Galgt2 gene therapy - Nationwide Children's Hospital/Sarepta Therapeutics

Drug Profile

Galgt2 gene therapy - Nationwide Children's Hospital/Sarepta Therapeutics

Alternative Names: GalNAc tranferase gene therapies; rAAVrh74.MCK.GALGT2

Latest Information Update: 15 Nov 2017

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At a glance

  • Originator Nationwide Children's Hospital
  • Developer Nationwide Children's Hospital; Sarepta Therapeutics
  • 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.

    No
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Duchenne muscular dystrophy

Most Recent Events

  • 03 Nov 2017 Sarepta Therapeutics plans a phase I/IIa trial for rAAVrh74.MCK.GALGT2 in Duchenne muscular dystrophy in USA in 2017
  • 03 Nov 2017 US FDA approves IND application for rAAVrh74.MCK.GALGT2 in Duchenne muscular dystrophy
  • 01 Nov 2017 Phase-I/II clinical trials in Duchenne muscular dystrophy (In children, In adolescents, In adults) in USA (Intra-arterial) (NCT03333590)
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