Investigating immune responses in severe hemophilia A across generations
Official title: A Severe Hemophilia A Intergenerational Cohort Research Program for the Study of Factor VIII Immunogenicity
Funded at University of Washington · NIH-10916298 · Data updated 2026-11-12
This study is looking for mothers and their babies with severe hemophilia A to learn how babies' immune systems respond to a treatment called Factor VIII during their first two years of life, so we can better understand and help manage this condition.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Washington NIH-funded |
| Lab location | 1 site (Seattle, United States) |
| Project start | September 21, 2022 |
| Project end | August 31, 2025 |
| Funding year | 2024 |
| Award (this year) | $2,465,319 |
| NIH project number | 5UG3HL165064-03 |
| Project ID | NIH-10916298 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include mothers who are genetic carriers of severe hemophilia A and their infants diagnosed with the condition.
✕Less likely to help
Patients who do not have severe hemophilia A or are not part of a mother-baby pair may not benefit from this research.
What this research studies
This research program aims to create a cohort of mother-baby pairs to study how immune responses to Factor VIII develop in infants with severe hemophilia A. By following these pairs over the first two years of life, the program will collect valuable data and biological samples to understand the factors that influence the development of FVIII inhibitors. The research will also establish a resource portal to share findings and samples with the broader research community, enhancing collaboration and knowledge in this area.
Why it matters
Potential benefit: If successful, this research could lead to improved understanding and management of immune responses in hemophilia A, potentially reducing complications for affected patients.
How similar studies have gone: Previous research has shown promise in studying immune responses in hemophilia, but this specific intergenerational approach is relatively novel.
Where this research is happening
-
University of Washington
Seattle, United States Active
Researchers
- Principal investigator: Johnsen, Jill Marie — University of Washington
- Study coordinator: Johnsen, Jill Marie
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.