Hydrodynamic gene delivery through the bile ducts for Wilson’s disease
Official title: Developing Hydrodynamic Injection through Biliary System for the Treatment of Wilson's Disease
Funded at Hydrogene Therapeutics INC · NIH-11405804 · Data updated 2026-11-12
Aimed at delivering a functional ATP7B gene variant to the liver using a special bile-duct delivery method to help people with Wilson’s disease avoid liver transplant.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Hydrogene Therapeutics INC NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project start | September 11, 2026 |
| Project end | June 30, 2028 |
| Funding year | 2026 |
| Award (this year) | $1,097,036 |
| NIH project number | 1R44DK148200-01 |
| Project ID | NIH-11405804 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults and adolescents with Wilson’s disease who require improved liver copper handling and could benefit from liver-directed gene therapy.
✕Less likely to help
Patients without Wilson’s disease or those unable to receive AAV-based or liver-directed therapies may not benefit.
What this research studies
This project explores a liver-directed, non-viral gene therapy approach to fix the faulty ATP7B gene in Wilson’s disease. It uses a hydrodynamic injection technique through the biliary system to deliver DNA directly to liver cells, with the goal of a repeatable treatment that can grow with pediatric patients. The work focuses on making redosing safe and feasible to sustain copper regulation over time, possibly reducing reliance on liver transplants. The grant supports preclinical and early development activities led by HydroGene Therapeutics, Inc. in Houston, under an NIH R44 award.
Why it matters
Potential benefit: If successful, patients could achieve lasting control of copper metabolism without liver transplantation or repeated invasive procedures.
How similar studies have gone: Previous AAV-based approaches showed partial improvements in WD but faced immune barriers to redosing, making this new approach potentially more durable.
Where this research is happening
-
Hydrogene Therapeutics INC
Houston, United States Active
Researchers
- Principal investigator: Kruse, Robert — Hydrogene Therapeutics INC
- Study coordinator: Kruse, Robert
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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.