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Sarepta Therapeutics: SRP-9001 (ENDEAVOR) Gene Therapy Study Update

May 20, 2021

Defeat Duchenne Canada is pleased to share an update from Sarepta Therapeutics on their Gene Therapy for DMD SRP-9001. The results from the first 11 patients enrolled showed an increase in micro-dystrophin expression and a consistent safety profile.

“We are delighted by these seminal results from the ENDEAVOR Study, our first trial results with SRP-9001 made by our commercial-scale manufacturing process. These data show strong transduction of the micro-dystrophin gene, resulting in robust expression of the properly localized micro-dystrophin protein, and did so with no new or unexpected safety signals. In addition to characterizing and differentiating SRP-9001, these results confirm the extraordinary work done over the last two and a half years to build an at-scale gene therapy manufacturing process and corresponding analytics sufficient to meet the needs of the Duchenne population with what we believe will be a potentially life-changing therapy. Armed with these data, we will seek a meeting with the FDA with the goal of rapidly starting our registrational study.”

Doug Ingram, president and chief executive officer, Sarepta

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If you have any questions about this press release, please reach out to:

Nicola Worsfold

Director of Research and Advocacy

Defeat Duchenne Canada

nicola.worsfold@defeatduchenne.ca

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