agreement concurrent with the Company’s IPO.

In May 2020, the Company entered into (i) amendment no. 1 (the “2020 Regeneron Amendment”) to the 2016 Regeneron Agreement, (ii) co-development and co-funding agreements for the treatment of hemophilia A and hemophilia B (the “Hemophilia Co/Co”) agreements and (iii) a stock purchase agreement. The collaboration expansion builds upon the jointly developed targeted transgene insertion capabilities designed to durably restore missing therapeutic protein, and to overcome the limitations of traditional gene therapy. The technology collaboration was extended until April 2024, at which point Regeneron would have an option to renew for an additional two years. The 2020 Regeneron Amendment also granted Regeneron exclusive rights to develop products for five additional in vivo CRISPR/Cas-based therapeutic liver targets and non-exclusive rights to independently develop and commercialize up to 10 ex vivo gene edited products made using certain defined cell types.

As part of the consideration for the 2020 Regeneron Amendment, Regeneron paid the Company an upfront payment of $70.0 million, which included the $25.0 million fee to extend the Technology Collaboration Term, as defined in the 2016 Regeneron Agreement, to April 2024. The potential future milestones and royalties remain unchanged from the 2016 Regeneron Agreement. In addition, on May 30, 2020, the Company and Regeneron entered into the 2020 Stock Purchase Agreement. Under the 2020 Stock Purchase Agreement, the Company sold to Regeneron 925,218 shares of its common stock, par value $0.0001 per share, for aggregate cash consideration of $30.0 million, or $32.42 per share (the “Equity Transaction”), representing a 100% premium over the volume-weighted average trading price of the Company’s common stock during the 30-day period prior to the closing of the Equity Transaction. Under the 2020 Stock Purchase Agreement, Regeneron will not dispose of any shares of common stock it beneficially owns in the Company until the termination of the Technology Collaboration Term.

In October 2023, Regeneron notified the Company that it was exercising its one-time option to extend the Technology Collaboration Term for an additional two years (the “2024 Technology Collaboration Extension”), until April 2026, in exchange for a nonrefundable payment of $30.0 million that was paid in April 2024.

2024 Technology Collaboration Extension: Accounting Analysis. The 2024 Technology Collaboration Extension was accounted for as a contract modification. The promised goods and services under the 2024 Technology Collaboration Extension are not

distinct from the combined performance obligations identified in the 2020 Regeneron Amendment, which was only partially satisfied at the date of option exercise. A cumulative catch-up adjustment was recorded during the fourth quarter of 2023 resulting in a charge of $10.3 million against revenue previously recognized.

The transaction price of the 2024 Technology Collaboration Extension was determined to be $51.7 million, which is comprised of the $11.4 million remaining consideration under the 2020 Regeneron Amendment as of the modification date, the $30.0 million extension fee and the $10.3 million cumulative catch-up adjustment. The $51.7 million transaction price was allocated to the performance obligations including the licenses to targets and associated research activities and evaluation plans and the combined performance obligation including the technology collaboration and associated research activities, on a relative standalone selling price basis.

As a result of this evaluation, the Company allocated $48.3 million to the combined performance obligation including the licenses to targets and associated research activities and evaluation plans and $3.4 million to the combined performance obligation including the technology collaboration and associated research activities, which are being recognized using a time elapsed inputs method from the October 2023 extension date through April 2026, the remaining period of the collaboration.

 

The Company recognized $20.7 million, $11.7 million and $22.5 million of collaboration revenue in the years ended December 31, 2024, 2023 and 2022, respectively, in the consolidated statements of operations and comprehensive loss related to the 2016 Regeneron Agreement, the 2020 Regeneron Amendment and the 2024 Technology Collaboration Extension.

As of December 31, 2024, there was approximately $26.4 million of the aggregate transaction price remaining to be recognized that will be recognized through April 2026, the remaining period of the collaboration.

In September 2023, Regeneron and Intellia further expanded the research collaboration (the “2023 Regeneron Amendment”) to develop additional in vivo CRISPR-based gene editing therapies focused on neurological and muscular diseases. The collaboration will leverage Intellia’s proprietary Nme2 CRISPR/Cas9 genome editing systems adapted for viral vector delivery and designed to precisely modify a target gene and Regeneron’s proprietary antibody-targeted adeno-associated virus vectors and delivery systems; each party will have the opportunity to lead potential development and commercialization for one product candidate, and the party that is not leading development and commercialization will have the option to enter into a co-development and co-promotion agreement for the target.

