Drug Profile
Research programme: gene editing therapeutics - Exonics Therapeutics
Alternative Names: CRISPR/Cas9 gene editing therapy - Exonics TherapeuticsLatest Information Update: 28 Sep 2022
Price :
$50
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At a glance
- Originator Exonics Therapeutics
- Developer Vertex Pharmaceuticals
- 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 No
Highest Development Phases
- No development reported Duchenne muscular dystrophy; Neuromuscular disorders
Most Recent Events
- 28 Sep 2022 No recent reports of development identified for preclinical development in Duchenne muscular dystrophy in USA (IM)
- 28 Mar 2021 No recent reports of development identified for preclinical development in Duchenne muscular dystrophy in USA (Parenteral)
- 28 Mar 2021 No recent reports of development identified for preclinical development in Neuromuscular-disorders in USA (Parenteral)