Nature Communications, Published online: 14 September 2026; doi:10.1038/s41467-026-77638-2 Treatment for frontotemporal dementia due to GRN haploinsufficiency (FTD-GRN) are lacking. Here, in this preclinical and Phase 1 clinical trial, the authors show that VES001, an oral small molecule sortilin inhibitor, is safe and well tolerated, and increased progranulin levels.
Preclinical and clinical investigation of VES001, an oral small molecule sortilin receptor inhibitor for the treatment of frontotemporal dementia: a first-in-human randomized placebo-controlled phase 1 trial
Mads Kjolby