Sarepta Therapeutics Enters Collaboration with Duke University to Develop Gene Editing to Treat DMD

Rare Daily Staff

Sarepta Therapeutics said it signed a research collaboration agreement with Duke University that grants the company an option to an exclusive license to intellectual property and technology related to CRISPR/Cas9 technology as a potential treatment for Duchenne muscular dystrophy, a progressive and fatal muscle disease.

The technology was developed in the laboratory of Charles Gersbach, an associate professor in Duke University’s Department of Biomedical Engineering. It is intended to be used to restore dystrophin expression by removing exons from the dystrophin gene. This includes a strategy to excise exons potentially enabling treatment for a majority of the DMD patient population.  The faulty gene in DMD results in abnormal express dystrophin, the key protein missing in children with Duchenne that helps stabilize and protect muscle fibers.

Under the terms of the agreement, Sarepta will collaborate with Gersbach’s lab to advance the CRISPR platform and take the lead on clinical development. The financial terms of were not been disclosed.

“Gene editing has the potential to revolutionize the treatment of diseases with genetic mutations. We are particularly excited about the potential it holds for DMD patients,” said Douglas Ingram, Sarepta’s president and CEO. “Today’s agreement exemplifies our strategy of investing in and advancing a multi-faceted 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.”

Gersbach said while the CRISPR technology is early in development, it represents hope for a large percentage of individuals with DMD.

“Excising certain exons has the potential to correct a majority of DMD mutations,” he said. “Toward that goal, we’ve shown in mouse models that we can excise exons from the dystrophin gene, leading to restoration of a functional dystrophin protein and improvements in muscle strength.”

November 1, 2017