Manusiran (sodium)

Manusiran (SLN124) sodium, a GalNAc conjugated 19-mer siRNA targeting TMPRSS6 (transmembrane protease serine 6), reduces plasma iron and increases hepcidin levels[1][2].

[1]Vadolas J, Ng GZ, Kysenius K, et al. SLN124, a GalNac-siRNA targeting transmembrane serine protease 6, in combination with deferiprone therapy reduces ineffective erythropoiesis and hepatic iron-overload in a mouse model of β-thalassaemia. Br J Haematol. 2021;194(1):200-210.|[2]Altamura S, Schaeper U, Dames S, et al. SLN124, a GalNAc-siRNA Conjugate Targeting TMPRSS6, Efficiently Prevents Iron Overload in Hereditary Haemochromatosis Type 1. Hemasphere. 2019;3(6):e301.