Drug Profile
GNT 0004
Alternative Names: GNT-0004; Micro-dystrophin program - GenethonLatest Information Update: 22 Mar 2022
Price :
$50
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At a glance
- Originator Genethon
- Developer Genethon; Sarepta Therapeutics
- Class Gene therapies
- Mechanism of Action Gene transference
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- Clinical Phase Unknown Duchenne muscular dystrophy
Most Recent Events
- 22 Mar 2022 GNT 0004 is still in preclinical trials for Duchenne muscular dystrophy in France (IV), prior to March 2022
- 22 Mar 2022 Clinical trials in Duchenne muscular dystrophy (IV), prior to March 2022
- 14 Jun 2018 Sarepta Therapeutics enters into a manufacturing agreement with Brammer Bio for its micro-dystrophin Duchenne muscular dystrophy and Limb girdle muscular dystrophy gene therapy programs