2023 Regeneron Amendment: Accounting Analysis. The Company concluded that the accounting for the 2023 Regeneron Amendment is within the scope of ASC 606. The Company identified one performance obligation, the transfer of the license and performance of collaborative research and development activities. There is no upfront consideration related to the 2023 Regeneron Amendment. As the 2023 Regeneron Amendment progresses, the Company and Regeneron will share research costs equally. Any cost reimbursements received from Regeneron will be recorded as a component of revenue and any payments made to Regeneron will be recorded as a reduction of revenue.

The Company recognized $2.3 million and $0.4 million of collaboration revenue in the years ended December 31, 2024 and 2023, respectively, in the consolidated statement of operations and comprehensive loss related to the 2023 Regeneron Amendment.

ATTR and Hemophilia Co/Co Agreements: Accounting Analysis. The Company concluded that the ATTR Co/Co and Hemophilia Co/Co agreements meet the definition of a collaborative arrangement per ASC 808, which is outside of the scope of ASC 606. Since ASC 808 does not provide recognition and measurement guidance for collaborative arrangements, the Company has analogized to ASC 606. As such, the Company classifies cumulative amounts paid or received under the cost sharing provisions of the ATTR Co/Co and the Hemophilia Co/Co agreements as a component of revenues in the consolidated statements of operations and comprehensive loss. The Company terminated the hemophilia B Co/Co agreement in September 2024. The Company will continue to support Regeneron with the development of gene editing products directed to hemophilia B, as applicable, under the 2016 Regeneron Agreement.

The Company recognized $21.9 million, $19.6 million, and $11.9 million, respectively, primarily representing payments due from Regeneron pursuant to the ATTR Co/Co agreement, in the years ended December 31, 2024, 2023 and 2022. The Company recognized contra-revenue related to the Hemophilia Co/Co agreements amounting to approximately $11.7 million in the year ended December 31, 2024, $10.7 million in the year ended December 31, 2023 and $10.4 million in the year ended December 31, 2022.

As of December 31, 2024 and December 31, 2023, the Company had accounts receivable of $7.2 million and $35.7 million, respectively, and deferred revenue of $26.4 million and $47.1 million, respectively, related to the Regeneron Agreements.

SparingVision SAS

In October 2021, the Company and SparingVision, a genomic medicine company developing vision saving treatments for ocular diseases, entered into a license and collaboration agreement (the “SparingVision LCA”) to develop novel genomic medicines utilizing CRISPR/Cas9 technology for the treatment of ocular diseases.

The Company granted SparingVision exclusive rights to its proprietary in vivo CRISPR/Cas9-based genome editing technology for up to three ocular targets addressing diseases with significant unmet medical need. In addition, the parties will research and develop novel self-inactivating adeno-associated virus (“AAV”) vectors and lipid nanoparticle (“LNP”)-based approaches to address delivery of CRISPR/Cas9 genome editing reagents to the retina.

 

SparingVision will lead and fund the preclinical and clinical development for the genome editing product candidates pursued under the collaboration.

 

The Company will have an option to obtain exclusive U.S. commercialization rights for product candidates arising from two of three collaboration targets. For product candidates the Company chooses to option, it will pay an opt-in fee between $10.0 million and $20.0 million depending on the stage of development of the target, reimburse certain costs, share in 50% of development costs and pay royalties to SparingVision on U.S. sales.

In exchange for the license, the Company received 83,316 shares of SparingVision’s Series A2 Preferred Stock (“Series A2”). Attached to each share of Series A2, the Company received three warrants for the right to purchase additional Series A2 shares at designated prices that are subject to certain vesting conditions. The Company will also be eligible to receive certain research, development and commercial milestone payments (up to approximately $200.0 million per product) as well as royalties on potential future sales of products arising from the collaboration.

SparingVision LCA: Accounting Analysis. The Company determined that the accounting for the SparingVision LCA is within the scope of ASC 606. The Company evaluated the promised goods and services and determined that it included one performance obligation: a combined performance obligation including the license to the CRISPR technology as well as ongoing research and support services, including participation in a joint steering committee (“JSC”).

