SOMERVILLE, Mass .(BUSINESS WIRE) and Bluebird bio, Inc. (BLUE) announced the support of U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) for betibeglogene autotemcel (beti-cel) for the treatment of people with β-thalassemia who require regular red blood cell transfusions. The advisory committee’s recommendation is based on the Biologics License Application (BLA) currently under priority review by the FDA with a decision goal date set for August 19, 2022. The BLA is based on data from bluebird bio’s Phase 3 studies HGB-207 (Northstar-2) and HGB-212 (Northstar-3), the Phase 1/2 HGB-204 (Northstar) and HGB-205 studies, and the long-term follow-up study LTF-303 as of March 2021. Additionally, as of the latest data cutoff date (August 2021), data from bluebird bio’s clinical development program represent 240 patient-years of experience with beti-cel and the longest available follow-up data in beta-thalassemia patients requiring regular RBC transfusions treated with a one-time gene therapy. If approved, beti-cel will be the first potentially curative gene therapy option for people with beta-thalassemia who require regular red blood cell transfusions and the first ex-vivo LVV gene therapy available in the U.S. For more information, here.