Evaluation Of The Human Factor Ix Gene Therapy Vector Tak-748 In Hemophilia: Results From Non-Clinical Studies In Factor Ix Knockout Mice And Rhesus Monkeys

Introduction: Adeno-associated virus (AAV)-based factor IX (FIX) gene therapy has the potential to provide clinical benefit in patients with hemophilia B. TAK-748 is a novel next-generation AAV vector for FIX gene therapy. The vector design includes the insertion of 3 hepatocyte-specific cis-regulatory elements to increase the strength of the liver-specific transthyretin promoter driving expression of a human FIX transgene.