Improved AAV3 gene delivery for hemophilia
Official title: Capsid- and genome-modified AAV3 vectors for hemophilia gene therapy.
Funded at University of Florida · NIH-11248395 · Data updated 2026-11-12
This work develops better AAV3-based gene delivery to help people with hemophilia make missing clotting factors using lower doses and fewer side effects.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Florida NIH-funded |
| Lab location | 1 site (Gainesville, United States) |
| Project start | December 15, 2024 |
| Project end | November 30, 2028 |
| Funding year | 2026 |
| Award (this year) | $668,207 |
| NIH project number | 5R01HL177230-02 |
| Project ID | NIH-11248395 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with moderate-to-severe hemophilia A or B who are considering gene therapy and meet clinical eligibility (for example, without prohibitive liver disease and with acceptable AAV antibody levels).
✕Less likely to help
Children, people with advanced liver disease, or those with high pre-existing antibodies to AAV may not be eligible or may not benefit from this approach.
What this research studies
This project creates next-generation AAV3 viral vectors with both capsid (shell) and genome (ITR) changes to boost delivery into human liver cells. The team will test these engineered vectors in primary human hepatocytes and preclinical models to measure how much clotting-factor gene expression can be achieved. The aim is to reach therapeutic levels of factor VIII or IX using much lower vector doses so patients would need less immune-suppression. If the lab and preclinical results are promising, the optimized vectors would be prepared for eventual clinical testing in people with hemophilia A or B.
Why it matters
Potential benefit: Could enable lower-dose, longer-lasting gene therapy for hemophilia with fewer immune-related complications.
How similar studies have gone: AAV-based gene therapies for hemophilia have led to FDA approvals, showing the overall approach can work, while AAV3 capsid and ITR genome modifications are newer and less tested in people.
Where this research is happening
-
University of Florida
Gainesville, United States Active
Researchers
- Principal investigator: Srivastava, Arun — University of Florida
- Study coordinator: Srivastava, Arun
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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.