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Single-dose autologous CRISPR-Cas9 edited CD34+ cells (CTX001/Casgevy) in severe sickle cell disease
2024-516067-83-00Phase 2Completed
- Sponsor
- Vertex Pharmaceuticals
- Start
- May 2019
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Cell Therapies
- Countries
- 4
01
Overview
Phase 2, open-label, single-arm trial of exagamglogene autotemcel in sickle cell disease (SCD), sponsored by Vertex Pharmaceuticals. Primary endpoints: Successful neutrophil engraftment, Time to neutrophil engraftment and 6 more. Started May 2019; status: completed.
02
Drugs
exagamglogene autotemcel
03
Conditions
sickle cell disease (SCD)
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