Sarepta Therapeutics (SRPT) announced early but encouraging results Wednesday from its second gene therapy program targeting a rare, muscle-wasting disease.
A one-time infusion of a corrective gene, delivered using an engineered virus, produced meaningful levels of a protein — an average of 51 percent — suggesting (but not yet proving) that muscle function can be improved, Sarepta said.
The three patients enrolled in the clinical trial have a form of limb-girdle muscular dystrophy, an inherited disorder that causes muscles around the hips and shoulders to weaken and atrophy over time.
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