Sarepta Therapeutics adds LGMD2A to their pipeline

Today, Sarepta Therapeutics, Inc. announced that it has signed an agreement with Nationwide Children’s Hospital giving Sarepta the exclusive option to Nationwide’s gene therapy candidate to treat limb girdle muscular dystrophy type 2A (LGMD2A), a form of calpainopathy. This program is currently in preclinical trials that are being led by Dr. Zarife Sahenk, attending neurologist at Nationwide Children’s Hospital, Director of Clinical and Experimental Neuromuscular Pathology at The Research Institute at Nationwide Children’s Hospital, and Professor of Pediatrics, Pathology and Neurology at The Ohio State University College of Medicine. These preclinical trials test the safety and efficacy of CAPN3 gene delivery throughout the body of a mouse model of LGMD2A. These results, if positive, will provide proof of principal data for CAPN3 gene therapy in LGMD2A patients.

 

Dr. Sahenk’s preclinical program was funded in part by Coalition to Cure Calpain 3 (C3) under the auspices of our Gene Therapy Initiative.

 

Sarepta has five other LGMD programs directed at LGMD2E, LGMD2D, LGMD2C, LGMD2B, and LGMD2L. Similar to these programs, the LGMD2A program employs the rh74 vector to deliver treatment to skeletal muscle. Positive early results from the LGMD2E clinical trial support this approach for other LGMDs.

 

“We are excited that Sarepta, a leader in developing genetic therapies for rare neuromuscular diseases, is adding LGMD2A to its LGMD pipeline,” said Dr. Jennifer Levy, C3 Scientific Director.  “We are proud to support the work of Dr. Sahenk, and we are hopeful that this program will ultimately lead to a treatment for those affected by LGMD2A.”