Huntington's disease clinical trials update: August 2026. (PubMed, J Huntingtons Dis) - Oct 20, 2026 - "We discuss the launch and subsequent discontinuation of POINT-HD, the first-in-human phase I study of RG6496 (Hoffmann-La Roche), an allele-selective antisense oligonucleotide designed to selectively lower mutant huntingtin while largely preserving wild-type huntingtin expression. We also discuss the launch of two phase III efficacy trials: INVEST-HD, evaluating votoplam (Novartis Pharmaceuticals), and PRECISE-HD, evaluating pridopidine (Prilenia). In addition, we summarise recent advances across the field, including preclinical updates for LQT-23 (LoQus23), SRP-1005 (Sarepta Therapeutics) and HRN001 (Harness Therapeutics); interim 12-month clinical results from SKY-0515 (Skyhawk Therapeutics); the announcement by uniQure that 3-year data from the phase I/II AMT-130 study may support a Biologics License Application seeking accelerated approval and Roche's discontinuation of the phase II GENERATION HD2 study of tominersen. Finally, we provide an updated..."
First-in-human • Journal • CNS Disorders • Huntington's Disease • Movement Disorders
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Edward J Wild; Mena Farag; Opeyemi Kinyomi; Sarah J Tabrizi
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