Three Researchers Granted Over $1 Million to De-Risk and Progress Preclinical Drug Candidates for Neurodegenerative Diseases

Inaugural funding through the Sprint to Lead program aims to translate scientific breakthroughs into therapeutic innovations

FORT WORTH, Texas, Nov. 10, 2026 /PRNewswire/ -- The Rainwater Charitable Foundation (RCF), one of the largest independent funders of neurodegenerative research, today announced over $1 million in grants to fund three innovative one-year research projects focused on targeting tau as an underlying disease driver, stemming from the 2026 Sprint to Lead (S2L) drug discovery grant program.

S2L fosters original ideas focused on accelerating the development of therapies for primary tauopathies within the hit-to-lead and lead optimization development phases of drug discovery. While the goal is to de-risk and advance preclinical drug candidates, successful projects may generate the evidence needed to support subsequent in vivo proof-of-concept and, where appropriate, later investigational new drug (IND)–enabling studies.

Primary tauopathies are a class of neurodegenerative diseases characterized by the abnormal accumulation of tau protein, including progressive supranuclear palsy (PSP), corticobasal degeneration (CBD), Pick's disease (PiD), and certain forms of frontotemporal degeneration (FTD). While emerging discoveries have deepened our understanding of tau-related neurodegeneration, substantial gaps remain in translating these insights into therapies that can deliver meaningful clinical benefit.

Through this inaugural S2L funding, three researchers will each receive $350,000 to support proposals selected for their potential to advance promising preclinical therapeutic candidates targeting tau-driven neurodegenerative diseases:

• Timothy Miller, MD, PhD, Washington University School of Medicine in St. Louis: Some primary tauopathies involve an imbalance between two forms of the tau protein, known as 3-repeat (3R) and 4-repeat (4R) tau. Dr. Miller and his team will advance a promising antisense oligonucleotide (ASO) designed to reduce excess 4R tau and restore a healthier balance of tau isoforms. In preclinical studies, the approach reduced excess 4R tau in disease-relevant mouse models and human neurons carrying disease-associated MAPT mutations, without reducing total tau in mice. Their work will continue to optimize the therapy for future clinical development and advance a potential disease-modifying treatment strategy for people living with 4R tauopathies.
• Neil Vasdev, PhD, MedChem Imaging: Rather than only trying to block harmful tau, Dr. Vasdev and his co-investigators are pursuing a strategy designed to remove it. They are developing compounds known as tau degraders, built to recognize tau and recruit the cell's natural protein-disposal machinery to tag it for destruction. Their work builds on tau-binding compounds developed as positron emission tomography (PET) imaging agents, including one that has advanced into first-in-human PET studies, providing a strong translational foundation for drug discovery. The team will seek to transform this tau-binding chemistry into potential therapeutics and identify promising degrader candidates for future testing in animal models of primary tauopathies.
• Markus Zweckstetter, PhD, brainQr Therapeutics: While many tau-targeted therapeutic approaches focus on clearing pathological tau after it has formed, Dr. Zweckstetter and his team are pursuing a differentiated strategy: developing brain-penetrant small molecules that directly bind tau and aim to prevent its conversion into disease-associated forms. This approach seeks to prevent pathological tau aggregation while preserving its physiological interactions with microtubules, an important consideration for neuronal function. Building on a promising brain-penetrant starting compound, the RCF-funded project will optimize a new series of tau-targeting molecules to identify a lead candidate for further preclinical development. The work could establish a novel therapeutic approach for tau-driven FTD and other primary tauopathies.

"Despite remarkable innovations in our understanding of tau pathology, transforming insights into effective therapies remains one of the field's greatest challenges," said Jeremy Smith, President of the Rainwater Charitable Foundation. "We believe that supporting bold, translational science at critical stages of drug discovery is imperative to help bridge that gap. Through Sprint to Lead, we aim to advance promising therapeutic approaches and bring renewed hope to people living with these devastating neurodegenerative diseases."

The RCF encourages applications from interdisciplinary teams that bring together diverse expertise. Details on the letter of intent (LOI) submission dates for the 2027 S2L program will be made available in the coming months. For more information, please click here.

About the Rainwater Charitable Foundation's Medical Research
The Rainwater Charitable Foundation (RCF) was created in the early 1990s by Richard E. Rainwater, the late private equity investor and philanthropist. When Richard was diagnosed with a rare neurodegenerative disease and primary tauopathy called Progressive Supranuclear Palsy (PSP), the RCF expanded its mission to accelerate the development of new diagnostics and treatments for tau-related neurodegenerative disorders. The RCF Medical Research Team supports this focus by managing the Tau Consortium, the Rainwater Prize, and the Tauopathy Challenge Workshop. With over $165 million invested in medical research to date, the RCF has helped to advance eight treatments into human trials. Currently, RCF supports medical research and a range of initiatives that expand opportunity, promote well-being, and strengthen communities in Fort Worth. For more information, please visit rainwatercharitablefoundation.org.

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Elizabeth Lentz
The Rainwater Charitable Foundation
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