For research use only. Not for therapeutic Use.
Fazirsiran Sodium (Cat No.: I040084) is an investigational RNA interference (RNAi) therapeutic designed to target mutant alpha-1 antitrypsin (Z-AAT) mRNA, reducing toxic protein accumulation in alpha-1 antitrypsin deficiency (AATD). By selectively silencing Z-AAT expression, it helps prevent liver damage and pulmonary complications. The sodium salt form enhances stability and bioavailability, making it suitable for in vivo and clinical applications. Fazirsiran is a promising candidate for AATD-related liver disease therapy, supporting precision medicine and RNA-based drug development for rare genetic disorders.
CAS Number | 2175009-09-1 |
Molecular Formula | C493H610F11N163Na43O312P43S6 |
Purity | ≥95% |
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. |