LICENSE AND COLLABORATION AGREEMENTS	6 Months Ended
Jun. 30, 2023
Organization, Consolidation and Presentation of Financial Statements [Abstract]
LICENSE AND COLLABORATION AGREEMENTS	3. LICENSE AND COLLABORATION AGREEMENTS F. Hoffman-La Roche Ltd. For the three and six months ended June 30, 2023 and 2022, the Company recognized $22.3 million and $44.3 million of collaboration revenue, respectively, associated with the license, collaboration and option agreement (the “Roche Agreement”) with F. Hoffman-La Roche Ltd. (“Roche”). As of June 30, 2023, the Company had total deferred revenue of $530.0 million associated with the Roche Agreement, of which $45.0 million is classified as current. The portion of deferred revenue related to the separate material rights for the options to acquire ex-U.S. rights to certain Duchenne-specific programs was $485.0 million as of June 30, 2023 and December 31, 2022. The costs associated with co-development activities performed under the Roche Agreement are included in operating expenses, with any reimbursement of costs by Roche reflected as a reduction of such expenses when the related expense is incurred. For the three and six months ended June 30, 2023, costs reimbursable by Roche and reflected as a reduction to operating expenses were $28.2 million and $48.5 million, respectively. For the three and six months ended June 30, 2022, costs reimbursable by Roche and reflected as a reduction to operating expenses were $26.4 million and $44.1 million, respectively. As of June 30, 2023, there was $28.6 million of collaboration receivable included in other current assets. Nationwide Children's Hospital In December 2016, the Company entered into an exclusive option agreement with Nationwide Children’s Hospital (“Nationwide”) from which the Company obtained an exclusive right to acquire a worldwide license of the micro-dystrophin gene therapy technology for Duchenne and Becker muscular dystrophy. In October 2018, the Company exercised the option and entered into a license agreement with Nationwide, which granted the Company exclusive worldwide rights to develop, manufacture and commercialize a micro-dystrophin gene therapy product candidate. In connection with the FDA approval of ELEVIDYS in June 2023, the Company recorded a milestone payment of $10.0 million to Nationwide as an in-licensed right intangible asset in its unaudited condensed consolidated balance sheets as of June 30, 2023. The in-licensed right is being amortized on a straight-line basis over the remaining life of the relevant patents and has a carrying value of approximately $10.0 million as of June 30, 2023. Upon commercial sale of ELEVIDYS, the Company is expected to make low-single-digit royalty payments based on net sales which will be recorded by the Company as cost of sales. Research and Option Agreements The Company has research and option agreements with third parties in order to develop various technologies and biologics that may be used in the administration of the Company’s genetic therapeutics. The agreements generally provide for research services related to pre-clinical development programs and options to license the technology for clinical development. Prior to the options under these agreements being executed, the Company may be required to make up to $39.8 million in research milestone payments. Under these agreements, there are $221.3 million in potential option payments to be made by the Company upon the determination to exercise the options. Additionally, if the options for each agreement are exercised and additional license agreements are executed, the Company would incur additional contingent obligations and may be required to make development, regulatory, and sales milestone payments and royalty payments based on the net sales of the developed products upon commercialization. In April 2023, the Company exercised one such option and recognized $7.5 million of up-front payment as research and development expense in the accompanying unaudited condensed consolidated statements of operations and comprehensive loss for the three and six months ended June 30, 2023. For the three and six months ended June 30, 2022, the Company recognized $6.0 million of research, option and milestone expense. As of June 30, 2023, no additional research milestone payments became probable of occurring. Milestone Obligations The Company has license and collaboration agreements in place for which it could be obligated to pay, in addition to the payment of up-front fees upon execution of the agreements, certain milestone payments as a product candidate proceeds from the submission of an investigational new drug application through approval for commercial sale and beyond. As of June 30, 2023, the Company may be obligated to make up to $3.2 billion of future development, regulatory, commercial and up-front royalty payments associated with its collaboration and license agreements. These obligations exclude potential future option and milestone payments for options that have yet to be exercised within agreements entered into by the Company as of June 30, 2023, which are discussed above. For the three and six months ended June 30, 2023, the Company recognized up-front, development milestone and other expenses of $7.6 million and $8.1 million, respectively, as research and development expense in the accompanying unaudited condensed consolidated statements of operations and comprehensive loss. For the three and six months ended June 30, 2022, the Company recognized up-front and development milestone expenses of approximately $8.8 million as research and development expense in the accompanying unaudited condensed consolidated statements of operations and comprehensive loss.

