Sarepta announces gene therapy program for LGMD2A/R1

Today, Sarepta Therapeutics, Inc. announced that it executed an exclusive license agreement for an LGMD2A/R1 gene therapy program developed by Dr. Zarife Sahenk at Nationwide Children’s Hospital. Preclinical research conducted by Dr. Sahenk provided early proof of concept data for AAV-mediated gene replacement of CAPN3 to treat LGMD2A/R1. The program uses the same AAVrh74 vector that is currently used in Sarepta’s Duchenne muscular dystrophy and other LGMD gene therapy programs. Sarepta’s six LGMD programs include LGMD2A, LGMD2B, LGMD2C, LGMD2D, LGMD2E, and LGMD2L, which together represent more than 70 percent of known LGMD cases.

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

“We are excited that Sarepta, a leader in developing genetic therapies for rare neuromuscular diseases, is licensing the LGMD2A/R1 program,” 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/R1.”