AATSNA-101: Advancing a Gene Therapy for a Rare Childhood Eye Disease
Official title: Manufacturing Development and Commercial Readiness of an Adeno-Associated Virus-Based Gene Therapy for the Treatment of Leber Congenital Amaurosis Type 1
Funded at Atsena Therapeutics, INC. · NIH-11414927 · Data updated 2026-11-12
A gene therapy treatment for Leber congenital amaurosis type 1 in young children is being prepared for Phase 3 and FDA approval.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Atsena Therapeutics, INC. NIH-funded |
| Lab location | 1 site (Durham, United States) |
| Project start | September 15, 2025 |
| Project end | August 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $731,026 |
| NIH project number | 5R44EY037156-02 |
| Project ID | NIH-11414927 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are children up to 11 years old with Leber congenital amaurosis type 1 who could participate in or benefit from later clinical trials and therapy development.
✕Less likely to help
Patients without LCA1 or those not eligible for future trials may not benefit from this grant.
What this research studies
This project focuses on making ATSN-101, an AAV-based gene therapy, ready for Phase 3 and commercial use to help children with LCA1 see better. It will optimize manufacturing processes, develop product-specific tests, and show that the final product is comparable to prior versions using analytical methods and a mouse model. The work leverages Atsena’s existing clinical data and manufacturing platform to ensure the therapy can be produced reliably and meet regulatory standards. If successful, this work could pave the way for a safe, effective treatment for a childhood blindness condition.
Why it matters
Potential benefit: If successful, ATSN-101 could become a approved gene therapy that improves vision for children with LCA1.
How similar studies have gone: Prior Phase 1/2 results showed meaningful improvements in retinal sensitivity and vision in LCA1 patients, supporting continued development.
Where this research is happening
-
Atsena Therapeutics, INC.
Durham, United States Active
Researchers
- Principal investigator: Mouillesseaux, Kevin P — Atsena Therapeutics, INC.
- Study coordinator: Mouillesseaux, Kevin P
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.