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Phase 3 Study of CTX001 (Casgevy) in Pediatric Transfusion-Dependent β-Thalassemia
2024-513349-35-00Phase 3Authorised
- Sponsor
- Vertex Pharmaceuticals
- Start
- Nov 2024
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Cell Therapies
- Countries
- 5
01
Overview
Phase 3, open-label, single-arm trial of exagamglogene autotemcel in beta thalassemia, sponsored by Vertex Pharmaceuticals. Primary endpoint: TI12 (12-month transfusion independence). Planned start Nov 2024; status: authorised.
02
Drugs
exagamglogene autotemcel
03
Conditions
beta thalassemia
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