Collaboration and License Agreements | 9. Collaboration and license agreements Vertex Agreement Summary of Agreement In July 2022, the Company entered into a Strategic Collaboration and License Agreement (the "Vertex Agreement") with Vertex Pharmaceuticals Incorporated ("Vertex") for an exclusive, four-year worldwide research collaboration focused on developing in vivo gene editing candidates toward an undisclosed target for the treatment of a single liver disease. Additionally, in connection with the execution of the Vertex Agreement, the Company entered into a stock purchase agreement (the "Vertex Stock Purchase Agreement") with Vertex, pursuant to which the Company sold 1,519,756 shares of its common stock to Vertex at a price of $ 23.03 per share, for an aggregate purchase price of $ 35.0 million. Pursuant to the Vertex Agreement, the Company is responsible for discovery, research and certain preclinical development of novel in vivo gene editing development candidates for the target of interest. The Company’s research activities are focused on (i) identifying and engineering specific gene editing systems and in vivo delivery systems directed to the target and (ii) evaluating and optimizing development candidates to achieve criteria specified in the Vertex Agreement. Vertex is obligated to reimburse the Company’s research expenses consistent with a mutually agreed-upon research plan and budget (“Vertex Research Plan”). The research term has an initial term of four years and may be extended by Vertex for up to one additional year (“Vertex Research Term”). The Vertex Research Plan is overseen by a Joint Research Committee (“Vertex JRC”) as detailed in the Vertex Agreement. Any material amendments to the Vertex Research Plan are required to be mutually agreed to by the Vertex JRC. During the Vertex Research Term, Vertex may select certain gene editing systems and in vivo delivery systems directed at the target to become a licensed agent. Upon the designation of the licensed agent, Vertex shall receive a license to exploit the licensed agent, and the licensed agent will continue to be developed under the Vertex Research Plan in order to achieve certain development candidate criteria agreed to by the Vertex JRC. Following the Vertex Research Term, Vertex will be solely responsible for subsequent development, manufacturing and commercialization of any product candidate resulting from the licensed agent. The Company received an upfront payment from Vertex of $ 25.0 million and is eligible to receive (i) success payments of up to $ 22.0 million for each product candidate (up to a maximum of $ 66.0 million) that achieves the applicable development criteria, (ii) up to an aggregate of $ 175.0 million in development milestones and (iii) up to an aggregate of $ 165.0 million in commercial milestone payments. The Company is also eligible to receive tiered single-digit royalties on net sales, with the rate dependent upon the type of product and subject to specified reductions. Such royalty payments will terminate on a country-by-country and product-by-product basis upon the later to occur of (i) the expiration of the last to expire valid claim under the patent rights covering such product in such country, (ii) the period of regulatory exclusivity associated with such product in such country or (iii) ten years after the first commercial sale of such product in such country. Prior to the first patient dosing in the first Phase 1 clinical trial for the first product candidate developed under the Vertex Agreement, the Company also has the right to opt-in to a profit share arrangement pursuant to which Vertex and the Company would share the costs and net profits for all product candidates emerging from the collaboration. If the Company exercises its opt-in right, in lieu of milestones and royalties, it will be obligated to pay for a specified percentage of the development and commercialization costs, and it will have the right to receive a specified percentage of the profits from any sales of any product candidates advanced under the collaboration. At the time the Company exercises the option, it may elect a profit/cost share of up to 40 % (with Vertex retaining a minimum of 60 %). In order to exercise its opt-in right, the Company is required to pay a fee ranging from $ 25.0 million to $ 70.0 million, depending on the profit/cost percentage elected by the Company and the Company’s licensed technology included in the most advanced product candidate at the time it exercises its opt-in right. Under all profit share scenarios, Vertex will control the worldwide development and commercialization of any product candidates resulting from the collaboration. The Vertex Agreement includes customary representations and warranties, covenants and indemnification obligations for a transaction of this nature. The Company and Vertex each have the right to terminate the agreement for material breach by, or insolvency of, the other party following notice, and if applicable, a cure period. Vertex may also terminate the Vertex Agreement in its entirety for convenience upon 90 days’ notice. Accounting Analysis The Company assessed the promised goods and services under the Vertex Agreement, in accordance with ASC Topic 606, "Revenue from Contracts with Customers" ("ASC 606"). At inception, the Vertex Agreement included the following performance obligations: (i) the research services obligation which relates to the research and development services to be provided under the Vertex Research Plan through June 30, 2023 (the “Initial Vertex Research Services”) and (ii) three licensed agent material rights related to the options to obtain licenses to exploit a licensed agent, at a discount. The