Summary of AAV-cFVIII—treated hemophilia A dog results
Dog . | Weight, kg . | Dose, vg/kg . | Ave WBCT, s . | Ave Coatest mU/mL, (%) . | Ave one-stage mU/mL, (%) . | CBT (timepoint, min) . | Inhibitory antibody . |
|---|---|---|---|---|---|---|---|
| Elisa | 10 | 6 × 1012 | 318 ± 38 | 179 ± 31 (2.6) | 124 ± 67 (1.8) | 3-4 (week 16) | — |
| Junior | 12.8 | 2.7 × 1013 | 314 ± 60 | 196 ± 52 (2.8) | 176 ± 53 (2.5) | 4-5 (week 35) | + (weeks 2-9) |
| Normal | NA | NA | 180-300 | 7000 (100) | 7000 (100) | <5 | NA |
| Hem A | NA | NA | >960 | <22.5 (0.3) | <44 (0.6) | >11 | NA |
Dog . | Weight, kg . | Dose, vg/kg . | Ave WBCT, s . | Ave Coatest mU/mL, (%) . | Ave one-stage mU/mL, (%) . | CBT (timepoint, min) . | Inhibitory antibody . |
|---|---|---|---|---|---|---|---|
| Elisa | 10 | 6 × 1012 | 318 ± 38 | 179 ± 31 (2.6) | 124 ± 67 (1.8) | 3-4 (week 16) | — |
| Junior | 12.8 | 2.7 × 1013 | 314 ± 60 | 196 ± 52 (2.8) | 176 ± 53 (2.5) | 4-5 (week 35) | + (weeks 2-9) |
| Normal | NA | NA | 180-300 | 7000 (100) | 7000 (100) | <5 | NA |
| Hem A | NA | NA | >960 | <22.5 (0.3) | <44 (0.6) | >11 | NA |
The Coatest and the one-stage assay are sensitive to 22.5 and 44 mU/mL, respectively. The WBCT, Coatest, and one-stage assay results were averaged over 14 months for Elisa and 7.5 months for Junior. The CBT was performed at a single time point for each injected dog.
A FVIII inhibitory antibody was detected by Bethesda assay in Junior between weeks 2 and 9.
NA indicates not applicable; WBCT, whole-blood clotting time; and CBT, cuticle bleeding time.