Improving gene therapy delivery to the liver using a special catheter
Official title: Enhanced Gene Therapy Delivery Through Electric Pulsing Double-Balloon Catheter
Funded at Vector Surgical, LLC · NIH-10821764 · Data updated 2026-11-12
This study is testing a new way to deliver gene therapy directly to the liver using a special catheter that uses gentle electrical pulses, which could make treatments for liver diseases safer and more effective for patients.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Vector Surgical, LLC NIH-funded |
| Lab location | 1 site (Waukesha, United States) |
| Project start | January 15, 2024 |
| Project end | January 14, 2026 |
| Funding year | 2024 |
| Award (this year) | $331,034 |
| NIH project number | 1R43TR004536-01A1 |
| Project ID | NIH-10821764 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals suffering from specific genetic liver disorders that could benefit from gene therapy.
✕Less likely to help
Patients with liver diseases not caused by monogenic factors or those who do not respond to gene therapy may not benefit from this research.
What this research studies
This research focuses on developing a novel Gene Therapy Catheter (GTC) that uses electrical pulsing to enhance the delivery of adeno-associated virus (AAV) gene therapy directly to the liver. By isolating the treatment area with double balloons and flushing out neutralizing antibodies, the GTC aims to improve the efficiency and safety of gene therapy for liver diseases. This innovative approach could potentially reduce the required dosage of the viral vector, minimizing the risk of immune responses and lowering treatment costs. The research will explore how this method can effectively treat various monogenic liver diseases.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective gene therapies for patients with monogenic liver diseases.
How similar studies have gone: Other research has shown promise in using adeno-associated viruses for gene therapy, but this specific approach with the Gene Therapy Catheter is novel.
Where this research is happening
-
Vector Surgical, LLC
Waukesha, United States Active
Researchers
- Principal investigator: Phillips, Janet — Vector Surgical, LLC
- Study coordinator: Phillips, Janet
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.