MAPT and progressive supranuclear palsy: BIIB080 (IONIS-MAPTRx / MAPTRx) targets MAPT mRNA via RNase H1 and achieved dose-dependent reductions in CSF total tau and p-tau181 in clinical trials, advancing to Phase 2 (CELIA), while NIO752 (Novartis) completed a Phase 1 trial in progressive supranuclear palsy (Harris et al. 2025).