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Updated: Jun 2, 2026
10:52
Efficient and Scalable Production of Full-length Human Huntingtin Variants in Mammalian Cells using a Transient Expression System
Published on: December 10, 2021
Systematic Review of the Huntington's Disease Drug Development Pipeline, 2014 to 2025
Pavlina Konstantinova1, Jenny Townhill1,2, Juliana Bronzova1
1European Huntington's Disease Network, University Hospital of Ulm, Ulm, Germany.
Summary
The Huntington's disease (HD) drug development pipeline has significantly expanded, with a strong shift towards disease-modifying therapies. Clinical trials now target earlier disease stages, reflecting advancements in HD research.
Area of Science:
- Neurology
- Clinical Research
- Drug Development
Background:
- Advances in understanding Huntington's disease (HD) pathobiology have identified potential therapeutic targets.
- The past decade has seen significant progress in HD research.
Purpose of the Study:
- To comprehensively describe the clinical pipeline for Huntington's disease drug development.
- To analyze trends in HD clinical trials over the past decade.
Main Methods:
- Analysis of HD clinical trials registered in clinicaltrials.gov and WHO ICTRP (2014-2025).
- Classification of studies into interventional (pharmacological, non-pharmacological) and observational categories.
- Categorization of interventional trials by phase, intervention type, and mechanism of action, including a distinct category for huntingtin lowering.
Main Results:
- 165 HD clinical studies involving 7501 participants were identified.
- 111 interventional and 54 observational trials were registered.
- Disease-modifying therapies constitute a growing majority of trials across all phases (Phase 1: 90.5%, Phase 2: 61.8%, Phase 3: 55.6%).
Conclusions:
- The HD clinical trial landscape has expanded significantly over the last decade.
- There is a clear shift towards disease-modifying strategies in HD drug development.
- Clinical trials are increasingly targeting earlier stages of Huntington's disease, reflecting evolving research and therapeutic approaches.
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