Sarepta Therapeutics has announced positive results from their gene therapy, SRP-9001, under investigation for Duchenne muscular dystrophy.
Preliminary data based upon four boys demonstrated the therapy to be safe and tolerable at one year in their clinical trial. Results were published in JAMA Neurology and further support the potential for SRP-9001 to provide clinically meaningful functional improvements in Duchenne patients according to the manufacture. Additional results will be coming in early 2021.
The phase two clinical trial is currently running in the USA. It is not clear yet if there will be trial opportunities in Canada. Still, Defeat Duchenne Canada remains committed to advocating for trials, like these, in Canada and keeping our community informed as plans develop.
To learn more about this trial, we encourage you to speak to your neuromuscular team and go to:
- Search for SRP-9001 (note: the current trial is limited to boys aged 4-7 years)
About Gene Therapy & SRP-9001
Gene therapy is a critical therapeutic approach. It works by correcting the defected DNA by delivering a synthetic dystrophin gene into the muscle cells that make a functional protein surrogate. SRP-9001 is an investigational gene transfer therapy intended to provide its micro-dystrophin-encoding gene to muscle tissue for the targeted production of micro-dystrophin protein.
Sarepta has 1 of 3 micro or mini dystrophins currently in clinical trials.Pfizer is working on PF-06939926, and Solid Biosciences is working on SGT-001. Click on the links to learn more about these molecules and their trials.
About Sarepta Therapeutics
Sarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for limb-girdle muscular dystrophy diseases (LGMD), Charcot-Marie-Tooth (CMT), MPS IIIA and other CNS-related disorders, totalling over 20 therapies in various stages of development. The Company’s programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases.