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

Fazirsiran (ARO-AAT) is a second-generation RNAi agent. Fazirsiran consistes of a cholesterol-conjugated RNAi trigger (chol-RNAi) to selectively degrade Alpha1-antitrypsin (AAT) mRNA by RNAi 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.

For research use only. We do not sell to patients.

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 Chemical Structure

CAS No. : 2175009-08-0

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Description

Fazirsiran (ARO-AAT) is a second-generation RNAi agent. Fazirsiran consistes of a cholesterol-conjugated RNAi trigger (chol-RNAi) to selectively degrade Alpha1-antitrypsin (AAT) mRNA by RNAi 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.

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.

Clinical Trial
Molecular Weight

15587.60

CAS No.
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

[ARO-AAT]

Shipping

Room temperature in continental US; may vary elsewhere.

Storage

-20°C, sealed storage, away from moisture

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

Purity & Documentation
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Fazirsiran Related Classifications

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  • 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.

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Product Name:
Fazirsiran
Cat. No.:
HY-132604
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