Sarepta goes back to the gene therapy well at Nationwide Children’s for its latest muscle-wasting program
Sarepta $SRPT is adding a preclinical gene therapy program to the pipeline, picking it up from their close contacts at Nationwide Children’s Hospital. The treatment is calpain 3 (CAPN-3), for Limb-girdle muscular dystrophy (LGMD) type 2A, which triggers the disease associated with weakness in muscles in the hips and shoulders.
This is the biotech’s 6th LGMD effort, which uses an AAVrh74 vector to target skeletal muscle. The work so far has been led by Zarife Sahenk, director of Clinical and Experimental Neuromuscular Pathology at the Research Institute at Nationwide Children’s in Ohio.
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