Tau antisense oligonucleotide (ASO)

Tau antisense oligonucleotides (ASOs) are a class of synthetic, single-stranded nucleic acid polymers designed to target microtubule-associated protein tau (MAPT) pre-mRNA or mRNA. By binding to the target transcript, these ASOs promote its degradation (typically via RNase H-mediated cleavage) or modulate splicing (e.g., to skip exon 10 and reduce 4R-tau isoforms), thereby reducing the expression and accumulation of tau protein. Pathological aggregation of tau into neurofibrillary tangles is a key driver of neurodegeneration in Alzheimer's disease, Down syndrome, progressive supranuclear palsy, and other tauopathies. Several tau-targeting ASOs, such as diranersen (BIIB080) developed by Ionis and Biogen, and NIO752 developed by Novartis, are in clinical development, while others are being explored preclinically by companies like Atalanta Therapeutics and Eli Lilly.

Other names

MAPT ASOMAPT antisense oligonucleotideTau ASOtau-targeting antisense oligonucleotide