Research programme: Galgt2 gene therapies - Nationwide Children's Hospital / Sarepta

Drug Profile

Research programme: Galgt2 gene therapies - Nationwide Children's Hospital / Sarepta

Alternative Names: GalNAc tranferase gene therapies

Latest Information Update: 20 Jan 2017

Price : $50

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.

  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Duchenne muscular dystrophy

Most Recent Events

  • 10 Jan 2017 Galgt2 gene therapy licensed to Sarepta Therapeutics
  • 10 Jan 2017 Nationwide Children's Hospital and Sarepta Therapeutics plan clinical trials for Duchenne muscular dystrophy during 2017
  • 01 Jan 2017 Preclinical trials in Duchenne muscular dystrophy in USA (Parenteral)
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