Therapeutics Accelerator

Launching a New Era in Rare Disease Treatment

The Therapeutics Accelerator – Advancing Treatments for Rare Diseases

The Therapeutics Accelerator is a pioneering model to advance the development of innovative treatments for rare diseases. Through this model, the Oxford-Harrington Rare Disease Centre (OHC) will identify, fund, and advance groundbreaking projects from academic partners in the UK and US to treatments for those living with rare diseases. Our goal is to deliver 40 new therapies for rare diseases into clinical trials over the next decade, with a focus on securing multiple approvals.

A Unique Non-Profit/For-Profit Model

The Therapeutics Accelerator operates on a unique non-profit/for-profit model, combining funding sources for maximum impact. More than 400 million people worldwide are living with a rare disease, and approximately 50 percent are children. There are more than 7,000 known rare diseases, with new diseases being discovered every day, the majority of which are genetic. Only five percent of rare diseases have a treatment approved by the US Food and Drug Administration (FDA); similar estimates have been made for treatments approved by the European Medicines 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.

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Powered by Collaboration

Our initiative is powered by a new partnership between Harrington Discovery Institute at University Hospitals in Cleveland, the University of Oxford, and Oxford Science Enterprises (OSE). This collaboration is set to redefine the landscape of rare disease therapeutics.

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Therapeutics Accelerator Signing (L-R): Dr Jonathan Stamler, Vice-Chancellor Professor Irene Tracey, Dr Cliff Megerian, James Wilkinson.

Leadership That Inspires Change

We are honoured to have had the former UK Prime Minister, David Cameron, as founding Chair of the Oxford-Harrington Rare Disease Centre Advisory Council. He brought his own experience of having had a child with a rare disease as well as leadership and expertise in driving the UK's leading role in genomics with the establishment of Genomics England during his tenure as Prime Minister.

Transatlantic Partnership

The OHC Therapeutics Accelerator bridges strengths between the UK and the US, delivering world-leading research and drug development expertise to find new treatments and cures for rare diseases. It combines the scientific leadership and resources of the University of Oxford, with over 250 principal investigator scientists working on more than 350 rare diseases, and the proven track record of Harrington Discovery Institute, which has advanced 177 medicines, launched 36 companies, progressed 19 medicines into the clinic, and facilitated 13 licenses to pharmaceutical companies since its founding in 2012.

Diagram highlighting the Institutes key achievements

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Prioritising Urgent Needs

The OHC places its focus on rare genetic diseases. We prioritise areas where there is an urgent need and opportunity for the greatest impact, particularly in the domains of rare neurological diseases, cancers, and developmental and metabolic diseases. Our projects are sourced from academic labs across the UK and US.

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Fostering Innovation

At the heart of the Accelerator is a commitment to innovation. We operate a unique non-profit/for-profit model, aiming to commit up to £200 million into new projects. Our funding is complemented by research, drug development, commercial strategy, and business development expertise. Our industry leaders bring a wealth of experience in bringing new drugs to market.

Pioneering Investment in Rare Lung Disease

AlveoGene was launched in September 2023 to develop unique inhaled gene therapies for rare respiratory disorders. This new gene therapy company was created through the OHC model to exemplify the power of partnership (i.e., funded by Oxford Science Enterprises, Harrington Discovery Institute, and Old College Capital in partnership with six leading scientists from the world-renowned UK Respiratory Gene Therapy Consortium (GTC)). AlveoGene employs a proprietary lentiviral gene therapy platform for inhaled delivery of nucleic acid therapies.

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You Can Transform Lives

We invite scientists, researchers, innovators, and philanthropists to join us on this transformative journey. Together, we can make a profound impact on the lives of millions worldwide, bringing hope and life-changing therapies to those who need them most.

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