The company’s lead program, VES001, is an oral, brain-penetrant sortilin inhibitor designed to increase levels of progranulin, a protein linked to neuroprotection. The therapy is being developed for frontotemporal dementia caused by GRN mutations (FTD-GRN), a genetic form of the disease with no approved treatments.

VES001 has completed Phase I trials in healthy volunteers, showing good safety, central nervous system penetration, and dose-dependent increases in progranulin. A Phase Ib/IIa trial (SORT-IN-2) is currently underway in asymptomatic GRN mutation carriers, with data expected in late 2025.

Vesper Bio originated from research at Aarhus University and has secured funding from investors including the Lundbeck Foundation and The Michael J. Fox Foundation. The latter supports exploration of sortilin inhibition in Parkinson’s disease. In 2025, Jacob Falck Hansen, formerly of Lundbeckfonden BioCapital, was appointed CEO to lead the company through its next phase of clinical development.