Testing a new enzyme therapy for Fabry disease
Official title: Preclinical Efficacy of Subcutaneous Enzyme Therapy for Lysosomal Fabry Disease
Funded at Kinetiq Therapeutics LLC · NIH-10919631 · Data updated 2026-11-12
This study is looking at a new way to give enzyme therapy for Fabry disease that involves a simple shot under the skin, making it easier and less painful for patients compared to regular IV treatments.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Kinetiq Therapeutics LLC NIH-funded |
| Lab location | 1 site (Fort Worth, United States) |
| Project start | July 1, 2024 |
| Project end | June 30, 2026 |
| Funding year | 2024 |
| Award (this year) | $295,923 |
| NIH project number | 1R43TR005111-01 |
| Project ID | NIH-10919631 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Fabry disease, including both children and adults.
✕Less likely to help
Patients who do not have Fabry disease or those who are not suitable for enzyme therapy may not benefit from this research.
What this research studies
This research investigates a new subcutaneous enzyme therapy for Fabry disease, a rare genetic disorder caused by a deficiency of the GLA enzyme. The goal is to develop a treatment that can be administered under the skin, reducing the need for frequent intravenous infusions that can be burdensome and painful for patients. By focusing on improving the delivery method of enzyme replacement therapy, the research aims to enhance patient quality of life and treatment adherence. The study will involve preclinical testing to evaluate the efficacy and safety of this new approach.
Why it matters
Potential benefit: If successful, this research could provide a more convenient and less invasive treatment option for patients with Fabry disease.
How similar studies have gone: While enzyme replacement therapies have been effective, this specific subcutaneous approach is novel and has not been widely tested in clinical settings.
Where this research is happening
-
Kinetiq Therapeutics LLC
Fort Worth, United States Active
Researchers
- Principal investigator: Cao, Mingju — Kinetiq Therapeutics LLC
- Study coordinator: Cao, Mingju
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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.