Research programme: gene editing therapeutics - Exonics Therapeutics

Drug Profile

Research programme: gene editing therapeutics - Exonics Therapeutics

Alternative Names: CRISPR/Cas9 gene editing therapy - Exonics Therapeutics

Latest Information Update: 28 Feb 2017

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At a glance

  • Originator Exonics Therapeutics
  • 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.

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Duchenne muscular dystrophy; Neuromuscular disorders

Most Recent Events

  • 28 Feb 2017 Preclinical trials in Duchenne muscular dystrophy in USA before February 2017 (Exonics pipeline and CureDuchenne pipeline, February 2017)
  • 28 Feb 2017 Preclinical trials in Neuromuscular disorders in USA (Exonics pipeline and CureDuchenne pipeline, February 2017)
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