Creating a universal gene therapy for hemophilia A and B
Development of a universal gene therapy for hemophilia A or B with or without inhibitors
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 ID | NIH-11073122 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals diagnosed with hemophilia A or B, particularly those who have developed inhibitors to traditional treatments.
Not a fit: Patients with bleeding disorders not classified as hemophilia A or B may not benefit from this research.
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 performed: 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
Raleigh, United States
- Geneventiv Therapeutics, INC. — Raleigh, United States (Active)
Researchers
- Principal investigator: Margaritis, Paris — Geneventiv Therapeutics, INC.
- Study coordinator: Margaritis, Paris
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 below.