Gene therapy for treating a common inherited eye disease.
A single mutation-independent AAV gene therapy for the treatment of autosomal-dominant retinitis pigmentosa.
['FUNDING_SBIR_2'] · OPUS GENETICS INC. · NIH-10919095
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
| Phase | ['FUNDING_SBIR_2'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | OPUS GENETICS INC. (nih funded) |
| Locations | 1 site (RALEIGH, UNITED STATES) |
| Trial ID | NIH-10919095 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with autosomal-dominant retinitis pigmentosa, particularly those with various mutations in the rhodopsin gene.
Not a fit: Patients with retinitis pigmentosa caused by mutations outside of the rhodopsin gene may not benefit from this research.
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 performed: 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
RALEIGH, UNITED STATES
- OPUS GENETICS INC. — RALEIGH, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: YERXA, BENJAMIN R — OPUS GENETICS INC.
- Study coordinator: YERXA, BENJAMIN R
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.