“What the AMT-130 trial shows us is that lowering mutant huntingtin is a feasible and effective way to progress in the treatment of Huntington’s disease, and that progression of the disease can potentially be slowed.”
Abi Pinchbeck (Editor, Cell & Gene Therapy Insights) speaks with Anne Rosser (Professor of Clinical Neuroscience, Cardiff University) about Cardiff’s leading role in the recent landmark Huntington’s disease trial of UniQure’s AMT-130 gene therapy trial in collaboration with UCL in the UK and sites in Poland and the US. This is the first gene therapy trial to report statistically significant slowing of Huntington’s progression [1][2]. They discuss the scientific breakthroughs behind the study, the challenges of neurosurgical delivery, possible implications for other neurodegenerative conditions, and the evolving relationship between gene and cell therapy approaches for Huntington’s disease.
