Gene Therapy

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.

Zarife Sahenk


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.

Sahenk is already working on a gene therapy candidate for Sarepta to treat Charcot-Marie-Tooth.

Doug Ingram

Researchers at Nationwide Children’s have achieved considerable attention for their work in gene therapies. Sahenk’s colleagues have been working on a micro-dystrophin strategy for Sarepta, while others have been engaged on a rival program for Audentes that encodes RNA and an antisense sequence together for exon-skipping. 

 “Recent positive early results from our LGMD2E program support expanding our development strategy to LGMD2A, as both programs utilize AAVrh74 vector, address sub-populations of LGMD, and address a well-characterized disease by directly replacing the missing protein which is the cause of the disease by transducing the native protein,” noted Sarepta CEO Doug Ingram.

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