Table 1.
Gene therapy dose in treated pigs
| Pig | Genotype | Sex | Age (weeks) | Weight (kg) | Vector | Route | Dose (mL) | Dose (TU/kg) | Study duration |
|---|---|---|---|---|---|---|---|---|---|
| No. 169 (Carmen) | FAH−/− | F | 6 | 11.8 | LV-FAH | Portal | 150 | 2.00 × 1010 | 2 daysa |
| No. 162 (Santiago) | FAH−/− | M | 6 | 10.4 | LV-FAH | Portal | 135 | 2.04 × 1010 | 60 days |
| No. 166 (Pepe) | FAH−/− | M | 6 | 10.8 | LV-FAH | Portal | 140 | 2.04 × 1010 | 337 days |
| No. 167 (Lola) | FAH−/− | F | 6 | 13.6 | LV-FAH | Portal | 170 | 1.96 × 1010 | 337 days |
| No. 762 | FAH−/− | M | 6 | 10.2 | LV-FAH | Systemic | 224 | 2.00 × 1010 | 14 days |
| No. 764 | FAH+/− | M | 6 | 9.2 | LV-GFP | Portal | 305 | 2.00 × 1010 | 13 days |
aAnimal recovered from anesthesia slowly and was found dead on Day 2 due to a suspected inflammatory reaction; TU transducing units.
Four animals (two male and two female) were assigned to dosing as indicated below. Animals were surgically anesthetized and dosed with 2 × 1010 TU/kg LV-FAH directly via the portal vein.