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The collaboration will involve developing therapies for at least eight CNS conditions as well as neuromuscular diseases.
Sarepta Therapeutics (NASDAQ:SRPT) said Thursday (November 14) that it is joining forces with privately held StrideBio to develop gene therapies targeting the central nervous system (CNS).
In a press release, the precision genetic medicine company said the collaboration between the two will involve developing therapies for at least eight CNS conditions as well as neuromuscular diseases.
StrideBio will work to develop the first four of the eight treatments for CNS conditions, which include Rett syndrome, Dravet syndrome, Angelman syndrome and Niemann-pick.
Sarepta will then pay StrideBio US$48 million in cash and stock — as well as future payments for development, regulatory and commercialization milestones — for the exclusive license to leverage StrideBio’s technology in developing the other four CNS gene therapy treatments.
Through this partnership, the companies will work to develop methods in order to “address re-dosing challenges in patients who have received (adeno-associated virus) AAV-delivered gene therapy,” according to the press release.
StrideBio will also have the opportunity to have co-commercialization rights to one of the therapies developed by Sarepta Therapeutics, should it go according to plan. Additionally, Sarepta will have the sole right to expand the collaboration, which could include four more therapeutic areas, for a cost of US$42.5 million, as well as future milestone payments.
“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,” Sarepta said.
Doug Ingram, president and CEO of Sarepta, said the partnership with StrideBio will help his company move forward in developing gene therapies for rare diseases.
“Our partnership with StrideBio expands our research portfolio by up to eight new targets,” he added.
Thursday’s announcement comes following the release of Sarepta’s Q3 2019 financial results on November 7. In the results, the company highlighted that it has raised its guidance from between US$365 million and US$375 million to a range of US$370 million to US$380 million.
Ingram also provided an update on the company’s gene therapy platform, noting that Sarepta increased the enrolment size and is now dosing patients at two sites. Ingram said dosing is expected to be completed by the end of the year.
Additionally, Ingram explained the company remains on track to begin its next trial by mid-2020 thanks to progress made on its gene therapy manufacturing capacity.
“While not without its challenges, 2019 has been one marked by a significant advancement of our platform,” Ingram commented.
Despite Thursday’s collaboration, shares of Sarepta dipped 3.55 percent to close the trading session at US$97.41. After-hours trading had bumped its share price down an additional 0.76 percent to US$96.67 as of 5:10 p.m. EST.
Based off 19 analyst ratings on TipRanks, Sarepta is currently ranked a “strong buy” with an average price target of US$191.19 — a 96 percent increase from its current price. It has a high estimate of US$270 and a low of US$160.
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Securities Disclosure: I, Jocelyn Aspa, hold no direct investment interest in any company mentioned in this article.