Michael Hayden, MB, ChB, PhD, FRCP, FRSC, The University of British Columbia, Vancouver, Canada, discusses the challenges faced in the development of gene therapy for Huntington’s disease, which, to date, has aimed to lower huntingtin levels. Despite early failures, ongoing Phase II studies are exploring different approaches, such as intrastriatal delivery and allele-specific silencing of mutant huntingtin. Concerns remain about the impact of lowering both mutant and wild-type huntingtin, which is neuroprotective. The research continues to be supported by strong preclinical data, but the negative readouts thus far have caused significant delays in the field. This interview took place at the American Academy of Neurology (AAN) Annual Meeting 2024 in Denver, CO.
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