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Exagamglogene autotemcel - CRISPR Therapeutics/Vertex Pharmaceuticals

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Drug Profile

Exagamglogene autotemcel - CRISPR Therapeutics/Vertex Pharmaceuticals

Alternative Names: Autologous CRISPR-Cas9 modified CD34+ hHSPCs - CRISPR Therapeutics/Vertex Pharmaceuticals; Autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells - CRISPR Therapeutics/Vertex Pharmaceuticals; CASGEVY; CRISPR/Cas9 gene-edited therapy - CRISPR Therapeutics/Vertex Pharmaceuticals; CTX-001; Exa-cel

Latest Information Update: 12 Apr 2024

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At a glance

  • Originator CRISPR Therapeutics
  • Developer CRISPR Therapeutics; Vertex Pharmaceuticals
  • Class Gene therapies; Haematopoietic stem cells therapies; Stem cell therapies
  • Mechanism of Action Cell replacements; Fetal haemoglobin expression stimulants
  • Orphan Drug Status

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

    Yes - Sickle cell anaemia; Beta-thalassaemia
  • New Molecular Entity No

Highest Development Phases

  • Marketed Beta-thalassaemia; Sickle cell anaemia

Most Recent Events

  • 01 Apr 2024 Health Canada accepts NDA for exagamglogene Autotemcel for Sickle Cell Disease and Beta-thalassemia for priority review
  • 01 Apr 2024 Preregistration for Beta-thalassaemia (In adolescents, In adults) in Canada (IV)
  • 01 Apr 2024 Preregistration for Sickle cell anaemia (In adolescents, In adults) in Canada (IV)
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