Sarepta Joins Duke’s CRISPR for New DMD Therapies


Sarepta is a commercial stage biopharmaceutical company which primarily focuses on finding and developing innovative ribonucleic acid (RNA) targeted therapies. Since the highly debated approval of its Duchenne muscular dystrophy (DMD) drug Exondys51 last fall, the company has been contracting a sequence of new research and development agreements. Most recently, Sarepta landed a deal in the sought-out area of Clustered Regularly Interspaced Short Palindromic Repeats or CRISPR.

In the deal with Duke University, Sarepta acquires the opportunity for an exclusive license for this form of the gene-editing technology. This technology comes from the lab of Charles Gersbach, Ph.D. Gersbach’s method is to restore dystrophin expression by removing exons from the dystrophin gene. By eliminating the exons this can add an advantage in treating most of the DMD patient population. Sarepta’s Exondys51 is limited in its treatment of DMD patients for only a specific mutation.

Douglas Ingram, CEO of Sarepta said, “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.”

Sarepta stated that the company will work diligently with Gersbach’s lab to “advance the CRISPR platform and take the lead on clinical development.” The company has entered comparable R&D agreements, which includes one for gene therapy with the hopes to generate more wide-ranging treatments for DMD patients.

“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” Ingrim said.

Adding, “Gene editing has the potential to revolutionize the treatment of diseases with genetic mutations.


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