Drug Profile

GNT 0004

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

Latest Information Update: 25 Apr 2024

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At a glance

Originator Genethon
Developer Genethon; Sarepta Therapeutics
Class Gene therapies
Mechanism of Action 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 I/II Duchenne muscular dystrophy

Most Recent Events

23 Apr 2024 Efficacy and adverse event data from a phase I/II/III trial in Duchenne muscular dystrophy released by Genethon
23 Apr 2024 Genethon plans a pivotal trial for Duchenne muscular dystrophy in Europe
22 Mar 2022 GNT 0004 is still in preclinical trials for Duchenne muscular dystrophy in France (IV), prior to March 2022

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