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Long-term Follow-up of CTX001 in β-thalassemia or Sickle Cell Disease
2024-512654-19-00Phase 3Authorised
- Sponsor
- Vertex Pharmaceuticals
- Start
- Jun 2024
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Cell Therapies
- Countries
- 6
01
Overview
Phase 3, open-label, single-arm trial of exagamglogene autotemcel in sickle cell disease (SCD) and beta thalassemia, sponsored by Vertex Pharmaceuticals. Primary endpoints: New malignancies, New or worsening hematologic disorders and 3 more. Planned start Jun 2024; status: authorised.
02
Drugs
exagamglogene autotemcel
03
Conditions
sickle cell disease (SCD)beta thalassemia
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