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
['FUNDING_SBIR_2'] · GENEVENTIV THERAPEUTICS, INC. · NIH-11181888
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
| Phase | ['FUNDING_SBIR_2'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | GENEVENTIV THERAPEUTICS, INC. (nih funded) |
| Locations | 1 site (RALEIGH, UNITED STATES) |
| Trial ID | NIH-11181888 on ClinicalTrials.gov |
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.
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 against standard treatments.
Not a fit: Patients with bleeding disorders other than hemophilia A or B may not benefit from this research.
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 performed: 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
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.
Conditions: bleeding disorder