ASO 56

ASO 56 is an experimental antisense oligonucleotide designed by researchers at the University of California San Diego for the treatment of liver fibrosis. It works by selectively targeting TEAD1 mRNA, modulating its splicing to suppress the production of the fibrosis-promoting variant of the TEAD1 protein, thereby reducing fibrotic protein expression in hepatic stellate cells. In mouse models, ASO 56 almost entirely prevented the development of primary sclerosing cholangitis (PSC) and effectively reduced liver fibrosis in models of metabolic dysfunction-associated steatohepatitis (MASH). This approach is notable for its specificity to liver stellate cells upon intravenous administration, helping to avoid off-target effects. ASO 56 remains investigational and is being considered for clinical development, with a primary focus on PSC, a rare and severe liver disease with limited treatment options.

Other names

antisense oligonucleotide 56