Biotechnology

Partnership for the advancement of neurodegenerative disorders

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IRBM, Rainwater Charitable Foundation (RCF, Fort Worth, Texas), a private family foundation, and Weill Cornell Medicine (based in New York) began a multi-year drug discovery collaboration to identify small molecule cGAS (cyclic GMP-AMP synthase) inhibitors for treatment of tauopathies and other neurodegenerative diseases.

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

Neurodegenerative disorders including dementia, Parkinson’s disease, Alzheimer’s disease (AD), Huntington’s disease, and tauopathies, are so called because they display large accumulations of tau protein aggregates in affected brain regions, often called tau tangles. Dissemination of tangles induces a toxic neuroinflammatory response, and often follows disease progression.

This collaboration will support the development of Li Gan’s work targeting neuroimmune pathways to maintain cognitive resilience to tau aggregates through cGAS inhibition. 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 chief science officer, Carlo Toniatti, said: “Neurodegenerative disorders can be devastating, because they are cruel, and ultimately fatal, stealing from individuals the ability to be themselves. Therefore, we are privileged to begin our work on Alzheimer’s with renowned specialists in this field: Rainwater Charitable Foundation and Weill Cornell Medicine. Together, we aim to bring many first-class pre-clinical assets to clinical trials to improve patient outcomes. As a first objective, we will provide our expertise in drug discovery and development to support the translation of Dr. The extraordinary gan becomes an effective therapy. I look forward to seeing how our collaboration will make a difference.”

Glenn Harris of the Rainwater Charitable Foundation, director of research partnerships, business development and drug discovery leader, said: “RCF is committed to funding neurodegenerative disease research with the goal of developing an advanced and effective treatment for primary tauopathy. To date, we have provided over $160 million in funding to promising research programs. Given the many challenges in developing drugs for these complex disorders, we look forward to continuing to support this important work. We acknowledge the great strides and continued potential of Dr. Gan and are honored to partner with him and Weill Cornell Medicine.”

Li Gan of Weill Cornell Medicine said: “Tau is a major driver of cognitive decline in AD and related dementias, but to date, we have had no clinical success in any anti-tau therapy. By advancing our new anti-know therapy, I see this collaboration making a fundamental difference to this devastating situation.”

Gan is the founder of Aeton Therapeutics Inc. Dr. Gan also holds equity in the company and serves as a consultant.

About six million people in the US have Alzheimer’s disease, and hundreds of thousands more have less well-known tau-related neurodegenerative disorders such as frontotemporal dementia and progressive supranuclear palsy (PSP).

Weill Cornell Medicine Enterprise Innovation negotiates and helps facilitate collaborations between Dr. Gan and RCF with the aim of supporting efforts to find new therapies for debilitating neuro-degenerative diseases.

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