Sarepta reveals positive results in limb-girdle gene therapy trial. What does that mean for DMD?
Sarepta on Monday unveiled a small batch of positive data for one of the gene therapies it bought out last year, sketching a path toward a pivotal study in 2021 and helping bolster the company’s broader gene therapy ambitions.
The gene therapy, known as SRP-9003, is designed to treat a rare degenerative disorder called limb-girdle muscular dystrophy by boosting production of a protein patients are missing, called beta-sarcoglycan. It was part of a basket of treatments Sarepta acquired in its $165 million-buyout of Myonexus last year. The biotech already showed a low dose of the therapy could lead to some increased expression of the missing protein and some improved muscle function in 3 kids, but it was unclear how long those results would last or if a higher dose would lead to better results.
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