Kavli Oxford’s Esther Becker wins support from the Oxford-Harrington Rare Disease Centre to advance rare disease therapies

Kavli Oxford’s Esther Becker wins support from the

Oxford-Harrington Rare Disease Centre to advance

rare disease therapies

Professor Esther Becker, a Kavli Institute for Nanoscience Discovery Principal Investigator, and Professor of Translational Neuroscience in the Nuffield Department of Clinical Neurosciences, has received support from the Oxford-Harrington Rare Disease Centre (OHC) to advance research into treatments for rare neurodegenerative diseases.

Professor Becker has been named one of ten recipients of the 2025 Rare Disease Scholar Award, which supports promising academic discoveries with the aim of accelerating their translation into clinical practice. She is the only 2025 Scholar selected from the UK and will receive £100,000 in drug and business development support for her work on new small molecules to treat spinocerebellar ataxia (SCA).

SCA is a group of rare inherited movement disorders involving progressive dysfunction and degeneration of the cerebellum. There is currently no approved effective treatment. Professor Becker’s research will focus on developing small, brain-penetrant inhibitors of TRPC3, a calcium-permeable ion channel that has emerged as a key player in spinocerebellar ataxia.  

 

 

"I am absolutely thrilled to have been selected as one of the 2025 Oxford-Harrington Rare Disease Scholars,” Professor Becker said. “The award will allow my team to work even more closely together with Paul Brennan’s group at the Centre for Medicines Discovery to develop and test novel TRPC3 inhibitors. The support from the OHC will make a huge difference in moving our research forward into life-changing therapies for patients with spinocerebellar ataxia."

Professor Becker has also received support through the FA Alliance Innovation Fund, coordinated by the OHC, for research into Friedreich’s ataxia (FA). She is one of five Oxford researchers awarded £100,000 each to explore novel therapeutic approaches for the condition, a debilitating and life-shortening multisystem rare disease for which there is currently no cure.

 

Professor Becker's FA research will investigate FXN locus silencing in cerebellar organoids, with the aim of identifying new opportunities for therapeutic intervention. The work forms part of a broader effort bringing together researchers and clinicians to accelerate the development of new therapies and cures for FA.

The OHC is a partnership between the University of Oxford and the Harrington Discovery Institute at University Hospitals, Cleveland, Ohio. Its programmes combine scientific expertise, philanthropic funding and translational drug development support to help move rare disease discoveries from the laboratory towards patient benefit.