Fazirsiran sodium is a second-generation RNAi agent. Fazirsiran sodium 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 sodium can be used in the study of Alpha-1 Antitrypsin Deficiency (AATD) liver disease.
Molekulargewicht:
16532.90
Reinheit:
93.86
CAS Nummer:
[2175009-09-1]
Formel:
C493H610F11N163Na43O312P43S6
Target-Kategorie:
Small Interfering RNA (siRNA)
Anwendungsbeschreibung:
Oligonucleotides
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