cGMP production of FVIII-making placental cells to help people with Hemophilia A
Official title: cGMP Manufacture Of FVIII-Expressing Placental Cells For Hemophilia A
Funded at Wake Forest University Health Sciences · NIH-11443970 · Data updated 2026-11-12
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.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Wake Forest University Health Sciences NIH-funded |
| Lab location | 1 site (Winston-Salem, United States) |
| Project start | September 1, 2026 |
| Project end | June 30, 2028 |
| Funding year | 2026 |
| Award (this year) | $816,782 |
| NIH project number | 2U01HL148681-03 |
| Project ID | NIH-11443970 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with Hemophilia A who have severe FVIII deficiency and may benefit from FVIII-producing cell therapies.
✕Less likely to help
People without Hemophilia A or with conditions not related to FVIII deficiency are unlikely to benefit.
What this research studies
This project focuses on creating placental cells that can produce FVIII under good manufacturing practices, with the goal of providing a durable therapy for Hemophilia A. Researchers will explore a cell+gene therapy approach that could bypass some immune barriers seen in adults and reduce the need for lifelong expensive treatments. The work involves early development steps, safety considerations, and strategies to scale up production for potential clinical use. If successful, this could lead to a new option for people with severe Hemophilia A who struggle with current therapies.
Why it matters
Potential benefit: If successful, patients could gain a longer-lasting, potentially more affordable treatment that reduces bleeding episodes and improves quality of life.
How similar studies have gone: Earlier liver-targeted FVIII gene therapies showed promise but had safety and durability concerns, making this approach exploratory but grounded in prior findings.
Where this research is happening
-
Wake Forest University Health Sciences
Winston-Salem, United States Active
Researchers
- Principal investigator: Almeida-Porada, Graca Duarte — Wake Forest University Health Sciences
- Study coordinator: Almeida-Porada, Graca Duarte
Related research
Other NIH-funded projects on the same topics.
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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.
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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.