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Fazirsiran

(Synonyms: ARO-AAT)

Fazirsiran (ARO-AAT) is a second-generation RNAi agent. Fazirsiran consistes of a cholesterol-conjugated RNAi trigger (chol-RNAi) to selectively reduce Alpha1-antitrypsin (AAT) synthesis and a melittin-derived peptide conjugated to N-acetylgalactosamine (NAG) formulated as the excipient EX1 to promote endosomal escape of the chol-RNAi in hepatocytes. Fazirsiran can be used in the study of Alpha-1 Antitrypsin Deficiency (AATD) liver disease. Alpha-1 antitrypsin deficiency (AATD) is caused by mutations in the alpha-1 antitrypsin (SERPINA1) gene.

The free form of the compound is prone to instability, it is advisable to consider the stable salt form (Fazirsiran sodium) that retains the same biological activity.

Para uso exclusivo en investigación. No vendemos a pacientes.

  • Almacenamiento:

    -20°C, sealed storage, away from moisture

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

  • Fazirsiran Chemical Structure
    Fazirsiran Chemical Structure
No. CAS: 2175009-08-0
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609-228-6898
[email protected] [email protected]
Encuentre su distribuidor local Your local distributor: Hycultec GmbHHycultec
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  • Fazirsiran Chemical Structure
    Fazirsiran Chemical Structure RNA, ([1'-de(6-amino-9H-purin-9-yl)]dA-(5'→5')-sp-Am-Gm-Cm-Gm-Um-Um- Um-Am-(2'-deoxy-2'-fluoro)G-(2'-deoxy-2'-fluoro)G-(2'-deoxy-2'-fluoro)C-Am-Um-Gm-Um-Um-Um-Am-Am-Cm-Am-(3'→3')-sp-[1'-de(6-amino-9H-purin-9-yl)]dA),3'-[O-[cis-4-[(3S,8S)-17-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-3,8-bis[[[2-[2-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]ethoxy]ethyl]amino]carbonyl]-1,6,11-trioxo-15-oxa-2,7,12-triazaheptadec-1-yl]cyclohexyl] hydrogen phosphorothioate], complex with RNA (Um-sp-(2'-deoxy-2'-fluoro)G-sp-Um-(2'-deoxy-2'-fluoro)U-Am-(2'- deoxy-2'-fluoro)A-Am-Cm-Am-Um-Gm-(2'-deoxy-2'-fluoro)C-Cm-(2'-deoxy-2'-fluoro)U-Am-(2'-deoxy-2'-fluoro)A-Am-(2'-deoxy-2'-fluoro)C-Gm-(2'-deoxy-2'- fluoro)C-sp-Um) (1:1)
    1/16
  • RNA, ([1'-de(6-amino-9H-purin-9-yl)]dA-(5'→5')-sp-Am-Gm-Cm-Gm-Um-Um- Um-Am-(2'-deoxy-2'-fluoro)G-(2'-deoxy-2'-fluoro)G-(2'-deoxy-2'-fluoro)C-Am-Um-Gm-Um-Um-Um-Am-Am-Cm-Am-(3'→3')-sp-[1'-de(6-amino-9H-purin-9-yl)]dA),3'-[O-[cis-4-[(3S,8S)-17-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-3,8-bis[[[2-[2-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]ethoxy]ethyl]amino]carbonyl]-1,6,11-trioxo-15-oxa-2,7,12-triazaheptadec-1-yl]cyclohexyl] hydrogen phosphorothioate], complex with RNA (Um-sp-(2'-deoxy-2'-fluoro)G-sp-Um-(2'-deoxy-2'-fluoro)U-Am-(2'- deoxy-2'-fluoro)A-Am-Cm-Am-Um-Gm-(2'-deoxy-2'-fluoro)C-Cm-(2'-deoxy-2'-fluoro)U-Am-(2'-deoxy-2'-fluoro)A-Am-(2'-deoxy-2'-fluoro)C-Gm-(2'-deoxy-2'- fluoro)C-sp-Um) (1:1)