|
|
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
|