New treatment approach for Stargardt disease
Official title: Polypharmacological approach to treatment of Stargardt disease
Funded at Columbia University Health Sciences · NIH-11044985 · Data updated 2026-11-12
This study is exploring a new way to help people with Stargardt disease by testing a treatment that could protect their eyesight by reducing harmful substances in the eye.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | March 1, 2023 |
| Project end | February 28, 2027 |
| Funding year | 2025 |
| Award (this year) | $619,139 |
| NIH project number | 5R01EY034461-03 |
| Project ID | NIH-11044985 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Stargardt disease, particularly those with mutations in the ABCA4 gene.
✕Less likely to help
Patients with other forms of macular degeneration or those without genetic mutations associated with Stargardt disease may not benefit from this research.
What this research studies
This research focuses on developing a new treatment for Stargardt disease, a genetic condition that leads to vision loss. The approach aims to reduce the harmful substances that accumulate in the retina by partially inhibiting a key enzyme involved in the visual cycle. By targeting this enzyme, the researchers hope to prolong the survival of retinal cells and improve vision outcomes for patients. The study will involve testing this method in laboratory models to assess its effectiveness and safety.
Why it matters
Potential benefit: If successful, this research could lead to a novel treatment that slows down or prevents vision loss in patients with Stargardt disease.
How similar studies have gone: Previous research has shown promise in targeting the visual cycle for similar retinal diseases, indicating potential for success in this novel approach.
Where this research is happening
-
Columbia University Health Sciences
New York, United States Active
Researchers
- Principal investigator: Petrukhin, Konstantin — Columbia University Health Sciences
- Study coordinator: Petrukhin, Konstantin
Related research
Other NIH-funded projects on the same topics.
-
Combination drug approach to slow vision loss in Stargardt disease
This project tests a combo-drug strategy to lower toxic eye pigments and help protect vision for people with Stargardt disease.
-
Investigating a new oral treatment for Stargardt disease to slow vision loss.
This study is testing a new pill called ALK-001 that aims to help people with Stargardt disease by slowing down vision loss caused by a problem with a specific gene.
-
ASP2020 eye injection safety in Stargardt-type macular dystrophy
A small study will test whether a single ASP2020 eye injection is safe and may help people with Stargardt-type macular dystrophy.
-
C.001 for retinal degeneration: a first-in-human dose-escalation safety test
A first-in-human study will test the safety and tolerability of a single subretinal injection of C.001 in adults with retinal diseases involving retinal pigment epithelium degeneration.
-
Functional visual field and navigation testing for moderate-to-profound inherited retinal disease
This project will test whether functional visual field and navigation tests can measure real-world vision and mobility in people with moderate-to-profound inherited retinal diseases.
-
Single intravitreal RTx-021 injection for Stargardt disease
This will test whether a single injection of RTx-021 into one eye is safe and may help people aged 16 and older with Stargardt disease.
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.