SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) is well tolerated and leads to an overall improvement in motor ability compared to baseline in patients with Duchenne muscular dystrophy (DMD), according to results from the SRP-9001-101 study reported at the World Muscle Society (WMS) 2021 Virtual Congress. These improvements were maintained over 3 years, meaning the response was durable.

Roche and partner Sarepta, who are codeveloping SRP-9001, also reported interim results from the ongoing, open-label, phase 1b study SRP-9001-103, which showed that SRP-9001 treatment resulted in robust microdystrophin expression in 11 boys with DMD, aged 4 to 7 years, who were still able to walk. The investigators identified no new safety signals.

Moreover, the ongoing, randomized, double-blind, placebo-controlled SRP-9001-102 study met its primary biological endpoint of change in microdystrophin protein expression from baseline following a single dose of SRP-9001. The study showed that boys treated with SRP-9001 had an increase in North Star Ambulatory Assessment (NSAA) total score compared to those treated with a placebo at 48 weeks, but the difference between the groups was not statistically significant. No new safety signals were identified during the study.

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A new phase 3 clinical trial of SRP-9001 is planned for the end of this year.

“The data from SRP-9001 have helped to optimize the design of the upcoming phase III trial for DMD,” Levi Garraway, MD, PhD, the chief medical officer and head of global product development at Roche, said in a press release. “Our goal is to continue to lead the way in developing transformative medicines for neuromuscular diseases.” 

SRP-9001 is an experimental gene therapy designed to deliver the gene coding for microdystrophin directly to the skeletal and cardiac muscles. The aim is to drive the production of microdystrophin protein and achieve a durable clinical response.


Roche presents new data at World Muscle Society (WMS) 2021 highlighting new advances for people living with rare neuromuscular disorders. News release. Roche; September 24, 2021.