Manusiran  (Synonyms: Divesiran)

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

14058.53

C₄₃₄H₅₇₀F₁₉N₁₄₅O₂₇₀P₄₀S₁₀

2646704-10-9

RNA, (Am-sp-(2′-deoxy-2′-fluoro)A-sp-Cm-(2′-deoxy-2′-fluoro)C-Am-(2′-deoxy-2′-fluoro)G-Am-(2′-deoxy-2′-fluoro)A-Gm-(2′-deoxy-2′-fluoro)A-Am-(2′-deoxy-2′-fluoro)G-Cm-(2′-deoxy-2′-fluoro)A-Gm-(2′-deoxy-2′-fluoro)G-Um-sp-(2′-deoxy-2′-fluoro)G-sp-Am), complex with RNA ((2′-deoxy-2′-fluoro)U-Cm-(2′-deoxy-2′-fluoro)A-Cm-(2′-deoxy-2′-fluoro)C-Um-(2′-deoxy-2′-fluoro)G-Cm-(2′-deoxy-2′-fluoro)U-Um-(2′-deoxy-2′-fluoro)C-Um-(2′-deoxy-2′-fluoro)U-Cm-(2′-deoxy-2′-fluoro)U-Gm-(2′-deoxy-2′-fluoro)G-sp-Um-sp-(2′-deoxy-2′-fluoro)U) 5′-[O-[19-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-7,7-bis[12-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-7-mercapto-7-oxido-2,6,8-trioxa-7-phosphadodec-1-yl]-14-mercapto-14-oxido-5,9,13,15-tetraoxa-14-phosphanonadec-1-yl] hydroxy phosphate] (1:1)

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• [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.  [Content Brief]

  [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.  [Content Brief]

  [3]. WHO Drug Information. International Nonproprietary Names for Pharmaceutical