Company identified $ 20.0 million of fixed transaction price consisting of the $ 25.0 million upfront fee offset by a discount of $ 5.0 million related to the 1,519,756 shares sold to Vertex under the Vertex Stock Purchase Agreement when measured at fair value on the date of issuance. The Company is also entitled to reimbursement of costs incurred associated with the delivery of the Initial Vertex Research Services. The Company utilized the most likely amount approach and estimated the expected cost reimbursement to be $ 5.8 million at inception. The Company concluded that these amounts do not require a constraint and are included in the transaction price at inception. The Company considers this estimate at each reporting date and updates the estimate based on information available. Additional consideration to be paid to the Company upon reaching certain milestones are excluded from the transaction price as that consideration may only be earned subsequent to an option exercise. The Company has concluded that the variable consideration related to the cost reimbursement of the Initial Vertex Research Services obligation will be allocated entirely to that obligation as the cost reimbursement relates specifically to the services being performed under the Vertex Research Plan. The reimbursement of the Initial Vertex Research Services is considered to be at a market rate and therefore depicts the estimated amount it would expect to receive for this obligation. As a result, the Company allocated the fixed consideration of $ 20.0 million to the three licensed agent material rights based on their relative standalone selling prices. The estimated standalone selling price for each material right was based on an adjusted market assessment approach. The Company concluded that the market would be willing to pay an equal amount for each licensed agent license on a standalone basis before being adjusted for the probability of the option becoming exercisable upon the successful completion of research activities to identify the licensed agents. The Company reached this conclusion after considering (i) the downstream economics including success fees, milestones and royalties related to each licensed agent being identical and (ii) all licensed agents are targeting the same gene. As such, based on the relative standalone selling price for each of the three material rights, the allocation of the transaction price to the separate performance obligations, at inception, is as follows: Performance obligation Amount (in thousands) Research services obligation $ 5,845 First licensed agent material right 6,667 Second licensed agent material right 6,667 Third licensed agent material right 6,666 Total $ 25,845 The amount allocated to the Initial Vertex Research Services obligation will be recognized on a proportional performance basis over the period of service using input-based measurements of total cost of research incurred to estimate the proportion performed and remeasured at the end of each reporting period. The amount allocated to the licensed agent material rights was recorded as deferred revenue and will commence recognition upon exercise of each option or, if an option is never exercised, it will be recognized in full upon expiry of the Vertex Research Term. The reimbursement related to the Initial Vertex Research Services was $ 5.4 million , based on the services rendered upon completion of the Initial Vertex Research Services as of June 30, 2023. Upon completion of the Initial Vertex Research Services, the parties agreed upon and estimated additional services to be rendered through December 31, 2024. The Company utilized the most likely amount approach and estimated the expected cost reimbursement to be $ 16.9 million which is being recognized on a proportional performance basis over the period of service using an input-based measurement. During the three and nine months ended September 30, 2023, the Company recognized $ 2.4 million and $ 5.9 million of revenue, respectively, associated with the Vertex Agreement related to research services performed during the period. As of September 30, 2023, the Company has recorded $ 20.0 million as non-current deferred revenue related to the unexercised material rights. During the three and nine months ended September 30, 2022 , the Company recognized $ 0.9 million of revenue associated with the Vertex Agreement related to research services performed during the period. Costs incurred relating to the Company’s collaboration programs under the Vertex Agreement consist of internal and external research costs, which primarily include: salaries and benefits, and preclinical research studies. These costs are included in research and development expenses in the Company’s condensed consolidated statements of operations. Lilly Agreement Summary of Agreement In June 2023, the Company entered into a Research and Collaboration Agreement (the “Lilly Agreement”) with Lilly for an exclusive, five-year worldwide research collaboration initially focused on advancing the Company’s discovery-stage in vivo gene editing lipoprotein(a) program. On July 26, 2023, following expiration of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, the Lilly Agreement became effective. Additionally, in June 2023, in connection with the execution of the Lilly Agreement, the Company entered into a stock purchase agreement with Lilly (the “Lilly Stock Purchase Agreement”), pursuant to which the Company sold 1,552,795 shares of the Company's common stock to Lilly at a price of $ 19.32 per share for an aggregate purchase price of $ 30.0 million (the “Private Placement”). The Private Placement closed on July 31, 2023. Pursuant to the Lilly Agreement, the Company