Skip to main content
. Author manuscript; available in PMC: 2016 Feb 17.
Published in final edited form as: Expert Opin Orphan Drugs. 2015 Jul 18;3(9):997–1010. doi: 10.1517/21678707.2015.1069179

Table 1.

Comparison of AAV-based liver-directed gene therapy for hemophilia B.

AAV2 liver-directed FIX trial [2]
 AAV8 liver-directed FIX trial [3]
Dose
cohort		 Vector
dose
(vg/kg)
*Empties
removed		 Total capsid
dose
(cp/kg)		 Initial
FIX level
(%)		 Prevalence of
transaminitis
(onset time)		 Vector dose
(vg/kg)
*Contains
fourfold
empties		 Total capsid
dose
(cp/kg)		 Initial
FIX level
(%)		 Prevalence of
transaminitis
(onset time)
Low 8 × 1010 8 × 1010 0 0/2 2 × 1011 1 × 1012 2 0/2
Medium 4 × 1011 4 × 1011 0 1/2 (4 weeks) 6 × 1011 3 × 1012 2 – 3 0/2
High 2 × 1012 2 × 1012 10 – 12 1/2 (4 weeks) 2 × 1012 1 × 1013 4 – 10 4/6 (7 – 9 weeks)

All subjects who developed transaminitis had anti-AAV NAbs < 1:5 in both trials.

AAV: Adeno-associated viral; FIX: Factor.