Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Long-term Follow-up of CTX001 in β-thalassemia and Sickle Cell Disease
2018-002935-88Phase 3Active
- Sponsor
- Vertex Pharmaceuticals
- Start
- Sep 2018
- 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 beta thalassemia and sickle cell disease (SCD), sponsored by Vertex Pharmaceuticals. Primary endpoint: New malignancies. Started Sep 2018; status: active.
02
Drugs
exagamglogene autotemcel
03
Conditions
beta thalassemiasickle cell disease (SCD)
Beyond the preview
Go deeper on 2018-002935-88.
Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on 2018-002935-88.