Activating a specific enzyme to treat a rare neurodegenerative disorder
Official title: PANK Activators for the treatment of pantothenate kinase-associated neurodegeneration
Funded at Virtus Therapeutics Corporation · NIH-10929351 · Data updated 2026-11-12
This study is looking for a way to help people with pantothenate kinase-associated neurodegeneration (PKAN) by finding new treatments that can boost a specific enzyme in their cells, which might help fix some of the problems caused by the disease.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Virtus Therapeutics Corporation NIH-funded |
| Lab location | 1 site (Groton, UNITED STATES) |
| Project start | September 15, 2023 |
| Project end | August 31, 2025 |
| Funding year | 2024 |
| Award (this year) | $338,249 |
| NIH project number | 5R43NS127740-02 |
| Project ID | NIH-10929351 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with pantothenate kinase-associated neurodegeneration (PKAN), particularly those with mutations in the PANK2 gene.
✕Less likely to help
Patients with neurodegenerative disorders other than PKAN or those without PANK2 mutations may not benefit from this research.
What this research studies
This research focuses on pantothenate kinase-associated neurodegeneration (PKAN), a rare and progressive brain disorder caused by mutations in the PANK2 gene. The study aims to activate the human PANK3 enzyme to enhance the production of coenzyme A in cells affected by PKAN, potentially reversing some of the metabolic and cellular defects caused by the disease. Researchers will utilize patient-derived cells and mouse models to evaluate the effectiveness of newly identified compounds that activate the PANK3 enzyme. The goal is to develop a targeted treatment that goes beyond symptom management and addresses the underlying cause of PKAN.
Why it matters
Potential benefit: If successful, this research could lead to a novel treatment that significantly improves the quality of life and outcomes for patients with PKAN.
How similar studies have gone: While research on PKAN is limited, the approach of targeting specific metabolic pathways in neurodegenerative disorders has shown promise in other studies, suggesting potential for success.
Where this research is happening
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Virtus Therapeutics Corporation
Groton, United States Active
Researchers
- Principal investigator: Regan, Jessica — Virtus Therapeutics Corporation
- Study coordinator: Regan, Jessica
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.