pyruvate carboxylase ASO

Pyruvate carboxylase ASO is an experimental antisense oligonucleotide drug designed to specifically reduce the expression of pyruvate carboxylase, a mitochondrial enzyme that catalyzes the conversion of pyruvate to oxaloacetate, the first step in gluconeogenesis. Mechanistically, the drug reduces hepatic and adipose tissue pyruvate carboxylase mRNA and protein, lowering endogenous glucose production and fasting plasma glucose levels. Animal studies have shown it can decrease adiposity, plasma lipid levels, hepatic steatosis, and improve hepatic insulin sensitivity, with good tolerability and no observed hepatotoxicity or hypoglycemia[1][3]. The use of this ASO thus offers a potential new pathway for treating metabolic disorders marked by excessive hepatic glucose production, such as type 2 diabetes and obesity[1][3]. As of now, it remains in preclinical or early clinical development settings and does not have an approved indication in humans.

Other names

pyruvate carboxylase antisense oligonucleotidePC ASO