Sarepta Acquires Rights to CMT Gene Therapy Program from Nationwide Children’s Hospital
Nationwide Children’s Hospital (NCH) has granted the exclusive rights of neutotrophin 3 (NT-3), its gene therapy candidate for the treatment of Charcot-Marie-Tooth (CMT) neuropathies, to Sarepta Therapeutics. Researchers plan to start a Phase 1 trial for the NT-3 therapy in CMT type 1A (CMT1A) patients next year.