Bluebird bio announced it has completed the rolling submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for approval of betibeglogene autotemcel (beti-cel) gene therapy in adult, adolescent and pediatric patients with β-thalassaemia who require regular red blood cell transfusions, across all β genotypes. Beti-cel is already cleared for market in Europe, where it's sold under the brand name Zynteglo. The BLA submission for beti-cel is based on data from Phase 1/2 and Phase 3 Northstar studies, which represent more than 220 patient-years of experience with beti-cel. The FDA previously granted beti-cel Orphan Drug status and Breakthrough Therapy designation for the treatment of transfusion-dependent β-thalassemia. If approved, beti-cel will be the first hematopoietic (blood) stem cell (HSC) ex-vivo gene therapy for patients in the United States. Source: bluebirdbio press release