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The Oxford-Harrington Rare Disease Centre Announces Newly Funded Grant Award Programmes to Develop New Therapies for Friedreich’s Ataxia

May 17, 2024

Microscope Friedrich's Ataxia

OXFORD, UK and CLEVELAND, Ohio, US, 17th May, 2024 – The Oxford-Harrington Rare Disease Centre (‘OHC’), a partnership (between the University of Oxford, UK and Harrington Discovery Institute at University Hospitals, Cleveland, Ohio) aimed at driving cutting-edge rare disease breakthroughs, is pleased to announce that it has received a philanthropic donation of up to £650,000 to enable the launch of two new grant award programmes to support the research and development of greatly needed therapeutics for Friedreich’s Ataxia (FA): The FA Alliance Catalyst Fund and The FA Alliance Innovation Fund.

In 2021, the OHC established a dedicated programme for recruiting leading disease area experts and coordinating substantial research efforts towards developing new treatments for FA. So far, 18 leading researchers have been recruited and a pipeline of FA projects has been created. This programme is supported by the non-profit organisations EndFA, The Friedreich’s Ataxia Research Alliance (FARA), and Ataxia UK, and is collectively known as the Friedreich’s Ataxia Alliance at Oxford (FA Alliance).

The FA Alliance Catalyst Fund and FA Alliance Innovation Fund will both initiate and accelerate cutting-edge research currently underway within the OHC for the development of a treatment for FA. The FA Alliance Catalyst Fund will leverage co-funding and support from the University of Oxford’s Translational Research Office to fund research scientists already tackling FA.  The FA Alliance Innovation Fund will specifically advance earlier-stage ideas with little or no preliminary data that aim to explore highly innovative, potentially ground-breaking concepts in FA research.

FA is a debilitating, life-shortening, degenerative multisystem rare disease that affects roughly 1 in 50,000 people in the US / UK[1], with onset typically between 10-15 years of age. Individuals with FA have genetic mutations that result in a deficiency in the production of a protein called frataxin, causing progressive damage and cell death. This leads to the initial symptoms of FA, such as tiredness, vision and hearing loss, chest pain, slow or slurred speech, difficulty walking and a loss of sensation and reflexes. Despite extensive research, there is no cure for FA, meaning that significant progress is still required to advance new therapeutics for patients in need.

Matthew Wood, Director and Chief Scientific Officer of the OHC, and Professor of Neuroscience in Oxford’s Department of Paediatrics, said:

Today’s announcement validates and underscores OHC’s commitment to driving innovation and collaboration in research towards new therapeutics to meet the major unmet need of Friedreich’s Ataxia. We are extremely grateful for the support received, which is enabling the launch of these two new award programmes. Through OHC and the FA Alliance, we are bringing a multidisciplinary team of leading researchers and clinicians together to tackle this challenging rare disease as one of our priority areas. Strengthened by the complementary expertise of the Harrington Discovery Institute, this new funding will both accelerate existing projects and advance new projects with the aim of developing a therapeutic to cure or treat FA.

For more information, visit: Friedreich’s Ataxia Research at OHC

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, UK and Harrington Discovery Institute at University Hospitals, Cleveland, Ohio, US. OHC combines world-leading strengths in research and therapeutics development from across these organisations to set the science and innovation agenda and drive 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 | X

About the University of Oxford

The University of Oxford 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. The University of Oxford’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. For more information, visit: www.ox.ac.uk 

About Harrington Discovery Institute

The Harrington Discovery Institute at University Hospitals in Cleveland, Ohio – 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.orgLinkedIn | X

Media contacts:

UK/ EU – MEDiSTRAVA
Sylvie Berrebi / Mark Swallow
OHC(at)medistrava.com

US – For University Hospitals
Ansley Kelm
ansley.kelm(at)UHhospitals.org


[1] The Friedreich’s Ataxia Research Alliance (FARA) - https://www.curefa.org/what-is-friedreichs-ataxia