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: 10 Aug 2018

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At a glance

  • Originator Editas Medicine
  • Class Antianaemics; Antivirals; 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 No

Highest Development Phases

  • Preclinical Alpha 1-antitrypsin deficiency; Cystic fibrosis; Duchenne muscular dystrophy; Eye-Disorders; Haematological disorders; Leber congenital amaurosis
  • Research Herpes simplex virus type 1 infections

Most Recent Events

  • 06 Aug 2018 EDIT 101 licensed to Allergan worldwide
  • 06 Aug 2018 Editas Medicine announces intention to submit an IND application for Leber Congenital Amaurosis in October 2018
  • 06 Aug 2018 Editas submits data package for human gene transfer clinical protocol registration to the United States National Institutes of Health (NIH) for potential review by the Recombinant DNA Advisory Committee for a potential trial in Leber congenital amaurosis
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