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Research programme: gene editing therapeutics - CRISPR Therapeutics

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Drug Profile

Research programme: gene editing therapeutics - CRISPR Therapeutics

Alternative Names: CRISPR-Cas9 gene therapies

Latest Information Update: 28 Jan 2025

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At a glance

  • Originator CRISPR Therapeutics
  • Developer CRISPR Therapeutics; Genethon; University of Alabama at Birmingham; University of Florida; Vertex Pharmaceuticals
  • Class Antianaemics; Antidementias; Antifibrinolytics; Antihyperglycaemics; Antineoplastics; Gene therapies; Neuropsychotherapeutics
  • 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

  • Preclinical Duchenne muscular dystrophy; Hypophosphataemia; Limb girdle muscular dystrophies; Type 1 diabetes mellitus
  • No development reported Amyotrophic lateral sclerosis; Cystic fibrosis; Friedreich's ataxia; Glycogen storage disease type I; HIV infections; Myotonic dystrophy
  • Discontinued Cancer; Dementia; Mucopolysaccharidosis I

Most Recent Events

  • 28 Jan 2025 No recent reports of development identified for research development in HIV-infections in USA
  • 28 Aug 2023 No recent reports of development identified for research development in Myotonic dystrophy in USA
  • 27 Mar 2023 Vertex Pharmaceuticals in-licenses CRISPR-Cas9 genome editing technology from CRISPR Therapeutics for the treatment of Type 1 diabetes mellitus

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