Human FIX concentration in rhesus plasma was determined at the indicated time points after administration of 1 × 1012 vg/kg (M1-sc, □/red line; M2-sc, ○/blue line) of scAAV2/8-LP1-hFIXco into the mesenteric vein of 2 rhesus macaques. Treatment of M1-sc with rituximab (Rit × 2 doses) and oral cyclosporine (CyA) is shown. M4-sc (▿/green line) was initially transduced with 4 × 1011 vg/kg scAAV2/8-LP1-hFIXco but did not respond and was and subsequently retransduced with 1 × 1012 vg/kg scAAV2/5-LP1-hFIXco as indicated by the arrow.

Human FIX concentration in rhesus plasma was determined at the indicated time points after administration of 1 × 1012 vg/kg (M1-sc, □/red line; M2-sc, ○/blue line) of scAAV2/8-LP1-hFIXco into the mesenteric vein of 2 rhesus macaques. Treatment of M1-sc with rituximab (Rit × 2 doses) and oral cyclosporine (CyA) is shown. M4-sc (▿/green line) was initially transduced with 4 × 1011 vg/kg scAAV2/8-LP1-hFIXco but did not respond and was and subsequently retransduced with 1 × 1012 vg/kg scAAV2/5-LP1-hFIXco as indicated by the arrow.

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