Collaborations | 9. 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 December 31, 2021, the Company’s accounts receivable were related to its collaborations with Regeneron and AvenCell . As of December 31, 2021 the Company's contract liabilities were related to its collaborations with Regeneron, AvenCell, SparingVision and Kyverna. As of December 31, 2020, the Company’s accounts receivable and contract liabilities were related to the Company’s collaboration with Regeneron. The following table presents changes in the Company’s accounts receivable and contract liabilities during the years ended December 31, 2021 and 2020 (in thousands): Balance at Additions Deductions Balance at End Year Ended December 31, 2021 Accounts receivable $ 2,130 $ 7,559 $ ( 7,658 ) $ 2,031 Contract liabilities: Deferred revenue $ 73,931 $ 84,659 $ ( 31,355 ) $ 127,235 Balance at Additions Deductions Balance at End Year Ended December 31, 2020 Accounts receivable $ 4,620 $ 103,116 $ ( 105,606 ) $ 2,130 Contract liabilities: Deferred revenue $ 28,810 $ 87,477 $ ( 42,356 ) $ 73,931 During the years ended December 31, 2021 and 2020, the Company recognized the following revenues as a result of changes in the contract liability balance (in thousands): Revenue recognized in the period from: Year Ended Year Ended Amounts included in the contract liability at the beginning of the period $ 22,544 $ 11,571 Costs to obtain and fulfill a contract The Company did no t incur any 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 (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”). On May 30, 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 collaboration was extended until April 2024, at which point Regeneron has an option to renew for an additional two years. The 2020 Regeneron Amendment also grants 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. Since December 31, 2020, there have been no material changes to the key terms of the 2016 Regeneron Agreement and the 2020 Regeneron Amendment (the “Amended Agreements”). For further information on the terms and conditions of these agreements, please see the notes to the consolidated financial statements included in the Company’s Annual Report for the year ended December 31, 2020. Revenue Recognition: Collaboration Revenue. Through December 31, 2021, excluding amounts allocated to Regeneron’s purchase of the Company’s common stock, the Company recorded $ 145.0 million in upfront payments under the Amended Agreements and $ 37.9 million for research and development services, primarily under the ATTR Co/Co agreement. Through December 31, 2021 , the Company has recognized $ 149.0 million of collaboration revenue under all arrangements, including $ 25.7 million, $ 53.0 million and $ 24.6 million of collaboration revenue in the years ended December 31, 2021, 2020 and 2019, respectively, in the consolidated statements of operations and comprehensive loss. This includes $ 5.9 million, $ 10.7 million, and $ 12.0 million, 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 $ 2.7 million in the year ended December 31, 2021 and $ 0 million in the years ended December 31, 2020 and 2019. As of December 31, 2021, there was approximately $ 51.4 million of the aggregate transaction price of the Amended Agreements remaining to be recognized, which the Company expects to be recognized during the research term through April 2024. As of December 31, 2021 and 2020, the Company had accounts receivable of $ 2.0 million and $ 2.1 million, respectively, and deferred revenue of $ 51.4 million and $ 73.9 million, respectively, related to the Amended Agreements. AvenCell Therapeutics, Inc. On July 30, 2021 (the “Effective Date”), the Company entered into two agreements with AvenCell, a privately held CAR-T cell therapy company formed on that date in a joint venture between the Company, Cellex and BXLS: (i) a license and collaboration agreement (the “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 will co-develop and co-commercialize allogeneic universal CAR-T cell products for an immuno-oncology indication. Scope: The Company granted AvenCell an exclusive license to combine the Company’s CRISPR/Cas9 technology platform with AvenCell’s switchable, universal CAR-T cell technology platform and made available to AvenCell certain know-how and materials. For an eighteen-month period after the Effective Date, the Company will provide to AvenCell any improvements with respect to the underlying technology that are developed. For the two-year period immediately following the Effective Date, the Company will perform certain activities, at the Company’s cost and expense, including providing to AvenCell certain know-how and materials to enable AvenCell to use the Company's CRISPR/Cas9 technology platform, as well as making available employees with requisite knowledge and experience to provide advice and answer questions regarding such know-how and materials for a limited number of hours per year (the “Knowledge Transfer Period”). In addition, the Company and AvenCell will collaborate on at least seven universal CAR-T ce ll products that combine the Company's allogeneic T cell technology with AvenCell's switchable, universal CAR-T cell technology, referred to as the (“Allo Collaboration”). AvenCell will pay the Company to provide supply and manufacturing services for them, including supplying GMP CRISPR reagents to support the research and development of all CRISPR Products (as defined in the LCA) under the Allo Collaboration until the completion of the first Pivotal Trial (as defined in the LCA) of the first such CRISPR Product. Financial Terms: In exchange for the license, the Company received a 33.33 % equity interest in AvenCell at the time of the initial closing and AvenCell is, therefore, considered to be a related party of the Company. Governance: The parties formed a joint steering committee (“JSC”), which is responsible for setting research objectives and overseeing the general strategies and research and development activities undertaken by the parties under the LCA. The