© Reuters.  FDA OKs Nationwide Children's Hospital's IND for Sarepta's microdystrophin gene therapy for DMD; shares up 2%© Reuters. FDA OKs Nationwide Children’s Hospital’s IND for Sarepta’s microdystrophin gene therapy for DMD; shares up 2%

  • The FDA has signed off on Nationwide Children’s Hospital’s IND application seeking approval to begin a Phase 1/2a study of evaluating a new gene therapy candidate, AAVrh74.MHCK7.micro-Dystrophin, in patients with Duchenne muscular dystrophy (DMD).
  • The study is the first in DMD patients as young as three months old. Enrollment is now open.
  • The gene therapy uses a construct called MHCK7 which was optimized for the trial after demonstrating high levels of gene expression in preclinical studies.
  • Sarepta Therapeutics (SRPT +1.5%) has the option to global exclusive rights.
  • Now read: Sarepta: Uncertainties And Controversies Surround Its Trials

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