Approved gene therapies

genetic modification of a patient's cells to produce a therapeutic effect

gene therapy product

Tisagenlecleucel, sold under the brand name Kymriah, is a CAR T cells medication for the treatment of B-cell acute lymphoblastic leukemia (ALL) which uses the body's own T cells to fight cancer (adoptive cell transfer).

pharmaceutical drug

modified herpes virus used for treatment of melanoma

experimental gene therapy for sickle cell disease and transfusion-dependent beta thalassemia

gene therapy medication for Leber congenital amaurosis (LCA), a genetic disorder that typically causes total blindness by early adulthood

medical treatment

pharmaceutical compound