Research programme: Duchenne muscular dystrophy gene therapy - Solid GT

Drug Profile

Research programme: Duchenne muscular dystrophy gene therapy - Solid GT

Alternative Names: SGT-001

Latest Information Update: 26 Oct 2016

Price : $50

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 expression stimulants
  • Orphan Drug Status

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

    Yes - Duchenne muscular dystrophy
  • 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.

    No
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Duchenne muscular dystrophy

Most Recent Events

  • 24 Oct 2016 SGT 001 receives Orphan Drug status for Duchenne muscular dystrophy in European Union
  • 24 Oct 2016 SGT 001 receives Orphan Drug status for Duchenne muscular dystrophy in USA
  • 24 Oct 2016 Solid GT plans a clinical trial for SGT 001 in USA (Parenteral)
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