Official websites use .gov

A .gov website belongs to an official government organization in the United States.

Secure .gov websites use HTTPS

A lock ( 🔒 ) or https:// means you've safely connected to the .gov website. Share sensitive information only on official, secure websites.

Need Help

Title: Development and Validation of Therapy for GM1 Gangliosidosis

Award Number: R43NS129414
ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 09/15/2023
PERIOD OF PERFORMANCE END DATE: 08/31/2025

Group Awards By:

View Award Description

Title: Development and Validation of Therapy for GM1 Gangliosidosis - PROJECT SUMMARY GM1 gangliosidosis is lysosomal storage disease in which a key hydrolase enzyme, known as beta- galactosidase is missing in the lysosome resulting in the toxic accumulation of complex sugars called gangliosides, in particular GM1 and GA1 principally in the central nervous system. There is currently no cure or effective treatment available. A primary approach for treating related types of disorders involves replacement of the missing enzyme by injection into the circulation. Although intravenous enzyme replacement therapy (ERT) resolves many aspects of the disease, unfortunately it does not resolve complications of the disease in the CNS. Although, ERT is not a cure for this disease it can have a marked effect on the patient’s development and thus quality of life. Intravenous ERT has been successfully commercialized for lysosomal mucopolysaccharidoses (MPS) disorders with approved drugs on the market, including (i) Laronidase for MPS I (Aldurazyme®, alpha-iduronidase, Hurler Syndrome), (ii) Idursulfatase for MPS II (Elaprase®, iduronate-2- sulfatase, Hunter Syndrome) and (iii) Vestronidase alfa for MPS VII (Mepsevii™, beta-glucuronidase, Sly Syndrome). Intracerebroventricular (ICV) ERT has also been successfully commercialized for a progressive neurodegenerative lysosomal disease called Batten disease. Cerliponase Alfa (Brineura®, tripeptidyl peptidase-1) is a lysosomal enzyme delivered via ICV infusion directly to the brain to replace the deficient enzyme. This therapy is the first safe and effective ICV ERT approved for direct delivery to the brain. A Phase II clinical trial is also underway using a modified lysosomal enzyme ICV-delivered directly to the brain. Together, the FDA and investors are familiar with ERT and its commercialization path forward, both of which are essential in reaching a clinical trial. This proposal focuses on the development of the ICV route of administration to perform ERT directly to the central nervous system and its application to treating GM1 gangliosidosis. GM1 gangliosidosis has severe neurodegenerative symptoms with no current therapies available due to poor transport across the blood-brain barrier. In our studies, we will engineer cells to produce sufficient quantities of recombinant human beta-galactosidase enzyme for testing in GM1 gangliosidosis knockout mice. These mice will be ICV-administered enzyme and analyzed for dose-dependent biodistribution of the enzyme and effects on biochemical and histological pathology will be evaluated. Efficacy and safety will be assessed in single intermittent dose; once a week for 8 weeks dosing study. The results will provide the preclinical information needed to proceed towards a novel treatment of the disease in humans.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2026 ( Subtotal = $0 )
2026	2023	TEGA THERAPEUTICS INC	3550 GENERAL ATOMICS CT STE G02-102	SAN DIEGO	CA	92121	SAN DIEGO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	1	4/22/2026	NEW	$0
														Subtotal = $0

Issue Date FY: 2023 ( Subtotal = $461,701 )
2023	2023	TEGA THERAPEUTICS, INC.	3550 GENERAL ATOMICS CT STE G02-102	SAN DIEGO	CA	92121	SAN DIEGO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	1	9/13/2023	NEW	$461,701
														Subtotal = $461,701

Grand Total All Awards = $461,701

Top

All Categories

About

Search

Reports

Data Submission

Award Information

Title: Development and Validation of Therapy for GM1 Gangliosidosis

Award Number: R43NS129414

ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 09/15/2023

PERIOD OF PERFORMANCE END DATE: 08/31/2025

Federal Websites

Department of Health & Human Services

HHS Operating Divisions

HHS Staff Divisions

Download A Document Viewer