Safer gene therapy options for Hemophilia A
Official title: Toward Safer Gene Therapy for Hemophilia A
Funded at Indiana University Indianapolis · NIH-11325354 · Data updated 2026-11-12
Developing liver-directed gene therapy that is safer and keeps factor VIII working longer for people with Hemophilia A.
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 | 2026 |
| Award (this year) | $2,492,072 |
| NIH project number | 5P01HL160472-05 |
| Project ID | NIH-11325354 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adult males with Hemophilia A (severe factor VIII deficiency) who are potential candidates for liver-directed AAV gene therapy.
✕Less likely to help
People with significant pre-existing liver disease, high levels of neutralizing antibodies to AAV, children, or those ineligible for liver-directed approaches may not benefit from these specific efforts.
What this research studies
If you have Hemophilia A, this project aims to find out why liver-directed gene therapy can sometimes cause liver injury and why factor VIII levels fall over time. Researchers will examine how AAV gene vectors and the factor VIII gene interact with liver cells and the immune system, using laboratory experiments and analysis of samples from clinical trials. They will test changes to the gene constructs and delivery methods to improve secretion of factor VIII and reduce stress on the liver. The overall aim is to produce approaches that maintain factor VIII levels longer while reducing liver side effects.
Why it matters
Potential benefit: Could lead to gene therapies that provide longer-lasting factor VIII correction with fewer liver-related side effects.
How similar studies have gone: Earlier AAV liver gene therapy trials for Hemophilia A showed promising early correction but have reported declining factor VIII over time and some liver toxicity, so this work builds on those mixed results.
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.
-
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.