Table 3.
Serotype (LoD) | 6E12 (n = 1) |
2E13 (n = 1) |
4E13 (n = 4)a |
6E13 (n = 7) |
||||
---|---|---|---|---|---|---|---|---|
Week 8 | End | Week 8 | End | Week 8 | End | Week 8 | End | |
AAV5 (33 ng/mL) | 10,504 | 87,235 | 357,751 | 8,596,124 | 748,831 | 2,896,108 | 421,222 | 3,413,365 |
AAV2 (60 ng/mL) | 30 | <MRD | 240 | 382 | 153,226a | 53,930a | 7,011 | 360 |
AAV6 (15 ng/mL) | 455 | 48 | 951 | 440 | 174,235a | 38,119a | 9,314 | 1,148 |
AAV8 (70 ng/mL) | 177 | 40 | 428 | 176 | 26,802 | 1,433 | 6,275 | 440 |
AAVrh10 (42 ng/mL) | 55 | <MRD | 377 | 287 | 17,600 | 1,408 | 6,283 | 394 |
LoD, limit of detection; End, last time point taken for cross-reactive antibody assessment ranges from week 76 to week 134; MRD, minimal required dilution.
One 4E13 patient with pre-existing antibodies to AAV2 and AAV6 developed comparatively higher antibody titers following dose administration and contributed to the elevated mean AAV2 and AAV6 titers at week 8 and the final time point.