BLUE - Bluebird bio updates long-term data for ?-thalassemia gene therapy

Announcing new data from a long-term follow-up study, Bluebird bio (NASDAQ:BLUE) said that 89% of ?-thalassemia patients from a key Phase 3 trial remained transfusion-free after receiving beti-cel, its experimental gene therapy for the disease. “These findings further support beti-cel as a potentially curative one-time treatment option that addresses the underlying genetic cause of beta-thal and mitigates the burdens associated with the practical management of the disease,” the company said. The updated data from the 13-year long-term follow-up study, LTF-303, will be part of a presentation at the ongoing 63rd American Society of Hematology (ASH) Annual Meeting and Exposition. The transfusion-dependent beta-thal is a hereditary condition caused by a mutation in the ?-globin gene. Patients can develop impairment in adult hemoglobin (Hb) production, leading to severe anemia, which in turn requires transfusions of red blood cells mostly every 3 – 4 weeks. In studies for beti-cel, a patient with transfusion

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Bluebird bio updates long-term data for ?-thalassemia gene therapy