On May 29, 2019, the European Commission approved the first gene therapy for the treatment of transfusion-dependent β-thalassemia, autologous CD34+ cells encoding βA-T87Q-globin gene.45,46 The βA-T87Q-globin gene is delivered to cells using a lentiviral vector.46,47

The therapy is indicated for patients who are at least 12 years of age with transfusion-dependent β-thalassemia who do not have a β0/β0 genotype, and for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen-matched, related HSC donor is not available.47