Research programme: gene editing therapeutics - Harvard University/Sarepta Therapeutics
Latest Information Update: 04 Nov 2022
At a glance
- Originator Harvard University; Sarepta Therapeutics
- Class Gene therapies
- Mechanism of Action Dystrophin expression modulators; 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 No
Highest Development Phases
- Preclinical Duchenne muscular dystrophy
Most Recent Events
- 04 Nov 2022 Preclinical development is ongoing in USA (Sarepta Therapeutics, November 2022)
- 28 Sep 2022 No recent reports of development identified for preclinical development in Duchenne muscular dystrophy in USA (IM)
- 28 Sep 2022 No recent reports of development identified for preclinical development in Duchenne muscular dystrophy in USA (IV)