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