A New Way to Deliver Factor VIII for Hemophilia A
Official title: Platelet-derived FVIII Gene Therapy of Hemophilia A
Funded at Medical College of Wisconsin · NIH-11128549 · Data updated 2026-11-12
This research explores a new gene therapy approach for hemophilia A that uses platelets to deliver Factor VIII, especially for patients who develop antibodies against current treatments.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Medical College of Wisconsin NIH-funded |
| Lab location | 1 site (Milwaukee, United States) |
| Project start | July 1, 2010 |
| Project end | June 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $572,808 |
| NIH project number | 5R01HL102035-15 |
| Project ID | NIH-11128549 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for future applications of this research would be individuals with hemophilia A, especially those who have developed inhibitory antibodies against Factor VIII.
✕Less likely to help
Patients without hemophilia A or those who do not develop inhibitory antibodies may not directly benefit from this specific gene therapy approach.
What this research studies
For people with hemophilia A, especially those who develop antibodies to standard Factor VIII treatments, this research offers a new hope. We are working on a gene therapy that teaches your body's platelets to make Factor VIII right where it's needed. This method aims to overcome the problem of your immune system attacking the Factor VIII, potentially helping your body accept the new protein. The goal is to provide a long-lasting and effective way to manage hemophilia A.
Why it matters
Potential benefit: If successful, this approach could offer a safe and effective long-term treatment for hemophilia A, particularly for patients who currently struggle with inhibitory antibodies.
How similar studies have gone: Previous studies in animal models have shown promising results, demonstrating sustained Factor VIII expression and immune tolerance using this platelet-targeted gene delivery method.
Where this research is happening
-
Medical College of Wisconsin
Milwaukee, United States Active
Researchers
- Principal investigator: Shi, Qizhen — Medical College of Wisconsin
- Study coordinator: Shi, Qizhen
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.
-
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.
-
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.