Hemophilia A: studying why some people develop inhibitors to FVIII across families
Official title: A Severe Hemophilia A Intergenerational Cohort Research Program for the Study of Factor VIII Immunogenicity
Funded at University of Washington · NIH-11405075 · Data updated 2026-11-12
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.
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, 2029 |
| Funding year | 2026 |
| Award (this year) | $2,841,864 |
| NIH project number | 5UH3HL165064-05 |
| Project ID | NIH-11405075 on NIH RePORTER |
Who could benefit from this research
✓Could help
Families with a child who has severe hemophilia A and potential mother–baby pairs willing to participate in a longitudinal study.
✕Less likely to help
Families without access to or interest in sharing long-term data and samples may not directly benefit.
What this research studies
We will build a long-term, intergenerational cohort starting before birth and continuing through early childhood to track immune responses to factor VIII in children with severe hemophilia A. The program will organize data and biospecimens, support biostatistics, and create a publicly accessible resource portal (F8portal.org) with well-annotated samples for researchers. Researchers will develop protocols across maternal, perinatal, and neonatal/pediatric immunology and multi-omics studies to understand FVIII immunogenicity and inhibitor development. Families participating could contribute genetic, clinical, and biological information, fostering advances in precision medicine for hemophilia A.
Why it matters
Potential benefit: If successful, this work could lead to personalized strategies to prevent or predict inhibitor formation in children with severe hemophilia A.
How similar studies have gone: Previous research shows immunogenic responses can vary; this program aims to build a comprehensive intergenerational resource to advance understanding beyond prior studies.
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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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.
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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.