Fazirsiran

• CAT Number: I045364
• CAS Number: 2175009-08-0
• Molecular Weight: 15587.60
• Purity: ≥95%

Fazirsiran (Cat No.: I045364) is an investigational RNA interference (RNAi) therapeutic targeting the mutant Z allele of the SERPINA1 gene, which encodes alpha-1 antitrypsin (AAT). In individuals with AAT deficiency, misfolded AAT accumulates in the liver, leading to liver disease. Fazirsiran reduces production of the abnormal protein by silencing the mutant gene transcript, thereby alleviating liver damage. Administered subcutaneously, it has shown promising results in early clinical trials. Fazirsiran offers a potential disease-modifying treatment for patients with AAT deficiency-related liver disease.

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.
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