- Sarepta Therapeutics (NASDAQ:SRPT) signs a research collaboration agreement with Duke University under which it has the option to exclusively license the intellectual property related to the use of gene editing technology CRISPR/Cas9 to restore dystrophin expression in patients with Duchenne muscular dystrophy (DMD).
- The company will collaborate with the laboratory of Charles A. Gersbach, Ph.D., to advance the platform and will take the lead on clinical development.
- Dr. Gerbach says, “Although early, CRISPR technology represents hope for a large percentage of individuals with DMD. 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."
- Financial terms are not disclosed.
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