Making gene therapy for hemophilia safer by improving immune control
Official title: Enhancing immune regulation in gene therapy for hemophilia
Funded at Indiana University Indianapolis · NIH-11243917 · Data updated 2026-11-12
This project aims to reduce harmful immune reactions so gene therapy for people with hemophilia works better and lasts longer.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Indiana University Indianapolis NIH-funded |
| Lab location | 1 site (Indianapolis, United States) |
| Project start | July 1, 2016 |
| Project end | June 30, 2029 |
| Funding year | 2025 |
| Award (this year) | $745,423 |
| NIH project number | 2R01HL131093-09 |
| Project ID | NIH-11243917 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with hemophilia A or B who are candidates for or interested in liver-directed AAV gene therapy, especially those with problematic antibodies to clotting factors, would be most directly relevant.
✕Less likely to help
People without hemophilia or those who cannot receive AAV-based liver gene therapy (for example due to high pre-existing anti-AAV antibodies) are unlikely to benefit from this work.
What this research studies
From a patient perspective, the team studies how AAV liver-directed gene therapy interacts with the immune system to either teach the body to tolerate the new clotting factor or trigger harmful responses. They use animal models and human-relevant systems to track regulatory T cells, CD8+ T cell activation, and signals from liver immune cells that influence those responses. The researchers are testing added immune-modulating approaches and exploring how conditions like fatty liver affect outcomes to find ways to prevent antibodies and other immune problems. Their methods combine gene transfer experiments, cellular and molecular immune studies, and translational models that reflect human biology.
Why it matters
Potential benefit: If successful, this work could lower immune-related risks and broaden who can safely benefit from AAV gene therapy for hemophilia.
How similar studies have gone: Liver-directed AAV gene therapies for hemophilia have shown promise and received regulatory approval, and animal studies plus early human reports suggest immune tolerance can occur, but immune responses remain a key challenge this work builds on.
Where this research is happening
-
Indiana University Indianapolis
Indianapolis, United States Active
Researchers
- Principal investigator: Herzog, Roland W. — Indiana University Indianapolis
- Study coordinator: Herzog, Roland W.
Related research
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A new, patient-friendly therapy aims to make FVIII-producing placental cells in the lab to help adults with Hemophilia A, potentially offering a longer-lasting and more affordable treatment.
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Enhancing immune regulation in gene therapy for hemophilia (new approaches to safer treatment)
A patient-focused overview of a long-running NIH grant exploring how to make gene therapy for hemophilia safer by teaching the body’s immune system to tolerate the therapeutic protein.
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A safer, longer-lasting gene delivery approach for Hemophilia A using improved nonviral vectors
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Non-viral gene therapy approach to permanently fix hemophilia B
A new gene therapy approach aims to permanently correct hemophilia B without using viruses, potentially reducing bleeding events for people with this condition.
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Hemophilia Point-of-Care Test Gets Regulatory Ready
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Hemophilia A: studying why some people develop inhibitors to FVIII across families
This NIH grant funds a multi-year program to follow families affected by severe hemophilia A to understand why some individuals develop antibodies against factor VIII.
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.