Gene therapy for Duchenne is centered on the goal of successfully introducing into a muscle cell the correct genetic code, or recipe, necessary to make the dystrophin protein. Because dystrophin is such a large protein, smaller versions (referred to as micro-dystrophins) are inserted into the delivery vehicle. Viral delivery using the adeno-associated virus (AAV) harnesses the virus’s natural ability to deposit genetic material right to the muscle cell nucleus. The result of this viral “infection” would be the successful recoding of each muscle cell in the patient’s body so that a smaller but functional dystrophin protein could be made.

Status

• EMBARK – A phase 3 placebo controlled clinical trial using commercial grade product is recruiting participants with mutations
• ENDEAVOR – A phase 1 clinical trial using commercial grade product is recruiting by invitation

Sponsor

This program is sponsored by Sarepta Therapeutics and has been partially funded by Parent Project Muscular Dystrophy.

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