Fitusiran
Based on 1 Customer Validation
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.
Para uso exclusivo en investigación. No vendemos a pacientes.
- Pureza: 98.70%
- No. CAS: 1499251-18-1
- Peso molecular:16248.2 (AS: 7675 + SS: 8573.2)
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Almacenamiento:
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Actividad biológica
Descripciòn
In Vitro
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.
Chemical Information
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No. CAS 1499251-18-1
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Appearance Solid
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Peso molecular 16248.2 (AS: 7675 + SS: 8573.2)
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Color White to off-white
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SMILES
[Fitusiran]
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Synonyms
ALN-AT3SC; SAR439774
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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)
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Envío
Room temperature in continental US; may vary elsewhere.
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Almacenamiento
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Solvente y solubilidad
In Vitro:
H2O : 50 mg/mL (Need ultrasonic)
Pureza y Documentación
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Ficha de datos (273 KB)
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SDS (252 KB)
- English - EN (252 KB)
- Français - FR (252 KB)
- Deutsch - DE (252 KB)
- Norwegian - NO (252 KB)
- Español - ES (252 KB)
- Swedish - SV (252 KB)
- Italian - IT (252 KB)
- Korean - KR (252 KB)
- Portuguese - PT (252 KB)
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Instrucciones de manejo (2242 KB)
Referencias
[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]
[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]
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)