Bioactivity | 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. |
CAS | 2175009-09-1 |
Formula | C493H610F11N163Na43O312P43S6 |
Molar Mass | 16532.90 |
Transport | Room temperature in continental US; may vary elsewhere. |
Storage | Please store the product under the recommended conditions in the Certificate of Analysis. |
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. |