Enhancing immune regulation in gene therapy for hemophilia (new approaches to safer treatment)
Official title: Enhancing immune regulation in gene therapy for hemophilia
Funded at Indiana University Indianapolis · NIH-11416511 · Data updated 2026-11-12
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.
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 | 2026 |
| Award (this year) | $701,982 |
| NIH project number | 5R01HL131093-10 |
| Project ID | NIH-11416511 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates include individuals with hemophilia A or B who may be considering or have received AAV-based gene therapy, and who can participate in preclinical-to-clinical translation studies.
✕Less likely to help
Patients not involved in liver-focused immune studies or those without hemophilia may not directly benefit.
What this research studies
This grant builds on gene therapy approaches for hemophilia that use a liver-targeted AAV delivery to produce the missing clotting factors. It aims to map how liver interactions influence immune responses and to refine immune-modulation strategies so patients maintain treatment benefits with fewer immune-related problems. Researchers plan to test models that reflect human biology and to examine how liver fat (steatosis) could affect therapy safety and effectiveness. The goal is to translate immune-regulation insights into safer, more reliable gene therapies for people with hemophilia A or B.
Why it matters
Potential benefit: If successful, the research could lead to safer gene therapies with fewer immune reactions, improving durability and quality of life for people with hemophilia.
How similar studies have gone: Prior work showed T cell tolerance and some immune insights in humans, suggesting potential for broader application; however, full translational success remains ongoing.
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
Other NIH-funded projects on the same topics.
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cGMP production of FVIII-making placental cells to help people with Hemophilia A
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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A safer, longer-lasting gene delivery approach for Hemophilia A using improved nonviral vectors
Researchers are testing a new type of nonviral gene delivery that could provide longer expression of a therapeutic gene for adults with Hemophilia A.
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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
A new point-of-care test for hemophilia A measures FVIII and emicizumab to help doctors manage bleeding risk quickly, with plans to obtain regulatory clearance.
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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.
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Intraosseous delivery of gene therapy vectors for hemophilia A
A gene therapy approach using vectors delivered into bone to help people with hemophilia A reduce or eliminate the need for frequent FVIII treatments.
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.