Rare disease centre's new initiative backed by former UK PM, David Cameron

The University of Oxford and Harrington Discovery Institute at University Hospitals in Cleveland, Ohio, announced the launch of the accelerator on Tuesday (October 3) at the UK university with an official signing event.

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.

Cameron, who was present at the launch event, will lead the international efforts of the cccelerator 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.” 

Breakthrough academic discoveries 

The university will offer its scientific leadership and resources which includes more than 250 principal investigator scientists working on at least 350 rare diseases while Harrington Discovery Institute has experience of advancing breakthrough medicines into clinical development. It has funded 177 medicines, launched 36 companies and brought 19 medicines into the clinic with 13 licenses to pharma companies since it was founded in 2012.

The accelerator is said to be 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.

It states 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 US, the UK and Europe.

Cameron went on to say: “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.”

Rare genetic diseases - urgent need for greatest impact

The OHC says it 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 UK and US.

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.”

The accelerator will operate a 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.

It 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.

Creation of Alvogene 

The first investment was made last month (September 2023) alongside the creation of Alveogene – a new UK company started to develop innovative inhaled gene therapies to transform outcomes for those 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.

Jonathan Stamler, 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.”