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SGT 003

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Drug Profile

SGT 003

Alternative Names: SGT-003

Latest Information Update: 23 Sep 2025

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At a glance

  • Originator Solid Biosciences
  • Class Gene therapies
  • Mechanism of Action Dystrophin replacements; 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

  • 08 Sep 2025 Solid Biosciences plans a phase III IMPACT DUCHENNE trial for Duchenne muscular dystrophy (In children) (IV, Infusion) in October 2025 (NCT07160634)
  • 12 Aug 2025 Adverse events data from a phase I/II trial in Duchenne muscular dystrophy released by Solid Biosciences
  • 15 May 2025 Phase-I/II clinical trials in Duchenne muscular dystrophy (In children) in Italy (IV)

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