Baseline demographics and disease characteristics
| Characteristic . | Participant 1 . | Participant 2 . | Participant 3 . |
|---|---|---|---|
| Age at enrollment (y) | 43 | 50 | 47 |
| Weight (kg) | 89 | 81 | 82 |
| HIV status | Nonreactive | Reactive (controlled) | Reactive (controlled) |
| HBV status | Negative | Negative | Negative |
| HCV status | Resolved | Resolved | Resolved |
| Baseline FIX activity levels (%) | 1 | <1 | <1 |
| Prescreening FIX treatment | Prophylaxis (EHL) | Prophylaxis (EHL) | Prophylaxis (EHL) |
| ABR 1 y before screening∗ | 3 | 1 | 5 |
| Anti-AAV5 NAb status at screening∗ (titer)†,‡ | Positive (48) | Positive (44) | Positive (25) |
| Anti-AAV5 NAb status at day of dosing∗ (titer)†,‡ | Positive (22) | Positive (33) | Positive (20) |
| Characteristic . | Participant 1 . | Participant 2 . | Participant 3 . |
|---|---|---|---|
| Age at enrollment (y) | 43 | 50 | 47 |
| Weight (kg) | 89 | 81 | 82 |
| HIV status | Nonreactive | Reactive (controlled) | Reactive (controlled) |
| HBV status | Negative | Negative | Negative |
| HCV status | Resolved | Resolved | Resolved |
| Baseline FIX activity levels (%) | 1 | <1 | <1 |
| Prescreening FIX treatment | Prophylaxis (EHL) | Prophylaxis (EHL) | Prophylaxis (EHL) |
| ABR 1 y before screening∗ | 3 | 1 | 5 |
| Anti-AAV5 NAb status at screening∗ (titer)†,‡ | Positive (48) | Positive (44) | Positive (25) |
| Anti-AAV5 NAb status at day of dosing∗ (titer)†,‡ | Positive (22) | Positive (33) | Positive (20) |
Participants 2 and 3 were previously excluded from another AAV-based gene therapy trial for hemophilia B based on anti-AAV NAb titer.
ABR, annualized bleeding rate; EHL, extended half-life; HBV, hepatitis B virus; HCV, hepatitis C virus.
Total bleeds (treated + untreated).
AAV5 NAb data considered positive if titer was ≥2.
Luciferase cell-based assay.