Correcting hereditary tyrosinemia type 1 and other liver diseases using gene therapy.
Official title: Lifelong Correction of Hereditary Tyrosinemia Type 1 and Other Monogenic Liver Diseases using Liver-specific Hydrodynamic Gene Delivery through the Biliary System
Funded at Hydrogene Therapeutics INC · NIH-11005449 · Data updated 2026-11-12
This study is testing a new way to treat hereditary tyrosinemia type 1 (HT1) by using a special gene therapy that delivers healthy genes directly to the liver, which could help patients avoid liver transplants and long-term medications.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Hydrogene Therapeutics INC NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project start | September 15, 2024 |
| Project end | June 30, 2025 |
| Funding year | 2024 |
| Award (this year) | $293,987 |
| NIH project number | 1R42DK141297-01 |
| Project ID | NIH-11005449 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with hereditary tyrosinemia type 1 or other similar monogenic liver diseases.
✕Less likely to help
Patients with liver diseases not caused by genetic defects or those who do not have hereditary tyrosinemia type 1 may not benefit from this research.
What this research studies
This research focuses on developing a new gene therapy approach to treat hereditary tyrosinemia type 1 (HT1) and other monogenic liver diseases. It utilizes a non-viral method to deliver functional genes directly to liver cells through the biliary system, aiming to replace the need for liver transplants and long-term medication. The therapy seeks to address the underlying genetic defect that causes HT1, potentially providing a lifelong solution for affected patients. By improving the delivery method, this research aims to enhance the safety and effectiveness of gene therapy for liver diseases.
Why it matters
Potential benefit: If successful, this research could provide a safe and effective lifelong treatment option for patients with hereditary tyrosinemia type 1, reducing the need for liver transplants and long-term medication.
How similar studies have gone: While gene therapy for liver diseases is an emerging field, this specific approach using hydrodynamic delivery via the biliary system is novel and has not been extensively tested in clinical settings.
Where this research is happening
-
Hydrogene Therapeutics INC
Houston, United States Active
Researchers
- Principal investigator: Kumbhari, Vivek — Hydrogene Therapeutics INC
- Study coordinator: Kumbhari, Vivek
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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.