IONIS-MAPTRx (BIIB080) is the first Tau-lowering antisense oligonucleotide (ASO). IONIS-MAPTRx has the potential for the research of Alzheimer Disease.
In Vivo
Intrathecal administrations of IONIS-MAPTRx in nonhuman primates showed a mean MAPT mRNA reduction of 77% in frontal cortex and 74% in hippocampus without dose-limiting side effects . MCE has not independently confirmed the accuracy of these methods. They are for reference only.