Transforming the Future of Rare Disease Medicines
The Oxford-Harrington Rare Disease Centre (OHC)—a partnership between the University of Oxford and Harrington Discovery Institute at University Hospitals—is a transatlantic initiative committed to delivering breakthrough treatments and cures for rare diseases.
Our goal: advance 40 new medicines into clinical trials by 2034, with a focus on rare neurological disorders, developmental diseases, and cancers.
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Why the Oxford-Harrington Rare Disease Centre is Different
Philanthropy Drives Breakthroughs
Donor support makes it possible to move bold scientific discoveries toward real treatments.
A Proven Pathway for Academic Scientists
Our scholars gain access to industry-level drug development capabilities, building a full translational pathway around each project. Each scholar’s discovery is supported with integrated expertise across drug development, mirroring the capabilities of a biotech company.
From Philanthropy to Venture
We uniquely bridge charitable giving to sustainable venture investment, ensuring discoveries can advance all the way to patients.
Focused on Medicines, Not Just Research
We prioritise advancing treatments and cures—not stopping at basic research.
Filling Critical Gaps in Drug Development
We advance treatments for diseases that lack sufficient investment, ensuring patients in underserved areas of medicine are not left behind.
Scaling for Greater Impact
Breakthroughs in rare diseases pave the way for treatments in more common conditions, such as Alzheimer’s.
A Global Network of Excellence
We source the most promising science from leading researchers across the UK, US, and Canada.
The Need for Rare Disease Treatments
- 500 million people worldwide are living with a rare disease.
- 1 in 2 patients diagnosed is a child.
- A third of children with a rare disease will not live to their 5th birthday.
- 95% of rare diseases—more than 10,000 in total—have no approved treatment.
Voices of Impact: Stories That Inspire and Drive Change
Rare diseases affect millions of families worldwide. Hear directly from leaders, advocates, and innovative scientists who are championing new rare disease treatments and advancing hope through the Oxford-Harrington Rare Disease Centre.
Gene Therapy Breakthrough for Rare Eye Cancer
Jacquelyn Bower, PhD, University of North Carolina at Chapel Hill, 2024 Oxford-Harrington Rare Disease Scholar, is developing a novel gene therapy to stop tumor growth and prevent metastases in uveal melanoma, advancing hope for patients with this rare eye cancer.
Advancing RNA Therapies for Rare Nerve Disorders
Haiyan Zhou, PhD, University College London, 2024 Oxford-Harrington Rare Disease Scholar, is developing a precision RNA therapy to tackle a rare and debilitating nerve disease, offering hope for patients and families.
Targeting Spinal and Bulbar Muscular Atrophy
Carlo Rinaldi, MA, MD, PhD, University of Oxford, 2024 Oxford-Harrington Rare Disease Scholar, is developing a splice-switching oligonucleotide therapy to target mutant androgen receptors in spinal and bulbar muscular atrophy, aiming to slow disease progression and improve patients’ quality of life.