Table 1.
Participant baseline demographics and clinical characteristics.
| Characteristic | AAV5 NAb+ participants (n = 21), n (%) |
|---|---|
| Age at screening (y), mean (SD; min-max) | 44.5 (17.5; 19-75) |
| Positive HIV status | 1 (4.8) |
| Prior hepatitis B | 5 (23.8) |
| Prior hepatitis C | 14 (66.7) |
| Severity of hemophilia B at diagnosis | |
| Severe (FIX < 1%) | 16 (76.2) |
| Moderately severe (FIX ≥ 1% and ≤2%) | 5 (23.8) |
| Prescreening for FIX treatment | |
| Extended half-life | 14 (66.7) |
| Standard half-life | 7 (33.3) |
| AAV5 NAb titer on the day of infusion | |
| Maximum | 3212 |
| Median (IQR) | 57 (23-199) |
| Participants with zero reported bleeds during the lead-in period | 3 (14.3) |
AAV5, adeno-associated viral vector serotype 5; FIX, factor IX; NAb, neutralizing antibody.