Drug Profile
Research programme: gene editing therapeutics - Sarepta Therapeutics
Alternative Names: CRISPR/Cas9 gene editing therapies - Sarepta; CRISPR/Cas9 gene therapies - SareptaLatest Information Update: 22 Mar 2022
Price :
$50
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At a glance
- Originator Duke University
- Developer Duke University; 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 No
Highest Development Phases
- Preclinical Duchenne muscular dystrophy
Most Recent Events
- 22 Mar 2022 Gene editing therapeutics are still in preclinical trials for Duchenne muscular dystrophy in USA (IV)
- 31 Oct 2017 Sarepta Therapeutics inters into a research collaboration agreement with the Duke University for use of gene editing CRISPR/Cas9 technology to develop therapeutics for Duchenne Muscular Dystrophy
- 31 Oct 2017 Preclinical trials in Duchenne muscular dystrophy in USA prior to October 2017 (IV)