Bluebird presents updated data from gene therapy program in TDT beta-thalassemia

Updated data from Bluebird Bio’s gene therapy program in transfusion-dependent beta-thalassemia were presented today at the 65th American Society of Hematology Annual Meeting & Exposition. Updated follow-up data showed sustained treatment effect, reduced iron management burden and improved quality of life measures in patients with beta-thalassemia who require regular red blood cell transfusions following treatment with betibeglogene autotemcel. “Long-term results presented at ASH 2023 showed durable transfusion independence and a continued positive safety profile in patients with beta-thalassemia treated with our beti-cel gene therapy through up to nine years of follow-up,” said Richard Colvin, chief medical officer, bluebird bio. “These data represent the longest follow-up with a gene therapy for patients with beta-thalassemia requiring regular transfusions and continue to show that beti-cel is a potentially curative therapy across ages and genotypes, through the achievement of durable transfusion independence and normal or near-normal hemoglobin levels.”