Trial Profile

A confirmatory pivotal trial of GNT-0004 in Europe

Status: Planning

Phase of Trial: Phase III

Latest Information Update: 08 Sep 2025

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At a glance

28 Jul 2025 According to a Genethon media release, Phase 3 double blind trials will begin in August and September in the UK and France
28 Jul 2025 According to a Genethon media release, based on data from Phase 1/2 study the company has received phase 3 authorizations.
28 Jul 2025 According to a Genethon media release, the company has has received approvals from regulatory authorities, MHRA and EMA*, to begin pivotal Phase 3 clinical trials in France and the UK of its gene therapy, GNT0004, for Duchenne muscular dystrophy (DMD).

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