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Research programme: genome editing therapeutics - Editas Medicine

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Drug Profile

Research programme: genome editing therapeutics - Editas Medicine

Alternative Names: LCA10 program

Latest Information Update: 28 Jun 2025

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At a glance

  • Originator Editas Medicine
  • Class Antiulcers; 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.

    No
  • New Molecular Entity No

Highest Development Phases

  • No development reported Alpha 1-antitrypsin deficiency; Cystic fibrosis; Duchenne muscular dystrophy; Eye disorders; Herpes simplex virus type 1 infections; Leber congenital amaurosis; Retinitis pigmentosa; Usher syndromes

Most Recent Events

  • 28 Jun 2025 No recent reports of development identified for preclinical development in Retinitis-pigmentosa in USA (Ophthalmic)
  • 12 May 2025 US Court of Appeals for the Federal Circuit remands CRISPR patent interference to Patent Trial and Appeal Board for further review
  • 08 May 2024 Editas Medicine initiates appeal of the Patent Trial and Appeal Board decision favouring Broad Institute for CRISPR patent interference in the USA

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