RNAi Therapeutics for Diseases Involving Protein Aggregation: Fazirsiran for Alpha-1 Antitrypsin Deficiency-associated Liver Disease

This article describes the role of misfolded proteins and protein aggregates in disease, and options for therapeutic approaches. The RNAi mechanism is discussed, along with how the siRNA therapeutic fazirsiran for the treatment of AATD was developed. We also describe implications of siRNA therapeutics in extrahepatic diseases.EXPERT OPINION: Using RNAi as a therapeutic approach is well suited to treat disease in conditions where an excess of a protein or the effect of an abnormal mutated protein causes disease. The results observed for the first few siRNA therapeutics that were approved or are in development provide an important promise for the development of future drugs that can address such conditions in a specific and targeted way. Current developments should enable the use of RNAi therapeutics outside the liver, where there are many more possible diseases to address.PMID:37470509 | DOI:10.1080/13543784.2023.2239707
Source: Expert Opinion on Investigational Drugs - Category: Drugs & Pharmacology Authors: Source Type: research