VES001

VES001 is a first-in-class, orally available, brain-penetrant small molecule sortilin antagonist developed by Vesper Bio for the treatment of frontotemporal dementia (FTD), particularly in patients with progranulin gene (GRN) mutations. The drug selectively binds to the sortilin receptor on neurons and inhibits its interaction with progranulin, thereby preventing the internalization and degradation of progranulin. This mechanism helps maintain and normalize extracellular progranulin levels, which are critical for neuronal health. By restoring progranulin concentrations, VES001 aims to modify disease progression in FTD(GRN). Clinical studies have demonstrated that VES001 is safe and well-tolerated in healthy volunteers, shows excellent pharmacokinetics with effective CNS penetration after oral administration, and results in significant increases in plasma and CNS progranulin levels[1][2][3][4][5][6][7].

Other names

VES-001VES001VES 001