Non-viral gene therapy approach to permanently fix hemophilia B
Official title: Non-viral hydrodynamic gene therapy for life-long correction of hemophilia
Funded at Hydrogene Therapeutics INC · NIH-11406850 · Data updated 2026-11-12
A new gene therapy approach aims to permanently correct hemophilia B without using viruses, potentially reducing bleeding events for people with this condition.
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 10, 2026 |
| Project end | August 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $1,027,445 |
| NIH project number | 1R44HL187020-01 |
| Project ID | NIH-11406850 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with hemophilia B seeking longer-lasting treatment options and who may have limitations with current AAV-based therapies.
✕Less likely to help
Patients unable to participate due to age, liver conditions, or contraindications to gene therapy modalities may not benefit.
What this research studies
This project looks for a long-lasting fix for hemophilia B by delivering a corrective gene without using viral vectors. Researchers plan to use non-viral methods to introduce the needed gene changes into liver cells, where the blood clotting factor IX is produced. The aim is to achieve stable FIX levels that reduce or replace daily or weekly treatments, improving quality of life for patients. The work will focus on safety, delivery efficiency, and sustained expression in a preclinical or early-stage setting.
Why it matters
Potential benefit: If successful, patients could require fewer or no regular FIX injections and have better control over bleeding episodes.
How similar studies have gone: Previous approaches using non-viral methods have shown promise in delivering therapeutic genes, but substantial work remains to demonstrate durability and safety in humans.
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.