2. Epigenetics
  3. Small Interfering RNA (siRNA)
  4. Fazirsiran

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.

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)

Fazirsiran Estructura química

No. CAS : 2175009-08-0

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1 mg Pide un presupuesto y un plazo de entrega
5 mg Pide un presupuesto y un plazo de entrega
10 mg Pide un presupuesto y un plazo de entrega

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Descripciòn

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[1]. 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.

In Vivo

Fazirsiran (ARO-AAT; sc; 4 mg/kg; on 1, 15, 29, 43 days during 57 days) deeply reduces Z-AAT mRNA and protein in five-week-old male PiZ mice[1].

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

15587.60

No. CAS
Appearance

Solid

Color

White to off-white

Sequence

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)
SMILES

[Fazirsiran]
Envío

Room temperature in continental US; may vary elsewhere.
Almacenamiento

-20°C, sealed storage, away from moisture

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

Pureza y Documentación
Referencias
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Fazirsiran Related Classifications

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Help & FAQs
  • Do most proteins show cross-species activity?

    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

Keywords:

Fazirsiran2175009-08-0ARO-AATSmall Interfering RNA (siRNA)RNAicholesterolchol-RNAiAATmRNAN-acetylgalactosamineNAGEX1hepatocytesAlpha-1 Antitrypsin DeficiencyAATDInhibitorinhibitorinhibit

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Nombre del producto:
Fazirsiran
Cat. No.:
HY-132604
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