Dual-action drug to slow dry age-related macular degeneration
Advancing the small molecule drug candidate with dual specificity as a therapy for dry AMD
A new medicine for people with dry age-related macular degeneration that lowers toxic retinal waste and stabilizes a protein linked to eye damage.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-11110474 on NIH RePORTER |
What this research studies
Researchers are developing a medicine that reduces retinol delivery to the retina to prevent buildup of toxic lipofuscin compounds that drive dry AMD. Older RBP4-lowering drugs reduced lesion growth in some patients but risked destabilizing transthyretin (TTR) and promoting amyloid. This project creates dual-action molecules that both lower RBP4-driven retinol transport and bind TTR at thyroxine sites to keep TTR stable. The lead candidate, (R)-ACPHS-6, will be optimized and advanced through lab and preclinical testing toward eventual human testing.
Who could benefit from this research
Good fit: People with dry (atrophic) age-related macular degeneration, particularly those with growing atrophic lesions or measurable RBP4-related biomarkers, would be ideal candidates.
Not a fit: People with wet (neovascular) AMD or those with advanced, irreversible vision loss are unlikely to benefit from this therapy.
Why it matters
Potential benefit: If successful, this therapy could slow or prevent vision loss in dry AMD by cutting toxic retinal waste while avoiding TTR-related amyloid risk.
How similar studies have performed: Earlier RBP4-lowering drugs like fenretinide slowed atrophy in some patients when serum RBP4 dropped below a threshold, but combining RBP4 lowering with TTR stabilization is a novel approach.
Where this research is happening
New York, United States
- Columbia University Health Sciences — New York, United States (Active)
Researchers
- Principal investigator: Petrukhin, Konstantin — Columbia University Health Sciences
- Study coordinator: Petrukhin, Konstantin
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 below.