Sarepta Therapeutics is set to pay $48 million upfront to work with StrideBio on gene therapies. The deal gives Sarepta exclusive licenses to four CNS targets, plus an option to pay a further $42.5 million to add another four targets to the deal.

In return for the initial outlay, Sarepta will secure the rights to gene therapies against Rett syndrome, Dravet syndrome, Angelman syndrome and Niemann-Pick. StrideBio will handle the development of the AAV capsid and all other IND-enabling R&D. Sarepta and StrideBio will then share early clinical development work for selected candidates.

Sarepta CEO Doug Ingram framed the division of responsibilities as a way for his company to “gain access to new technology and targets” without distracting it from its near-term priorities.

StrideBio’s AAV capsids are the centerpiece of the technology covered by the deal. The capsids could set StrideBio apart from the growing pack of gene therapy biotechs. Notably, StrideBio is working on capsids capable of evading neutralizing antibodies, thereby enabling repeat dosing. Sarepta called out re-dosing as a focal point of its collaboration with StrideBio. 

By granting Sarepta rights to gene therapies based on the technology, StrideBio has positioned itself to receive “significant” but currently undisclosed milestones. The deal also grants StrideBio royalties and the chance to co-develop and co-commercialize one of the gene therapies. CRISPR Therapeutics and Takeda are among the companies that work with StrideBio in other contexts.

For Sarepta, the StrideBio deal furthers its move into gene therapies and beyond Duchenne muscular dystrophy, the indication in which it made its name. Over the past two years, Sarepta has teamed up with academic labs to work on treatments for Rett, cardiomyopathy and multiple sclerosis, while also partnering with Lysogene and Lacerta Therapeutics on Sanfilippo syndrome and Pompe disease.