Alexander Kistner, Jessica A Chichester, Lili Wang, Roberto Calcedo, Jenny A Greig, Leah N Cardwell, Margaret C Wright, Julien Couthouis, Sunjay Sethi, Brian E McIntosh, Kathleen McKeever, Samuel Wadsworth, James M Wilson, Emil Kakkis, Barbara A Sullivan
Adeno-associated virus (AAV) vector gene therapy is a promising approach to treat rare genetic diseases; however, an ongoing challenge is how to best modulate host immunity to improve transduction efficiency and therapeutic outcomes. This report presents two studies characterizing multiple prophylactic immunosuppression regimens in male cynomolgus macaques receiving an AAVrh10 gene therapy vector expressing human coagulation factor VIII (hFVIII). In study 1, no immunosuppression was compared with prednisolone, rapamycin (or sirolimus), rapamycin and cyclosporin A in combination, and cyclosporin A and azathioprine in combination...
October 13, 2023: Gene Therapy