Improving Gene Therapy for Hemophilia A
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-11169708 · Data updated 2026-11-12
This project aims to make gene therapy safer and more effective for people with hemophilia A, potentially even before birth.
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, 2027 |
| Funding year | 2025 |
| Award (this year) | $652,874 |
| NIH project number | 5R01HL161290-04 |
| Project ID | NIH-11169708 on NIH RePORTER |
Who could benefit from this research
✓Could help
This foundational research does not directly recruit patients, but future clinical applications would target individuals with hemophilia A, including those with pre-existing immunity to AAV vectors or those who might benefit from early intervention.
✕Less likely to help
Patients who do not have hemophilia A or related blood coagulation disorders would not directly benefit from this specific gene therapy approach.
What this research studies
Gene therapy offers the hope of a lasting solution for hemophilia A with a single treatment. However, current gene therapies for adults have shown some risks, like liver issues and potential genetic changes, and many patients already have immunity to the treatment delivery method. This work explores new ways to deliver gene therapy, including during pregnancy, to overcome these challenges and prevent the body from rejecting the treatment or developing new problems. The goal is to find a way to safely correct hemophilia A early in life, potentially avoiding complications seen in adult treatments.
Why it matters
Potential benefit: If successful, this work could lead to a safer, more effective, and potentially lifelong gene therapy for hemophilia A, possibly even for newborns or before birth.
How similar studies have gone: While gene therapy for hemophilia A has shown promise in clinical trials, this project addresses known challenges like immune responses and potential side effects to improve upon existing approaches.
Where this research is happening
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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.
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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.