APOC3-GalNAc ASO

APOC3-GalNAc ASO is an antisense oligonucleotide (ASO) conjugated with N-acetylgalactosamine (GalNAc), designed as a genetically targeted therapy for metabolic diseases, including nonalcoholic steatohepatitis (NASH). The GalNAc conjugate facilitates targeted delivery of the ASO to hepatocytes (liver cells) by binding to the asialoglycoprotein receptor (ASGPR). Once internalized, the ASO binds to the messenger RNA (mRNA) encoding Apolipoprotein C-III (APOC3), leading to its degradation via RNase H-mediated cleavage. This process prevents the translation of the APOC3 protein, which is a key regulator of triglyceride metabolism. By reducing APOC3 levels, the drug aims to lower circulating triglyceride levels. This drug is being developed through a collaboration between Wave Life Sciences, leveraging its stereopure chemistry platform, and Pfizer, contributing its hepatic targeting technology.