Transgene protein Gene therapy Predicted human dose which induces 5% normal FIX or 12% normal FVIII activity (vg/kg) # Predicted human dose which induces 5% normal FIX or 12% normal FVIII activity (vg/kg) # Predicted human dose which induces 5% normal FIX or 12% normal FVIII activity (vg/kg) # Clinical dose (vg/kg) Observed FIX or FVIII activity (% of normal) Refs
Direct vg/kg conversion Allometric scaling Dose-response normalization
Factor IX (FIX) rAAV2-hAAT-FIX 1.03 × 1012 1.20 × 1012 4.13 × 1011 2.0 × 1012 7.0 19
scAAV2/8-LP1-hFIXco 1.79 × 1011 9.57 × 1011 2.20 × 1011 2.0 × 1012 5.1 13
rAAV-Spark100-FIX-R338L (SPK-9001) 5.84 × 1011 9.62 × 1011 6.19 × 1010 5.0 × 1011 33.7
Factor VIII (FVIII)
rAAV8-HLP-hFVIII-V3 (GO-8)
3.68 × 1011
1.85 × 1012
1.21 × 1012
6.0 × 1011 2.0 × 1012 7.0 37.5
20
Giroctocogene fitelparvovec (SB-525)
7.16 × 1011
2.86 × 1012
7.85 × 1011
1.0 × 1013 3.0 × 1013 9.0 80.1
Valoctocogene roxaparvovec (BMN270)
7.81 × 1012
4.43 × 1013
2.41 × 1013
4.0 × 1013 6.0 × 1013 20.8 59.0
21-23
Peboctocogene camaparvovec (DTX201)
2.51 × 1012
1.34 × 1014
1.08× 1014
5.0 × 1012 1.0 × 1013 6.0 × 1013 6.8 12.5 48.5
Dirloctocogene samoparvovec (SPK-8011)
1.52 × 1012
2.23 × 1013
4.50 × 1012
1.0 × 1012 2.0 × 1012 11.3 43.6
24