Sarepta Therapeutics is beefing up its gene therapy pipeline with a collaboration and licensing agreement with StrideBio to develop in vivo AAV-based therapies for up to eight central nervous system and neuromuscular targets involved in rare diseases.

Under the terms of their agreement, Sarepta has been granted an exclusive license on selected targets to leverage StrideBio’s novel, structure-driven capsid technology, which is 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 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: MECP2 (Rett syndrome), SCN1A (Dravet syndrome), UBE3A (Angelman syndrome), and NPC1 (Niemann-Pick). Sarepta will also have an exclusive option to four additional targets based on StrideBio’s capsid technology.

Sarepta will pay StrideBio $48 million in an upfront payment 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 $42.5 million 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. Sarepta has also committed to invest in StrideBio’s next financing round.

“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,” said Douglas Ingram, Sarepta’s president and CEO.

According to Sarepta, 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. The privately-held biotech was co-founded by Mavis Agbandje-McKenna, and Aravind Asokan, leading scientists in the field of AAV biology and gene therapy. StrideBio’s portfolio of novel AAV capsids evolved in non-human primates show reduced seroprevalence and potential for improved tropism to targeted tissues, potentially addressing an unanswered question in the fast-paced field of the possible need for re-treatment to gene therapies.

For StrideBio, the partnership will enable the rapid expansion of its research and manufacturing platform, says DEO Sapan Shah, 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,” he says.

Photo: Douglas Ingram, president and CEO of Sarepta Therapeutics

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