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

Sarepta Therapeutics (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, announced today it has signed an agreement with Nationwide Children’s Hospital (Nationwide Children’s) giving Sarepta the exclusive option to a Nationwide Children’s gene therapy candidate, neurotrophin 3 (NT-3), to treat Charcot-Marie-Tooth (CMT) neuropathies, including CMT type 1A.

As quoted in the press release:

CMT is a group of hereditary, degenerative nerve diseases that can affect motor skills, resulting in muscle weakness, and limiting patients’ ability to walk or use their hands. CMT is the most common inherited neuromuscular disorder, affecting over 2.8 million people worldwide. CMT type 1A is most often caused by an extra copy of the PMP22 gene, and affects approximately 50,000 patients in the U.S. alone. Currently, there are no available treatment options.

The clinical trial to test NT-3 gene therapy is planned to commence dosing in 2019 for CMT type 1A. The delivery of the NT-3 gene may have applicability to other sub-types of CMT in addition to other muscle-wasting diseases. Pre-clinical research has shown the ability of the NT-3 gene construct to regenerate nerves.  Further research is under way to explore its potential.

Dr. Zarife Sahenk, M.D., Ph.D. is the founder of the NT-3 program at Nationwide Children’s. Over the past 15 years, Dr. Sahenk has accumulated a large body of evidence to support the efficacy of neurotrophin NT-3 to improve nerve regeneration and myelination, associated with increased Schwann Cell (SC) survival and differentiation along with the normalization of axonal neurofilament (NF) cytoskeleton.

Click here to read the full press release.