Research programme: genome editing therapeutics - Editas Medicine

Drug Profile

Research programme: genome editing therapeutics - Editas Medicine

Alternative Names: EDIT 101; LCA10 program

Latest Information Update: 25 Oct 2017

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At a glance

  • Originator Editas Medicine
  • Class Antianaemics; Eye disorder therapies; 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.

    Yes - Leber congenital amaurosis
  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Alpha 1-antitrypsin deficiency; Cystic fibrosis; Duchenne muscular dystrophy; Eye disorders; Haematological disorders; Leber congenital amaurosis

Most Recent Events

  • 19 Oct 2017 Pharmacodynamics data from a preclinical trial in Leber Congenital Amaurosis released by Editas Medicine
  • 26 Sep 2017 EDIT 101 receives Orphan Drug status for Leber congenital amaurosis in European Union
  • 25 Jul 2017 University of California, University of Vienna, and Charpentier submit appellate brief seeking reversal of decision by PTAB, which dismissed interference proceedings against Broad Institute, MIT, and Harvard University for a patent related to CRISPR-Cas9 genome editing technology, in USA
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