N Engl J Med 2022 01 12;386(2):138-147. Epub 2021 Dec 12.
From Bluebird Bio, Cambridge, MA (S.G., D.W., H.B., P.D.G., G.P., M.F., A.Y., M.G., S.B.V., A.M., J.L., R.A.C., M.B.); the Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute-National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD (J.T.); GeneWerk, Heidelberg, Germany (M.S.); the University of Alabama at Birmingham, Birmingham (J.K.); and the Division of Pediatric Hematology-Oncology, Medical University of South Carolina, Charleston (J.J.).
Gene therapy with LentiGlobin for sickle cell disease (bb1111, lovotibeglogene autotemcel) consists of autologous transplantation of a patient's hematopoietic stem cells transduced with the BB305 lentiviral vector that encodes the β-globin gene. Acute myeloid leukemia developed in a woman approximately 5.5 years after she had received LentiGlobin for sickle cell disease as part of the initial cohort (Group A) of the HGB-206 study. Read More