Sarepta Announces Agreement With Nationwide Children’s Hospital for Rights to its Gene Therapy Program

Sarepta Therapeutics, Inc. recently announced it has recently signed an agreement with the Research Institute at Nationwide Children’s Hospital (Nationwide Children’s) giving Sarepta the exclusive option to a Nationwide Children’s gene therapy candidate, calpain 3 (CAPN-3), to treat Limb-girdle muscular dystrophy type 2A (LGMD2A).

LGMDs represent a group of distinct genetic neuromuscular diseases with a generally common set of symptoms, including progressive, debilitating weakness, and wasting that begins in muscles around the hips and shoulders before progressing to muscles in the arms and legs. Many LGMD sub-types are seriously life-limiting and often life-ending diseases. Also known as calpainopathy, LGMD2A is caused by mutations in the CAPN-3 gene and is the most common type of LGMD, accounting for almost a third of cases.

Like Sarepta’s micro-dystrophin and five other LGMD programs, the LGMD2A program employs the AAVrh74 vector, designed to systematically and robustly deliver treatment to skeletal muscle, including the diaphragm, without promiscuously crossing the blood brain barrier, making it an ideal candidate to treat muscle disease.

The CAPN-3 program is currently in pre-clinical trials. The program is led by Zarife Sahenk, MD, PhD, an attending neurologist at Nationwide Children’s, Director of Clinical and Experimental Neuromuscular Pathology at The Research Institute at Nationwide Children’s and Professor of Pediatrics, Pathology and Neurology at The Ohio State University College of Medicine.

“We are pleased to expand and deepen our working relationship with Nationwide Children’s and Dr. Sahenk, with whom we are already working on a gene therapy candidate to treat Charcot-Marie-Tooth. With six LGMD gene therapy programs now in our portfolio, our commitment and investment in research for this group of neuromuscular diseases is unparalleled,” said Doug Ingram, Sarepta’s President and Chief Executive Officer. “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. We continue to fuel our gene therapy development engine aimed at building an enduring model that delivers potentially transformative therapies to treat genetically based diseases.”

“LGMD2A is the most common form of limb-girdle muscular dystrophy and its relentless progression causes patients to lose the ability to walk in early adulthood,” said Dr. Zahenk. “Our preclinical work suggests that a gene therapy approach has the potential to help those living with LGMD2A and we look forward to collaborating with Sarepta to advance this program in the clinic.”

Limb-girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begins in muscles around the hips and shoulders before progressing to muscles in the arms and legs. Sarepta’s six LGMD gene therapy programs in development now include LGMD2E, LGMD2D, LGMD2C, LGMD2B, LGMD2L and LGMD2A.

Sarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for Limb-girdle muscular dystrophy diseases (LGMD), Charcot-Marie-Tooth (CMT), MPS IIIA, Pompe and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Company’s programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases. For more information, visit