Therapeutic Factor IX (FIX) Activity After Single Treatment With AMT-060 (AAV5-FIX) In Hemophilia B Patients With Pre-existing Anti-AAV5 Humoral Immunity Anna Majowicz, Bart Nijmeijer, Margit Lampen, Lisa Spronck, Martin de Haan, Harald Petry, Sander van Deventer, Christian Meyer, Marco Tangelder, Valerie Ferreira
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Therapeutic Factor IX (FIX) Activity After Single Treatment With AMT-060 (AAV5-FIX… · 2018. 5. 18. · Therapeutic Factor IX (FIX) Activity After Single Treatment With AMT-060
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Therapeutic Factor IX (FIX) Activity After Single
Treatment With AMT-060 (AAV5-FIX) In Hemophilia B
Patients With Pre-existing Anti-AAV5 Humoral
Immunity
Anna Majowicz, Bart Nijmeijer, Margit Lampen, Lisa Spronck, Martin de Haan,
Harald Petry, Sander van Deventer, Christian Meyer, Marco Tangelder,
Valerie Ferreira
A S G C T 2 0 1 8 1 9 M a y 2 0 1 8 | 33
▪ 1:5 anti-AAV8 NAB titer totally blocks in vivo liver transduction in Non-
human Primates (NHP) with AAV8-hFIX (Jiang et al., Blood 2006)
▪ 1:17 titer of pre-existing anti-AAV2 NAB is described to “modulate linear
dose response” in humans to AAV2-hFIX treatment:
1:17 mean hFIX activity of 3%
1:2 mean hFIX activity of 11%
(Manno et al., Nature Medicine 2006)
▪ 1:1 titer of bioengineerd AAV-Spark100 capsid was reported to be
associated with reduced therapeutic efficacy (George et al., NEJM 2017)