Huntington's disease clinical trials update: October 2025
In this edition of the Huntington's Disease Clinical Trials Update, we expand on the launch of the phase II/III clinical trial of SKY-0515 from Skyhawk Therapeutics and the phase I/II clinical trial of SPK-10001 from Spark Therapeutics. We also report positive topline data from uniQure's phase I/II clinical trial of AMT-130 after 36 months of follow-up. Further updates include recent developments in Roche's tominersen programme within GENERATION HD2, progress with votoplam (PTC518) in PIVOT-HD by PTC Therapeutics and developments in the collaborative PTC Therapeutics/Novartis programme. We additionally discuss regulatory developments regarding pridopidine following the negative PROOF-HD study. Finally, we provide an updated listing of all registered and ongoing clinical trials in Huntington's disease.
Authors
- Edward J. Wild (ORCID: https://orcid.org/0000-0002-6921-7887)
- Sarah J. Tabrizi (ORCID: https://orcid.org/0000-0003-2716-2045)
- Mena S. Farag (ORCID: https://orcid.org/0000-0002-0679-0117)
Institutions
- Huntington's Disease Association (GB)
- National Hospital for Neurology and Neurosurgery (GB)
- University College London (GB)
Publication Details
- Journal
- Journal of Huntington s Disease
- Published
- 2025-11-26
- DOI
- https://doi.org/10.1177/18796397251399751
- Citations
- 1
- Primary Topic
- Genetic Neurodegenerative Diseases
- Type
- article
- Field-Weighted Citation Impact
- 0.91