Collaborations and Other Arrangements |
9 Months Ended | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Sep. 30, 2023 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Collaborations and Other Arrangements | 7. Collaborations and Other Arrangements To accelerate the development and commercialization of CRISPR/Cas9-based 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 September 30, 2023, the Company’s accounts receivable were related to its collaborations with Regeneron Pharmaceuticals, Inc. (“Regeneron”), SparingVision SAS (“SparingVision”), Kyverna Therapeutics, Inc. (“Kyverna”), AvenCell Therapeutics, Inc. (“AvenCell”) and ONK Therapeutics, Ltd. (“ONK”), and the Company’s contract liabilities were related to its collaborations with Regeneron and SparingVision. As of December 31, 2022, the Company’s accounts receivable were related to its collaborations with Regeneron, AvenCell, SparingVision and ONK, and the Company’s contract liabilities were related to its collaborations with Regeneron, AvenCell, SparingVision and Kyverna. The following table presents changes in the Company’s accounts receivable and contract liabilities during the nine months ended September 30, 2023 and 2022 (in thousands):
During the nine months ended September 30, 2023 and 2022, the Company recognized the following revenues as a result of changes in the contract liability balance (in thousands):
Costs to obtain and fulfill a contract 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. License and Collaboration Agreement 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 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. 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, until April 2026, in exchange for a nonrefundable payment of $30 million due in April 2024. 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. Under the terms of the expanded research collaboration, the companies will initially research two in vivo non-liver targets. Intellia will lead the design of the editing methodology and Regeneron will lead the design of the targeted viral vector delivery approach and the parties will share research costs equally. 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. Since December 31, 2022, there have been no material changes to the key terms of the 2016 Regeneron Agreement, ATTR Co/Co or Hemophilia Co/Co (the “Regeneron Agreements”), other than as described above. Revenue Recognition – Collaboration Revenue. Through September 30, 2023, excluding amounts allocated to Regeneron’s purchase of the Company’s common stock, the Company recorded $145.0 million in upfront payments under the Regeneron Agreements and $46.1 million for research and development services, primarily under the ATTR Co/Co agreement. Through September 30, 2023, the Company has recognized $196.5 million of collaboration revenue under all arrangements, including $9.3 million and $23.5 million during the three and nine months ended September 30, 2023, respectively, and $6.5 million and $18.6 million during the three and nine months ended September 30, 2022, respectively, in the condensed consolidated statements of operations and comprehensive loss. This includes $5.5 million and $14.2 million during the three and nine months ended September 30, 2023 and $4.0 million and $8.7 million during the three and nine months ended September 30, 2022, respectively, primarily representing payments due from Regeneron pursuant to the ATTR Co/Co agreement. These revenues are offset in part by contra-revenue related to the Hemophilia Co/Co agreements amounting to approximately $1.8 million and $7.6 million during the three and nine months ended September 30, 2023 and $3.2 million and $6.9 million during the three and nine months ended September 30, 2022, respectively. As of September 30, 2023, there was approximately $12.0 million of the aggregate transaction price remaining to be recognized, which the Company has been recognizing through April 2024 in accordance with the terms of the 2020 Regeneron Amendment. As of September 30, 2023 and December 31, 2022, the Company had accounts receivable of $5.5 million and $3.2 million, respectively, and deferred revenue of $12.0 million and $28.8 million, respectively, related to Regeneron Agreements. AvenCell Therapeutics, Inc. In July 2021, the Company entered into two agreements with AvenCell, a privately held chimeric antigen receptor T (“CAR-T”) cell therapy company formed on that date in a joint venture between the Company, Cellex Cell Professionals GmbH (“Cellex”) and funds managed by Blackstone Life Sciences Advisors L.L.C. (“BXLS”): (i) a license and collaboration agreement (the “AvenCell LCA”), under which the Company will collaborate to develop allogeneic universal CAR-T cell therapies and which granted AvenCell a license to develop and commercialize genome edited universal CAR-T cell therapies (limited to its use with their switchable, universal CAR-T cell UniCAR and RevCAR platforms); and (ii) a co-development and co-funding