Sarepta announces collaboration, license agreement with StrideBio
arepta Therapeutics announced the signing of a collaboration and license agreement to develop in vivo AAV-based therapies for up to eight central nervous system and neuromuscular targets. Pursuant to the agreement, Sarepta is granted an exclusive license on selected targets to leverage StrideBio's novel, structure-driven capsid technology, intended to enhance specific tropism to tissues of interest and evade neutralizing antibodies. The parties also plan to focus on strategies intended to address re-dosing challenges in patients who have received AAV-delivered gene therapy. StrideBio will conduct all investigational new drug enabling research, development and manufacturing for the first four CNS targets, which are MECP2, SCN1A, UBE3A, and NPC1. Additionally, Sarepta will have an exclusive option to four additional targets based on StrideBio's capsid technology. StrideBio has created a growing portfolio of novel AAV capsids evolved in non-human primates that show reduced seroprevalence and potential for improved tropism to targeted tissues. Under the terms of the agreement, StrideBio will be responsible for AAV capsid development, non-clinical development and manufacturing of preclinical candidates to be selected for advancement into clinical studies. The parties will also share early clinical development activities for selected targets, with Sarepta responsible for late stage development and commercialization. A total of four initial targets are specified under the collaboration. StrideBio will receive a $48M upfront payment in the form of cash and Sarepta stock, in addition to significant future development, regulatory and commercial milestones for the four programs. StrideBio will also receive royalties on worldwide net sales of any commercial products developed through the collaboration. Sarepta has also obtained an exclusive option to expand the collaboration to include up to an additional four targets with an upfront payment of up to $42.5M along with future downstream milestone payments, while StrideBio has an option to obtain co-development and co-commercial rights to one of the collaboration targets. In addition, Sarepta has made a commitment to invest in StrideBio's next financing round. Further financial terms were not disclosed.