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Single-dose autologous CRISPR-Cas9 modified CD34+ HSPC therapy (Casgevy/CTX001) in transfusion-dependent β-thalassemia
2024-516894-57-00Phase 3Completed
- Sponsor
- Vertex Pharmaceuticals
- Start
- Sep 2024
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Cell Therapies
- Countries
- 4
01
Overview
Phase 3, open-label, single-arm trial of exagamglogene autotemcel in beta thalassemia, sponsored by Vertex Pharmaceuticals. Primary endpoints: Successful neutrophil engraftment, Time to neutrophil engraftment and 6 more. Started Sep 2024; status: completed.
02
Drugs
exagamglogene autotemcel
03
Conditions
beta thalassemia
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