Targeting FVIII production to eye-opening blood vessels in hemophilia A
Official title: Endothelial cell targeting of factor VIII for hemophilia A
Funded at Children's Hosp of Philadelphia · NIH-11382316 · Data updated 2026-11-12
Aims to improve treatment for people with hemophilia A by directing how the body makes clotting factor VIII, using a targeted gene-delivery approach.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | August 1, 2025 |
| Project end | May 31, 2029 |
| Funding year | 2026 |
| Award (this year) | $857,161 |
| NIH project number | 5R01HL177014-02 |
| Project ID | NIH-11382316 on NIH RePORTER |
Who could benefit from this research
✓Could help
Individuals with hemophilia A or their caregivers who are interested in future gene therapy approaches and meet study-specific eligibility criteria.
✕Less likely to help
Patients not eligible for gene therapy or those with contraindications to viral vectors or genome editing may not benefit.
What this research studies
This project explores where the body should make factor VIII to best control bleeding in hemophilia A. By comparing endothelial cell targeting in the liver’s blood vessels with traditional liver-targeted approaches, researchers will test different gene delivery methods and genome editing tools. The study uses engineered AAV capsids and targeted genome editing to see which strategy yields stable, long-lasting FVIII production with fewer side effects. If successful, this could lead to more reliable, durable therapies for patients with hemophilia A.
Why it matters
Potential benefit: Potentially more durable and predictable treatment for hemophilia A by improving how and where factor VIII is produced in the body.
How similar studies have gone: Previous liver-targeted FVIII gene therapy has shown promise but with variable responses, making this endothelial-targeted approach a novel next step.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Sabatino, Denise E — Children's Hosp of Philadelphia
- Study coordinator: Sabatino, Denise E
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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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.