Benzinga | Aug 4, 2021 09:28AM EDT

Sarepta Therapeutics Earlier Announced Execution Of Licensing Agreement For Gene Therapy Program From Nationwide Children's Hospital To Treat Limb-Girdle Muscular Dystrophy Type 2A; Financial Details Not Disclosed

-- Limb-girdle muscular dystrophy type 2A is the most common form of LGMD, accounting for a third of LGMD diagnoses

-- Sarepta's unrivaled portfolio of investigational gene therapies for LGMD offers the potential to address six LGMD subtypes, which together represent more than 70% of all known LGMDs

CAMBRIDGE, Mass., Aug. 04, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that upon completion of a number of preclinical and safety studies, it had executed an exclusive license agreement for an investigational gene therapy candidate, calpain 3 (CAPN-3), to treat Limb-girdle muscular dystrophy type 2A (LGMD2A), developed by the Abigail Wexner Research Institute at Nationwide Children's Hospital (Nationwide Children's).

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 significantly 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.

"Treatment plans for LGMD2A are currently limited to physical therapy, assistive devices and surgery for complications. We're excited about the opportunity to transform patient care for this significantly life-limiting disease by advancing the CAPN-3 program following extensive pre-clinical work by the team at Nationwide Children's. Preclinical research conducted to date has provided early proof of concept for CAPN-3 in LGMD2A and supports further advancement," said Louise Rodino-Klapac, Sarepta's executive vice president and chief scientific officer. "We intend to build off the knowledge we have gained from our lead investigational gene transfer programs for Duchenne muscular dystrophy and LGMD2E, as the CAPN-3 program also uses the AAVrh74 vector to address another well-characterized genetic disease. Sarepta's commitment and research investment in LGMD is unparalleled and we continue to work towards advancing all of our LGMD programs as quickly as possible."

Like SRP-9001, Sarepta's lead investigational gene transfer therapy for Duchenne muscular dystrophy, and the Company's five other LGMD programs, the LGMD2A program uses the AAVrh74 vector, designed to systematically and robustly deliver treatment to skeletal muscle, including the diaphragm, making it an ideal candidate to treat muscle disease.

The preclinical work on the CAPN-3 program in LGMD2A has been led by Zarife Sahenk, M.D., Ph.D., 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.

About Limb-girdle Muscular Dystrophy

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 include LGMD2E, LGMD2D, LGMD2C, LGMD2B, LGMD2L and LGMD2A, which together represent more than 70 percent of known LGMD cases.

About Sarepta Therapeutics

Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visit www.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.