Table 2.
Biodistribution Data in NHP Brain at 6 Weeks Post-injection
| Animal | #169I | #763E | ||
|---|---|---|---|---|
| Hemisphere: | Right | Left | Right | Left |
| Vector concentration (vg/mL) | 3.6E+12 | 3.6E+12 | 3.6E+12 | 3.6E+12 |
| No. of deposits per hemisphere | 2 | 2 | 2 | 2 |
| Volume per deposit (μL) | 50 | 50 | 50 | 50 |
| Speed of injection (μL/min) | 5 | 5 | 5 | 5 |
| Total dose per hemisphere (vg) | 3.6E+11 | 3.6E+11 | 3.6E+11 | 3.6E+11 |
| No. of punches for qPCR analysis | 46 | 48 | 48 | 43 |
| No. of punches >0.1 cp/cell | 6 | 5 | 4 | 5 |
| No. of punches >0.1 cp/cell | 13% | 10% | 8% | 12% |
| No. of punches for enzyme analysis | 51 | 53 | 52 | 50 |
| No. of punches >20% enzyme increase | 50 | 53 | 50 | 46 |
| % of punches >20% enzyme increase | 98% | 100% | 96% | 92% |
Data from four hemispheres injected with LYS-SAF302 from two NHPs with 6 weeks post-injection endpoint. cp, copies.