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-11181888 · Data updated 2026-11-12
This study is testing a new gene therapy for people with hemophilia A and B to help their bodies make the clotting factors they need, aiming to provide a longer-lasting solution than the usual treatments, and it’s designed for both patients who have developed antibodies against these treatments and those who haven’t.
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) | $45,344 |
| NIH project number | 3R44HL174236-02S1 |
| Project ID | NIH-11181888 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 against standard treatments.
✕Less likely to help
Patients with bleeding disorders other than hemophilia A or B may not benefit from this research.
What this research studies
This research focuses on developing a gene therapy that aims to treat hemophilia A and B, which are inherited bleeding disorders caused by deficiencies in specific clotting factors. The approach involves using adeno-associated viruses to deliver the necessary genes to produce these factors, potentially offering a long-lasting solution compared to current treatments that require frequent infusions. The study also addresses the challenge of inhibitors, which are antibodies that some patients develop against clotting factors, making traditional therapies ineffective. By targeting both patients with and without inhibitors, this research seeks to improve treatment outcomes for a broader range of hemophilia patients.
Why it matters
Potential benefit: If successful, this research could provide a more effective and durable treatment option for hemophilia patients, reducing the need for frequent infusions and improving their quality of life.
How similar studies have gone: While gene therapies for hemophilia A have shown promise, this specific approach targeting both inhibitor and non-inhibitor patients is relatively novel and has not yet been tested in approved therapies.
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.
-
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.
-
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.