Research programme: gene editing therapeutics - CRISPR Therapeutics

Drug Profile

Research programme: gene editing therapeutics - CRISPR Therapeutics

Alternative Names: CRISPR-Cas9 gene therapies; CTX 001

Latest Information Update: 19 Dec 2017

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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; Antineoplastics; Gene therapies; Neuropsychotherapeutics; Stem cell therapies
  • Mechanism of Action Gene modulators; Immunomodulators
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Beta-thalassaemia; Cancer; Haemoglobinopathies; Mucopolysaccharidosis I; Sickle cell anaemia
  • Research Amyotrophic lateral sclerosis; Cystic fibrosis; Dementia; Duchenne muscular dystrophy; Friedreich's ataxia; Glycogen storage disease type I; Unspecified

Most Recent Events

  • 31 Jan 2018 Vertex plans a phase I/II trial of CTX 001 for Sickle cell anaemia in USA in 2018
  • 13 Dec 2017 Vertex and CRISPR Therapeutics enter into a collaboration to co-develop and co-commercialise CTX 001 for Sickle cell disease and Beta-thalassemia
  • 12 Dec 2017 Preclinical data in Beta-thalassaemia and Sickle cell anaemia presented at the 59th Annual Meeting and Exposition of the American Society of Haematology (ASH-Hem-2017)
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