autologous CD34+ peripheral blood stem cells transduced with a lentiviral vector encoding a novel coagulation factor VIII transgene

This is an investigational ex vivo gene and cell therapy in which autologous CD34+ hematopoietic stem cells (HSCs) are collected from the patient, then genetically modified ex vivo using a self-inactivating lentiviral vector (CD68-ET3-LV) that encodes a novel coagulation factor VIII (F8) transgene under the control of a myeloid-specific promoter. The modified cells are reinfused into the patient following myeloablative conditioning. The goal is to achieve stable endogenous production of functional factor VIII by engrafted hematopoietic progeny, providing long-term correction of severe hemophilia A. Early clinical data show durable FVIII expression and prevention of spontaneous bleeding events in adults with severe hemophilia A who lack inhibitors to FVIII[1][4][7]. This approach may offer advantages over current adeno-associated virus-based therapies, including applicability to patients regardless of pre-existing antibodies and potential for use in pediatric populations[4].

Other names

autologous CD34+ HSCs transduced with lentiviral vector encoding F8lentiviral gene therapy for hemophilia A (CD68-ET3-LV)ET3 gene therapyET-3 gene therapyET 3 gene therapy