REGENXBIO Inc. and AveXis Inc. announced that they have entered into an amendment which expands upon the exclusive, worldwide license agreement they entered into in March 2014 (2014 License Agreement) for the development and commercialization of products to treat spinal muscular atrophy (SMA). AveXis’ initial proprietary gene therapy candidate, AVXS-101, is in a pivotal trial for the treatment of SMA Type 1, and a Phase 1 trial for SMA Type 2. AVXS-101 uses REGENXBIO’s NAV AAV9 vector. Under the terms of the amendment, REGENXBIO granted AveXis exclusive, worldwide rights to all vectors in REGENXBIO’s NAV Technology Platform for the treatment of SMA in addition to adding and amending certain terms of the 2014 License Agreement, including the modification of the assignment provision to now permit assignment in the event of a change of control by AveXis without REGENXBIO’s consent. REGENXBIO will receive, in addition to the payments and royalties owed under the original 2014 License Agreement, an upfront payment of $80 million, an additional payment of $30 million after one year and an additional payment of $30 million after two years, and REGENXBIO is eligible to receive potential commercial milestone payments of up to $120 million. For any product developed for the treatment of SMA using the NAV AAV9 vector, REGENXBIO will continue to receive mid-single to low double-digit royalties on net sales as defined in the original 2014 License Agreement, and for any product developed for the treatment of SMA using a NAV vector, other than NAV AAV9, REGENXBIO will receive a low double-digit royalty on net sales.