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
Click the button below to read more about this announcement.
If you have any questions about this press release, please reach out to:
Nicola Worsfold
Director of Research and Advocacy
Defeat Duchenne Canada