Table 1.
Characteristics of Hemophilia B Patients Participating in the CT-AMT-060-01 Clinical Study, Including Levels of hFIX Activity Pre- and Post-treatment with AMT-060 (AAV5-hFIX)
| Patient No | FIX Activity, IU/dL (Basal) | Mean Steady-State FIX Activity, IU/dL (Posttreatment) | Pre-existing Anti-AAV5 NABs Titer (Luciferase-Based Bioassay) | Pre-existing Anti-AAV5 IgG (ELISA) | Pre-existing Anti-AAV5 NABs Titer (GFP-Based Bioassay) |
|---|---|---|---|---|---|
| Low-Dose Cohort | |||||
| 1 | <1 | 6.2 | <4 | negative | negative |
| 2 | <1 | 4.7 | <4 | negative | negative |
| 3 | <1 | 1.3 | 210 | positive (87) | negative |
| 4 | 1.5 | 6.8 | 340 | positive (256) | negative |
| 5 | <1 | 3.0 | 21 | negative | negative |
| High-Dose Cohort | |||||
| 6 | <1 | 12.7 | <4 | negative | negative |
| 7 | <1 | 6.4 | <4 | negative | negative |
| 8 | <1 | 6.8 | <4 | negative | negative |
| 9 | <1 | 3.1 | <4 | negative | negative |
| 10 | <1 | 5.8 | <4 | negative | negative |
Pre-existing anti-AAV5 NABs titers were measured using luciferase- and GFP-based assays, and pre-existing anti-AAV5 IgG titers were measured using ELISA. AAV, adeno-associated virus; hFIX, human factor IX; IgG, immunoglobulin G; NABs, neutralizing antibodies.