Betibeglogene autotemcel

Betibeglogene autotemcel is an autologous gene therapy that adds functional copies of the β-globin gene (β-globin) to hematopoietic stem cells in order to treat β-thalassemia. β-thalassemia is a condition caused by mutations in the β-globin gene (HBB) that leads to a significant decrease in the production of β-globin. This affects hemoglobin levels, and in patients with severe forms of this disease, long-term red cell transfusions are required for survival and the prevention of serious complications. Allogeneic hematopoietic-cell transplantation would be a therapeutic option in patients with β-thalassemia; however, this process is reserved for young children with an HLA-identical sibling donor due to the risks of graft rejection, graft-versus-host disease, and other treatment-related toxic effects. Patients treated with betibeglogene autotemcel are given an infusion of their own hematopoietic stem cells, previously enriched for CD34+ and transduced ex vivo with BB305 LVV, a self-inactivating lentiviral vector (LVV). BB305 LVV encodes β-globin, compensating for the lack of β-globin in these patients. The use of betibeglogene autotemcel is more effective in patients with β-thalassemia with residual β-globin synthesis (non-β/β genotype) compared to those with no detectable β-globin production (β/β). Betibeglogene autotemcel was approved by the FDA in August 2022 and is the first ex-vivo lentiviral vector gene therapy available in the U.S. for the treatment of people with β-thalassemia.