JSC will meet quarterly until the expiration or termination of the Allo Collaboration. Term and Termination: The term of the Allo Collaboration is from the Effective Date of the LCA until the completion of all activities under the then-current Allo Collaboration with respect to all relevant CRISPR Products. The LCA contains termination provisions, including termination for insolvency, material breach, patent challenge, convenience, and cessation. Co-Development and Co-Promotion Agreement: Under the AvenCell Co/Co the parties will co-develop and co-commercialize in the U.S. and key European countries certain allogeneic universal CAR-T products directed to an immuno-oncology target. The Company is the lead commercialization party in the U.S., and AvenCell is the lead commercialization party in the European countries. The parties will share equally in the profits and development costs. The Company will have one additional option to enter into a second co-development and co-funding agreement from selected allogeneic universal CAR-T cell therapy products that the parties intend to develop under the Allo Collaboration for a payment of $ 30.0 million to AvenCell. AvenCell LCA - Accounting Analysis: The Company concluded that the accounting treatment for the LCA is within the scope of ASC 606 . The Company evaluated the promised goods and services under the LCA and determined that it included one performance obligation: a combined performance obligation including the license to the allogeneic technology, initial know-how and ongoing support services, including participation in the JSC during the two-year Knowledge Transfer Period. The transaction price was determined to be $ 62.9 million, which represents the fair value of the Company's equity interest in AvenCell as of the Effective Date. The Company allocated the full transaction price to the combined performance obligation including the license to allogeneic technology, the JSC, initial-know-how and ongoing support services. The Company will recognize the $62.9 million using a time elapsed input method over the Knowledge Transfer Period, which in management’s judgement is the best measure of progress towards satisfying the performance obligation as this method provides the most faithful depiction of the entity’s performance in transferring control of the goods and services promised to AvenCell. This represents the Company’s best estimate of the obligation, as after this period AvenCell will be able to fully benefit from the licensed IP on its own or with readily available resources. Revenue recorded during each period will be eliminated in part by an amount representing the Company's 33.33 % ownership interest in AvenCell at that time, as this represents the intra-entity profit related to the transaction. The Company will re-evaluate the measure of progress in each reporting period and, if necessary, adjust the measure of performance and related revenue recognition. The Company completed the initial transfer of know-how in the third quarter of 2021. The Company recognized $ 5.9 million in revenue related to the LCA for the year ended December 31, 2021 after eliminating $ 2.9 million 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, the $ 2.9 million of revenue is indefinitely deferred and excluded from the results of operations of the Company. As of December 31, 2021 the Company had deferred revenue of $ 54.1 million related to the AvenCell LCA, which the Company expects to recognize through July 2023 . The payments attributable to the supply and manufacturing services are variable and are commensurate with the standalone selling prices of the services, and as such, will be attributed to those services. The Company did not record any consideration related to the supply and manufacturing services in 2021. AvenCell Co/Co - Accounting Analysis: The Company concluded that the AvenCell Co/Co agreement meets 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 AvenCell Co/Co as a component of revenues in the consolidated statements of operations and comprehensive loss, to the extent that this does not result in a cumulative “negative revenue” amount, in which case the cumulative shortfall would be reclassified as an expense. The Company recognized $ 0.2 million in revenues related to the AvenCell Co/Co agreement for the year ended December 31, 2021. 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. Scope: 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-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. Financial Terms: In exchange for the license, the Company received 83,316 shares of Series A2 Preferred Stock (“Series A2”) which represented an equity ownership of approximately 11 % at the time of closing. 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 million per product) as well as royalties on potential future sales of products arising from the collaboration. Governance : The parties formed a JSC, which is responsible for monitoring and managing the collaboration prior to program completion. 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 the 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. See Note 10 for the determination of the fair value of the Company’s investment. The Company allocated the full transaction price to the combined performance obligation. 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. These costs will be recorded as revenue when the expenses are incurred. There was no revenue recognized in the year ended December 31, 2021 related to the SparingVision LCA. As of December 31, 2021, the Company had deferred revenue of $ 14.8 million, which is expected to be recognized over a three to five year period. 