Research programme: gene editing neuromuscular disorder therapeutics - Genevant Sciences/Sarepta Therapeutics
Latest Information Update: 18 Jan 2021
At a glance
- Originator Genevant Sciences; Sarepta Therapeutics
- Class Gene therapies
- Mechanism of Action Gene modulators
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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 Yes
Highest Development Phases
- Research Duchenne muscular dystrophy; Neuromuscular disorders
Most Recent Events
- 13 Jan 2021 Genevant Sciences and Sarepta Therapeutics collaborate to develop lipid nanoparticle-bsed gene editing therapeutics for Duchenne muscular dystrophy and other neuromuscular disorders
- 13 Jan 2021 Early research in Duchenne muscular dystrophy in Canada and USA (Parenteral)
- 13 Jan 2021 Early research in Neuromuscular disorders in Canada and USA (Parenteral)