Matthew Gentry, Ph.D., an MBI researcher and chair of UF’s Department of Biochemistry and Molecular Biology, is among 10 recipients of the Oxford-Harrington Rare Disease Centre’s 2025 Rare Disease Scholar Awards. The $100,000 awards aim to advance promising lab studies into clinical practice.
Gentry’s project is focused on testing a novel enzyme therapy for Lafora disease, a recessive neurodegenerative disorder that causes childhood dementia.
“This is a devastating and fatal disease,” Gentry said. “Until their early teen years, these children seem to develop like their peers, and then they experience seizures followed by rapid Alzheimer’s-like dementia in their late teen years. Often they die within 10 years of the first symptoms, and there currently is no disease-modifying treatment.”
The funds will support advanced preclinical testing of the enzyme therapy, which has been shown in previous studies to cross the blood-brain barrier, penetrate cells and degrade the disease-causing accumulation of abnormal sugar molecules. If successful, this biological drug offers a potential strategy for treating many neurological diseases, Gentry said.
As part of the award, an advisory team will provide expert support for drug and business development, and there is an opportunity to compete for additional support and investment funds of up to $1 million.
The Oxford-Harrington Rare Disease Centre is a partnership between the University of Oxford in the United Kingdom and the Harrington Discovery Institute at University Hospitals in Cleveland, with a mission to accelerate progress by combining philanthropy, world-class science and entrepreneurial drug development.