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Phase 2b gene therapy with autologous CD34+ cells transduced with GLOBE lentiviral vector in transfusion-dependent beta-thalassemia
2025-522160-32-00Phase 2Authorised
- Sponsor
- Fondazione Telethon
- Start
- Dec 2025
- Modality
- Stem Cell Therapies → Cell Therapies, Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of betibeglogene autotemcel in beta thalassemia, sponsored by Fondazione Telethon. Primary endpoint: Transfusion independence for ≥12 months (TI12). Planned start Dec 2025; status: authorised.
02
Drugs
betibeglogene autotemcel
03
Conditions
beta thalassemia
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