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A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease

Trial Profile

A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease

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

Latest Information Update: 25 Mar 2024

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At a glance

  • Drugs Exagamglogene autotemcel (Primary) ; Busulfan; Plerixafor
  • Indications Sickle cell anaemia
  • Focus Adverse reactions; Proof of concept; Registrational; Therapeutic Use
  • Acronyms CLIMB-121
  • Sponsors Vertex Pharmaceuticals
  • Most Recent Events

    • 13 Feb 2024 According to a Vertex Pharmaceuticals media release, company that the European Commission has granted conditional marketing authorization to CASGEVY (exagamglogene autotemcel) for the treatment of patients who are 12 years of age and older with severe sickle cell disease and transfusion-dependent beta thalassemia.
    • 16 Jan 2024 According to a Vertex Pharmaceuticals media release, company announced that the U.S. Food and Drug Administration (FDA) has approved CASGEVY (exagamglogene autotemcel) for the treatment of transfusion-dependent beta thalassemia in patients 12 years and older.
    • 15 Dec 2023 According to a Vertex Pharmaceuticals media release, the Committee for Advanced Therapies (CAT), EMAs expert committee for cell and gene-based medicines, found that the benefits of Casgevy outweighed the possible risks in patients with beta thalassemia and sickle cell disease. The CHMP, EMAs human medicines committee, agreed with the CATs assessment and positive opinion, and recommended approval of this medicine.
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