Table 3.
Summary of genome editing in liver biopsies
| Indication (study no.) | Dose (vg/kg) | Subject no.a |
Baseline |
Week 24 |
Week 48 |
||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| On-target indels | Off-target indels | Fusion transcript | On-target indels | Off-target indels | Fusion transcript | On-target indels | Off-target indels | Fusion transcript | |||
| MPS II (SB-913-1602) | 5e12 | 1 | - b | – | – | – | – | – | – | – | – |
| 1e13 | 3 | – | – | – | – | – | + c | – | – | + | |
| 4 | – | – | – | – | – | + | – | – | + | ||
| 5e13 | 6 | – | – | – | – | – | – | N/Ad | |||
| 8 | – | – | – | -e | -e | -e | – | – | – | ||
| MPS I (SB-318-1502) | 1e13 | 10 | – | – | – | – | – | + | – | – | + |
| 5e13 | 12 | – | – | – | N/A | – | – | – | |||
a
Biopsy was not available for SB-FIX-1501, SB-913-1602 subjects 2, 5, 7, 9, or SB-318-1502 subject 11.
b
–, not detected.
c
+, detected.
d
N/A, not available.
e
Week 24 liver biopsy for subject 8 was collected at an unscheduled time point past week 24 (approximately 7 months after baseline).