A phase 1b trial by Biogen and Ionis Pharmaceuticals testing IONIS-MAPTRX for the treatment of Alzheimer’s disease has reported a robust time and dose-dependent reduction in total tau and phospho-tau in the cerebrospinal fluid; results from mouse and non-human primates were also encouraging but the effects on cognition are as yet unknown [86]. The gene discussed is MAPT; the disease is Alzheimer disease.