Tau Protein Modulator

PTC-553 is a microtubule-associated protein tau (MAPT) mRNA splicing modulator. PTC-553 promotes exon 10 exclusion by modulating MAPT pre-mRNA splicing, corrects the 4R/3R tau isoform imbalance, thereby reducing the pathological accumulation of 4R tau protein and alleviating neurotoxicity. PTC-553 can be used for research on frontotemporal dementia and tauopathies.

PTC-003 is an orally active and blood-brain barrier-penetrant MAPT pre-mRNA splicing modulator. PTC-003 promotes skipping of exon 10 of the MAPT gene, shifting the protein isoform balance from toxic 4R-tau toward 3R-tau. PTC-003 reduces the accumulation of hyperphosphorylated tau, oligomeric tau, and insoluble tau protein. PTC-003 protects neurons from glutamate-induced neuronal stress injury, and in humanized MAPT gene-replaced N279K mice, it corrects the 3R/4R-tau isoform ratio in brain tissue and reduces the level of pathogenic pTau T231. PTC-003 can be used in research related to neurodegenerative diseases.