Figure 1.

Expression of hFIX in NHPs after Peripheral Administration of AMT-061 and AMT-060

(A) hFIX protein in the monkey plasma was determined at different time points after AAV administration. Mean of n = 3 ± SD, n = 2 for 2.5 × 10¹³ gc/kg group. (B) FIX activity as measured by activated partial thromboplastin (APTT) assay in the plasma of the NHPs post-treatment after baseline correction. Endogenous baseline FIX activity levels were determined by three pre-treatment clotting activity determinations. Mean of n = 3 ± SD, n = 2 for 2.5 × 10¹³ gc/kg group. (C) Correlation of the expressed hFIX protein and measured FIX activity from week 4–13.