Title: new clinical biomarkers for GM1 gamgliosidosis
Active Project Grant Awarded Last action
- Award ID
- R43NS139721
- Recipient
-
Tega Therapeutics Inc. UEI C1LJLCS3PM51
- Awarding agency
-
National Institute of Neurological Disorders and Stroke
National Institutes of Health, Department of Health and Human Services
- Obligated
- $1,000,000
Award facts
- Assistance listing
- 93.853 Extramural Research Programs in the Neurosciences and Neurological Disorders
- Completion date 63% complete
- Originally
- Place of performance
- California, USA
Award details come from USAspending.gov.
Description
- Abstract
- GM1 gangliosidosis is a lysosomal storage disease in which the key hydrolase enzyme, beta-galactosidase is missing, resulting in toxic accumulation of gangliosides and other glycan metabolites, principally in the central nervous system. Gangliosides are glycolipids while the glycan metabolites are derived from oligosaccharides both accumulate due to the lack of the enzyme. Identifying these diverse glycan substrates is essential for understanding the neurodegenerative progression of the disease. Research has shown that these metabolites accumulate in urine and cerebral spinal fluid (CSF) and in tissues in a mouse model. This was also observed in patients so these metabolites could potentially be used as biomarkers. To facilitate use of these metabolites as non-invasive biomarkers, levels in urine and/or CSF should correspond to the levels in tissues. Previous enzyme replacement therapy studies showed promising results in a GM1 gangliosidosis mouse model. Administering beta-galactosidase enzyme directly to the brain reduced GM1 ganglioside levels in the brain and systemically in the liver. Here, we propose using this mouse model to confirm whether the levels of the metabolites in urine and/or CSF reflect the changes observed in tissues following therapy. With success, these metabolites would serve as clinical biomarkers that would be valuable for monitoring therapy in clinical trials and diagnosing newborn patients.
Transactions
| Action | Obligated | Date |
|---|---|---|
| Amendment | $0 | |
| Amendment | $0 | |
| Amendment | $0 | |
| Amendment | $500,000 | |
| Original award | $500,000 |
Quick answers
Who received award R43NS139721?
R43NS139721 was awarded to Tega Therapeutics Inc. by National Institute of Neurological Disorders and Stroke (National Institutes of Health) on August 5, 2025.
How much is award R43NS139721 worth?
$1,000,000 has been obligated on R43NS139721.
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