Sarepta turns to Duke’s CRISPR work for next-gen DMD therapies

The CRISPR tech could help more DMD patients than Sarepta's current drug.

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.


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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.

RELATED: Sarepta signs another Duchenne gene therapy pact as it aims for wider treatment

“Gene editing has the potential to revolutionize the treatment of diseases with genetic mutations,” said Douglas Ingram, Sarepta’s new CEO, in a release. “We are particularly excited about the potential it holds for DMD patients. We will work closely with Gersbach, a pioneer in applying the CRISPR technology to treat Duchenne, to advance a program that builds upon the established body of research by Gersbach and his team.

“Today’s agreement exemplifies our strategy of investing in and advancing a multifaceted array of potential therapies for the largest number of individuals with DMD by leveraging our own research and development efforts, as well as forging external partnerships with the field’s best and brightest minds.”

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