The transaction price was determined to be $14.8 million, which represents the fair value of the Company's equity interest in SparingVision at the time of closing. The Company allocated the full transaction price to the combined performance obligation, which was recorded as deferred revenue upon execution of the agreement. The Company will use a costs-incurred input method to recognize revenue, measuring the progress of the programs based on the costs incurred against budget, which in management's judgment is the best measure of progress towards satisfying the performance obligation.

Effective November 2024, SparingVision provided notice to the Company to terminate one of their three ocular targets due to a reprioritization strategy.

The Company recognized $2.5 million, $1.8 million and $0.2 million in revenue related to the SparingVision LCA for the years ended December 31, 2024, 2023 and 2022, respectively. As of December 31, 2024 and December 31, 2023, the Company had $0.6 million and $0.5 million in accounts receivable, respectively, related to the SparingVision LCA. As of December 31, 2024 and December 31, 2023, the Company had deferred revenue of $12.5 million and $13.9 million, respectively, related to the SparingVision LCA, which is expected to be recognized over a six to nine year period from the signing of the agreement.

ReCode Therapeutics, Inc. (“ReCode”)

On February 14, 2024, the Company entered into a license, collaboration and option agreement with ReCode (the “ReCode LCA”), a clinical-stage genetic medicines company, to develop novel genomic medicines for the treatment of cystic fibrosis (“CF”). The ReCode LCA leverages the Company’s proprietary CRISPR-based gene editing platform, including its deoxyribonucleic acid (“DNA”) writing technology, and ReCode’s proprietary Selective Organ Targeting (“SORT”) LNP delivery platform to precisely correct one or more CF disease-causing gene mutations. As part of the agreement, the companies will focus initial research efforts on therapeutic approaches that address CF for patients who have limited or no treatment options available, with the opportunity to expand the scope of the collaboration in later phases. The Company will be responsible for the design of the editing strategy and research-grade components for the investigational therapies. ReCode will lead the subsequent preclinical and clinical development and worldwide commercialization for certain programs arising from the collaboration. The Company also has an option to lead commercialization in the U.S. for certain programs (the “Co/Co option”).

The ReCode LCA did not include an exchange of upfront consideration between the parties. The Company will be eligible to receive pre-specified development and commercial milestone payments, up to $262.0 million per product, as well as single digit

royalties on potential sales. Certain milestone and royalty payments may be removed or reduced for a product if the Company exercises the Co/Co option. The Company is entitled to cost reimbursements for certain research activities, which will be recorded as revenue. The Company did not recognize material revenue from the ReCode LCA during the year ended December 31, 2024.

Other Agreements

The Company has existing license and collaboration agreements with AvenCell, Kyverna, and ONK Therapeutics, Ltd. (“ONK”). Since December 31, 2023, there have been no material changes to the key terms of the AvenCell, Kyverna and ONK license and collaboration agreements. For further information on the terms and conditions of these agreements, see the notes to the consolidated financial statements included in the Company’s Annual Report for the year ended December 31, 2023.

During the year ended December 31, 2024, the Company recognized $21.0 million of previously eliminated intra-entity profit related to its license and collaboration agreement with AvenCell (the “AvenCell LCA”) in the consolidated statements of operations and comprehensive loss. During the years ended December 31, 2023 and 2022, the Company recognized $13.2 million and $22.8 million in revenue related to the AvenCell LCA, after eliminating $6.6 million and $11.4 million in intra-entity profits during those respective periods. The eliminated revenue was deferred and excluded from the results of operations of the Company until the first quarter of 2024. The Company did not recognize material revenue related to materials shipments under the AvenCell LCA during the years ended December 31, 2024, 2023 and 2022 and did not recognize material contra-revenue under the AvenCell Co/Co agreement during the years ended December 31, 2024 and 2023. The Company recognized $2.0 million of contra-revenue during the year ended December 31, 2022. The Company had no material accounts receivable from AvenCell or accrued expenses related to AvenCell agreements as of December 31, 2024 and 2023.

The Company did not recognize material revenue from Kyverna during the years ended December 31, 2024 and 2023 and recognized $6.6 million during the year ended December 31, 2022. The Company did not recognize material revenue from ONK during the years ended December 31, 2024, 2023 and 2022.