Investigating gene therapy for hemophilia A using liver-targeted AAV vectors
Official title: Defining the therapeutic efficacy, tolerogenic potential, and genotoxicity of liver-targeted AAV gene therapy for hemophilia A
Funded at Wake Forest University Health Sciences · NIH-10889145 · Data updated 2026-11-12
This study is looking at a new gene therapy for hemophilia A that uses a special virus to help the liver produce the missing clotting factor, and it’s testing different ways to give this treatment safely to babies before they are born or shortly after.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Wake Forest University Health Sciences NIH-funded |
| Lab location | 1 site (Winston-Salem, United States) |
| Project start | September 15, 2022 |
| Project end | June 30, 2026 |
| Funding year | 2024 |
| Award (this year) | $648,076 |
| NIH project number | 5R01HL161290-03 |
| Project ID | NIH-10889145 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include newborns and adults diagnosed with hemophilia A who have not developed inhibitors to clotting factors.
✕Less likely to help
Patients with severe pre-existing AAV immunity or those who have already developed inhibitors to clotting factors may not benefit from this research.
What this research studies
This research focuses on developing a gene therapy approach for hemophilia A that targets the liver using adeno-associated virus (AAV) vectors. It aims to evaluate the effectiveness of this therapy, its potential to induce tolerance in the immune system, and any risks of genetic damage. The study will explore both neonatal and in utero gene therapy methods to address challenges such as pre-existing immunity to AAV and the risk of developing inhibitors to clotting factors. By using animal models, the research seeks to identify optimal conditions for safe and effective treatment.
Why it matters
Potential benefit: If successful, this research could provide a long-lasting treatment option for hemophilia A, potentially reducing or eliminating the need for regular factor replacement therapy.
How similar studies have gone: Previous research has shown promise in gene therapy approaches for hemophilia, but this specific method using liver-targeted AAV vectors is still being explored and is considered novel.
Where this research is happening
-
Wake Forest University Health Sciences
Winston-Salem, United States Active
Researchers
- Principal investigator: Almeida-Porada, Graca Duarte — Wake Forest University Health Sciences
- Study coordinator: Almeida-Porada, Graca Duarte
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.