eloengaltugagene autoleucel

eloengaltugagene autoleucel is an investigational autologous lentiviral gene therapy developed by Aruvant Sciences for the treatment of sickle cell disease. It involves genetically modifying a patient's own hematopoietic stem cells with a lentiviral vector to express a modified fetal hemoglobin gene (γ-globin). This aims to increase the production of fetal hemoglobin, which is known to reduce the severity of sickle cell disease symptoms, such as vaso-occlusive crises, by improving red blood cell function. The therapy is designed to be administered with reduced intensity conditioning, offering a potentially safer alternative to traditional myeloablative chemotherapy.

Other names

GbGm lentiviral gamma-globin vectorGbGm lentiviral vectormodified gamma-globin gene therapy