Table 2.

AAV vector quality attributes

AAV sampleqPCR titer, vg/mLTotal AAV concentration, Cp/mLCapsid occupancy SEC-MALS, vg/CpCapsid occupancy qPCR, vg/Cp
hFIX-Padua 7.74 × 1013 2.1 ± 0.1 × 1013 0.86 ± 0.05 3.67 
An96 2.52 × 1013 7.5 ± 0.5 × 1012 0.86 ± 0.03 3.36 
An96-Padua 1.15 × 1013 3.4 ± 0.3 × 1012 0.99 ± 0.34 3.41 
AAV sampleqPCR titer, vg/mLTotal AAV concentration, Cp/mLCapsid occupancy SEC-MALS, vg/CpCapsid occupancy qPCR, vg/Cp
hFIX-Padua 7.74 × 1013 2.1 ± 0.1 × 1013 0.86 ± 0.05 3.67 
An96 2.52 × 1013 7.5 ± 0.5 × 1012 0.86 ± 0.03 3.36 
An96-Padua 1.15 × 1013 3.4 ± 0.3 × 1012 0.99 ± 0.34 3.41 

Values are mean and standard deviation of 2 injections except An96-Padua (n = 1 because of lower titer and limited sample availability).

Cp, capsid particle.

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