Fig. 4: Tolerability and efficacy of in vivo LV gene therapy in NHP. | Nature Communications

Fig. 4: Tolerability and efficacy of in vivo LV gene therapy in NHP.

From: Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates

Fig. 4: Tolerability and efficacy of in vivo LV gene therapy in NHP.

a–e Mean with SEM (n = 3) or range (n = 2) of the serum concentration of alanine aminotransferase (ALT, a), aspartate aminotransferase (AST, b), body temperature (c), counts of white blood cells (WBC, d) and lymphocytes (e) of NHP treated with LV.coFVIII (n = 3 6 × 109 TU/kg; n = 2 3 × 109 TU/kg), or with LV.coFVIII.XTEN (n = 3 3 × 109 TU/kg; n = 2 1 × 109 TU/kg) at the indicated time after administration. The black dashed lines show the mean±3 standard deviations (SD) calculated on a pool of 38 pre-LV samples taken from 19 animals; the blue dashed lines show the normal reference values for Macaca fascicularis. f, g Single values of the concentration of hFVIII antigen (f) or activity (g) measured in the plasma of LV-treated NHP (as indicated) at the indicated time after administration. h Mean of the concentration of hFVIII activity before Abs development in NHP treated as indicated at the indicated dose. i, j Single values of the concentration of total anti-hFVIII Abs (i), or neutralizing anti-hFVIII Abs (j) in the serum of LV-treated NHP (as indicated) at the indicated time after administration. BU: Bethesda Units.

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