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A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease

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Trial Profile

A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease

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

Latest Information Update: 08 Dec 2025

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At a glance

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

Most Recent Events

  • 10 Nov 2025 According to Vertex Pharmaceuticals media release, dosing is on track to complete this quarter. Initial data from these studies will be presented at the American Society of Hematology (ASH) annual meeting on December 6th, 2025.
  • 03 Apr 2025 Planned End Date changed from 1 Feb 2025 to 9 Jun 2027.
  • 03 Apr 2025 Planned primary completion date changed from 1 Feb 2025 to 9 Jun 2027.

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