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Phase 3b Study of Exagamglogene Autotemcel (CTX001) in Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease
2021-006390-37Phase 3Active
- Sponsor
- Vertex Pharmaceuticals
- Start
- Mar 2022
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Cell Therapies
- Countries
- 1
01
Overview
Phase 3, open-label trial of exagamglogene autotemcel in beta thalassemia, sickle cell disease (SCD) and sickle cell anemia (SCA) (2 arms), sponsored by Vertex Pharmaceuticals. Primary endpoint: HbF and total hemoglobin levels over time. Started Mar 2022; status: active.
02
Drugs
exagamglogene autotemcel
03
Conditions
beta thalassemiasickle cell disease (SCD)sickle cell anemia (SCA)
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