Sarepta's Gene Therapy for Duchenne Muscular Dystrophy Clears a Major Safety Hurdle
A once-and-done gene therapy candidate that could halt the progression of a lethal muscle-wasting disorder took an important step on the path toward approval Monday. Results of a clinical trial published by the Journal of the American Medical Association strongly suggest that Sarepta Therapeutics' (NASDAQ: SRPT) SRP-9001 will become a safe treatment option for boys born with Duchenne muscular dystrophy (DMD).
Back in 2017, Sarepta began a safety study with four boys born with DMD, a rare inherited disorder caused by a mutated dystrophin gene. Without functional dystrophin to protect their muscles, DMD patients inflict muscle damage on themselves with every movement, breath, and heartbeat. SRP-9001 is a non-replicating adenovirus that delivers a portion of the working dystrophin gene to muscle cells. So far, none of the four participants have reported adverse events serious enough to require hospitalization.
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