New small-molecule therapy for Huntington's disease aimed at lowering disease-causing protein
Official title: Translational Development and Mechanistic Characterization of Small Molecule Drug for Huntington's Disease Therapy
Funded at University of Mississippi Med Ctr · NIH-11428277 · Data updated 2026-11-12
A potential drug candidate called GLYN122 is being developed to reach the brain and lower the harmful huntingtin protein in Huntington’s disease patients.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Mississippi Med Ctr NIH-funded |
| Lab location | 1 site (Jackson, United States) |
| Project start | August 15, 2026 |
| Project end | April 30, 2031 |
| Funding year | 2026 |
| Award (this year) | $554,565 |
| NIH project number | 1R01NS150332-01 |
| Project ID | NIH-11428277 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults diagnosed with Huntington’s disease or those in early disease stages who could participate in preclinical and translational studies linked to GLYN122 development.
✕Less likely to help
Persons without Huntington’s disease or those not eligible for the translational components of this program may not benefit.
What this research studies
This grant supports translating a computer-designed small molecule into a therapy that can cross the blood-brain barrier and reduce mutant huntingtin levels in the brain. The work includes lab studies showing GLYN122 binds mHTT and promotes neuron-autophagy, plus animal studies to confirm brain delivery and target engagement. If successful, it could lead to a new disease-modifying option for adults with Huntington’s disease who currently have limited treatment choices. The project is led by researchers at the University of Mississippi Medical Center and runs from 2026 to 2031.
Why it matters
Potential benefit: If successful, patients with Huntington’s disease could gain a therapy that directly lowers the disease-causing protein and potentially slows disease progression.
How similar studies have gone: Earlier work showed that similar small molecules can engage the target and influence disease-related pathways in models, supporting further development.
Where this research is happening
-
University of Mississippi Med Ctr
Jackson, United States Active
Researchers
- Principal investigator: Raucher, Drazen — University of Mississippi Med Ctr
- Study coordinator: Raucher, Drazen
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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.