Fazirsiran

• CAT Number: I045364
• CAS Number: 2175009-08-0
• Molecular Weight: 15587.60
• Purity: ≥95%

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.
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].

Reference

[1]. Christine I Wooddell, et al. Development of an RNAi therapeutic for alpha-1-antitrypsin liver disease. JCI Insight. 2020 Jun 18;5(12):e135348.
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