Ten scientists selected for second annual award to pursue innovations in gene, RNA, small molecule and cell therapies to treat rare and ultra-rare diseases
OXFORD, England and CLEVELAND, Nov. 20, 2025 /PRNewswire/ -- The Oxford-Harrington Rare Disease Centre ('OHC') today announced the recipients of the 2025 Rare Disease Scholar Award, which advances promising discoveries from academic labs into clinical practice. Each Scholar will receive an advisory team providing advanced drug and business development support, $100,000 (or £100,000 in the UK), and the opportunity to compete for additional advisory support, acceleration funds, and to qualify for investment funds up to $1,000,000 according to project requirements.
Rare diseases affect an estimated 500 million people worldwide-half of whom are children. Yet, fewer than 5% of the 7,000 known rare diseases have approved treatments. The OHC, a unique partnership between the University of Oxford, UK and Harrington Discovery Institute at University Hospitals, Cleveland, Ohio, is on a mission to accelerate progress by combining philanthropy, world-class science and entrepreneurial drug development.
"Behind every rare disease is a person, a family and a story of resilience," said Lord Cameron, Chair of the OHC Advisory Council. "At OHC, we believe that by bringing together the best of academia and cutting-edge medical research, with life sciences, pharma, philanthropy and venture capital from around the world, we can achieve transformational results, closing the gap between research and drug development and turning hope into the reality of new treatments for patients and their families. These awards are an important step toward achieving our mission of 40 new treatments for rare diseases within a decade."
Through the Rare Disease Scholar Award, the 2025 cohort is working on treatments for a wide range of rare conditions impacting the brain, immune system, metabolism, and cancer. Cutting-edge approaches include gene and RNA therapies, enzyme replacement and targeted medicines-tools that could help correct or manage the underlying causes of these diseases.
"We've seen remarkable progress from our inaugural class of OHC Scholars, with several projects already moving closer to clinical application," said Matthew Wood, MD, PhD, Director and Chief Scientific Officer of the OHC. "With the 2025 Scholar Awards, we are adding ten new OHC programs and tackling some of the most challenging rare diseases through pioneering gene, RNA, small molecule and cell-based therapies."
OHC seeks to advance 40 drugs into clinical trials by 2034 and offers a new model for rare disease innovation that progresses Scholar discoveries through a structured, industry-informed approach.
"The OHC builds on Oxford University's outstanding scientific capabilities with Harrington's track record in drug development," said Matthew P. Anderson, MD, PhD, Co-Director of the OHC. "Our vision for these programs is not just for each to address a single rare disease but ultimately to identify new platforms for treating multiple rare diseases-and even common diseases."
The 2025 OHC Rare Disease Scholar Award recipients, their organizations and fields of research include:
Rachel Bailey, PhD - University of Texas Southwestern Medical Center, Dallas, TX, USA
Gene Therapy to Cure an Ultra-Rare, Devastating Neurodevelopmental Metabolic Disorder of the SLC13A5 Citrate Transporter
Esther Becker, PhD - University of Oxford, Oxford, UK
New Small Molecules to Inhibit Abnormal Nerve Function to Preserve and Restore Muscle Function in Spinocerebellar Ataxia
Joseph Buxbaum, PhD - Icahn School of Medicine at Mount Sinai, New York, NY, USA
Treatment of the Autism-Spectrum Neurodevelopmental ADNP Syndrome with Small Molecule Drugs
Matthew Gentry, PhD - University of Florida, Gainesville, FL, USA
Advanced Brain Penetrant Enzyme Therapy to Treat Neurological Glycogen Storage Diseases
Albert La Spada, MD, PhD - University of California, Irvine, Irvine, CA, USA
Silencer RNA Therapy to Treat Rare, Juvenile-onset ALS Neuromuscular Disease, ALS4
Michael Lin, MD, PhD - Stanford University, Stanford, CA, USA
A Genetically Programmed Therapeutic for Sensing and Killing Rare Brain Cancers
Pengfei Liu, PhD - Baylor College of Medicine, Houston, TX, USA
Precision Nucleic Acid Therapeutics for the Ultra-Rare CAMK2B Neurodevelopmental Disorder
David Segal, PhD - University of California, Davis, Davis, CA, USA
Treatment of the Autism-Spectrum Neurodevelopmental ADNP Syndrome with Nucleic Acid Drugs
Anthony Shum, MD - University of California, San Francisco, San Francisco, CA, USA
Gene Therapy for the Progressive, Debilitating, Multisystem COPA Syndrome
Mingshan Xue, PhD - Baylor College of Medicine, Houston, TX, USA
Gene Therapy for the Catastrophic CASK-Related Neurodevelopmental Disorder
For those interested in submitting a proposal for the Oxford-Harrington Rare Disease Scholar Award, the next funding call will open on the 14th of January 2026. For more information, visit: OxfordHarrington.org/funding
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