Gene therapy aims to free Beta-Thalassemia patients from lifelong transfusions
Disease control
Not yet recruiting
This study tests a single-dose gene therapy for people with transfusion-dependent beta-thalassemia, a blood disorder that requires regular red blood cell transfusions. The therapy uses a patient's own stem cells, modified with a virus to produce healthy hemoglobin, and then infus…
Phase: PHASE2 • Sponsor: Fondazione Telethon • Aim: Disease control
Last updated Jul 04, 2026 00:00 UTC