Improving gene therapy for hemophilia A
Official title: Toward Safer Gene Therapy for Hemophilia A
Funded at Indiana University Indianapolis · NIH-11023034 · Data updated 2026-11-12
This study is looking at ways to make gene therapy for hemophilia A safer and more effective by using a special virus to deliver the missing gene to the liver, so that patients can have a better and longer-lasting treatment.
Quick facts
| Grant type | P01 program project |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Indiana University Indianapolis NIH-funded |
| Lab location | 1 site (Indianapolis, United States) |
| Project start | February 5, 2022 |
| Project end | January 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $2,288,637 |
| NIH project number | 5P01HL160472-04 |
| Project ID | NIH-11023034 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are males diagnosed with severe hemophilia A who are experiencing challenges with current treatment options.
✕Less likely to help
Patients with mild hemophilia or those who do not have hemophilia A may not benefit from this research.
What this research studies
This research focuses on enhancing the safety and effectiveness of gene therapy for hemophilia A, a bleeding disorder caused by a deficiency in factor VIII. The team is investigating the use of adeno-associated viral (AAV) gene transfer to deliver the necessary gene to the liver, which has shown promise in initial trials but faces challenges such as declining factor levels and liver toxicity. By addressing these safety concerns and understanding the interactions between the gene therapy vector and liver cells, the research aims to provide a more durable and safer treatment option for patients. The approach includes studying the immune response and potential risks associated with the therapy.
Why it matters
Potential benefit: If successful, this research could lead to a safer and more effective gene therapy that provides lasting treatment for hemophilia A.
How similar studies have gone: Previous clinical trials using similar gene therapy approaches have shown initial success, but this research aims to address critical safety and efficacy concerns that have emerged.
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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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.
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Targeting FVIII production to eye-opening blood vessels in hemophilia A
Aims to improve treatment for people with hemophilia A by directing how the body makes clotting factor VIII, using a targeted gene-delivery approach.
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Platelet-targeted FVIII Gene Therapy for Hemophilia A
A gene therapy approach aims to make platelets carry FVIII to help people with hemophilia A, even if they have inhibitors against FVIII.
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.