ET3-101

ET3-101 (also known as CD68-ET3-LV) is an investigational ex vivo lentiviral gene therapy developed by Expression Therapeutics for the treatment of severe hemophilia A. The therapy involves the collection of a patient's autologous hematopoietic stem cells (HSCs), which are then genetically modified using a lentiviral vector. This vector contains a bioengineered Factor VIII (FVIII) transgene, termed ET3, driven by a myeloid-specific CD68 promoter. Once reinfused into the patient following low-dose conditioning, the modified stem cells differentiate into myeloid cells that continuously produce and secrete therapeutic levels of Factor VIII. This approach aims to provide a durable, potentially curative treatment by establishing a steady endogenous supply of the clotting factor, thereby preventing spontaneous bleeding episodes.

Other names

ET3ET-3ET 3