Table 1.
Plasma α-Gal A enzyme activity in wild-type mice following treatment with AAV8-FLT190 or enzyme replacement therapy.
| Treatment Group | Cmax (nmol/hr/mL) | AUC2week (U*hr/L) | Fold vs agalsidase alfa Cmax | Fold vs agalsidase alfa AUC | Fold vs agalsidase beta Cmax | Fold vs agalsidase beta AUC |
|---|---|---|---|---|---|---|
| Agalsidase alfa 0.2 mg/kg | 4516 | 13,678 | 1 | 1 | - | - |
| Agalsidase beta 1 mg/kg | 67,932 | 62,111 | - | - | 1 | 1 |
| AAV8-FLT190 6 x 1011 vg/kg | 5134 | 103,501 | 1.14 | 7.6 | 0.08 | 1.7 |
| AAV8-FLT190 2 × 1012 vg/kg | 7648 | 154,183 | 1.69 | 11.3 | 0.11 | 2.5 |
| AAV8-FLT190 6 × 1012 vg/kg | 16,031 | 323,184 | 3.55 | 23.6 | 0.24 | 5.2 |
Comparing Cmax of GLA enzyme activity levels in the plasma of C57BL6 wild type male mice administered at 6 × 1011, 2 x 1012 or 6 × 1012 vg/kg of FLT190 or ERT at 2 months of age. Table shows Cmax and AUC comparison of 3 dose groups vs ERT and fold changes. AUC is expressed as U*hr/L where one unit is defined as the amount of enzyme required to hydrolyze 1 µmol/h of 4-methylumbelliferyl-α-D-galactopyranoside substrate at 37 °C.
α-Gal A alpha galactosidase A, AAV8 adeno-associated virus serotype 8, AUC area under the curve, Cmax highest concentration of α-Gal A in plasma, ERT enzyme replacement therapy, GLA galactosidase, vg vector genomes.