Partnership to progress neurodegenerative disorder treatments

amyloid plaque tau

IRBM, Rainwater Charitable Foundation (RCF, Fort Worth, Texas), a private family foundation, and Weill Cornell Medicine (based in New York) are starting 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 Li Gan to pursue innovative therapies for neurodegenerative disorders. 

Neurodegenerative disorders include dementias, Parkinson’s disease, Alzheimer’s disease (AD), Huntington’s disease, and tauopathies, so called as they feature the accumulation in affected brain regions of large aggregates of tau proteins, often called tau tangles. The spread of tangles induces toxic neuroinflammatory responses, and often tracks closely with disease progression. 

The collaboration will support the progression of Li Gan’s work 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: “Neurodegenerative disorders can be devasting, as they cruelly, and eventually fatally, steal away an individual’s ability to be themselves. We are therefore privileged to start our work on Alzheimer’s with renowned specialists in the area: Rainwater Charitable Foundation and Weill Cornell Medicine. 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.” 

The Rainwater Charitable Foundation’s Glenn Harris, director of research partnerships, business development and the drug discovery lead, said: “The RCF is committed to funding neurodegenerative disease research with the goal of developing effective, cutting-edge treatments for primary tauopathies. To date, we have provided over $160 million in funding to promising research programs. Given the myriad challenges of developing drugs for these complex disorders, we hope to continue supporting this important work. We recognize the great strides and continued potential of Dr. Gan’s neuroscience drug discovery endeavors and are honored to partner with her and Weill Cornell Medicine.”

Weill Cornell Medicine’s Li Gan said: “Tau is a major driver of cognitive decline in AD 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.”

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

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

Weill Cornell Medicine Enterprise Innovation negotiated and helped facilitate the collaboration between Dr. Gan and the RCF with the aim to support efforts to discover new therapies for debilitating neuro-degenerative diseases.

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