Academia, Pre-clinical

Expanding its preclinical attack on Duchenne MD, Sarepta grabs a license for Duke’s CRISPR tech

Sarepta $SRPT announced this morning that it’s teaming up with Duke University to explore new CRISPR-based methods in the hunt for an effective Duchenne muscular dystrophy therapy.

The company says it’s specifically interested in DMD, the rare and lethal condition it aimed to treat with its drug Exondys51. But remember, Sarepta got a controversial regulatory approval for Exondys51 based on data critics say never met a proper threshold for efficacy, and which they argue led the FDA to an erroneous conclusion.

Charles Gersbach

Now, the Cambridge, MA-based company is making a show of effort for DMD patients with this new research collaboration with Duke. The collaboration gets Sarepta an exclusive license to IP and technology related to CRISPR/Cas9. The tech was developed in the lab of Charles Gersbach, whose approach is to restore dystrophin expression by removing exons from the dystrophin gene, which can be applied to DMD patients.

“Although early, CRISPR technology represents hope for a large percentage of individuals with DMD,” said Gersbach, an associate professor in Duke’s department of biomedical engineering. “Excising certain exons has the potential to correct a majority of DMD mutations. 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.”

This gene editing collaboration is just one of several next-generation efforts under way to treat this rare neuromuscular disease. Gene therapies, which aim to deliver a functional copy of the dystrophin gene missing in afflicted boys, are now underway at Solid Biosciences, Pfizer, and Sarepta too.


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