LICENSE AND COLLABORATION AGREEMENTS

6 Months Ended

Jun. 30, 2023

Organization, Consolidation and Presentation of Financial Statements [Abstract]

3. LICENSE AND COLLABORATION AGREEMENTS

F. Hoffman-La Roche Ltd.

For the three and six months ended June 30, 2023 and 2022, the Company recognized $22.3 million and $44.3 million of collaboration revenue, respectively, associated with the license, collaboration and option agreement (the “Roche Agreement”) with F. Hoffman-La Roche Ltd. (“Roche”). As of June 30, 2023, the Company had total deferred revenue of $530.0 million associated with the Roche Agreement, of which $45.0 million is classified as current. The portion of deferred revenue related to the separate material rights for the options to acquire ex-U.S. rights to certain Duchenne-specific programs was $485.0 million as of June 30, 2023 and December 31, 2022.

The costs associated with co-development activities performed under the Roche Agreement are included in operating expenses, with any reimbursement of costs by Roche reflected as a reduction of such expenses when the related expense is incurred. For the three and six months ended June 30, 2023, costs reimbursable by Roche and reflected as a reduction to operating expenses were $28.2 million and $48.5 million, respectively. For the three and six months ended June 30, 2022, costs reimbursable by Roche and reflected as a reduction to operating expenses were $26.4 million and $44.1 million, respectively. As of June 30, 2023, there was $28.6 million of collaboration receivable included in other current assets.

Nationwide Children's Hospital

In December 2016, the Company entered into an exclusive option agreement with Nationwide Children’s Hospital (“Nationwide”) from which the Company obtained an exclusive right to acquire a worldwide license of the micro-dystrophin gene therapy technology for Duchenne and Becker muscular dystrophy. In October 2018, the Company exercised the option and entered into a license agreement with Nationwide, which granted the Company exclusive worldwide rights to develop, manufacture and commercialize a micro-dystrophin gene therapy product candidate. In connection with the FDA approval of ELEVIDYS in June 2023, the Company recorded a milestone payment of $10.0 million to Nationwide as an in-licensed right intangible asset in its unaudited condensed consolidated balance sheets as of June 30, 2023. The in-licensed right is being amortized on a straight-line basis over the remaining life of the relevant patents and has a carrying value of approximately $10.0 million as of June 30, 2023. Upon commercial sale of ELEVIDYS, the Company is expected to make low-single-digit royalty payments based on net sales which will be recorded by the Company as cost of sales.

Research and Option Agreements

The Company has research and option agreements with third parties in order to develop various technologies and biologics that may be used in the administration of the Company’s genetic therapeutics. The agreements generally provide for research services related to pre-clinical development programs and options to license the technology for clinical development. Prior to the options under these agreements being executed, the Company may be required to make up to $39.8 million in research milestone payments. Under these agreements, there are $221.3 million in potential option payments to be made by the Company upon the determination to exercise the options. Additionally, if the options for each agreement are exercised and additional license agreements are executed, the Company would incur additional contingent obligations and may be required to make development, regulatory, and sales milestone payments and royalty payments based on the net sales of the developed products upon commercialization. In April 2023, the Company exercised one such option and recognized $7.5 million of up-front payment as research and development expense in the accompanying unaudited condensed consolidated statements of operations and comprehensive loss for the three and six months ended June 30, 2023. For the three and six months ended June 30, 2022, the Company recognized $6.0 million of research, option and milestone expense. As of June 30, 2023, no additional research milestone payments became probable of occurring.

Milestone Obligations

The Company has license and collaboration agreements in place for which it could be obligated to pay, in addition to the payment of up-front fees upon execution of the agreements, certain milestone payments as a product candidate proceeds from the submission of an investigational new drug application through approval for commercial sale and beyond. As of June 30, 2023, the Company may be obligated to make up to $3.2 billion of future development, regulatory, commercial and up-front royalty payments associated with its collaboration and license agreements. These obligations exclude potential future option and milestone payments for options that have yet to be exercised within agreements entered into by the Company as of June 30, 2023, which are discussed above. For the three and six months ended June 30, 2023, the Company recognized up-front, development milestone and other expenses of $7.6 million and $8.1 million, respectively, as research and development expense in the accompanying unaudited condensed consolidated statements of operations and comprehensive loss. For the three and six months ended June 30, 2022, the Company recognized up-front and development milestone expenses of approximately $8.8 million as research and development expense in the accompanying unaudited condensed consolidated statements of operations and comprehensive loss.