Pharma Focus Asia

Sarepta and StrideBio Announce Multi-target Strategic Collaboration to Advance Novel Gene Therapies

Friday, November 15, 2019

Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, and StrideBio, Inc., a leading developer of novel adeno-associated viral (AAV) based gene therapies, today announced the signing of a collaboration and license agreement to develop in vivo AAV-based therapies for up to eight central nervous system (CNS) 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 (IND) enabling research, development and manufacturing for the first four CNS targets, which are MECP2 (Rett syndrome), SCN1A (Dravet syndrome), UBE3A (Angelman syndrome), and NPC1 (Niemann-Pick). Additionally, Sarepta will have an exclusive option to four additional targets based on StrideBio’s capsid technology.

StrideBio possesses an innovative and proprietary platform that is enabled by a deep knowledge of AAV structure and a unique approach to engineering capsids with novel functions, and was co-founded by Mavis Agbandje-McKenna, Ph.D., and Aravind Asokan, Ph.D., leading scientists in the field of AAV biology and gene therapy. 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.

“With our partnership with StrideBio, Sarepta continues to build on its leadership position in gene therapies to treat rare diseases. We are excited to work with StrideBio and access its innovative AAV platform for next-generation capsids,” said Doug Ingram, Sarepta’s President and Chief Executive Officer. “Our partnership with StrideBio expands our research portfolio by up to eight new targets and, through our strategic partnering approach that has our collaborator lead all IND-enabling research and development, ensures that we gain access to new technology and targets while not distracting Sarepta from its near-term priorities.”   

“We are very excited to initiate this multi-target collaboration with Sarepta, an established leader in the development and commercialization of genetic medicines,” stated Sapan Shah, Ph.D., Chief Executive Officer, StrideBio. “This partnership will provide significant resources and expertise to enable StrideBio’s continued rapid expansion of our research and manufacturing platform, as well as accelerate the development of AAV gene therapies for multiple rare disease targets. We are looking forward to working together with Sarepta to bring novel treatments to patients as quickly as possible.”

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