A Novel Agent-based Computational Model for Liver-targeting, AAV-based Gene Therapies Could Predict Response Durability in Hemophilia B Patients Treated with Etranacogene Dezaparvovec

Co-developed with CSL Behring, this research introduces a high-resolution model that simulates hepatocyte turnover,  episomal retention and coagulation factor IX (FIX) expression  in virtual patients treated with Etranacogene Dezaparvovec ( HEMGENIX).  The model captured clinical variability before recent clinical readouts were available, and projects FIX expression durability  over two decades.