Collaborators pursue anti-tau therapy for neurodegeneration

Tau proteins aggregate to neurofibrillary tangles in a neuron axon

IRBM, Rainwater Charitable Foundation and Weill Cornell Medicine will start of a multi-year drug discovery collaboration to identify small molecule cGAS (cyclic GMP-AMP synthase) inhibitors for the treatment of tauopathies and other neurodegenerative diseases.

The collaboration brings together IRBM’s capabilities in drug discovery and development, RCF’s medical research resources via the Tau Consortium, and the expertise of Weill Cornell Medicine neuroscientist Dr Li Gan to pursue innovative therapies for neurodegenerative disorders.

This collaboration will support the progression of Dr Li Gan’s innovative work in targeting neuroimmune pathways to preserve cognitive resilience against tau aggregates through inhibition of cGAS. This protein is the primary sensor of cytosolic double-stranded DNA (dsDNA), and when dysfunctional, is involved in toxic neuroimmune responses to tau tangles.

IRBM’s Chief Science Officer Carlo Toniatti said: “Together, we aim to bring a wealth of first-in-class, pre-clinical assets into clinical trials to improve patient outcomes. As a first goal, we will provide our expertise in drug discovery and development to support the translation of Dr Gan’s outstanding neuroscience research into effective therapeutics. I look forward to seeing how our collaboration will make a difference.”

Dr Li Gan added: “Tau is a major driver of cognitive decline in Alzheimer’s disaese and related dementia, but to date we have had no clinical success in any anti-tau therapies. By advancing our novel anti-tau therapy, I see this collaboration leading to a fundamental difference to this devastating situation.”

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