Oxford-Harrington Rare Disease Centre Launches Pioneering Therapeutics Accelerator to Advance Innovative Treatments for Rare Diseases

The OHC unites world-leading research across the U.K. and U.S. with expertise in therapeutics development by combining University of Oxford’s scientific leadership and resources (250+ principal investigator scientists working on over 350 rare diseases) with Harrington Discovery Institute’s track record of advancing breakthrough medicines into clinical development (177 medicines funded, 36 companies launched, 19 medicines into the clinic, and 13 licenses to pharma companies since its founding in 2012).

The Accelerator is 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.

David Cameron, the former U.K. Prime Minister, who was present at the launch event, will lead the international efforts of the Accelerator under his role as Chair of the Oxford-Harrington Rare Disease Centre Advisory Board.

David Cameron said: 'I know from my own experience of having a son born with an incredibly rare and incurable disease, how little we really know about rare diseases. Although individually they may only affect small numbers of patients like my son, Ivan, collectively they represent a major global health crisis that affects millions of patients and their families in life-changing ways.

Therapeutics Accelerator Launch (L-R) Sir John Bell, Jill Harrington, Dr Jonathan Stamler, Professor Matthew Wood, Rt Hon David Cameron, Nancy Harrington, Professor Georg Hollander, Ronald Harrington and Dr Cliff Megerian.

Image credit: Cyrus Mower

'As Prime Minister and since, I have been fortunate to follow the enormous scientific breakthroughs that have taken place over the last decade, such as the use of genomics, which opened my eyes to how much we could know about rare diseases. That’s why I set up Genomics England and personally championed the 100,000 Genomes Project - now expanded to sequence 5 million adults across the UK - and that is why today I am so pleased to back this unique Rare Disease Therapeutics Accelerator, designed to make a transformational, global impact on the treatment of these devastating and often neglected diseases.

'I have long said that beating these diseases is within our grasp. This Accelerator will deliver new, real and tangible therapies for patients and, by doing so, offer huge benefits for patients, their families and wider society. I am delighted to play my part in making it a reality.'

The OHC prioritizes rare genetic diseases affecting children or adults where there is urgent need and opportunity for greatest impact and that fall into the areas of rare neurological diseases, cancers and developmental diseases. Projects will be sourced from academic labs across the U.K. and U.S.

The Accelerator will operate a unique non-profit/for-profit model with the intention to commit up to £200 million into new projects. Project funding will be paired with research, drug development, commercial strategy and business development expertise from pharma-experienced industry leaders with a track record of bringing new drugs to market.

The Accelerator will receive support from a new co-funding and co-investment agreement between University Hospitals in Cleveland, Ohio, University of Oxford and investment company Oxford Science Enterprises, and aims to ensure that capital is deployed efficiently to advance a sustainable pipeline of new therapies either through academia or via the creation of venture-backed spinout companies.

The first investment by the Accelerator was made in September 2023 in conjunction with the creation of AlveoGene, a new U.K. company launched to develop innovative inhaled gene therapies and transform outcomes in patients with rare respiratory diseases.

The partners are also collaborating to create a dedicated Rare Disease Impact Fund to enable investments in mission-aligned projects and accelerate the development of rare disease therapeutics.

Sir John Bell, Regius Professor of Medicine at the University of Oxford and member of the OHC Steering Committee, said: 'It requires true innovation, collaboration, vision and commitment to make meaningful progress in changing the global rare disease landscape for patients, their families and society. Since the creation of the OHC in 2019 we have made good progress along this path – and the hope is that by building on this foundation with this exciting new initiative, we can really create momentum to drive the translation of cutting-edge science into life-changing therapies targeting some of the most debilitating diseases.'

Jonathan Stamler, MD, President and Founder of Harrington Discovery Institute, said: 'Harrington has been supporting the advancement of treatments for rare and other diseases for more than a decade, committing significant funds and resources to this global challenge with considerable success. Our commitment is to major unmet needs in society to better the world. This new Accelerator and extended collaboration with Oxford and Oxford Science Enterprises represents a major step forward towards finding the best medicines that improve outcomes for the millions of individuals and families affected by rare diseases.'

Matthew Wood, Professor of Neuroscience and Director of the OHC, said: 'OHC was set up with a bold vision and ambitious goals to make an impact on the treatment of rare diseases. This new Accelerator initiative takes this a step further with its unique collaboration and for-profit and not-for-profit model. It is only by combining resources and adopting innovative approaches that we can truly accelerate the development of new drugs for patients in need. Already we have seen many exciting opportunities and look forward to engaging with scientists at the forefront of rare disease research.'

Georg Holländer, Head of Department of Paediatrics at the University of Oxford, said: 'This is a great moment for Paediatrics at Oxford as it expands its commitment to childhood diseases across the U.K. and the U.S. There is so much great research being done on these diseases and only through exciting multi-organizational collaborations like the OHC and the Accelerator can we make the progress we need to bring hope to patients and their families around the world.'

Ed Bussey, CEO at Oxford Science Enterprises, said: 'As a company focused on translating scientific breakthroughs into commercially successful businesses, we are thrilled to partner with OHC on the introduction of a Rare Disease Therapeutics Accelerator. Through this initiative we hope to uncover the most promising opportunities, creating and building innovative new companies with the goal of improving patient outcomes for people impacted by rare diseases globally.'

Ronald G. Harrington, Entrepreneur and Philanthropist, said: 'Our family is thrilled to see the extraordinary progress that Harrington Discovery Institute has made since we launched in 2012. It is humbling and gratifying that our efforts are recognized and supported by so many participants in the industry from disease foundations to prestigious universities and innovative biopharmaceutical companies. The launch of this new rare disease initiative and extension of our relationship with Oxford is a further example of our ambition to make a meaningful positive impact on global health challenges affecting millions of people around the world.'

Cliff A. Megerian, MD, FACS, CEO, University Hospitals, said: 'Every day, through the work we do at University Hospitals, we are inspired by the courage and determination of patients with rare diseases and their families. This gives us a real sense of urgency and motivation to make a difference in the lives of these families and we are fully committed to supporting this new Accelerator and the efforts of our transatlantic team and partners in their mission.'

Daniel I. Simon, MD, President, Academic & External Affairs and Chief Scientific Officer, University Hospitals, said: 'Our health system is focused on translating discoveries to address unmet clinical needs. Leveraging the unique Harrington model in the Oxford-Harrington Rare Disease Centre will catalyze the development of new medicines for hundreds of thousands of patients across the globe.'

For further information contact Julie Stevens or visit the Oxford-Harrington Rare Disease Centre  website.