Huntington's disease clinical trials update: March 2025
Farag M. Tabrizi S. Wild E (2025). Huntington's disease clinical trials update: March 2025. Journal of Huntington's Disease, 14(2), 191-206. https://doi.org/10.1177/18796397251337000
- Authors
- Mena Farag, Sarah J Tabrizi, Edward J Wild
- Journal
- Journal of Huntington's Disease
- First published
- 2025
- Number of citations
- 2
- Type
- Journal Article
- DOI
- 10.1177/18796397251337000
Abstract
In this edition of the Huntington's Disease Clinical Trials Update, we expand on the ongoing phase I clinical trial of ALN-HTT02 from Alynlam Pharmaceuticals. We also report on the SAGE-718 (also known as dalzanemdor) program from Sage Therapeutics, with results of the phase II DIMENSION study and the recent termination of the open-label phase III PURVIEW study. Additionally, we discuss recent developments in the regulatory pathway for AMT-130, following discussions between uniQure and the U.S. Food and Drug Administration regarding key aspects of accelerated approval. Finally, we provide a comprehensive listing of all currently registered and ongoing clinical trials in Huntington's disease.