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 licensing 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. Scope: The Company granted Kyverna rights to its proprietary ex vivo CRISPR/Cas9-based allogeneic platform for the development of KYV-201, an allogeneic CD19 CAR-T cell investigational candidate for the treatment of select autoimmune diseases. This is a novel approach aimed at targeting CD19 for inflammatory diseases as compared to traditional oncology indications. Kyverna will lead and fund preclinical and clinical development for KYV-201. The Company will have an option to lead U.S. commercialization for KYV-201 under a co-development and co-commercialization agreement. If the Company chooses to co-develop and co-commercialize KYV-201, it will pay an opt-in fee of $ 5.0 million and share in 50 % of development costs and future net profit and/or loss arising from commercializing KYV-201 in the U.S. Kyverna retains all rights outside of the U.S. , and the Company will receive low-to-mid-single-digit royalties on net sales generated outside of the U.S. Kyverna is considered to be a related party, as they have a board member in common with the Company. Financial Terms: In exchange for the license, the Company received an equity ownership of approximately 7% in Kyverna at the time of closing. The Company will be eligible to receive certain development and commercial milestone payments, as well as low-to-mid-single-digit royalties on potential future sales of KYV-201. Kyverna LCA – Accounting Analysis: The Company determined that the accounting for the Kyverna 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 related to the transfer of the license related to the allogeneic platform technology, a technology transfer, and other supply and research and development activities. The transaction price was determined to be $ 7.0 million, which represents the fair value of the Company's equity interest in Kyverna at the time of closing. See Note 10 for the determination of the fair value of the Company's investment. The Company allocated the full transaction price to the combined performance obligation. Revenue will be recognized under a time-elapsed input model starting at the completion of the technology transfer, which in management's judgment is the best measure of progress towards satisfying the performance obligation. Progress will be measured and reassessed quarterly. There was no revenue recognized in the year ended December 31, 2021 related to the Kyverna LCA. As of December 31, 2021, the Company had deferred revenue of $ 7.0 million which is expected to be recognized over a nine to twelve month period. Novartis Institutes for BioMedical Research, Inc. In December 2014, the Company entered into a strategic collaboration agreement with Novartis (the “2014 Novartis Agreement”), primarily focused on the research of new ex vivo CRISPR/Cas9-edited therapies using CAR-T cells and hematopoietic stem cells (“HSCs”). The agreement was amended in December 2018 (the “Novartis Amendment”) to also include research on ocular stem cells (“OSCs”). In December 2019, per the terms of the 2014 Novartis Agreement, the research term ended, although the 2014 Novartis Agreement remains in effect, for which the Company will be eligible to receive milestone and royalty payments in the future. In June 2021, the Company entered into Amendment No. 3 (the “Amendment”) to the 2014 Novartis Agreement. The Amendment amends Novartis’ rights with respect to all of the CAR-T Therapeutic Targets (as defined in the 2014 Novartis Agreement) that Novartis selected under the 2014 Novartis Agreement, including (a) making Novartis’ license non-exclusive for such CAR-T Therapeutic Targets, (b) removing Novartis’ diligence and related reporting obligations for such CAR-T Therapeutic Targets, and (c) refining the scope of Novartis’ sublicense rights for such CAR-T Therapeutic Targets. The Company made a one-time payment to Novartis of $ 10.0 million within 30 days after the effective date of the Amendment, which was recorded as research and development expense in the consolidated statement of operations and comprehensive loss for the year ended December 31, 2021. Since December 31, 2020, there have been no other material changes to the key terms of the 2014 Novartis Agreement and the Novartis Amendment. For further information on the terms and conditions of these agreements, please see the notes to the consolidated financial statements included in the Company’s Annual Report for the year ended December 31, 2020. Revenue Recognition – Collaboration Revenue. Through December 31, 2021, excluding amounts allocated to Novartis’ purchase of the Company’s Class A-1 and Class A-2 Preferred Units, the Company had recorded a total of $62.4 million in cash under the 2014 Novartis Agreement and the Novartis Amendment. Through December 31, 2021, the Company recognized $ 62.4 million of collaboration revenue. No revenue was recognized during the years ended December 31, 2021 or 2020 related to the 2014 Novartis Agreement and the Novartis Amendment. The Company recognized $ 18.5 million during the year ended December 31, 2019, in the consolidated statement of operations and comprehensive loss, related to the 2014 Novartis Agreement and the Novartis Amendment. As of December 31, 2019, the aggregate transaction price had been recognized in full. Revenue Recognition – Milestone . In March 2020, the U.S. Food and Drug Administration (“FDA”) accepted the Investigational New Drug ( “ IND ” ) application submitted by Novartis for a CRISPR/Cas9-based engineered cell therapy for the treatment of sickle cell disease. As a result of meeting this milestone, the Company recognized $ 5.0 million as collaboration revenue within the consolidated statement of operations and comprehensive loss. In September 2021, an additional milestone was reached and, as a result, the Company recognized $ 0.3 million as collaboration revenue within the consolidated statement of operations and comprehensive loss. No other milestones under the 2014 Novartis Agreement and the Novartis Amendment were achieved during the years ended December 31, 2021, 2020 or 2019. The Company is eligible to receive additional downstream success-based milestones and royalties. As of December 31, 2021 and 2020, the Company had no accounts receivable or deferred revenue related to the 2014 Novartis Agreement and the Novartis Amendment. |