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GNT 0004

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Drug Profile

GNT 0004

Alternative Names: GNT-0004; Micro-dystrophin program - Genethon

Latest Information Update: 16 Oct 2025

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At a glance

  • Originator Genethon
  • 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.

    No
  • New Molecular Entity No

Highest Development Phases

  • Phase III Duchenne muscular dystrophy

Most Recent Events

  • 09 Oct 2025 Efficacy, pharmacodynamics and adverse event data from a phase I/II/III trial in Duchenne muscular dystrophy released by Genethon
  • 01 Oct 2025 Phase-III clinical trials in Duchenne muscular dystrophy (In children) in France (IV)
  • 28 Jul 2025 Genethon plans a pivotal phase III trial for Duchenne muscular dystrophy (In children) in France, in September 2025

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