betibeglogene autotemcel

betibeglogene autotemcel is an **autologous ex vivo lentiviral vector gene therapy** developed by bluebird bio for transfusion-dependent \u03b2-thalassemia. The product consists of a patient's own CD34-positive hematopoietic stem cells collected and transduced ex vivo with the BB305 self-inactivating lentiviral vector encoding a functional modified \u03b2-globin gene, **\u03b2A-T87Q-globin**. After myeloablative conditioning and one-time intravenous infusion, the genetically modified stem cells engraft in bone marrow and produce HbAT87Q-containing erythroid cells, aiming to restore effective hemoglobin production and reduce or eliminate the need for chronic red blood cell transfusions. The therapy is marketed in the United States as Zynteglo for adult and pediatric patients with \u03b2-thalassemia who require regular RBC transfusions.

Brand names

Zynteglo

Other names

betibeglogene autotemcel