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.
Molecular Weight:
15587.60
Purity:
93.86
CAS Number:
[2175009-08-0]
Formula:
C493H65 F11N163O312P43S6
Target:
Small Interfering RNA (siRNA)
Application Notes:
Oligonucleotides
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