Sarepta Therapeutics Executes Licensing Agreement for Gene Therapy Program from Nationwide Children’s Hospital to Treat Limb-Girdle Muscular Dystrophy Type 2A
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).
- Treatment plans for LGMD2A are currently limited to physical therapy, assistive devices and surgery for complications.
- Were 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 Childrens.
- Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing.
- Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.