The interim results of the early-phase (1/2) randomized controlled trials (RCTs), comprising three ongoing gene replacement studies (PROCLAIM, ASPIRE-FTD, upliFT-D) and one immune-modulating monoclonal antibody (INFRONT, now in phase 3)—all targeting the FTD-GRN mutation—show safety, tolerability, and effectiveness in restoring progranulin levels. The gene discussed is GRN; the disease is frontotemporal dementia.