SGT 001

Drug Profile

SGT 001

Alternative Names: SGT-001

Latest Information Update: 23 Apr 2018

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At a glance

  • Originator University of Missouri-Columbia
  • Developer Solid GT
  • 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

  • Phase I/II Duchenne muscular dystrophy

Most Recent Events

  • 18 Apr 2018 Solid Biosciences receives a full clinical hold letter from the US FDA for the phase I/II IGNITE DMD trial
  • 14 Mar 2018 Solid Biosciences suspends patient enrolment after the company reported a Suspected Unexpected Serious Adverse Reaction (SUSAR) in the first dosed patient in a phase I/II trial in Duchenne muscular dystrophy (In children, In adolescents) in USA (IV) (NCT03368742)
  • 14 Mar 2018 US FDA puts clinical hold on the phase I/II IGNITE DMD trial in Duchenne muscular dystrophy (In children, In adolescents)
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