Several clinical trials testing experimental, potentially disease-modifying treatments are underway for FTD caused by the progranulin gene mutation (FTD-GRN), employing unique strategies to restore progranulin levels, including a monoclonal antibody trial to block the progranulin/sortilin pathway (NCT04374136), two separate gene therapy trials using adeno-associated viral vectors (NCT04408625; NCT04747431), and a protein replacement therapy trial (NCT05262023). The gene discussed is SORT1; the disease is frontotemporal dementia.