Gene transfer strategies for hemophilia A and B.
| Trial . | No. patients . | Vector dose . | Endpoints . | Reference . |
|---|---|---|---|---|
| Intravenous infusion of retroviral vector expressing F.VIII | 13 | Up to 4.4 × 108 TU/kg | Vector DNA detected in PBMCs of 8/8 subjects treated at ≥ 9.2 × 107 TU/kg. No patients with consistent detection of F.VIII > 1%. | Powell et al3 |
| Ex vivo plasmid-F.VIII transfection of autologous fibroblasts | 12 | Up to 400 × 106 transfected autologous cells | F.VIII 1% to 2% in higher-dose cohort. | Roth et al2 |
| AAV-F.IX into skeletal muscle | 8 | Up to 2 × 1012 μg/kg | Persistent F.IX expression in muscle biopsy. No patients with consistent F.IX levels > 1%. | Manno et al5 |
| AAV-F.IX into liver | 7 | Up to 2 × 1012 μg/kg | F.IX levels > 5% × 4 weeks in top dose cohort. | Manno et al6 |
| Gutted adenoviral vector expressing F.VIII | 1 | 4.3 × 1010 particles/kg | Not published |
| Trial . | No. patients . | Vector dose . | Endpoints . | Reference . |
|---|---|---|---|---|
| Intravenous infusion of retroviral vector expressing F.VIII | 13 | Up to 4.4 × 108 TU/kg | Vector DNA detected in PBMCs of 8/8 subjects treated at ≥ 9.2 × 107 TU/kg. No patients with consistent detection of F.VIII > 1%. | Powell et al3 |
| Ex vivo plasmid-F.VIII transfection of autologous fibroblasts | 12 | Up to 400 × 106 transfected autologous cells | F.VIII 1% to 2% in higher-dose cohort. | Roth et al2 |
| AAV-F.IX into skeletal muscle | 8 | Up to 2 × 1012 μg/kg | Persistent F.IX expression in muscle biopsy. No patients with consistent F.IX levels > 1%. | Manno et al5 |
| AAV-F.IX into liver | 7 | Up to 2 × 1012 μg/kg | F.IX levels > 5% × 4 weeks in top dose cohort. | Manno et al6 |
| Gutted adenoviral vector expressing F.VIII | 1 | 4.3 × 1010 particles/kg | Not published |