EHDN 2022 | Strategies to improve ASOs in Huntington’s disease

Patrick Weydt, MD, PhD, University of Bonn Medical Center, Bonn, Germany, talks on strategies to improve the efficacy of antisense oligonucleotides (ASOs) for the treatment of Huntington’s disease (HD). Several putative huntingtin (HTT) lowering ASOs have been in clinical development in recent years, with disappointing results to date. Dr Weydt highlights the importance of thoroughly analyzing the data from these trials in order to fully understand why the ASOs did not provide the hypothesized benefit. Additionally, further research into central nervous system distribution, improved delivery techniques, and the value of allele-specific approaches are key investigational avenues. This interview took place during the European Huntington’s Disease Network 2022 Plenary Meeting.

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