Sarepta has been signing a series of new R&D pacts since its controversial Duchenne muscular dystrophy (DMD) drug approval last fall, and today it’s penned a deal for one of the hottest research areas around: CRISPR.
In a deal with Duke University (financial terms were not disclosed), Sarepta gains an option for an exclusive license for this form of the gene-editing tech, which comes out of the lab of Charles Gersbach, Ph.D.
Gersbach’s approach is to restore dystrophin expression by removing, or “excising,” exons from the dystrophin gene. This also entails excising exons that could help treat the majority of the DMD patient population. Sarepta’s current med, Exondys 51, can only treat a small number of DMD patients with a certain mutation.
Sarepta says it will collaborate with Gersbach’s lab to “advance the CRISPR platform and take the lead on clinical development.” It’s signed a series of similar deals, including in gene therapy, to help it create better, broader treatments for the muscle-wasting disease in the future.
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