New strategies to prevent bleeding in people with hemophilia A
Official title: Targeted, adaptive and time-variant strategies for bleed prevention across the lifespan for persons with hemophilia A
Funded at Yale University · NIH-10949401 · Data updated 2026-11-12
This study is looking for better ways to help kids with hemophilia A avoid bleeding problems by exploring different treatment options, including new medicines and gene therapy, so they can find the safest and most effective choices for their needs.
Quick facts
| Grant type | Career grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Yale University NIH-funded |
| Lab location | 1 site (New Haven, United States) |
| Project start | September 17, 2024 |
| Project end | September 30, 2029 |
| Funding year | 2024 |
| Award (this year) | $173,448 |
| NIH project number | 1K01HL175220-01 |
| Project ID | NIH-10949401 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include children and young individuals diagnosed with hemophilia A.
✕Less likely to help
Patients with other bleeding disorders or those who do not have hemophilia A may not benefit from this research.
What this research studies
This research focuses on developing and evaluating new strategies to reduce bleeding risks for individuals with hemophilia A, particularly in children. It aims to create a transparent and independent health economic model that assesses various treatment options, including standard and extended half-life factor products, non-factor substitutes, and gene therapy. By analyzing these options, the research seeks to provide valuable insights into the most effective and equitable treatment strategies for different subpopulations. The project will involve collaboration with experts in health decision science, policy, and hematology.
Why it matters
Potential benefit: If successful, this research could lead to improved treatment options and better health outcomes for patients with hemophilia A.
How similar studies have gone: Previous research has shown promise in developing treatment strategies for hemophilia A, but this approach aims to create a novel, independent economic model.
Where this research is happening
-
Yale University
New Haven, United States Active
Researchers
- Principal investigator: Goshua, George — Yale University
- Study coordinator: Goshua, George
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.