Gene therapy for aging-related Parkinson’s disease (idPD) tested in animals and moved toward humans
Official title: Gene Therapy for Idiopathic Parkinson's Disease: In Vivo Validation
Funded at Mark Therapeutics, INC. · NIH-11316942 · Data updated 2026-11-12
A patient-facing overview of a gene therapy approach aiming to slow or modify idiopathic Parkinson’s disease by delivering a therapeutic gene in the brain, with plans to test safety and efficacy in advanced animal models before human studies.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Mark Therapeutics, INC. NIH-funded |
| Lab location | 1 site (San Diego, United States) |
| Project start | September 1, 2024 |
| Project end | July 31, 2029 |
| Funding year | 2026 |
| Award (this year) | $862,457 |
| NIH project number | 2R44NS134446-03 |
| Project ID | NIH-11316942 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with idiopathic Parkinson’s disease who are in the age-related disease trajectory and could be eligible for future gene therapy trials.
✕Less likely to help
Individuals without Parkinson’s disease or those not in a progression stage suitable for disease-modifying therapy may not benefit.
What this research studies
This program aims to refine a one-of-a-kind gene therapy for idiopathic Parkinson’s disease, focusing on restoring cellular functions in brain regions that control movement. Researchers will optimize animal models that resemble age-related disease progression and test how well the leading therapy candidate, Mark202, protects dopamine neurons and supports brain cell health. The project will also evaluate safety and potential benefits in these models to inform future human studies. If successful, this work could pave the way for a disease-modifying option for many patients with idPD who currently have limited treatment choices.
Why it matters
Potential benefit: If successful, this research could lead to a first-in-class gene therapy that slows or halts progression of idPD and preserves motor function.
How similar studies have gone: Prior gene therapy approaches in neurodegenerative models have shown partial disease-modifying potential; this program builds on that by targeting idPD-specific cellular deficits.
Where this research is happening
-
Mark Therapeutics, INC.
San Diego, United States Active
Researchers
- Principal investigator: Bolotina, Victoria M — Mark Therapeutics, INC.
- Study coordinator: Bolotina, Victoria M
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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.