Article

Clinical investigation of VES001, a novel oral small molecule sortilin receptor inhibitor, for the treatment of frontotemporal dementia

2026-03-31

Abstract excerpt

<title>Abstract</title> <p>GRN gene haploinsufficiency reduces progranulin and causes frontotemporal dementia (FTD-GRN). Restoring progranulin to physiological levels counteracts the molecular deficit underlying the disease. VES001 is an orally administered small molecule sortilin inhibitor that increases progranulin by blocking its interaction with sortilin without downregulating sortilin. In preclinical studies...

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Literature Corpus work: ae93ab63-62e7-5efa-88a4-0d899cf2a098
DOI: 10.21203/rs.3.rs-8405910/v1

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