Making gene therapy safer and more effective for Friedreich's ataxia
Official title: Immunomodulation Approaches to Improve Safety And Efficacy of Gene Therapy Treatment in Friedreich’s Ataxia
Funded at University of Florida · NIH-11179356 · Data updated 2026-11-12
Looks at using immune-calming strategies to help AAV gene therapy work better and be safer for people with Friedreich's ataxia.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Florida NIH-funded |
| Lab location | 1 site (Gainesville, United States) |
| Project start | May 1, 2021 |
| Project end | August 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $730,189 |
| NIH project number | 5U01NS116752-05 |
| Project ID | NIH-11179356 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with genetically confirmed Friedreich's ataxia, typically those with early-onset disease who meet the trial's age and health criteria, are the intended candidates.
✕Less likely to help
People without Friedreich's ataxia, those ineligible for AAV-delivered gene therapy, or those who cannot receive immunomodulation would not be expected to benefit.
What this research studies
If I have Friedreich's ataxia, this project is developing a gene replacement treatment that uses a harmless AAV virus to deliver a working frataxin gene to affected tissues. Because the body can react to the AAV capsid and cause side effects or shorten benefit, the team is adding immune-modulating approaches to reduce those reactions. The researchers have completed animal toxicology and biodistribution studies and are preparing a first-in-human trial under an IND. When approved, patients would be enrolled at clinical sites to receive the gene therapy with added immune management and be followed for safety and durability of benefit.
Why it matters
Potential benefit: Could make AAV gene replacement safer and increase how long the treatment helps people with Friedreich's ataxia.
How similar studies have gone: AAV-based gene replacement has shown promise in FA animal models and in other genetic diseases, but combining AAV delivery with targeted immune suppression for FA is a newer approach.
Where this research is happening
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University of Florida
Gainesville, United States Active
Researchers
- Principal investigator: Corti, Manuela — University of Florida
- Study coordinator: Corti, Manuela
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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.