Research programme: gene editing therapeutics - Sarepta Therapeutics

Drug Profile

Research programme: gene editing therapeutics - Sarepta Therapeutics

Alternative Names: CRISPR/Cas9 gene editing therapies - Sarepta; CRISPR/Cas9 gene therapies - Sarepta

Latest Information Update: 06 Nov 2017

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At a glance

  • Originator Duke University
  • Class Gene therapies
  • Mechanism of Action Gene modulators
  • 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

  • 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)
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