Table 1.
Study design to evaluate subretinal injection of AGTC-501 in RPGR mutant dogs
| Group | No. of Animals | Dog ID and Sex | Dosage Level |
||
|---|---|---|---|---|---|
| Vector Concentration (vg/mL) | Injection Volume (mL) | Total Dose (vg/eye) | |||
| Vehicle | 3 | Z559 F, Z569 M, Z587 M | 0 (control) | 0.07 | 0 |
| Low dose | 4 | Z558 F, Z566 M, Z576 M, Z605 M | 1.2 × 1011 | 0.07 | 8.4 × 109 |
| Mid dose | 4 | Z555 M, Z574 M, Z594 M, Z603 M | 6.0 × 1011 | 0.07 | 42 × 109 |
| High dose | 5 | Z570 M, Z575 M, Z592 M, Z596 M, Z604 M | 30 × 1011 | 0.07 | 210 × 109 |
Each animal received a subretinal injection in the right eye. The left eye was left untreated and used as control.
RPGR, retinitis pigmentosa GTPase regulator.