720-ROCK2 Inhibition for Treatment of Liver FibrosisPaper Talk

720-ROCK2 Inhibition for Treatment of Liver Fibrosis

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This research identifies ROCK2 as a critical driver of liver fibrosis, specifically appearing in elevated levels within the vascular endothelial cells and hepatic stellate cells of diseased livers. Scientists developed TDI01, a highly selective and potent inhibitor designed to block this protein’s activity and restore healthy vessel function. The study demonstrates that TDI01 successfully reduces scarring and inflammation in rodent and minipig models of metabolic dysfunction-associated steatohepatitis (MASH). Early human trials confirm the drug is safe and well-tolerated, showing favorable absorption and metabolism in healthy volunteers. Furthermore, an extended clinical study of six patients suggests a promising trend toward reversing liver fibrosis and improving hepatic health. These findings highlight ROCK2 as a viable therapeutic target for treating chronic liver diseases that currently lack effective medical options.

References:

  • Hu Y, Yang B, Xiao C, et al. Selective targeting of endothelial and perivascular angiocrine ROCK2 treats liver fibrosis[J]. Cell, 2026.