Skip to main content
Home » Rare diseases » Pioneering pathways to rare disease therapeutics

Matthew Wood

Professor of Neuroscience, Oxford Department of Paediatrics and Director and Chief Scientific Officer, Oxford-Harrington Rare Disease Centre

Jonathan Stamler, MD

President and Co-Founder,
Harrington Discovery Institute

Julie M. Stevens

Director of Operations, Oxford-Harrington Rare Disease Centre

A rare disease drug development partnership aims to deliver 40 new therapies to clinic by 2033. The last year has seen significant progress in realising this vision for the 400 million people affected by rare diseases globally.

Matthew Wood, Professor of Neuroscience and Director and Chief Scientific Officer of the Oxford-Harrington Rare Disease Centre (OHC), says: “Our Centre was established with a bold and ambitious global vision to transform the treatment of rare diseases. Our new Accelerator takes this a step further with its unique, collaborative, integrated for-profit and not-for-profit model. By combining resources and adopting innovative approaches, we can truly accelerate the development of new drugs for patients in need.”

Helping families affected by rare disease

Jonathan Stamler, MD, President and Co-Founder of Harrington Discovery Institute, says: “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. This new Accelerator and extended collaboration with Oxford, and Oxford Science Enterprises, is a major step forward towards finding the best medicines for the millions of individuals and families affected by rare diseases.”

It pairs academic project funding with drug
and business development expertise from
seasoned pharmaceutical industry leaders.

Accelerating new drugs for rare diseases

Launched in October 2023, the Oxford-Harrington Therapeutics Accelerator will co-invest up to £200 million from University Hospitals in Cleveland, Ohio and Oxford Science Enterprises. It pairs academic project funding with drug and business development expertise from seasoned pharmaceutical industry leaders.

David Cameron, former UK Prime Minister and founding Chair of the OHC Advisory Council remarks: “I know, from 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.”

The first investment by the Accelerator was made in a new company, AlveoGene, which is developing inhaled gene therapies for patients with rare respiratory diseases.

Delivering pathways to individualised medicines

Furthermore, the OHC is pivotal in the UK’s new Rare Therapies Launch Pad, aiming to provide personalised anti-sense oligonucleotides for children with severe neurological conditions in collaboration with Genomics England, the UK regulator MHRA, patient representatives and others.

Funding for rare disease drug development

The Centre has launched its first call for funding for promising UK and North American academic drug development projects, which will support ten rare disease Scholars per year and add to its therapeutics pipeline.

Next article