Date: 2018-10-05

Type of information: Licensing agreement

Compound: AAV1 neurotrophin 3 (NT-3) gene therapy

Company: Sarepta Therapeutics (USA - MA) Nationwide Children’s Hospital (USA - OH)

Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases

Type agreement: licensing

Action mechanism:

  • gene therapy.

Disease: Charcot-Marie-Tooth (CMT) neuropathies including CMT type 1A


  • • On October 5, 2018, -- Sarepta Therapeutics announced 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.
  • 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.
  • Dr. Sahenk is an 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, and a lead researcher in the anatomical and molecular basis of nerve degeneration and impaired regeneration in hereditary peripheral neuropathies.

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