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Expansion Therapeutics Announces In-Licensing of Two New Research Programs from Scripps Research...

Key Program Targeting Toxic Brain Protein Tau Supported by over $3 Million in Grants to Scripps Research from the Rainwater Charitable Foundation

BOSTON--(BUSINESS WIRE)--Expansion Therapeutics, Inc., a biotechnology company advancing transformative medicines to patients with RNA-mediated diseases, today announced the in-licensing of two new research programs from Scripps Research. One program targets tau, a key driver of dementia disorders, while the second program targets another crucial protein related to neurodegeneration. The tau program was supported by over $3 million in grants to Scripps Research from the Rainwater Charitable Foundation over nine years to the company’s scientific founder and professor at Scripps Research, Matthew D. Disney, Ph.D.

“We are very excited about the addition of these two new programs, which complement and strengthen our portfolio of small molecule medicines to treat RNA-mediated diseases, including neurodegenerative and neuromuscular disorders,” said Renato Skerlj, Ph.D., Chief Executive Officer and President of Expansion Therapeutics. “We look forward to advancing the preclinical and clinical development of these novel agents to ultimately help patients suffering from these devastating diseases, who currently have limited or no treatment options.”

The tau program builds on the observation that defective pre-mRNA encoding the production of tau protein is involved in the formation of neurofibrillary tangles and has been linked to tauopathies such as Alzheimer’s Disease, Frontotemporal Dementias, Progressive Supranuclear Palsy and other neurodegenerative diseases. Dr. Disney has shown tau pre-mRNA splicing modulators to be effective in animal models of tau-mediated disease, reducing brain tau aggregates and ameliorating behavioral deficits.

“We recognized the potential of Matt Disney’s approach to create oral, brain-penetrant treatments for tauopathies. We are pleased to have supported Matt’s work at Scripps Research for nine years and helped enable an integrated drug discovery approach, bringing this program to the point where Expansion Therapeutics will continue its development towards the clinic,” said Leticia M. Toledo-Sherman, Ph.D., Senior Director of Drug Discovery for the Rainwater Charitable Foundation. “We look forward to continued interaction with the team as the program progresses into clinical testing in patients who so desperately need novel medicines.”

“I am delighted that our work on tau splicing modulators has been in-licensed by Expansion Therapeutics and truly appreciate the support from the Rainwater Charitable Foundation on this program, from inception through to this critical inflection point. This program, along with others in the Expansion pipeline, have the potential to make a meaningful impact on the lives of people living with neurodegenerative diseases,” said Disney, a chemistry professor at Scripps Research in Jupiter, Florida, and scientific founder of Expansion Therapeutics.

About Expansion Therapeutics

Expansion Therapeutics is a drug discovery and development company pursuing the vast potential of small molecule medicines for RNA-mediated diseases. Based on exclusive worldwide rights to groundbreaking research from the laboratory of Matthew D. Disney, Ph.D., at Scripps Research, Expansion has assembled the intellectual property, know-how, and proprietary enabling technologies and tools necessary to facilitate the creation of potent and specific small molecule binders of RNA. Through this unique platform, Expansion is building a portfolio of novel RNA-targeted drug candidates with activity across a broad number of disease indications. Headquartered in Boston, Massachusetts, Expansion’s research facility is located in Jupiter, Florida. For more information, visit

About the Rainwater Charitable Foundation

The Rainwater Charitable Foundation (RCF) was created in the early 1990s by renowned private equity investor and philanthropist Richard E. Rainwater. RCF supports a range of programs in K-12 education, medical research and other worthy causes. In order to deliver on its mission to accelerate the development of new diagnostics and treatments for tau-related neurodegenerative disorders, the Rainwater Charitable Foundation Medical Research team manages the Tau Consortium and the Rainwater Prize Program. With over $145 million invested to date, the Rainwater family has helped to advance eight treatments into human trials. For more information, please visit, and

About Scripps Research

Scripps Research is an independent, non-profit biomedical institute ranked the most influential in the world for its impact on innovation. With campuses in La Jolla, California and Jupiter, Florida, the institute is advancing human health through profound discoveries that address pressing medical concerns around the globe. The Scripps Research drug discovery and development division, Calibr, works hand-in-hand with scientists across disciplines to bring new medicines to patients as quickly and efficiently as possible, while teams at Scripps Research Translational Institute harness genomics, digital medicine and cutting-edge informatics to understand individual health and render more effective healthcare. Scripps Research also trains the next generation of leading scientists at its Skaggs Graduate School, consistently named among the top 10 U.S. programs for chemistry and biological sciences. Learn more at


Media: Mike Beyer Sam Brown Inc. 312-961-2502



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