Table 1.
Biodistribution Data in Dog Brain 4 Weeks Post-injection
| Animal | #106 | #108 | #109 | |||
|---|---|---|---|---|---|---|
| Hemisphere: | Right | Left | Right | Left | Right | Left |
| Vector concentration (vg/mL) | 1.0E+12 | 1.0E+12 | 1.0E+12 | 1.0E+12 | 1.0E+12 | 1.0E+12 |
| No. of deposits per hemisphere | 1 | 1 | 1 | 1 | 2 | 2 |
| Volume per deposit (μL) | 500 | 500 | 500 | 500 | 500 | 500 |
| Speed of injection (μL/min) | 10 | 10 | 10 | 10 | 10 | 10 |
| Total dose per hemisphere (vg) | 5.0E+11 | 5.0E+11 | 5.0E+11 | 5.0E+11 | 1.0E+12 | 1.0E+12 |
| Total volume injected (vi) per hemisphere (cm3) | 0.50 | 0.50 | 0.50 | 0.50 | 1.00 | 1.00 |
| Volume of distribution (vd) of gadolinium (cm3) | 1.56 | 1.43 | 1.50 | 1.39 | 3.63 | 2.92 |
| vd gadolinium/vi | 3.1 | 2.9 | 3.0 | 2.8 | 3.6 | 2.9 |
| No. of punches for qPCR analysis | 40 | 40 | 40 | 40 | 33 | 33 |
| No. of punches >0.1 cp/cell | 14 | 15 | 13 | 17 | 10 | 14 |
| % of punches >0.1 cp/cell | 35% | 38% | 33% | 43% | 30% | 42% |
| No. of punches for enzyme analysis | 40 | 40 | 40 | 40 | 33 | 33 |
| No. of punches >20% enzyme increase | 29 | 33 | 30 | 27 | 30 | 26 |
| % of punches >20% enzyme increase | 73% | 83% | 75% | 68% | 91% | 79% |
Data from six hemispheres injected with LYS-SAF302 from three dogs with 4 weeks post-injection endpoint. cp, copies.