| Chemical Properties | Back Directory | [storage temp. ]
Store at -20°C | [form ]
Solid | [color ]
White to off-white | [Sequence]
RNA, ((2'-deoxy-2'-fluoro)G-sp-Gm-sp-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)A-Am- (2'-deoxy-2'-fluoro)C-Am-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)A-Um-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)A-Cm-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)C-Am-(2'-deoxy-2'-fluoro)A),3'-[[(2S,4R)-1-[29-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-14,14-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-Dgalactopyranosyl]oxy]-1-oxopentyl]amino]propyl]amino]-3-oxopropoxy]methyl]-1,12,19,25-tetraoxo-16-oxa-13,20,24-triazanonacos-1-yl]-4-hydroxy-2-pyrrolidinyl]methyl hydrogen phosphate], complex with RNA (Um-sp-(2'-deoxy-2'-fluoro)U-sp-Gm-(2'-deoxy2'-fluoro)A-Am-(2'-deoxy-2'-fluoro)G-Um-(2'-deoxy-2'-fluoro)A-Am-(2'-deoxy-2'-fluoro)A-Um-Gm-Gm-(2'-deoxy-2'-fluoro)U-Gm-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)A-Am-(2'-deoxy-2'-fluoro)C-Cm-sp-Am-sp-Gm) |
| Hazard Information | Back Directory | [Uses]
Fitusiran (ALN-AT3SC), an small interfering RNA, specifically targets antithrombin (AT) messenger RNA to lower production of AT in the liver. Fitusiran increases thrombin generation and has the potential for the research of the hemophilia[1]. | [storage]
Store at -20°C | [References]
[1] Pasi KJ, et al. Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran-Results of the phase 1 inhibitor cohort. J Thromb Haemost. 2021;19(6):1436-1446. DOI:10.1111/jth.15270 |
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