ISSN : 2146-3123
E-ISSN : 2146-3131

FDA approved first gene therapy in United States

CAR T-cell therapy approved to treat certain children and young adults with B-cell acute lymphoblastic leukemia

The U.S. Food and Drug Administration (FDA) approved Kymriah (tisagenlecleucel), a cell-based gene therapy, for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).


Kymriah is a genetically-modified autologous T-cell immunotherapy. The patient’s T-cells are collected and genetically modified. The genetically-modified T-cells have a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. These cells are given back to the patient to kill the cancer cells.