Coadministration of AAV8-hFIX and immunosuppressants was well tolerated in rhesus macaques. Six AAV8-naive macaques were assigned to 2 groups. The No IS group consisted of R11, R12, and R13 and received 2 × 1013 vg/kg AAV8-hFIX16. The IS group consisted of R14, R15, and R16 and received the same dose of the vector in combination with MMF and tacrolimus. (A) Serum ALT levels. Dotted lines represent the normal reference range at the study facility. (B) Levels of hFIX detected in macaque plasma by ELISA (left y-axis) and their corresponding percentage of normal FIX levels (5000 ng/mL; right y-axis). Dotted line indicates when immunomodulatory therapy was stopped. (C) The relative levels of anti-AAV8 IgG detected in rhesus macaque plasma by ELISA before and after AAV8-hFIX infusion. Dotted line indicates when immunomodulatory therapy was stopped.
