RTP gene tech firm lands deal worth up to $90M for 8 nervous system therapies

By Rick Smith, WRAL TechWire

RESEARCH TRIANGLE PARK – StrideBio, a life science startup focused on gene editing technology, has a deal in place with a Massachusetts  drug firm that could be worth nearly $90 million plus royalties in years to come for rights to as many as eight potential gene-based therapies.

Any developed drug would focus on central nervous system and neuromuscular “targets,” StrideBio and its partner Sarepta Therapeutics announced Thursday. However, initial investor reaction was not positive. Sarpeta stock fell nearly 5 percent after the news broke.

StrideBio, which launched in 2015, is focused on what it calls engineered viral vectors, or AAV, for gene therapy.

The firm has also struck a deal with Crispr Therapeutics to develop in vivo gene delivery applications. As part of that deal, StrideBio will receive development funding, milestones and royalties on licensed vectors, and retain certain rights to use the novel AAV vectors for gene therapy applications.

The technology at StrideBio is based on the work of Dr. Aravind Asokan at UNC-Chapel Hill and Dr. Mavis Agbandje-McKenna at the University of Florida.

Asokan leads the Laboratory for Synthetic Virology & Gene Therapy at UNC-Chapel Hill. Originally trained as a chemist, he has blended the fields of protein engineering, virology and genetics to generate several gene therapy platforms currently being evaluated in preclinical and clinical studies.

StrideBio raised nearly $16 million from investors in what it described as an oversubscribed round of venture capital last year and named a new CEO earlier this year.

“We are very excited to initiate this multi-target collaboration with Sarepta, an established leader in the development and commercialization of genetic medicines,” said Sapan Shah, Chief Executive Officer of StrideBio about the deal. “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.”

Hatteras Ventures in Durham is among StrideBio’s investors.

“StrideBio will receive a $48 million upfront payment in the form of cash and Sarepta stock, in addition to significant future development, regulatory and commercial milestones for the four programs,” the companies said.

“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 also agreed to invest in StrideBio’s next financing round.

Original Article Source: WRAL TechWire