Creating a universal gene therapy for hemophilia A and B
Official title: Development of a universal gene therapy for hemophilia A or B with or without inhibitors
Funded at Geneventiv Therapeutics, INC. · NIH-11073122 · Data updated 2026-11-12
This study is testing a new gene therapy that could help people with hemophilia A and B by providing a long-lasting treatment, so they won’t need to get regular infusions of clotting factors anymore, especially for those who have trouble with traditional treatments.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Geneventiv Therapeutics, INC. NIH-funded |
| Lab location | 1 site (Raleigh, United States) |
| Project start | April 10, 2024 |
| Project end | March 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $1,266,694 |
| NIH project number | 5R44HL174236-02 |
| Project ID | NIH-11073122 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with hemophilia A or B, particularly those who have developed inhibitors to traditional treatments.
✕Less likely to help
Patients with bleeding disorders not classified as hemophilia A or B may not benefit from this research.
What this research studies
This research focuses on developing a universal gene therapy aimed at treating hemophilia A and B, which are inherited bleeding disorders caused by deficiencies in specific clotting factors. The approach involves using gene therapy to provide a long-lasting solution, potentially eliminating the need for frequent infusions of clotting factors. The study addresses the challenge of inhibitors, which are antibodies that some patients develop against traditional treatments, making them less effective. By targeting both hemophilia types, this therapy aims to improve patient outcomes significantly.
Why it matters
Potential benefit: If successful, this research could provide a long-term treatment option for hemophilia patients, reducing the frequency of bleeding episodes and the need for infusions.
How similar studies have gone: While gene therapies for hemophilia A have shown some promise, this specific approach targeting both hemophilia types and addressing inhibitors is novel and has not yet been tested.
Where this research is happening
-
Geneventiv Therapeutics, INC.
Raleigh, United States Active
Researchers
- Principal investigator: Margaritis, Paris — Geneventiv Therapeutics, INC.
- Study coordinator: Margaritis, Paris
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.
-
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.