SGT 001

Drug Profile

SGT 001

Alternative Names: SGT-001

Latest Information Update: 22 May 2017

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At a glance

  • Originator University of Missouri-Columbia
  • Developer Solid GT; University of Missouri-Columbia
  • Class Gene therapies
  • Mechanism of Action Dystrophin expression stimulants; Gene transference
  • Orphan Drug Status

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

    Yes - Duchenne muscular dystrophy
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Duchenne muscular dystrophy

Most Recent Events

  • 08 Jun 2017 Solid Biosciences plans an adaptive phase I/II trial to evaluate the safety and efficacy of intravenous SGT 001 for Duchenne muscular dystrophy in USA, in the second half of 2017 (Solid Biosciences website, September 2017)
  • 11 May 2017 Pharmacokinetics, immunogenicity and pharmacodynamics data from preclinical studies in Duchenne muscular dystrophy released by Solid Biosciences
  • 24 Oct 2016 SGT 001 receives Orphan Drug status for Duchenne muscular dystrophy in European Union
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