The Rainwater Charitable Foundation (RCF), one of the largest independent funders of neurodegenerative disease research, has announced a partnership with Charles River Laboratories to accelerate the development of treatments that arise from discoveries made in academic labs sponsored by the foundation.
The partnership will initially support an integrated drug discovery program between RCF’s Tau Consortium, Charles River and Dr. Matthew Disney from Scripps Research to develop a brain penetrant 4R tau mRNA splicing modulator for the treatment of primary tauopathies, a news release said.
The program builds on initial discoveries made by Disney, who has developed tools and techniques that enable the design of drug-like compounds that bind, and destroy or repair, defective non-coding RNA.
With support from Fort Worth-based RCF and others, Disney has discovered compounds that bind and rescue defective tau pre-mRNA involved in the formation of the neurofibrillary tangles that has been linked to tauopathies such as Alzheimer’s Disease, Frontal Temporal Dementias and Progressive Supranuclear Palsy.
Building on these compounds as starting points, in continued collaboration with the Disney lab at Scripps, Charles River scientists will provide RCF and Scripps with drug discovery and safety assessment services and expertise. Funding for this program is provided by RCF under the Tau Consortium and its Tau Pipeline Enabling Program (T-PEP), a partnership between RCF and the Alzheimer’s Association.
“This partnership is a significant step forward in bringing potential treatments closer to the clinic for patients suffering from tauopathies, and therefore closing the gap between drug candidates and the cutting-edge research that can help them,” said Leticia Toledo-Sherman, Ph.D., Senior Director of Drug Discovery for the Tau Consortium at the Rainwater Charitable Foundation.
“Given the difficulties in developing therapies for these complex disorders, the impressive track record of Charles River in drug discovery and development – including delivery of several clinical-stage molecules in neurodegenerative diseases such as Parkinson’s Disease to their clients – was a key driver in creating this partnership,” Toledo-Sherman said.
“We are excited to work with the RCF,” said Wilbert Frieling, Corporate Senior Vice President, Global Discovery Services, Charles River.
“This research is focused on an area of unmet medical need, and it builds upon the biology, medicinal chemistry and biophysical expertise Charles River has gained through experience in small molecule programs targeting mRNA, including in Huntington’s disease,” Frieling said.
“Academic research serves as an engine of ideas and innovation. To advance academic discoveries into the clinic, partnerships between nonprofit and corporate organizations are needed. We applaud the Rainwater Charitable Foundation for its vision and leadership in this area,” said Scripps Research President and CEO Peter Schultz, Ph.D.
“We are grateful for the financial support from the Rainwater Charitable Foundation and to be working with the Charles River team,” adds Dr. Disney at Scripps Research. “Rainwater’s commitment to an integrated drug discovery model will make a key difference in producing the breakthrough that people affected by tau-related illnesses need,” Schultz said in the announcement.
“The Alzheimer’s Association’s collaboration with the Rainwater Charitable Foundation supports important research investigating ways to target abnormal tau, a toxic brain protein that is a hallmark of Alzheimer’s, Frontotemporal dementia, Lewy body dementia and over 20 other brain diseases,” said Maria C. Carrillo, Ph.D., Alzheimer’s Association chief science officer. “We are excited to leverage this new partnership with Charles River Labs to further advance this promising field of drug research and enrich the pipeline for possible treatments, bringing us closer to more effective therapies, and even prevention strategies for these diseases.”
The Rainwater Charitable Foundation (RCF) was created in the early 1990s by renowned private equity investor and philanthropist Richard E. Rainwater.
Mr. Rainwater died in 2015 after a long, debilitating battle with a rare and incurable brain disease called progressive supranuclear palsy.