Platelet-targeted FVIII Gene Therapy for Hemophilia A
Official title: Platelet-derived FVIII Gene Therapy of Hemophilia A
Funded at Medical College of Wisconsin · NIH-11377676 · Data updated 2026-11-12
A gene therapy approach aims to make platelets carry FVIII to help people with hemophilia A, even if they have inhibitors against FVIII.
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 | 2026 |
| Award (this year) | $561,352 |
| NIH project number | 5R01HL102035-16 |
| Project ID | NIH-11377676 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with hemophilia A, including those with FVIII inhibitors, who could benefit from platelet-targeted FVIII gene therapy.
✕Less likely to help
Individuals without hemophilia A or those unwilling to undergo gene therapy procedures may not benefit.
What this research studies
This project focuses on delivering the FVIII gene specifically to blood platelets using a lentiviral system and a platelet-specific promoter, then transplanting these modified cells. The goal is for platelets to release FVIII at sites of injury, potentially avoiding immune inhibitors. The work builds on prior lab studies showing platelet-FVIII expression can be sustained and promote immune tolerance. The research will explore safety and effectiveness in a translational pathway toward patients who need durable, inhibitor-tolerant FVIII activity.
Why it matters
Potential benefit: If successful, this approach could provide a long-lasting, inhibitor-tolerant source of FVIII and reduce bleeding risks for adults with hemophilia A.
How similar studies have gone: Previous animal studies showed sustained platelet-FVIII expression and immune tolerance; human translation remains exploratory.
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.
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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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Intergenerational Hemophilia A Program to Understand Factor VIII Immune Responses
This program follows mothers who carry hemophilia A and their babies with severe hemophilia to learn how immune reactions to factor VIII develop in early life.
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.