WVE-006

WVE-006 is a first-in-class, GalNAc-conjugated, subcutaneously administered A-to-I RNA editing oligonucleotide (AIMer) developed by Wave Life Sciences. It is designed to correct the single base mutation in messenger RNA coded by the SERPINA1 Z allele, which causes alpha-1 antitrypsin deficiency (AATD). By enabling precise adenosine-to-inosine editing of the mutant mRNA transcript in hepatocytes, WVE-006 restores production and circulation of functional wild-type M-AAT protein. This approach aims to address both lung and liver manifestations of AATD by increasing levels of functional alpha-1 antitrypsin and reducing toxic Z-AAT protein aggregation. The drug leverages endogenous ADAR enzymes for targeted RNA editing and has demonstrated potent activity in preclinical models as well as positive proof-of-mechanism data in early clinical trials[1][5][6][7][8].