One-vector gene therapy for dominant retinitis pigmentosa
Official title: A single mutation-independent AAV gene therapy for the treatment of autosomal-dominant retinitis pigmentosa.
Funded at Opus Genetics INC. · NIH-11187134 · Data updated 2026-11-12
This gene therapy aims to lower harmful rhodopsin and replace it with a healthy copy to help people with autosomal-dominant retinitis pigmentosa caused by many RHO mutations.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Opus Genetics INC. NIH-funded |
| Lab location | 1 site (Raleigh, United States) |
| Project start | September 30, 2024 |
| Project end | August 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $836,933 |
| NIH project number | 5R44EY036301-02 |
| Project ID | NIH-11187134 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are people diagnosed with autosomal-dominant retinitis pigmentosa caused by mutations in the RHO gene who retain some photoreceptor cells.
✕Less likely to help
People with retinitis pigmentosa caused by non-RHO genes, end-stage retinal degeneration with little or no remaining photoreceptors, or unrelated eye diseases are unlikely to benefit.
What this research studies
You or a loved one with dominant retinitis pigmentosa caused by RHO mutations would get a single AAV5 viral vector that both knocks down the patient's own rhodopsin (mutant and wild-type) and delivers a codon‑optimized healthy RHO gene. The 'knockdown-and-replace' design is intended to work across many different RHO mutations so one product can help many patients instead of tailoring to each mutation. The team is testing delivery, safety, and retinal protection in preclinical models including canine studies to measure retinal function and structure after treatment. If preclinical results are favorable, the program aims to progress toward human clinical trials.
Why it matters
Potential benefit: If successful, the therapy could preserve night and peripheral vision and slow or stop progression of vision loss in people with RHO-related dominant RP.
How similar studies have gone: Gene therapy has produced clinical benefits for other inherited retinal diseases like RPE65-related vision loss, but mutation-independent knockdown-and-replace approaches for RHO are largely preclinical and not yet proven in humans.
Where this research is happening
-
Opus Genetics INC.
Raleigh, United States Active
Researchers
- Principal investigator: Jayagopal, Ashwath — Opus Genetics INC.
- Study coordinator: Jayagopal, Ashwath
Related research
Other NIH-funded projects on the same topics.
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Inflammation in MERTK-related retinitis pigmentosa
This project aims to understand whether losing the MERTK gene causes retinal inflammation that leads to vision loss in people with MERTK-related retinitis pigmentosa.
-
Enhancing cone survival in RP through targeted cell-specific CRISPR therapy
A patient-friendly look at a new approach to protect cone cells in retinitis pigmentosa using precise gene editing to support vision.
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A gene-mutation-agnostic pill (MC16) is being studied to help people with retinitis pigmentosa by addressing a common cellular problem in the eye.
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Role of IPM Proteoglycans in Retinal Health and Inherited Blindness
This grant studies how specific retinal extracellular matrix proteins (IMPG1 and IMPG2) affect eye health and contribute to retinitis pigmentosa, with potential implications for people who have or are at risk for inherited blindness.
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NAC Attack: a late-stage trial for preserving vision in RP patients
A large, multi-site study is testing whether taking a daily NAC pill can help people with retinitis pigmentosa keep more of their vision.
-
Non-invasive biomarker for mitochondria health in retinitis pigmentosa
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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.