Fitusiran_product_DataBase

Bioactivity	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].
Name	Fitusiran
CAS	1499251-18-1
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)
Molar Mass	16248.2 (AS: 7675 + SS: 8573.2)
Appearance	Solid
Transport	Room temperature in continental US; may vary elsewhere.
Storage	4°C, sealed storage, away from moisture *In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Reference	[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.

PeptideDB

Fitusiran

CAS: 1499251-18-1 F: W: 16248.2 (AS: 7675 + SS: 8573.2)