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Gene Therapy: Bluebird’s Beti-Cel Sails Through US Panel Review On Strength of ‘Impressive’ Efficacy

Executive Summary

Treatment is a potential game-changer for patients with transfusion-dependent beta-thalassemia, committee members said; panel was hesitant to extrapolate the hematological malignancies seen with bluebird’s other lentiviral vector programs to beti-cel.

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