Oxford-Harrington Rare Disease Centre to research rare diseases therapies

The University of Oxford and University Hospitals in Cleveland, Ohio, have announced a multi-year affiliation to establish a global centre for rare diseases. The Oxford-Harrington Rare Disease Centre will bring together the capabilities, resources and expertise of both institutions to deliver new treatments for rare diseases, for which therapeutic options are lacking.

More than 350 million people worldwide are living with a rare disease, and approximately 50 percent are children. There are about 7,000 known rare diseases, with new diseases being discovered every day. A rare disease affects one in 10 Americans, or 10 percent of the US population. Similarly, Europe has approximately 30 million people who suffer from a rare disease. The majority of all rare diseases are genetic in origin, which means they are present throughout a person’s life. Only five percent of rare diseases have a treatment approved by the US Food and Drug Administration (FDA) and similar estimates have been made for treatments approved by the European Medicine Agency (EMA). Therefore, someone with a rare disease today faces a lifelong, often life-threatening, condition with little hope for a cure, or even an effective treatment option.

In this partnership, the University of Oxford and Harrington Discovery Institute commit to addressing unmet need in rare disease. Through their combined resources, the new Centre will set the science and innovation agenda to support cutting-edge breakthroughs across the UK and US with the greatest chance for clinical impact.

The University of Oxford has more than 250 Principal Investigator scientists working on over 350 rare diseases. The Oxford Rare Disease Initiative, established in 2012, is a collaboration between the University of Oxford and the Oxford University Hospitals Trust, which provides the opportunity to combine pre-clinical and clinical expertise in areas such as neurology, inflammation and immunology, hematology, cardiovascular and metabolic diseases and has created an extensive rare disease network.

Since its founding in 2012, Harrington Discovery Institute – part of The Harrington Project for Discovery & Development – has supported more than 100 drugs-in-the-making across disease areas and academic institutions, with a concentration in diseases where unmet need is greatest. The Harrington Project was established to advance scientific discoveries over the ‘Valley of Death’ – the stage in the drug development process when a new discovery is seen as promising, but is insufficiently validated to attract the funding necessary for clinical trials. Harrington Discovery Institute advances these promising discoveries towards the clinic by aligning, through mission and structure, scientific and drug discovery expertise into a new model for drug development.

'Our partnership with Harrington Discovery Institute recognises the combined experience needed in world-class science and drug development if we are to change the rare disease landscape in a meaningful way – and Oxford is committed to doing so through this unique, open model supporting the most impactful innovation throughout the UK,' said Sir John Bell, Regius Professor of Medicine at Oxford.

'Advancing breakthroughs in rare diseases will require bold, new approaches that can overcome scientific challenges and create new medicines. This affiliation represents a commitment to patients first and a tremendous opportunity to improve the health and outcomes of those living with a rare disease,' said Jonathan S. Stamler, MD, President, Harrington Discovery Institute and Reitman Family Distinguished Chair of Cardiovascular Innovation at University Hospitals and Case Western Reserve University School of Medicine.

The Oxford-Harrington Rare Disease Centre has been several years in planning through efforts of Sir John Bell, Regius Professor of Medicine at Oxford, and with the help of David U’Prichard, ex-Global Head of Pharma R&D and Advisor to The Harrington Project. The Centre will be headquartered both in Oxford, under the leadership of Professor Georg Holländer and Professor Matthew Wood, and in Cleveland within the Harrington Discovery Institute, and managed by its leadership. The Centre will be leveraged by researchers worldwide, and raise awareness for rare diseases.

'Our family is thrilled to see the incredible evolution of the Harrington Project in only 7 years. Our US program has generated interest from disease foundations to biotech and pharma, and we are so pleased to see it extend to the UK. We are honoured and privileged to be collaborating with perhaps the most illustrious university in the world, and we welcome them as true partners in our efforts to cure disease,” said Ronald G. Harrington, whose family co-founded the Harrington Project with Dr. Stamler at University Hospitals.

“Our Centre promises to advance the best scientific breakthroughs not only at Oxford but also across the UK. This is a unique feature of our commitment to science and patients and one I am most proud of,” said Georg Holländer, Head of the Department of Paediatrics. Matthew Wood, Professor of Neuroscience and Director of the Centre remarked, “The enormous challenge of bringing new medicines into the rare disease space requires not only scientific excellence but also mechanisms to share knowledge and ambitious approaches to translate this successfully into new drugs for patients. We are thrilled that the new Centre will partner with Harrington Discovery Institute and benefit directly from their extraordinary track record in new drug development and commitment to patients.'

'This is a wonderful example of the synergy in science and innovation that is needed to move discovery forward, and it is gratifying to see our institutions in Cleveland and Oxford combine their strengths to this end,' said University Hospitals CEO Thomas F. Zenty III.

The Centre is supported in part by a grant from the Cleveland Foundation. 'This is a phenomenal opportunity for Cleveland to further distinguish itself as a leader in biomedical innovation,' said Cleveland Foundation President and CEO Ronn Richard. 'We are excited by the capabilities of Harrington Discovery Institute, which will support medical breakthroughs across the US and UK through its affiliation with one of the world’s premier medical institutions.'