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AAN 2022 | Ongoing trials with importance for Huntington’s disease

Jee Bang, MD, Johns Hopkins University School of Medicine, Baltimore, MD, discusses ongoing Huntington’s disease clinical trials which are generating excitement. Gene-based therapies continue to a focus in the development of treatments for Huntington’s. Tominersen, an antisense oligonucleotide (ASO), was investigated in the Phase III GENERATION HD1 (NCT03761849) study and while it did reduce mutant huntingtin levels, there was no evidence of clinical efficacy. Positive findings from a recent post-hoc analysis of GENERATION HD1 has led to a new Phase II trial of tominersen in younger adults with a lower disease burden. Another ASO drug in development is WVE-003, which targets a single nucleotide polymorphism in the mutant huntingtin mRNA. Compared to its predecessors, WVE-003 has novel backbone chemistry which is thought to lead to better distribution and potency. This interview took place at the American Academy of Neurology 2022 Congress in Seattle, WA.