LICENSE AND COLLABORATION AGREEMENTS	6 Months Ended
Jun. 30, 2022
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, 2022 and 2021, the Company recognized $22.3 million and $44.3 million of collaboration revenue associated with the license, collaboration and option agreement (the “Roche Agreement”) with F. Hoffman-La Roche Ltd. (“Roche”). As of June 30, 2022, the Company has total deferred revenue of $619.2 million associated with the Roche Agreement, of which $89.2 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, 2022 and December 31, 2021. 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 months 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. For the three and six months ended June 30, 2021, costs reimbursable by Roche and reflected as a reduction to operating expenses were $18.0 million and $31.4 million, respectively. As of June 30, 2022, there was $26.4 million of collaboration receivable included in other current assets. Lysogene SA and Henogen SA In October 2018, the Company entered into a license and collaboration agreement to develop and commercialize LYS-SAF302, a gene therapy to treat Mucopolysaccharidosis type IIIA as well as an equity investment agreement with Lysogene SA (“Lysogene”). Under the license and collaboration agreement, in addition to the payment of up-front fees, the Company may be liable for a total of $102.8 million in development, regulatory and sales milestones. Furthermore, the Company may be required to make tiered royalty payments based on net sales of the LYS-SAF302 product subsequent to its commercialization. Beginning January 1, 2020, the Company began to reimburse Lysogene for expenses incurred in connection with development activities of LYS-SAF302. As of June 30, 2022, the Company owns 1,140,728 shares of common stock issued by Lysogene and recorded $0.8 million of equity investment in Lysogene as an other non-current asset in the Company’s consolidated balance sheets. The Company sent a termination notice to Lysogene on January 11, 2022 to notify them of the Company's intent to terminate the license and collaboration agreement. The termination became effective July 11, 2022. The Company is not obligated to pay early termination penalties to Lysogene but has agreed to pay certain research and development reimbursements incurred in the six months following termination, which are not expected to be material. The Company entered into a development, manufacturing and supply agreement with Henogen SA ("Henogen") in December 2019. Pursuant to the terms of the agreement, Henogen agreed to reserve manufacturing capacity within their facility to develop, manufacture and supply the Company with LYS-SAF302. On June 9, 2022, the Company and Henogen entered into an agreement to terminate the development, manufacturing and supply agreement. As a result, the Company recorded a charge of $17.1 million during the three months ended June 30, 2022, which was recorded in research and development expenses in the accompanying unaudited condensed consolidated statements of operations and comprehensive loss. 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 exercised, the Company may be required to make up to $7.0 million in research milestone payments. Under these agreements, there are $187.1 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 executed, the Company would incur additional contingent obligations and may be required to make development, regulatory, and sales milestone payments and tiered royalty payments based on the net sales of the developed products upon commercialization. For the three and six months ended June 30, 2022 and 2021, the Company recognized $6.0 million and $3.0 million of research, option and milestone expense, respectively. As of June 30, 2022, the Company has not exercised any options nor have any additional research milestone payments become 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, 2022, the Company may be obligated to make up to $3.7 billion in future development, regulatory, commercial and up-front royalty milestone payments associated with its license and collaboration 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, 2022, which are discussed above. For the three months and six months ended June 30, 2022, the Company recognized up-front and development milestone expenses approximately $8.8 million 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, 2021, the Company recognized up-front, development milestone, settlement and other expenses of $31.7 million and $45.7 million, respectively, 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, 2022

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, 2022 and 2021, the Company recognized $22.3 million and $44.3 million of collaboration revenue associated with the license, collaboration and option agreement (the “Roche Agreement”) with F. Hoffman-La Roche Ltd. (“Roche”). As of June 30, 2022, the Company has total deferred revenue of $619.2 million associated with the Roche Agreement, of which $89.2 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, 2022 and December 31, 2021.

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 months 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. For the three and six months ended June 30, 2021, costs reimbursable by Roche and reflected as a reduction to operating expenses were $18.0 million and $31.4 million, respectively. As of June 30, 2022, there was $26.4 million of collaboration receivable included in other current assets.

Lysogene SA and Henogen SA

In October 2018, the Company entered into a license and collaboration agreement to develop and commercialize LYS-SAF302, a gene therapy to treat Mucopolysaccharidosis type IIIA as well as an equity investment agreement with Lysogene SA (“Lysogene”). Under the license and collaboration agreement, in addition to the payment of up-front fees, the Company may be liable for a total of $102.8 million in development, regulatory and sales milestones. Furthermore, the Company may be required to make tiered royalty payments based on net sales of the LYS-SAF302 product subsequent to its commercialization. Beginning January 1, 2020, the Company began to reimburse Lysogene for expenses incurred in connection with development activities of LYS-SAF302. As of June 30, 2022, the Company owns 1,140,728 shares of common stock issued by Lysogene and recorded $0.8 million of equity investment in Lysogene as an other non-current asset in the Company’s consolidated balance sheets.

The Company sent a termination notice to Lysogene on January 11, 2022 to notify them of the Company's intent to terminate the license and collaboration agreement. The termination became effective July 11, 2022. The Company is not obligated to pay early termination penalties to Lysogene but has agreed to pay certain research and development reimbursements incurred in the six months following termination, which are not expected to be material.

The Company entered into a development, manufacturing and supply agreement with Henogen SA ("Henogen") in December 2019. Pursuant to the terms of the agreement, Henogen agreed to reserve manufacturing capacity within their facility to develop,

manufacture and supply the Company with LYS-SAF302. On June 9, 2022, the Company and Henogen entered into an agreement to terminate the development, manufacturing and supply agreement. As a result, the Company recorded a charge of $17.1 million during the three months ended June 30, 2022, which was recorded in research and development expenses in the accompanying unaudited condensed consolidated statements of operations and comprehensive loss.

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 exercised, the Company may be required to make up to $7.0 million in research milestone payments. Under these agreements, there are $187.1 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 executed, the Company would incur additional contingent obligations and may be required to make development, regulatory, and sales milestone payments and tiered royalty payments based on the net sales of the developed products upon commercialization. For the three and six months ended June 30, 2022 and 2021, the Company recognized $6.0 million and $3.0 million of research, option and milestone expense, respectively. As of June 30, 2022, the Company has not exercised any options nor have any additional research milestone payments become 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, 2022, the Company may be obligated to make up to $3.7 billion in future development, regulatory, commercial and up-front royalty milestone payments associated with its license and collaboration 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, 2022, which are discussed above. For the three months and six months ended June 30, 2022, the Company recognized up-front and development milestone expenses approximately $8.8 million 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, 2021, the Company recognized up-front, development milestone, settlement and other expenses of $31.7 million and $45.7 million, respectively, as research and development expense in the accompanying unaudited condensed consolidated statements of operations and comprehensive loss.