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CTX001 in Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease
2024-514641-12-00Phase 3Authorised
- Sponsor
- Vertex Pharmaceuticals
- Start
- Jul 2024
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Cell Therapies
- Countries
- 5
01
Overview
Phase 3, open-label trial of exagamglogene autotemcel in beta thalassemia and sickle cell disease (SCD) (2 arms), sponsored by Vertex Pharmaceuticals. Primary endpoint: HbF and hemoglobin levels over time. Planned start Jul 2024; status: authorised.
02
Drugs
exagamglogene autotemcel
03
Conditions
beta thalassemiasickle cell disease (SCD)
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