Table 3.
Summary of microscopic findings in mice—terminal euthanasia (weeks 3, 6/7 and 12).
| Males | Females | |||||||
|---|---|---|---|---|---|---|---|---|
| Group | 1 | 2 | 3 | 4 | 1 | 2 | 3 | 4 |
| Test article virus concentration (vg/kg) | 0 | 7.9 × 1013 | 2.37 × 1014 | 3.91 × 1014 | 0 | 7.9 × 1013 | 2.37 × 1014 | 3.91 × 1014 |
| No. Animals Examined | 5 | 5 | 5 | 5 | 5 | 5 | 5 | 5 |
| Heart (No. Examined) | (5)a | (5) | (5) | (5) | (5) | (5) | (5) | (5) |
| Week 3 | ||||||||
| No. Animals Examined | (5)a | (5) | (5) | (5) | (5) | (5) | (5) | (5) |
| Degeneration/regeneration | 0 | 3 | 5 | 5 | 1 | 5 | 5 | 5 |
| Minimal | – | 3 | 0 | 0 | 1 | 5 | 0 | 0 |
| Mild | – | 0 | 5 | 5 | 0 | 0 | 5 | 5 |
| Week 6/7 | ||||||||
| Heart (No. Examined) | (5)a | (5) | (5) | (4) | (5) | (5) | (5) | (5) |
| Degeneration/regeneration | 0 | 1 | 5 | 4 | 0 | 0 | 5 | 5 |
| Minimal | – | 1 | 4 | 2 | – | – | 1 | 0 |
| Mild | – | 0 | 1 | 2 | – | – | 4 | 5 |
| Thrombus | 0 | 0 | 0 | 1 | 0 | 0 | 0 | 1 |
| Moderate | – | – | – | 1 | – | – | – | 1 |
| Week 12 | ||||||||
| Heart (No. Examined) | (5)a | (5) | (5) | (0) | (5) | (5) | (5) | (0) |
| Degeneration/regeneration | 0 | 2 | 5 | – | 0 | 2 | 5 | – |
| Minimal | – | 2 | 5 | – | – | 2 | 5 | – |
As this is a gene therapy that is administered using a viral vector, the amount of virus contained in the product is very important. The bolded row represents that viral concentration.
aNumbers in parentheses represent the number of animals with the finding.
- = Not applicable