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Phase 3 pediatric CTX001 study in severe sickle cell disease
2021-002173-26Phase 3Active
- Sponsor
- Vertex Pharmaceuticals
- Start
- Aug 2021
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Cell Therapies
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of exagamglogene autotemcel in sickle cell disease (SCD), sponsored by Vertex Pharmaceuticals. Primary endpoint: Proportion of subjects achieving VF12. Started Aug 2021; status: active.
02
Drugs
exagamglogene autotemcel
03
Conditions
sickle cell disease (SCD)
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