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betibeglogene autotemcel (beti-cel) One-Time Gene Therapy for β-thalassemia Continues to Demonstrate Durable Efficacy Across Pediatric and Adult Patient Populations and All ...

With 51 patients enrolled, data from the long-term follow-up study (LTF-303) show that all patients treated with beti-cel who achieve transfusion independence (TI) remain free from transfusions, with ...
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Long-Term Data Presented at ASH Support beti-cel as a Potentially Curative Gene Therapy for β-Thalassemia Patients Who Require Regular Transfusions Through Achievement of ...

All four parent studies are complete, and all patients have transitioned to a long-term follow-up study (LTF-303) SOMERVILLE, Mass.--(BUSINESS WIRE)--Updated data from bluebird bio inc.’s (NASDAQ: ...