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
- 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
- Natalia Gromak, Associate Professor in Pathology - Design of GAA-repeat proteomics to identify factors interacting with expanded repeats in Friedreich’s Ataxia
- 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.
Professors Becker and Németh said:
We are truly grateful to receive this award from the FA Alliance Innovation Fund. This support will enable us to advance the modelling of Friedreich’s Ataxia using cerebellar organoids, with the goal of identifying new therapeutic strategies for patients. We are excited about the opportunities this funding creates and look forward to working closely with our colleagues across the FA Alliance and FARA.
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.