will be responsible for all research activities and Phase 1 clinical development of the initial target of interest— LPA . The Company’s research and development activities will be focused on (i) identifying and engineering specific gene editing systems and in vivo delivery technologies directed to the relevant target; (ii) evaluating and optimizing development candidates to achieve criteria specified in the Lilly Agreement; and (iii) Phase 1 clinical development ("Lilly Research Services"). Lilly will reimburse the Company’s research expenses and Phase 1 clinical development expenses consistent with an agreed-upon budget (“Lilly Research and Development Plan”). The research term for the initial target is five years and may be extended by Lilly for up to one additional year (“Lilly Research Term”). The Lilly Research and Development Plan is overseen by a Joint Steering Committee (“Lilly JSC”) as detailed in the Lilly Agreement. Any material amendments to the Lilly Research and Development Plan are required to be mutually agreed to by the Lilly JSC. A licensed product under the Lilly Agreement is an in vivo gene editing product that includes a candidate developed from the Lilly Research and Development Plan. A candidate is agreed upon when reviewed by the Lilly JSC against the candidate success criteria described in the Lilly Agreement. Upon approval of a candidate, Lilly will receive a license to exploit the licensed product, and the licensed product will continue to be developed under the Lilly Research and Development Plan through completion of the Phase 1 clinical trial for the applicable licensed product. Following completion of Phase 1 clinical trials with respect to any licensed product candidate under the Lilly Agreement, Lilly will be solely responsible for subsequent development, manufacturing and commercialization of each such product candidate resulting from the Company’s research efforts. Under the Lilly Agreement, the Company received an upfront payment from Lilly of $ 30.0 million in August 2023. The Company is also eligible to receive (i) up to an aggregate of $ 190.0 million in research and development milestone payments and (ii) up to an aggregate of $ 275.0 million in commercial milestone payments. The Company is also eligible to receive tiered and incremental high single and low-double digit royalties on global net sales, subject to specified reductions. Such royalty payments will terminate on a country-by-country and product-by-product basis upon the latest to occur of (i) the expiration of the last-to-expire valid claim under the patent rights covering such product in such country, (ii) expiration of the period of regulatory and market exclusivity associated with such product in such country or (iii) 10 years after the first commercial sale of such product in such country. Following completion of Phase 1 clinical development, the Company has the right to opt-in to a cost and margin share arrangement pursuant to which Lilly and the Company would share the costs and net margins for all product candidates emerging from the collaboration. If the Company exercises its opt-in right, the Company will be obligated to pay an opt-in fee in addition to funding 40 % of the development and commercialization costs, and it will have the right to receive, in lieu of the milestones and royalties described above, 40 % of the gross margin less eligible expenses from any sales of any product candidates advanced under the collaboration, with Lilly retaining 60 % of the cost and margin share. Notwithstanding this opt-in right, Lilly will control the worldwide development and commercialization of any product candidates resulting from the collaboration. Beyond the initial target of interest, upon the achievement of certain criteria and payment of additional upfront consideration, Lilly has the right to elect one additional, pre-determined target to the collaboration. The research, clinical development and commercialization of such additional target would be subject to the same terms under the Lilly Agreement as the initial target, including the Company’s right to receive up to an additional $ 465.0 million in research, development and commercial milestone payments, the Company’s right to receive tiered and incremental high single and low-double digit royalties on global net sales and the Company’s right to opt-in to a cost and margin share arrangement. During the Lilly Research Term, Lilly can make the determination subject to the terms of the Lilly Agreement, to replace the initial target or additional target with a different specified option target. Such replacement target will be the subject of a new licensed program under the research and development program. All rights granted with respect to the replaced target will cease upon Lilly’s election to select a different specified option target. The Lilly Agreement includes customary representations and warranties, covenants and indemnification obligations for a transaction of this nature. The Company and Lilly each have the right to terminate the agreement for material breach by the other party following notice, and if applicable, a cure period. Lilly may also terminate the Lilly Agreement in its entirety for convenience upon 180 days’ notice or in part, on a research plan, licensed target or product basis, for convenience upon 90 days’ notice. The Company may terminate the Lilly Agreement, in part with respect to its licensed patents, if Lilly directly or indirectly challenges the enforceability, validity or scope of such patent rights, or on a licensed product-by-licensed product basis, if such licensed product ceases to be developed for a period of time. Concurrently with the Lilly Agreement, the