Developing a treatment for GM1 gangliosidosis
Official title: Title: Development and Validation of Therapy for GM1 Gangliosidosis
Funded at Tega Therapeutics, INC. · NIH-10678309 · Data updated 2026-11-12
This study is looking at a new way to help people with GM1 gangliosidosis by testing a special treatment that delivers a missing enzyme directly to the brain, hoping to make life better for those dealing with the challenges of this condition.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Tega Therapeutics, INC. NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project start | September 15, 2023 |
| Project end | August 31, 2025 |
| Funding year | 2023 |
| Award (this year) | $461,701 |
| NIH project number | 1R43NS129414-01A1 |
| Project ID | NIH-10678309 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adults diagnosed with GM1 gangliosidosis who are experiencing neurological symptoms.
✕Less likely to help
Patients with other types of lysosomal storage diseases unrelated to GM1 gangliosidosis may not benefit from this research.
What this research studies
This research focuses on GM1 gangliosidosis, a lysosomal storage disease caused by the deficiency of the enzyme beta-galactosidase, leading to harmful sugar accumulation in the brain. The approach involves enzyme replacement therapy (ERT), which aims to restore the missing enzyme through injections. While intravenous ERT has shown benefits for similar disorders, this research explores intracerebroventricular (ICV) delivery methods to target the central nervous system more effectively. The goal is to improve patient quality of life by addressing neurological complications associated with the disease.
Why it matters
Potential benefit: If successful, this research could provide a new treatment option that significantly improves the quality of life for patients with GM1 gangliosidosis.
How similar studies have gone: Previous research has successfully utilized enzyme replacement therapies for related lysosomal disorders, indicating potential for success in this approach.
Where this research is happening
-
Tega Therapeutics, INC.
La Jolla, United States Active
Researchers
- Principal investigator: Brown, Jillian R — Tega Therapeutics, INC.
- Study coordinator: Brown, Jillian R
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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.