Table 1 Plasma α-Gal A enzyme activity in wild-type mice following treatment with AAV8-FLT190 or enzyme replacement therapy.

From: Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease

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

  1. 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.
  2. 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.
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