Gene therapy to protect vision in age-related macular degeneration
Official title: Developing a gene therapy-based solution for early intervention in age related macular degeneration (AMD) to prevent disease progression
Funded at Oculogenex · NIH-11311499 · Data updated 2026-11-12
A patient-focused gene therapy approach aims to slow or prevent progression of intermediate AMD to more advanced stages.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Oculogenex NIH-funded |
| Lab location | 1 site (Dallas, UNITED STATES) |
| Project start | August 1, 2026 |
| Project end | May 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $1,092,536 |
| NIH project number | 1R44EY038510-01 |
| Project ID | NIH-11311499 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with intermediate AMD who are at risk of progressing to advanced AMD.
✕Less likely to help
Patients without AMD or those with late-stage disease are unlikely to benefit.
What this research studies
This project centers on developing a gene therapy called OCLX-001 to halt the progression of intermediate AMD by addressing mitochondrial dysfunction and oxidative stress in the retina. The work includes creating a scalable manufacturing process for the therapy and performing safety studies to support future human trials. If successful, this could lead to a treatment that preserves vision and quality of life for people with AMD who are at risk of rapid disease progression.
Why it matters
Potential benefit: If successful, patients with intermediate AMD could delay vision loss and maintain independence longer.
How similar studies have gone: Preclinical results have shown protective effects on photoreceptors and retinal function in models of AMD.
Where this research is happening
-
Oculogenex
Dallas, United States Active
Researchers
- Principal investigator: Ramkumar, Ramaswamy — Oculogenex
- Study coordinator: Ramkumar, Ramaswamy
Related research
Other NIH-funded projects on the same topics.
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Active cycling program to support vision and daily living in Veterans with AMD
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-
Transplanting stem-cell–derived retinal cells to treat dry AMD
A medical team is testing a stem cell therapy to replace damaged retinal cells in adults with dry age-related macular degeneration.
-
RPESC-derived RPE Transplant for Dry AMD in adults
A new cell-based therapy derived from RPESCs is being tested to help adults with dry age-related macular degeneration regain vision.
-
New high-resolution OCT biomarkers to map early AMD changes
A new imaging approach using advanced OCT methods will help doctors see early changes in age-related macular degeneration to guide treatment and improve quality of life for older adults.
-
Targeting inflammation in age-related macular degeneration
Aims to understand how inflammation drives age-related macular degeneration in people and explore new targets to slow or stop vision loss.
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.