The Oxford-Harrington Rare Disease Centre ('OHC'), a partnership between the University of Oxford, UK and Harrington Discovery Institute at University Hospitals, Cleveland, Ohio, dedicated to creating new medicines for rare diseases, has announced that the FA Alliance Innovation Fund has awarded funding to five researchers from the University of Oxford, each of whom receive £100,000 and specialist support to advance pioneering research projects exploring novel approaches to discover treatments for Friedreich's Ataxia (FA). FA is a debilitating, life-shortening, degenerative multisystem rare disease for which there is currently no cure.
The OHC coordinates the Friedreich's Ataxia Alliance at Oxford (FA Alliance), which brings together researchers and clinicians from across the University of Oxford, supported by philanthropic funding, to develop new therapies or cures for FA. The alliance comprises established experts in FA research together with emerging investigators at the forefront of developing advanced therapeutic modalities and novel delivery systems to ensure appropriate targeting to relevant tissues, which is critical in FA.
The FA Alliance Innovation Fund was established by the OHC in collaboration with a philanthropic foundation that provided up to £650,000 to advance cutting-edge therapeutic research at Oxford into FA. A total of £500,000 was awarded to support research into innovative therapeutic approaches for this devastating neurodegenerative disease.
Following a rigorous selection process, the researchers leading the five projects at the University of Oxford are:
Esther Becker, Professor of Translational Neuroscience
Investigating FXN locus silencing in FRDA cerebellar organoids for therapeutic intervention
Natalia Gromak, Associate Professor in Pathology
Design of GAA-repeat proteomics to identify factors interacting with expanded repeats in Friedreich's Ataxia
Andrea Németh, Professor and Consultant in Neurogenetics
Fusion of therapeutic AI exercise gaming and motion capture to create a novel digital clinical outcome assessment in FA
Carlo Rinaldi, Professor of Molecular and Translational Neuroscience and Honorary Consultant Neurologist
Reversing FXN locus silencing via precision epigenetic therapy
Christopher Toepfer, Associate Professor of Cardiovascular Science
Investigating epigenetic upregulation of FXN in FRDA cardiac organoids for treating FRDA cardiomyopathy.
Professor Matthew Wood, Director and Chief Scientific Officer of OHC, said: 'The first FA Alliance Innovation Fund awards mark a major milestone for OHC and the FA Alliance. These projects capture the creativity and ambition needed to change the future for people with Friedreich's Ataxia. They also show how philanthropy is being deployed to transform early ideas into real progress for patients. We're deeply grateful to our partners, whose support enables this life-changing work. Philanthropy is central to our mission, and we warmly welcome others who share this vision to join us in accelerating breakthroughs for rare diseases.'
Professor Carlo Rinaldi, a recipient of the award, commented: 'As researchers working on rare disease therapies, early support from philanthropy is often the difference between an idea that stalls and one that has the potential to change lives. This award allows us to pursue a high-risk, high-reward approach to Friedreich's Ataxia that could generate entirely new therapeutic possibilities. It is a privilege to have this opportunity and to collaborate with the other researchers in the FA Alliance.'
Jennifer Farmer, CEO of Friedreich's Ataxia Research Alliance - FARA, commented: 'The FA Alliance established by Oxford-Harrington, brings together basic, translational and clinical researchers, and a multi-disciplinary approach, which we believe is essential for translating discoveries to treatments. The new research projects announced today through the FA Alliance Innovation Fund give individuals and families living with FA hope and confidence in our mission to treat and cure FA. We are grateful for this partnership and commitment from the OHC and its philanthropic partner and look forward to supporting the research teams as they begin this important work.'
Dr Julie Greenfield, Head of Research of Ataxia UK, noted: 'As the leading national charity for the more than 10,000 people in the UK affected by ataxia, we're delighted to see advancements at Oxford in driving new therapeutic approaches for Friedreich's, which is the most common form of hereditary ataxia.'
The FA Alliance Innovation Fund complements the FA Alliance Catalyst Fund to support translational projects at the OHC. Together, these initiatives represent a comprehensive and coordinated strategy to accelerate research into FA – from the earliest ideas to late-stage development – demonstrating the vital role philanthropy plays in advancing discoveries for rare diseases.