New treatments for inherited retinal diseases
Official title: Triple threat therapeutics for inherited retinal degenerations
Funded at University of California, San Francisco · NIH-11005398 · Data updated 2026-11-12
This study is looking at new ways to help people with inherited retinal dystrophies, which cause vision loss, by testing two small drugs that might protect the eyes from damage, no matter what specific gene is involved.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project start | February 1, 2024 |
| Project end | January 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $471,569 |
| NIH project number | 5R01EY035514-02 |
| Project ID | NIH-11005398 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with inherited retinal dystrophies, such as Stargardt disease or Batten disease.
✕Less likely to help
Patients with retinal conditions caused by non-genetic factors or those not classified as inherited retinal dystrophies may not benefit from this research.
What this research studies
This research investigates innovative therapies for inherited retinal dystrophies (IRDs), which lead to progressive vision loss. Instead of focusing on specific genetic causes, the study explores the use of small molecule drugs that can potentially treat multiple IRDs regardless of their genetic background. The researchers will test two drugs in mouse models that target harmful lipid accumulation in the retina, aiming to protect retinal cells and preserve vision. By understanding how these drugs work, the goal is to develop effective treatments for patients suffering from these conditions.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that significantly improve or preserve vision for patients with inherited retinal diseases.
How similar studies have gone: This approach of using gene-agnostic therapies for retinal diseases is relatively novel, but similar strategies have shown promise in other areas of medical research.
Where this research is happening
-
University of California, San Francisco
San Francisco, United States Active
Researchers
- Principal investigator: Lakkaraju, Aparna — University of California, San Francisco
- Study coordinator: Lakkaraju, Aparna
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.