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Sarepta options CMT gene therapy candidate from NCH

Executive Summary

Sarepta Therapeutics Inc. gained an exclusive option to Nationwide Children's Hospital's neurotrophin 3 (NT-3) gene therapy candidate for Charcot-Marie-Tooth (CMT) disease, a group of rare, inherited, degenerative motor and sensory neuropathies that affects the peripheral nerves and can lead to muscle wasting and numbness.
Deal Industry
  • Pharmaceuticals
  • Biotechnology
    • Gene Therapy, Cell Therapy
Deal Status
  • Final
Deal Type
  • Alliance
    • R&D and Marketing (Licensing)

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