Fig. 2: FIX activity after gene therapy in phase 1/2 and phase 3 studies.
From: Factor IX-Padua AAV gene therapy in hemophilia B: phases 1/2 and 3 trials
a, Mean FIX activity postgene therapy within 52 weeks in the phase 1/2 study. FIX:C was measured with the OS method with both actin-FSL and SynthAsil APTT reagents. The data are presented as mean ± s.d. b, Median FIX activity between 52 and 104 weeks postgene therapy with BBM-H901 in the phase 1/2 study. FIX activity was measured with the OS actin-FSL method. Data are presented as box plots with center depicting median, bounds of box depicting interquartile range, and whiskers depicting minima and maxima. c. Long-term FIX activity up in phase 1/2 study. FIX activity was measured with the OS actin-FSL method. All participants completed a 104-week follow-up. d. Long-term FIX activity in the phase 1 pilot study. FIX activity was measured with the OS actin-FSL method.
