RARE Daily

Sarepta Enters Licensing Agreement for Nationwide Children’s Gene Therapy Program for Limb-Girdle Muscular Dystrophy

August 5, 2021

Rare Daily Staff

Sarepta Therapeutics said 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, to treat Limb-girdle muscular dystrophy type 2A, developed by the Abigail Wexner Research Institute at Nationwide Children’s Hospital.

Photo: Louise Rodino-Klapac, Sarepta’s executive vice president and chief scientific officer

Limb-girdle muscular dystrophy (LGMD) 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. LGMD type 2A, also known as calpainopathy, 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.

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 deliver treatment systematically and robustly to skeletal muscle, including the diaphragm, making it an ideal candidate to treat muscle disease.

“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.”

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