agreement (the “AvenCell Co/Co”), under which the Company would co-develop and co-commercialize allogeneic universal CAR-T cell products for an immuno-oncology indication. In November 2022, the Company decided to re-prioritize its ex vivo programs and terminated the AvenCell Co/Co, effectively turning over control of the program to AvenCell. The Company’s obligations under the terminated agreement were completed in the second quarter of 2023. The Company also has one option to enter into an additional co-development and co-funding agreement for a payment of $30.0 million to AvenCell. Since December 31, 2022, there have been no other material changes to the key terms of the AvenCell LCA and AvenCell Co/Co agreements. Revenue Recognition – Collaboration Revenue. The Company recognized $1.9 million and $13.2 million in revenue related to the AvenCell LCA for the three and nine months ended September 30, 2023, respectively, after eliminating $1.0 million and $6.6 million during those respective periods in intra-entity profits, which will be deferred and recognized if and when AvenCell commercializes a product with the Company’s license or abandons the related project. Until such time, this revenue is indefinitely deferred and excluded from the results of operations of the Company. The Company recognized no revenue in the three months ended September 30, 2023 and $0.6 million in contra-revenue in the nine months ended September 30, 2023 related to the AvenCell Co/Co agreement. The Company recognized $1.3 million and $1.4 million in contra-revenue in the three and nine months ended September 30, 2022, respectively, related to the AvenCell Co/Co agreement. As of September 30, 2023, there was no remaining transaction price of the AvenCell LCA remaining to be recognized. The Company had $9.0 thousand in accounts receivable and no deferred revenue related to the AvenCell agreements as of September 30, 2023. As of December 31, 2022, the Company had $0.3 million in accounts receivable and deferred revenue of $19.9 million related to the AvenCell 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. Since December 31, 2022, there have been no material changes to the key terms of the SparingVision LCA agreement. Revenue Recognition – Collaboration Revenue. The Company recognized $0.4 million and $1.3 million in revenue related to the SparingVision LCA for the three and nine months ended September 30, 2023, respectively. The Company did not recognize collaboration revenue in the three and nine months ended September 30, 2022 related to the SparingVision LCA. As of September 30, 2023 and December 31, 2022, the Company had $0.7 million and $0.1 million in accounts receivable, respectively, related to the SparingVision LCA. As of September 30, 2023 and December 31, 2022, the Company had deferred revenue of $14.1 million and $14.7 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. Kyverna Therapeutics, Inc. In December 2021, the Company and Kyverna, a cell therapy company engineering a new class of therapies for autoimmune and inflammatory diseases, entered into a license and collaboration agreement (the “Kyverna LCA”), for the development of an allogeneic CD19 CAR-T cell therapy for the treatment of a variety of B cell-mediated autoimmune diseases. Since December 31, 2022, there have been no material changes to the key terms of the Kyverna LCA agreement. Revenue Recognition – Collaboration Revenue. The Company had recognized revenue from the Kyverna LCA in full as of March 31, 2023, including $0.4 million in revenue recorded in 2023. The Company recognized approximately $0.1 million in revenue in the three and nine months ended September 30, 2023 related to materials shipped to Kyverna. The Company recognized $2.3 million and $4.3 million in collaboration revenue for the three and nine months ended September 30, 2022, respectively, related to the Kyverna LCA. As of September 30, 2023, the Company had $0.1 million in accounts receivable related to the shipments made to Kyverna. As of December 31, 2022, the Company had no accounts receivable and deferred revenue of $0.4 million related to Kyverna. ONK Therapeutics, Ltd. On February 12, 2022, the Company entered into a license, collaboration and option agreement (the “ONK LCA”) with ONK, an innovative company dedicated to developing optimally engineered natural killer (“NK”) cell therapies to cure patients with cancer. Since December 31, 2022, there have been no material changes to the key terms of the ONK LCA agreement. Revenue Recognition – Collaboration Revenue. The Company recognized $0.2 million in revenue for each of the three and nine month periods ended September 30, 2023 related to materials shipped in accordance with the ONK LCA. There was no revenue recognized in the three and nine months ended September 30, 2022 related to the ONK LCA. The Company had $0.2 million and $0.1 million in accounts receivable related to the ONK LCA at September 30, 2023 and December 31, 2022, respectively. |