Ribozyme treatment to reduce toxic retinal pigments in dry macular degeneration
Official title: A Ribozyme Rescue Strategy for Dry Age-Related Macular Degeneration
Funded at VA Western New York Healthcare System · NIH-11218694 · Data updated 2026-11-12
This project will develop a ribozyme treatment to break down toxic pigments in the retina and help protect vision for people with dry age-related and juvenile macular degeneration.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | VA Western New York Healthcare System NIH-funded |
| Lab location | 1 site (Buffalo, United States) |
| Project start | January 1, 2020 |
| Project end | June 30, 2026 |
| Funding year | 2025 |
| NIH project number | 5I01BX000669-14 |
| Project ID | NIH-11218694 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with dry age-related macular degeneration or juvenile macular degeneration (for example, Stargardt disease) who have early-to-moderate disease and remaining retinal cells could be ideal candidates.
✕Less likely to help
People with advanced geographic atrophy or extensive photoreceptor loss are unlikely to benefit because too much retinal tissue is already damaged.
What this research studies
The team is developing a small RNA enzyme (a ribozyme) designed to cut the toxic bis-retinoid pigments that build up in retinal pigment epithelium (RPE) cells. They use lab tests and preclinical models to deliver the ribozyme to RPE cells and measure changes in lipofuscin, drusen-like deposits, cell survival, and retinal function. If lab and model results are promising, the work would support moving toward clinical testing in people with dry AMD or juvenile macular degeneration.
Why it matters
Potential benefit: If successful, this approach could slow or stop vision loss by reducing toxic pigment buildup and preserving retinal cells.
How similar studies have gone: Gene-based retinal therapies have shown promise, but ribozyme-based treatments are relatively novel and have limited clinical proof to date.
Where this research is happening
-
VA Western New York Healthcare System
Buffalo, United States Active
Researchers
- Principal investigator: Sullivan, John M. — VA Western New York Healthcare System
- Study coordinator: Sullivan, John 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.