Company and Lilly entered into a Sublicense Option Agreement, pursuant to which the Company granted Lilly an option to obtain a non-exclusive sublicense under patent rights licensed to the Company under the Harvard/Broad License Agreement (“Lilly Option”). In consideration for the Lilly Option, Lilly paid the Company an upfront fee of $ 0.3 million . For accounting purposes, the upfront payments for the Lilly Agreement and the Sublicense Option Agreement will be combined. If Lilly exercises the Lilly Option, Lilly will be required to pay a sublicense fee to the Company and Lilly will reimburse the Company for royalties and milestones incurred related to the sublicensed product under the Harvard/Broad License Agreement. Accounting Analysis The Company assessed the promised goods and services under the Lilly Agreement, in accordance with ASC 606. At inception, the Lilly Agreement included the following performance obligations: (i) the Lilly Research Services obligation which relates to the research and development services to be provided under the Lilly Research and Development Plan and (ii) two licensed product material rights related to the right to obtain licenses to exploit a licensed product associated with the initial target upon achievement of certain development criteria. The Company identified $ 28.5 million of fixed transaction price consisting of (i) the $ 30.0 million upfront fee in the Lilly Agreement, (ii) the $ 0.3 million upfront fee in the Lilly Option, which is combined for accounting purposes, (iii) offset by a discount of $ 1.7 million related to the 1,552,795 shares sold to Lilly under the Lilly Stock Purchase Agreement when measured at fair value on the date of issuance. The Company is also entitled to reimbursement of costs incurred associated with the delivery of the Lilly Research Services. The Company utilized the most likely amount approach and estimated the expected cost reimbursement to be $ 16.4 million at inception, which represents the budget for the first year of the Lilly Research Services. The remainder of the Lilly Research and Development Plan budget is subject to annual review by Lilly and the Company. The Company concluded that these amounts do not require a constraint and are included in the transaction price at inception. The Company considers this estimate at each reporting date and updates the estimate based on information available. Additional consideration to be paid to the Company upon reaching certain milestones are excluded from the transaction price at contract inception as they are not probable at this time. The probability will be re-evaluated each reporting period, and the transaction price will be updated accordingly. The Company has concluded that the variable consideration related to the cost reimbursement of the Lilly Research Services obligation will be allocated entirely to that obligation as the cost reimbursement relates specifically to the services being performed under the Research and Development Plan. The reimbursement of the Research and Development Services is considered to be at a market rate and therefore depicts the estimated amount it would expect to receive for this obligation. As a result, the Company allocated the fixed consideration of $ 28.5 million to the two licensed product material rights based on their relative standalone selling prices. The estimated standalone selling price for each material right was based on an adjusted market assessment approach. The Company concluded that the market would be willing to pay an equal amount for each license on a standalone basis before being adjusted for the probability of the option becoming exercisable upon the successful completion of research and development activities to identify the licensed products. The Company reached this conclusion after considering (i) the downstream economics including success fees, milestones and royalties related to each licensed product being identical and (ii) both licensed products are targeting the same gene. As such, based on the relative standalone selling price for each of the material rights, the allocation of the transaction price to the separate performance obligations, at inception, is as follows: Performance obligation Amount (in thousands) Research services obligation $ 16,398 First licensed product material right 14,270 Second licensed product material right 14,270 Total $ 44,938 The amount allocated to the Lilly Research Services obligation will be recognized on a proportional performance basis over the period of service using input-based measurements of total cost of research incurred to estimate the proportion performed and remeasured at the end of each reporting period. The amount allocated to the licensed product material rights was recorded as deferred revenue and will commence recognition upon the achievement of the candidate selection criteria or, if never achieved, it will be recognized in full upon expiry of the Lilly Research Term. During three and nine months ended September 30, 2023, the Company recognized $ 0.7 million of revenue associated with the Lilly Agreement related to the Lilly Research Services performed during the period. As of September 30, 2023, the Company has recorded $ 28.5 million as non-current deferred revenue related to the unexercised material rights. Costs incurred relating to the Company’s collaboration programs under the Lilly Agreement consist of internal and external research costs, which primarily include: salaries and benefits, and preclinical research studies. These costs are included in research and development expenses in the Company’s condensed consolidated statements of operations during the three and nine months ended September 30, 2023 . |