Rajeev Kumar, MD, Rocky Mountain Movement Disorders Center, Englewood, CO, and Ann Morgan, PhD, Annexon Biosciences, discuss huntingtin-lowering strategies currently under clinical investigation in Huntington’s disease (HD). Antisense oligonucleotide (ASO), microRNA, and sRNA strategies, as well as zinc finger protein and CRISPR-Cas9 techniques, are currently being investigated, with many already being evaluated in clinical trials. The largest ASO trial in HD evaluating tominersen (GENERATION HD1) was halted after it was seen that the drug did not have any benefit, and those receiving the most frequent dosing were performing worse. Other allele-specific ASO strategies and RNA splicing modulators, such as branaplam, continue to be evaluated as HD therapies, although some trials have been put on hold due to safety concerns. This interview took place at the 2022 International Congress of Parkinson’s Disease and Movement Disorders in Madrid, Spain.
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