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: 15 Jan 2018

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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
  • Research Herpes simplex virus type 1 infections

Most Recent Events

  • 08 Jan 2018 Early research in Herpes simplex virus type-1 infections in USA (Ophthalmic)
  • 11 Dec 2017 Pharmacodynamics data from preclinical studies in Haematological disorders presented at the 59th Annual Meeting of the American Society of Haematology (ASH-2017)
  • 19 Oct 2017 Pharmacodynamics data from a preclinical trial in Leber Congenital Amaurosis released by Editas Medicine
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