Testing a new gene therapy to improve blood clotting in patients with severe hemophilia A.
Official title: A Phase I Clinical Trial Testing Feasibility of Hematopoietic Stem Cell Gene Therapy Using Platelet Factor VIII to Safely Improve Hemostasis for Severe Hemophilia A with Inhibitory Antibodies
Funded at Medical College of Wisconsin · NIH-10599276 · Data updated 2026-11-12
This study is testing a new gene therapy for people with severe hemophilia A to see if it can help their bodies make a key protein that helps stop bleeding, which could mean fewer trips for treatments and less hassle with current therapies.
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 | April 5, 2019 |
| Project end | March 31, 2026 |
| Funding year | 2023 |
| Award (this year) | $1,577,015 |
| NIH project number | 5R01HL142791-05 |
| Project ID | NIH-10599276 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with severe hemophilia A who have developed inhibitory antibodies against standard treatments.
✕Less likely to help
Patients with mild hemophilia or those who do not have inhibitory antibodies may not benefit from this research.
What this research studies
This research investigates a novel gene therapy approach aimed at treating severe hemophilia A, a condition characterized by a significant deficiency in a crucial blood clotting factor. The therapy involves using hematopoietic stem cells to deliver platelet factor VIII, which could potentially enhance the body's ability to stop bleeding. Patients will be monitored for safety and effectiveness as they receive this treatment, which may reduce the need for frequent infusions of traditional clotting factor therapies. The study aims to address challenges faced by patients, such as the development of inhibitory antibodies that complicate treatment.
Why it matters
Potential benefit: If successful, this research could significantly improve the quality of life for patients with severe hemophilia A by reducing bleeding episodes and the need for frequent treatments.
How similar studies have gone: Previous research has shown success in using gene therapy for hemophilia, indicating potential for this novel approach to also yield positive outcomes.
Where this research is happening
-
Medical College of Wisconsin
Milwaukee, United States Active
Researchers
- Principal investigator: Wilcox, David Allen — Medical College of Wisconsin
- Study coordinator: Wilcox, David Allen
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.
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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.