BLUE - Bluebird's gene therapy for ?-thalassemia shows sustained and durable efficacy across pediatric and adult patients

bluebird bio (BLUE) presents data from several studies of betibeglogene autotemcel (beti-cel) gene therapy in adult, adolescent and pediatric patients with transfusion-dependent ?-thalassemia ((TDT)). A total of 63 patients have been treated with beti-cel in Phase 1/2 HGB-204 and HGB-205 studies and the Phase 3 HGB-207 and HGB-212 studies. As of data cut-off date (March 9), 51 of 63 beti-cel-treated patients completed two years of follow-up and were enrolled in LTF-303 with a median post-infusion follow-up of 44.2 months. Of the 51 patients enrolled, 40 achieved transfusion independence ((TI)): 15/22 (68%) patients treated in Phase 1/2 and 25/29 (86%) in Phase 3. All patients who achieved TI remained free from transfusions through their last follow-up (n=40).Phase 1/2 and Phase 3 patients had a median duration of ongoing TI of 57.1 months and 26.3 months, respectively. Weighted average hemoglobin (Hb) in patients who achieved TI reached normal or near-normal levels in Phase 1/2 studies

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Bluebird's gene therapy for ?-thalassemia shows sustained and durable efficacy across pediatric and adult patients