Fitusiran  (Synonyms: ALN-AT3SC; SAR439774)

Fitusiran (ALN-AT3SC) is a siRNA strategy. Fitusiran delivers siRNA targeting the SERPINC1 gene in hepatocytes to inhibit antithrombin synthesis. Fitusiran enhances thrombin activity and increases Thrombin generation, thereby restoring hemostatic function. Fitusiran can be used in hemophilia-related research^[1][2][3].

Fitusiran delivers siRNA targeting the SERPINC1 gene in hepatocytes to inhibit antithrombin synthesis. Fitusiran enhances thrombin activity and increases thrombin generation, thereby restoring hemostatic function^[1][2][3].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

16248.2 (AS: 7675 + SS: 8573.2)

1499251-18-1

Solid

White to off-white

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)

[Fitusiran]

Room temperature in continental US; may vary elsewhere.

4°C, sealed storage, away from moisture

*In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)

• [1]. Lu D, et al. Fitusiran: The first approved siRNA therapy for hemophilia via reducing plasma antithrombin levels. Drug Discov Ther. 2025;19(2):131-132.  [Content Brief]

  [2]. Srivastava A, et al. Fitusiran, an investigational siRNA therapeutic targeting antithrombin for the treatment of hemophilia: first results from a phase 3 study to evaluate efficacy and safety in people with hemophilia A or B without inhibitors (ATLAS-A/B)[J]. Blood, 2021, 138: LBA-3.

  [3]. 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.  [Content Brief]