Sarepta Acquires Investigational Gene Therapy for LGMD2A
Sarepta Acquires Investigational Gene Therapy for LGMD2A
Sarepta Therapeutics has executed an exclusive license agreement for a gene therapy candidate that aims to treat limb-girdle muscular dystrophy type 2A (LGMD2A), the company announced.
The investigational therapy, called calpain 3 or CAPN-3, was developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital, in Columbus, Ohio.
The deal followed a number of successful preclinical and safety studies led by Zarife Sahenk, MD, PhD, an attending neurologist at the hospital.
