Gene therapy for treating a common inherited eye disease.
Official title: A single mutation-independent AAV gene therapy for the treatment of autosomal-dominant retinitis pigmentosa.
Funded at Opus Genetics INC. · NIH-10919095 · Data updated 2026-11-12
This study is testing a new gene therapy for people with autosomal-dominant retinitis pigmentosa, a genetic eye condition that causes vision loss, by using a special delivery method to fix the faulty gene and help improve their sight.
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 | 2024 |
| Award (this year) | $882,971 |
| NIH project number | 1R44EY036301-01 |
| Project ID | NIH-10919095 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with autosomal-dominant retinitis pigmentosa, particularly those with various mutations in the rhodopsin gene.
✕Less likely to help
Patients with retinitis pigmentosa caused by mutations outside of the rhodopsin gene may not benefit from this research.
What this research studies
This research focuses on developing a gene therapy approach for autosomal-dominant retinitis pigmentosa (adRP), a genetic eye disorder that leads to vision loss. The therapy aims to address the underlying genetic mutations causing the disease by using a viral vector to deliver a treatment that can knock down both the mutant and normal versions of the rhodopsin gene, while also providing a healthy copy of the gene. This innovative approach is designed to be effective for a wide range of mutations, making it a potentially universal treatment for patients with adRP. The research will involve preclinical models to evaluate the safety and efficacy of this therapy before moving to clinical trials.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for patients suffering from a currently untreatable form of retinitis pigmentosa.
How similar studies have gone: While gene therapy for retinal diseases is an emerging field, this specific mutation-independent approach is novel and has not been extensively tested in clinical settings.
Where this research is happening
-
Opus Genetics INC.
Raleigh, United States Active
Researchers
- Principal investigator: Yerxa, Benjamin R — Opus Genetics INC.
- Study coordinator: Yerxa, Benjamin R
Related research
Other NIH-funded projects on the same topics.
-
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.
-
Oral, once-daily MC16 medicine for retinitis pigmentosa
A gene-mutation-agnostic pill (MC16) is being studied to help people with retinitis pigmentosa by addressing a common cellular problem in the eye.
-
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
Aims to test a new, non-invasive OCT-based biomarker to explain and predict how mitochondria function affects cone cells in people with retinitis pigmentosa.
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.