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

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Oct 03, 2023, 04:00 ET

The Therapeutics Accelerator (the 'Accelerator') will identify, fund and advance innovative projects from academic partners in the U.K. and U.S. with the goal to deliver 40 new potentially life-changing therapies for rare diseases into clinical trials over the next ten years, targeting multiple approvals in key markets.
The Accelerator will operate a unique non-profit/for-profit model with the intention to commit up to £200 million ($250 million).
The Accelerator will receive support from a new partnership between University Hospitals in Cleveland, Ohio, University of Oxford and investment company Oxford Science Enterprises.
Former U.K. Prime Minister David Cameron to lead the international efforts of the Accelerator as Chair of the Oxford-Harrington Rare Disease Centre Advisory Board.

OXFORD, United Kingdom and CLEVELAND, Oct. 3, 2023 /PRNewswire/ -- The University of Oxford and Harrington Discovery Institute at University Hospitals in Cleveland, Ohio announced the launch of the Oxford-Harrington Rare Disease Centre Therapeutics Accelerator with an official signing event at the University of Oxford. The event was hosted by the Oxford-Harrington Rare Disease Centre (OHC), a partnership established in 2019 between the University of Oxford and Harrington Discovery Institute. Attendance at the signing included leaders from OHC, the University of Oxford, the Harrington family, University Hospitals, and Oxford Science Enterprises.

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.

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 academics and researchers to find out more about the Therapeutics Accelerator at the Oxford-Harrington Rare Disease Centre and how to apply, please go to OxfordHarrington.org.

Notes to Editors:

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 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.

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, OH, USA. OHC combines world-leading strengths in research and therapeutics development from across these organizations, to set the science and innovation agenda, driving 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 | Twitter

About the University of Oxford Medical Sciences Division
Oxford University'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. The University is rated the best in the world for medicine and life sciences, and it is home to the UK's top-ranked medical school. It has one of the largest clinical trial portfolios in the UK 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.

About Oxford Science Enterprises
Oxford Science Enterprises (OSE) is an independent, billion-pound investment company, created in 2015 to found, fund and build transformational businesses via its unique partnership with the University of Oxford, the world's #1 research university. This partnership enables OSE to work with the brightest academic minds tackling the world's toughest challenges and guarantees unrivalled access to their scientific research. In collaboration with its global network of entrepreneurs and advisers, OSE goes well beyond funding to shape and nurture these complex ideas into successful businesses. Actively focused on a core portfolio of around 40 companies spanning three high-growth, high-impact sectors – Life Sciences, Health Tech, and Deep Tech – the company adopts a flexible, long-term investment approach, recognising the path from ground-breaking research to global markets takes time and resilience. To date, OSE has invested £0.6 billion in over 80 ambitious companies built on Oxford science. For more information, visit: oxfordscienceenterprises.com | Twitter | LinkedIn

About Harrington Discovery Institute
The Harrington Discovery Institute at University Hospitals in Cleveland, OH – 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 |Twitter

About University Hospitals, Cleveland, Ohio
Founded in 1866, University Hospitals serves the needs of patients through an integrated network of 21 hospitals (including five joint ventures), more than 50 health centers and outpatient facilities, and over 200 physician offices in 16 counties throughout northern Ohio. The system's flagship quaternary care, academic medical center, University Hospitals Cleveland Medical Center, is affiliated with Case Western Reserve University School of Medicine, Northeast Ohio Medical University, Oxford University, the Technion Israel Institute of Technology and National Taiwan University College of Medicine. The main campus also includes the UH Rainbow Babies & Children's Hospital, ranked among the top children's hospitals in the nation; UH MacDonald Women's Hospital, Ohio's only hospital for women; and UH Seidman Cancer Center, part of the NCI-designated Case Comprehensive Cancer Center. UH is home to some of the most prestigious clinical and research programs in the nation, with more than 3,000 active clinical trials and research studies underway. UH Cleveland Medical Center is perennially among the highest performers in national ranking surveys, including "America's Best Hospitals" from U.S. News & World Report. UH is also home to 19 Clinical Care Delivery and Research Institutes. UH is one of the largest employers in Northeast Ohio with more than 30,000 employees. Follow UH on LinkedIn, Facebook and Twitter. For more information, visit UHhospitals.org.

SOURCE Harrington Discovery Institute