Request Demo

Last update 08 May 2025

GM2A

Last update 08 May 2025

Basic Info

Synonyms

Cerebroside sulfate activator protein, Ganglioside GM2 activator, Ganglioside GM2 activator isoform short

+ [6]

Introduction

The large binding pocket can accommodate several single chain phospholipids and fatty acids, GM2A also exhibits some calcium-independent phospholipase activity (By similarity). Binds gangliosides and stimulates ganglioside GM2 degradation. It stimulates only the breakdown of ganglioside GM2 and glycolipid GA2 by beta-hexosaminidase A. It extracts single GM2 molecules from membranes and presents them in soluble form to beta-hexosaminidase A for cleavage of N-acetyl-D-galactosamine and conversion to GM3 (By similarity). Has cholesterol transfer activity (PubMed:17552909).

Drugs associated with GM2A

AL-801

Target

GM2A

Mechanism

GM2A inhibitors

Active Org.

Alps Biotech Co., LTD

Originator Org.

Alps Biotech Co., LTD

Active Indication

Lung Cancer

Inactive Indication-

Drug Highest PhasePreclinical

First Approval Ctry. / Loc.-

First Approval Date20 Jan 1800

100 Clinical Results associated with GM2A

100 Translational Medicine associated with GM2A

0 Patents (Medical) associated with GM2A

319

Literatures (Medical) associated with GM2A

01 Jun 2025·Molecular Immunology

Steap3 is a key node in regulating the phagosome escape of Listeria monocytogenes

Article

Author: Liu, Xiao ; Yuan, Jiangbei ; Shao, Yan ; Wan, Xiaoyuan ; Xiong, Pei ; Wang, Liting ; Li, Zhangfu

Listeria monocytogenes, a foodborne pathogen, poses a significant threat to human health due to its high mortality rate and increasing antibiotic resistance. This study investigates the role of Steap3 in regulating the early phagosomal escape of Listeria monocytogenes. We found that Steap3 expression is downregulated in dendritic and intestinal epithelial cells following infection, and its deficiency exacerbates bacterial proliferation both in vitro and in vivo. Mechanistically, Steap3 interacts with Gm2a and Sting to inhibit Listeria monocytogenes infection. Our results highlight Steap3 as a key regulator in dendritic and intestinal epithelial cells' defense against Listeria monocytogenes infection, suggesting the Steap3-STING/Gm2a axis is a potential therapeutic target for listeriosis. This study provides valuable insights into the molecular mechanisms underlying Listeria monocytogenes pathogenesis and host immune response, offering new directions for developing anti-Listeria monocytogenes therapies.

01 Mar 2025·Clinical and Translational Science

Evaluation of the Landscape of Pharmacodynamic Biomarkers in GM1 and GM2 Gangliosidosis

Review

Author: Schuck, Robert ; Crisamore, Karryn ; Pacanowski, Michael ; Stern, Sydney ; Li, Ruo‐Jing

ABSTRACTGM1 and GM2 gangliosidosis are inherited, progressive, neurodegenerative lysosomal disorders of variable onset and disease progression. GM1 gangliosidosis is a result of biallelic pathogenic variants in the GLB1 gene, which confer absent or reduced β‐galactosidase enzyme activity and lead to the accumulation of glycoconjugates such as glycosphingolipid GM1‐gangliosides. GM2 is caused by biallelic pathogenic variants in one of the three genes (HEXA, HEXB, and GM2A) which confer deficiency of β‐hexosaminidase or the GM2 ganglioside activator protein, responsible for the catabolism of GM2 gangliosides. In both gangliosidoses, glycosphingolipids accumulate primarily in neurons, with subsequent neuronal death, which translates to early mortality for patients. The clinical course is commonly differentiated by age of symptom onset. To date, no disease‐modifying therapy has been approved globally, and treatment is typically supportive. The lack of mature biomarker development in these diseases contributes to challenges associated with quantifying treatment response. However, recent advancements in the detection of neurodegenerative biomarkers and treatment innovation have spurred interest in biomarker identification in plasma and cerebrospinal fluid in patients with GM1 and GM2 gangliosidosis as pharmacodynamic endpoints to support clinical trials and regulatory decision‐making. In this review, we assess the landscape of lipid and protein biomarkers, the extent of evidence, and propose considerations for future biomarker development to measure treatment response and support drug development in GM1 and GM2 gangliosidosis.

01 Jan 2025·Molecular Genetics and Metabolism

Clinical and biochemical abnormalities in a feline model of GM2 activator deficiency

Article

Author: Kell, Pamela ; Maguire, Anne S ; Martin, Douglas R ; Beecy, Sidney J ; Gray-Edwards, Heather L ; Gross, Amanda L ; Cuddon, Paul ; Diffie, Elise B ; Jiang, Xuntian ; Hoffman, Leah M K

Though it has no catalytic activity toward GM2 ganglioside, the GM2 activator protein (GM2A) is essential for ganglioside hydrolysis by facilitating the action of lysosomal ß-N-acetylhexosaminidase. GM2A deficiency results in death in early childhood due to rapid central nervous system deterioration similar to the related GM2 gangliosidoses, Tay-Sachs disease and Sandhoff disease. This manuscript further characterizes a feline model of GM2A deficiency with a focus on clinical and biochemical parameters that may be useful as benchmarks for translational therapeutic research. The GM2A deficient cat has clinical features consistent with the human condition, including isointensity of gray and white matter of the brain on T2-weighted MRI; MR spectroscopic changes of brain metabolites consistent with gliosis, neuronal injury and demyelination; rhythmical slowing of cerebral cortical activation on electroencephalography; and elevation of aspartate aminotransferase and lactate dehydrogenase in cerebrospinal fluid. Biochemically, the brain of GM2A deficient cats has storage of GM2 and GA2 ganglioside coincident with increased hexosaminidase activity toward a standard synthetic substrate. Also, the brain of GM2A deficient cats has increased levels of lyso-platelet activating factor and lyso-phosphatidylcholine, which may serve as novel biomarkers of disease progression and provide insights into pathogenic mechanisms.

Analysis

Perform a panoramic analysis of this field.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis

GM2A

Basic Info

Related

Analysis