Newswise — OXFORD, U.K. and CLEVELAND, Ohio, U.S., Feb. 28, 2024 – The Oxford-Harrington Rare Disease Centre (‘OHC’), a partnership between the University of Oxford, U.K. and Harrington Discovery Institute at University Hospitals, Cleveland, Ohio, aimed at driving cutting-edge rare disease breakthroughs, is delighted to announce it will be hosting a webinar entitled Pioneering a Path to Rare Disease Therapeutics. The event will be held today in acknowledgement of Rare Disease Day on February 29 and will highlight important initiatives underway at OHC designed to accelerate the discovery and advancement of brilliant science from around the world into technologies to help those living with rare diseases.
During the webinar, participants will hear from leading experts in the field of rare diseases and therapeutics development discussing the role of OHC and the recent launch of its Therapeutics Accelerator, a first-in-kind, transatlantic initiative to identify, fund and advance breakthrough academic discoveries to deliver new treatments for the 400 million people worldwide who suffer from rare diseases. Its goal is to deliver 40 new potentially life-changing therapies for rare diseases into clinical trials over the next ten years and target multiple approvals from regulators in key markets including the U.S., the U.K. and Europe.
The event will also cover a recent example of new company creation via the Accelerator with support from the investment firm Oxford Science Enterprises; that of AlveoGene, a new U.K. company launched to develop innovative inhaled gene therapies and transform outcomes in patients with rare respiratory diseases.
Baroness Nicola Blackwood, Chair of Genomics England and of Oxford University Innovation, reflecting on her journey as an Ehlers-Danlos patient, will emphasise the dire need for improved diagnosis and care for rare diseases: "My own experience underscores the urgent necessity to improve diagnosis and care for rare disease patients. Because in the U.K. the average diagnostic period lasts for between four to five years. And at the current rate of discovery, it will take 2000 years for there to be therapies for all rare conditions. This is far too long, and impacts far too many. That is why initiatives like Genomics England, the Rare Therapies Launch Pad and the OHC Therapeutics Accelerator are mission critical, as they will pave the way for a transformative future in rare disease treatment, bridging the gap between diagnosis and clinical care.”
Finally, details of the Oxford-Harrington Rare Disease Scholar Award will be shared. The Award provides funding and expert therapeutics development support to researchers in the U.K., U.S. or Canada to accelerate academic and preclinical projects into clinical practice towards becoming new treatments for patients.
“Rare Disease Day has grown into a crucial annual event to engage the rare disease community at all levels, and elevate the stories of people living with a rare disease diagnosis and their families. In support of this mission, we are putting a spotlight on some of the important initiatives underway at OHC to support groundbreaking research by passionate and committed researchers around the world towards the creation of urgently needed treatments for patients,” said Matthew Wood, Professor of Neuroscience in Oxford’s Department of Paediatrics, Director and Chief Scientific Officer of the OHC. “With over 400 million individuals globally affected by rare diseases, rapid and decisive action is imperative. OHC was set up with a bold vision and ambitious goals to make a positive impact on the treatment of these debilitating and often fatal diseases. Through collaborative efforts and increased awareness, we can drive meaningful change and improve the lives of those affected.”
Panellists for the webinar will include:
- Professor Eric Alton, Coordinator of The U.K. Respiratory Gene Therapy Consortium, Imperial College London
- Baroness Nicola Blackwood, Chair of Genomics England, Chair of Oxford University Innovation
- Claire Brown, PhD, MBA, Partner Life Sciences, Oxford Science Enterprises
- David Hipkiss, Executive Chair at AlveoGene
- Jonathan S. Stamler, MD, President & Co-Founder, Harrington Discovery Institute
- Diana Wetmore, PhD, Vice President, Therapeutics Development Center, Harrington Discovery Institute
- Professor Matthew Wood, Professor of Neuroscience, Oxford’s Department of Paediatrics, Director and Chief Scientific Officer, Oxford-Harrington Rare Disease Centre
For more information and to register for the webinar, please click here. Questions can be submitted to panellists by contacting [email protected].
Join us today, as we unite in solidarity with the rare disease community and pioneer a path towards innovative therapeutics.
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About Rare Diseases
More than 400 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 U.S. 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 U.S. 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.
About Oxford-Harrington Rare Disease Centre
The Oxford-Harrington Rare Disease Centre (OHC) is a partnership between the University of Oxford, U.K. and Harrington Discovery Institute at University Hospitals, Cleveland, Ohio, U.S. OHC combines world-leading strengths in research and therapeutics development from across these organizations to set the science and innovation agenda and drive cutting-edge rare disease breakthroughs to address the unmet need in rare disease across the globe to deliver major clinical impact for patients. For more information, visit: OxfordHarrington.org | X
About the University of Oxford
The University of Oxford is rated the best in the world for medicine and life sciences, and it is home to the U.K.’s top-ranked medical school. It has one of the largest clinical trial portfolios in the U.K. and great expertise in taking discoveries from the lab into the clinic. Partnerships with the local NHS Trusts enable patients to benefit from close links between medical research and healthcare delivery. The University of Oxford’s Medical Sciences Division is one of the largest biomedical research centres in Europe, with over 2,500 people involved in research and more than 2,800 students. For more information, visit: www.ox.ac.uk
About Harrington Discovery Institute
The Harrington Discovery Institute at University Hospitals in Cleveland, Ohio – part of The Harrington Project for Discovery & Development – is a translational accelerator that advances promising discoveries from academic labs into the clinic for the benefit of patients and society. The institute was created in 2012 with a $50 million founding gift from the Harrington family and is a prime example of the commitment they share with University Hospitals to a Vision for a ‘Better World’. For more information, visit: HarringtonDiscovery.org| LinkedIn | X
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