Table 1.

In vitro characterization of AAV-cFVIII vectors


Vector lot

Genome titer, vg/mL

Capsid titer, cp/mL

Ratio of total to full capsids

Transduced HepG2, vg/well

In vitro activity, mU/mL

Dog injected
17-26   2.7 × 1012  3.6 × 1013  13   1 × 106  260   Elisa  
510-1
 
4.5 × 1012
 
4.3 × 1013
 
10
 
1 × 106
 
275
 
Junior
 

Vector lot

Genome titer, vg/mL

Capsid titer, cp/mL

Ratio of total to full capsids

Transduced HepG2, vg/well

In vitro activity, mU/mL

Dog injected
17-26   2.7 × 1012  3.6 × 1013  13   1 × 106  260   Elisa  
510-1
 
4.5 × 1012
 
4.3 × 1013
 
10
 
1 × 106
 
275
 
Junior
 

Ratio of total to full capsids is equivalent to the ratio of the capsid titer to genome titer.

vg/mL indicates vector genomes/mL; and cp/mL, capsid (empty and fulls) particles/mL.

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