EV-α lentiviral vector

EV-α (erythroid vector-alpha) is a lentiviral vector gene therapy designed for the treatment of alpha thalassemia major (ATM), a severe genetic blood disorder caused by the deletion of all four α-globin genes. The vector delivers a functional copy of the HBA2 gene, regulated by optimized β-globin locus control region (LCR) enhancers, to restore α-globin production in erythroid cells. The therapy is administered via autologous hematopoietic stem cell transplantation (HSCT), where a patient's hematopoietic stem and progenitor cells (HSPCs) are harvested, transduced with the EV-α vector ex vivo, and then reinfused. Preclinical data indicates that EV-α can achieve high transduction efficiency and restore hemoglobin levels toward therapeutic ranges, potentially offering a curative alternative to lifelong transfusions or allogeneic transplantation.

Other names

erythroid vector-alphaEV-α-UV