One-vector gene therapy for dominant retinitis pigmentosa
A single mutation-independent AAV gene therapy for the treatment of autosomal-dominant retinitis pigmentosa.
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 ID | NIH-11187134 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates are people diagnosed with autosomal-dominant retinitis pigmentosa caused by mutations in the RHO gene who retain some photoreceptor cells.
Not a fit: 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.
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 performed: 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
Raleigh, United States
- Opus Genetics INC. — Raleigh, United States (Active)
Researchers
- Principal investigator: Jayagopal, Ashwath — Opus Genetics INC.
- Study coordinator: Jayagopal, Ashwath
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.