Intergenerational Hemophilia A Program to Understand Factor VIII Immune Responses
Official title: A Severe Hemophilia A Intergenerational Cohort Research Program for the Study of Factor VIII Immunogenicity
Funded at University of Washington · NIH-11376434 · Data updated 2026-11-12
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.
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 | 2025 |
| Award (this year) | $2,849,004 |
| NIH project number | 4UH3HL165064-04 |
| Project ID | NIH-11376434 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are pregnant women who are known genetic carriers of hemophilia A and their babies who are diagnosed with or expected to have severe hemophilia A, especially during pregnancy and the first two years of life.
✕Less likely to help
People without a hemophilia A carrier pregnancy or babies/children who do not have severe hemophilia A (for example those with mild or moderate disease or unrelated conditions) are unlikely to receive direct benefit from participating.
What this research studies
If you join, you and your baby would be followed from pregnancy through at least the first two years of life as part of a mother-baby paired cohort. The program will collect medical information and biological samples like blood to look for genetic markers and immune signals linked to development of inhibitors against factor VIII. Samples and de-identified data will be stored in a shared portal and biorepository so other researchers can use them to study why immune responses happen. The project coordinates protocols across clinical centers to focus on antenatal, perinatal, neonatal, and event-driven pediatric research.
Why it matters
Potential benefit: If successful, this work could identify early markers or causes of factor VIII inhibitors and help guide ways to prevent or reduce harmful immune reactions in children with severe hemophilia A.
How similar studies have gone: Previous studies have identified some genetic and immune risk factors for factor VIII inhibitors, but this intergenerational, longitudinal mother-to-infant cohort approach is relatively new.
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.
-
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.
-
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.
-
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.
-
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.
-
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.