Investigating small molecules that reduce harmful huntingtin protein for Huntington's disease treatment
Official title: Characterization of small molecules that lower mutant huntingtin protein as potential therapeutics for Huntington’s disease
Funded at Origami Therapeutics, INC. · NIH-10929461 · Data updated 2026-11-12
This study is looking at new small molecules that could help lower the harmful protein causing Huntington's disease, with the hope of slowing down the disease and improving life for those affected.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Origami Therapeutics, INC. NIH-funded |
| Lab location | 1 site (San Diego, United States) |
| Project start | September 15, 2023 |
| Project end | July 31, 2025 |
| Funding year | 2024 |
| Award (this year) | $107,065 |
| NIH project number | 5R43NS127716-02 |
| Project ID | NIH-10929461 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Huntington's disease, particularly those in the early to mid-stages of the condition.
✕Less likely to help
Patients with advanced Huntington's disease or those who do not carry the genetic mutation associated with the condition may not benefit from this research.
What this research studies
This research focuses on developing small molecules that can lower the levels of mutant huntingtin protein, which is responsible for the neurodegenerative effects of Huntington's disease. By targeting the abnormal protein, the study aims to halt or slow down the progression of the disease, which currently has no effective treatment. The approach involves testing these molecules in preclinical models to assess their efficacy and safety before potential clinical application. Patients may benefit from new therapeutic options that could improve their quality of life and disease management.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that significantly improve outcomes for patients with Huntington's disease.
How similar studies have gone: Previous research has shown promise in lowering mutant huntingtin protein in animal models, indicating potential for success in translating these findings to human therapies.
Where this research is happening
-
Origami Therapeutics, INC.
San Diego, United States Active
Researchers
- Principal investigator: Hoffman, Beth J — Origami Therapeutics, INC.
- Study coordinator: Hoffman, Beth J
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.