Gene therapy candidate for RDH12-related Leber congenital amaurosis
Official title: Development of OPGx-RDH12 as a gene therapy for RDH12 deficiency associated Leber congenital amaurosis (LCA)
Funded at Opus Genetics INC. · NIH-11405186 · Data updated 2026-11-12
A gene therapy approach using an AAV8 vector to deliver RDH12 to retinal cells in people with RDH12-related LCA, aiming to protect vision.
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, 2026 |
| Project end | June 30, 2028 |
| Funding year | 2026 |
| Award (this year) | $1,004,925 |
| NIH project number | 1R44EY038152-01A1 |
| Project ID | NIH-11405186 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with RDH12-related LCA/IRD, typically presenting early-onset retinal degeneration.
✕Less likely to help
Patients without RDH12-related LCA or without eligibility for AAV-based retinal gene therapy may not benefit.
What this research studies
This project aims to develop a one-time gene therapy for RDH12-IRD, a form of severe inherited vision loss starting in childhood. The therapy uses a non-self-replicating AAV8 vector to deliver a codon-optimized RDH12 gene specifically to photoreceptor cells. Researchers will test the therapy in models and prepare for human studies, focusing on safety, dosing, and potential vision preservation. If successful, the treatment could slow or prevent early vision loss for people with RDH12-IRD.
Why it matters
Potential benefit: If successful, patients with RDH12-IRD could retain more vision and quality of life through a one-time gene therapy.
How similar studies have gone: Gene therapies using AAV vectors have shown promise for inherited retinal diseases, with several progressing to human trials.
Where this research is happening
-
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.