Long-Term Follow-Up Data Continue to Support Beti-Cel as a Potentially Curative Gene Therapy for β-Thalassemia Patients Who Require Regular Transfusions Through Achievement of Durable Transfusion Independence and Normal or Near-Normal Adult Hb Levels

bluebird bio shared long-term data at the ASH Annual Meeting on ZYNTEGLO® (betibeglogene autotemcel), a one-time gene therapy for beta-thalassemia patients requiring regular transfusions. Results from up to 10 years of follow-up show durable transfusion independence, improved hemoglobin levels, reduced iron burden, and sustained safety outcomes. Most participants achieved long-term benefits, including discontinuation of iron chelation therapy and enhanced quality of life. The therapy continues to demonstrate its potential to transform the management of severe genetic diseases.

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