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scAAV1.tMCK.NTF3 - Nationwide Children's Hospital/Sarepta Therapeutics

Drug Profile

scAAV1.tMCK.NTF3 - Nationwide Children's Hospital/Sarepta Therapeutics

Alternative Names: AAV1.NT-3 gene therapy - Nationwide Children's Hospital; Neurotrophin-3 gene therapy - Nationwide Children's Hospital; rAAV1.NT-3 gene therapy - Nationwide Children's Hospital

Latest Information Update: 12 Oct 2018

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At a glance

  • Originator Nationwide Children's Hospital
  • Class Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Charcot-Marie-Tooth disease

Most Recent Events

  • 27 May 2019 Sarepta Therapeutics plans a clinical trial for Charcot-Marie-Tooth disease in 2019
  • 08 Oct 2018 Nationwide Children’s Hospital and Sarepta Therapeutics enter an option agreement for neurotrophin-3 gene therapy for the treatment of Charcot-Marie-Tooth disease
  • 01 Oct 2018 Phase-I/II clinical trials in Charcot-Marie-Tooth disease (In adolescents, In adults) in USA (IM) (NCT03520751)
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