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A Long-term Follow-up Study of Subjects With β-thalassemia or Sickle Cell Disease Treated With Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)

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A Long-term Follow-up Study of Subjects With β-thalassemia or Sickle Cell Disease Treated With Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)

Status: Active, no longer recruiting
Phase of Trial: Phase III

Latest Information Update: 13 Feb 2025

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At a glance

  • Drugs Exagamglogene autotemcel (Primary)
  • Indications Beta-thalassaemia; Sickle cell anaemia
  • Focus Adverse reactions; Registrational; Therapeutic Use
  • Acronyms CLIMB-131
  • Sponsors Vertex Pharmaceuticals

    • Most Recent Events

    • 11 Feb 2025 According to Vertex Pharmaceuticals media release, the company expects to complete dosing of this age group in 2025.
    • 11 Feb 2025 Status changed from recruiting to active, no longer recruiting.
    • 14 Jun 2024 According to a Vertex Pharmaceuticals media, company today announced longer-term data for CASGEVY (exagamglogene autotemcel [exa-cel]) from global clinical trials (CLIMB-111, -121 and -131) in people with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). The results, presented at the annual European Hematology Association (EHA) Congress.

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