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Mar 02, 2015
 |  BioCentury  |  Emerging Company Profile

The road to Tau

Asceneuron plans to show power of tau in AD by first tackling Orphan tauopathy

Asceneuron S.A. is working to treat neurodegeneration by preventing formation of neurofibrillary tangles in the hopes this mechanism will fare better in treating Alzheimer's disease than amyloid-targeted products.

Neurofibrillary tangles arise when microtubule-associated protein tau (tau; MAPT; FTDP-17) aggregates into helical filaments and larger structures, a process associated with tau hyperphosphorylation. Glycosylation by enzymes such as O-linked N-acetylglucosamine (GlcNAc) transferase (OGT) protects against tau hyperphosphorylation; however, this glycosylation is reversed by the enzyme O-linked N-acetylglucosaminidase (O-GlcNAcase).

Asceneuron's ASN-561 is a small molecule O-GlcNAcase inhibitor. Inhibiting O-GlcNAcase may promote glycosylated tau, thereby preventing hyperphosphorylation and subsequent tangle formation.

CEO Dirk Beher said Asceneuron chose to focus on tau, the lesser explored of the two major AD pathologies, because of potential partners' interest in the space and the chance to establish proof of concept in progressive supranuclear palsy (PSP). PSP is an Orphan neurodegenerative disorder...

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