23rd October 2025
Gene therapy appears to slow Huntington’s disease progression
A global clinical trial for a new Huntington’s disease treatment, which involved academics from Cardiff University, has posted positive results.
The research team at UCL found that patients receiving the treatment experienced 75% less progression of the disease overall, compared to a matched cohort of people with Huntington’s who were not receiving the treatment.
This is the first time a drug trial has reported continuing, statistically significant slowing of Huntington’s progression.
Trial sponsor uniQure plans to submit an application to the US Food and Drug Administration early next year requesting accelerated approval to market the drug, with applications in the UK and Europe to follow.
The treatment is a type of gene therapy given during 12 to 18 hours of delicate brain surgery.
Professor William Gray, Professor of Neurosurgery and Director of the Advanced Neurotherapies Centre at Cardiff University, performed the gene therapy surgeries in the UK. The Advanced Neurotherapeutics Centre (ANTC), funded by Health and Care Research Wales, is the only centre in the UK that can perform the surgeries included in the trial – directly delivering the gene therapy to the brain.
Professor Gray said: “This is a landmark result for patients and families affected by this devastating disease. Professor Anne Rosser and I are proud to have collaborated with Professors Tabrizi and Wild in London, recruiting our patients and their patients to surgically deliver this innovative therapy directly into the brain in Cardiff.”
“Cardiff is the only UK centre – and only one of a few centres worldwide – performing these gene therapy surgeries.”
“We wish to acknowledge the courage of our patients undergoing these experimental clinical trials, the work of Uniqure, and the fantastic clinical and research teams here in Cardiff making these therapies a reality,” added Professor Gray.
“Huntington’s Disease is an absolutely devastating condition that runs through families, affecting movement, cognition and behaviour. For Welsh researchers to have contributed to this study by carrying out complex neurosurgeries in Cardiff – the only site in the UK with the capability to perform them – is a testament to how strongly Wales is able to support research into life-changing and even life-saving medicines for the benefit of people around the world,” said the Cabinet Secretary for Health and Social Care, Jeremy Miles.
The study took place in clinical centres across the US, and in two European sites – including the UK. The findings represent years of work, in particular by the sponsor, UniQure, but also by HD investigators, patients and families, and the Huntington’s Disease community. Data was sourced from the global Enroll-HD study as a comparator group for the study.