Gene therapy for NMNAT1-related Leber congenital amaurosis
Official title: Development of OPGx-NMNAT1 as a gene therapy for NMNAT1 associated LCA.
Funded at Opus Genetics INC. · NIH-11409872 · Data updated 2026-11-12
A gene therapy using an AAV9 vector to deliver NMNAT1 could potentially help young patients with NMNAT1-related retinal degeneration improve or 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 15, 2026 |
| Project end | May 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $1,012,469 |
| NIH project number | 1R44EY038149-01A1 |
| Project ID | NIH-11409872 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children and adolescents with NMNAT1-related Leber congenital amaurosis or related NMNAT1 retinal degeneration.
✕Less likely to help
Patients without NMNAT1 mutations or those with non-eligible retinal conditions would likely not benefit.
What this research studies
This project aims to develop a gene augmentation therapy called OPGx-NMNAT1 for Leber congenital amaurosis type 9 caused by NMNAT1 mutations. The approach uses a recombinant AAV9 vector to deliver a codon-optimized NMNAT1 gene under a strong promoter to retinal cells. It focuses on advancing a potential treatment through preclinical development and early testing relevant to affected patients, with safety and efficacy considerations in mind. If successful, this work could pave the way for a therapy that preserves vision in children affected by NMNAT1-related retinal degeneration.
Why it matters
Potential benefit: Potential to preserve or improve vision for patients with NMNAT1-associated retinal degeneration.
How similar studies have gone: Other NMNAT1-related gene therapies are in early stages, with promising preclinical signals but no approved treatments yet.
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.