The FDA has accepted Bluebird bio Inc's (NASDAQ: BLUE) marketing application for betibeglogene autotemcel (beti-cel) for β-thalassemia. The agency has granted priority review for Beti-cel, gene therapy for β-thalassemia across all genotypes who require regular red blood cell transfusions. If approved, beti-cel will be the first one-time treatment that addresses the underlying genetic cause of disease—offering an alternative to regular RBC transfusions and iron chelation therapy. The agency has s
CAMBRIDGE, Mass., November 22, 2021--bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for betibeglogene autotemcel (beti-cel) for priority review. Beti-cel is a potentially transformative gene therapy for adult, adolescent and pediatric patients with β-thalassemia across all genotypes who require regular red blood cell (RBC) transfusions. If approved, beti-cel will be the first one-time treatme
"Regaining confidence and credibility is something that I'm very focused on," bluebird bio CEO Andrew Obenshain told the Business Journal. "The only thing I can do in this circumstance is meet the deliverables I say I'm going to meet."