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Sarepta's Gene Therapy for Duchenne Muscular Dystrophy Clears a Major Safety Hurdle

By Cory Renauer – Updated Jun 22, 2020 at 11:46AM

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Early safety data for the potential one-shot cure for the lethal muscle wasting disorder looks encouraging.

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' (SRPT -0.03%) SRP-9001 will become a safe treatment option for boys born with Duchenne muscular dystrophy (DMD). 

Positive clinical trial results 

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.

Scientist reading a report.

Image source: Getty Images.

About SRP-9001's efficacy

That lack of serious adverse events means the safety study was a success, and it looks like future outcome assessments will hit the mark as well. Investigators took muscle tissue samples 12 weeks after patients received SRP-9001 and found proof of micro-dystrophin generation in 82% of fibers sampled.

Creatine kinase (CK) leaks from damaged muscles into the bloodstream, but one year after treatment with SRP-9001, patients' CK levels were much lower than expected, with the exception of one who was probably a little too active just ahead of his 52-week assessment. Since the average age of these patients was less than 5 years old, getting them to sit still is much easier said than done.

Cory Renauer has no position in any of the stocks mentioned. The Motley Fool has no position in any of the stocks mentioned. The Motley Fool has a disclosure policy.

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