The Oxford-Harrington Rare Disease Centre (OHC) has unveiled the recipients of the 2025 Rare Disease Scholar Award, a program dedicated to propelling innovative treatments from academic research into practical applications. This year’s cohort consists of ten exemplary researchers who are engaging in groundbreaking work aimed at tackling rare and ultra-rare diseases. Each scholar is set to benefit from extensive support, including an advisory team for advanced drug and business development, a grant of $100,000, and a chance to vie for additional funding up to $1,000,000 based on their project's criteria.
Rare diseases impact an estimated 500 million individuals globally, with children constituting approximately half of this population. Alarmingly, fewer than 5% of the 7,000 recognized rare diseases currently have approved treatment options available. To address this significant gap, the OHC, a unique collaboration between the University of Oxford and the Harrington Discovery Institute at University Hospitals in Cleveland, Ohio, seeks to foster progress by merging philanthropic resources, elite scientific research, and entrepreneurial drug development strategies.
As Lord Cameron, Chair of the OHC Advisory Council, articulates, "Behind every rare disease is a person, a family, and a story of resilience. At OHC, we are determined to unite outstanding academic endeavors with pioneering medical research, alongside partnerships from the life sciences, pharmaceutical industries, philanthropy, and venture capital. This comprehensive approach aims to translate hope into a tangible reality for those affected by rare diseases, ultimately targeting the introduction of 40 new treatments within the next decade."
Among the recipients of the 2025 Rare Disease Scholar Awards are researchers working on diverse therapies employing the latest innovations in gene therapy, RNA technologies, enzyme replacement, and precision medicines. These advanced approaches are focused on correcting or managing the root causes of various conditions that primarily affect the brain, immune system, metabolism, and even cancers.
Dr. Matthew Wood, the Director and Chief Scientific Officer at OHC, emphasized the significance of this initiative by reflecting on the progress of the inaugural cohort of Scholars, many of whom have already moved closer to clinical application. With the introduction of the 2025 cohort, OHC has ambitious plans to launch ten new programs targeting some of the most challenging rare diseases through state-of-the-art gene, RNA, small molecule, and cellular therapies.
Dr. Matthew P. Anderson, Co-Director of the OHC, further elaborated on the center's vision, stating, "Our goal extends beyond simply addressing individual rare diseases; we aim to identify novel platforms that can potentially treat multiple rare diseases and even some common ailments. This represents a transformative blueprint for rare disease innovation that can help empower new therapies through a structured, industry-informed framework."
The selected 2025 OHC Rare Disease Scholars and their research domains include:
- - Rachel Bailey, PhD – University of Texas Southwestern Medical Center, Dallas, TX
Focus: Gene Therapy for an ultra-rare, devastating neurodevelopmental metabolic disorder affecting the SLC13A5 citrate transporter.
- - Esther Becker, PhD – University of Oxford, Oxford, UK
Focus: Development of small molecules to inhibit abnormal nerve function and preserve muscle function in Spinocerebellar Ataxia.
- - Joseph Buxbaum, PhD – Icahn School of Medicine at Mount Sinai, New York, NY
Focus: Treatment for Autism-Spectrum Neurodevelopmental ADNP Syndrome using small molecule drugs.
- - Matthew Gentry, PhD – University of Florida, Gainesville, FL
Focus: Advanced brain-penetrant enzyme therapy for neurological glycogen storage diseases.
- - Albert La Spada, MD, PhD – University of California, Irvine, CA
Focus: Silencer RNA Therapy for juvenile-onset ALS (ALS4) neuromuscular disease.
- - Michael Lin, MD, PhD – Stanford University, Stanford, CA
Focus: Genetically programmed therapeutic approaches for sensing and targeting rare brain cancers.
- - Pengfei Liu, PhD – Baylor College of Medicine, Houston, TX
Focus: Precision nucleic acid therapeutics for ultra-rare CAMK2B neurodevelopmental disorders.
- - David Segal, PhD – University of California, Davis, CA
Focus: Nucleic acid drug treatments for Autism-Spectrum Neurodevelopmental ADNP Syndrome.
- - Anthony Shum, MD – University of California, San Francisco, CA
Focus: Gene therapy for the progressive, debilitating multisystem COPA syndrome.
- - Mingshan Xue, PhD – Baylor College of Medicine, Houston, TX
Focus: Gene therapy for the catastrophic CASK-related neurodevelopmental disorder.
Those interested in submitting proposals for the next Oxford-Harrington Rare Disease Scholar Award can anticipate the funding application window to open on January 14, 2026. For additional details, visit
OxfordHarrington.org/funding.