Matthew Anderson, MD, PhD
Co-Director
Dr Matthew Anderson, MD, PhD joined the Oxford-Harrington Rare Disease Centre as Co-Director in early 2025.
Dr Anderson brings an extraordinary depth of expertise to the OHC mission, with a background that bridges medicine, neuroscience, and biotechnology. He holds a BS in Chemistry from Cornell College, followed by an MD and PhD from the University of Iowa, where he studied medicine, physiology, and biophysics. His training in pathology and neuropathology took him from the University of Iowa Hospitals to Harvard Medical School, where he later became Chief of Neuropathology.
He joined OHC from a position as VP and Head, Neuroscience Therapeutic Focus Area at Regeneron Pharmaceuticals, leading a team of 45 scientists. He has also conducted pioneering research in neuroscience and immunology as a research fellow at MIT.
Dr Anderson is also Sylvia K. Reitman Chair in Discovery and Innovation, and Senior Attending Physician at University Hospitals as well as Professor of Pathology at Case Western Reserve University.
Key Scientific Contributions
Dr Anderson’s work has directly shaped modern approaches to treating genetic diseases. His research into cystic fibrosis helped define disease mechanisms and therapeutic targets that underpinned the development of transformative small-molecule therapies, contributing to a shift from markedly shortened life expectancy to near-normal lifespans for many patients.
His past and ongoing work in epilepsy led to the discovery that arrested neurodevelopment underlies a genetic form of human epilepsy caused by dominant-negative mutations in the LGI1 gene — a field demonstrating the therapeutic potential of nucleic acid–based approaches.
Dr Anderson's work has also advanced our understanding of neuroimmune interactions by identifying CD8 T-cell-mediated pathology targeting the astrocyte glial limitans, helping to explain inflammatory signals observed in autism.