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[1]. Fazirsiran can be used in the study of Alpha-1 Antitrypsin Deficiency (AATD) liver disease.
Product Specifications
CAS Number
[2175009-08-0]
Product Name Alternative
ARO-AAT
UNSPSC
12352208
Target
Small Interfering RNA (siRNA)
Type
Oligonucleotides
Related Pathways
Epigenetics
Applications
Metabolism-protein/nucleotide metabolism
Field of Research
Metabolic Disease
Assay Protocol
https://www.medchemexpress.com/fazirsiran.html
Purity
93.86
Solubility
10 mM in H2O
Smiles
[Fazirsiran]
Molecular Formula
C493H65 F11N163O312P43S6
Molecular Weight
15587.60
References & Citations
[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.
Shipping Conditions
Blue Ice
Storage Conditions
-20°C (Powder, sealed storage, away from moisture)
Scientific Category
Oligonucleotides
Clinical Information
Phase 3
Available Sizes
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