Target intelligence / Profile preview

GM2 ganglioside (GM2) (GM2)

Target
GM2
Molecular classification
Ganglioside, Glycosphingolipid, Tumor-associated carbohydrate antigen
01

Overview

GM2 ganglioside is a sialic acid-containing glycosphingolipid primarily located on the outer leaflet of the plasma membrane (Livingston, 1995 [1]). While its expression in normal adult tissues is largely restricted to the central nervous system, it is significantly overexpressed on the surface of various neuroectoderm-derived tumors, such as melanoma, neuroblastoma, and sarcomas (Ragupathi, 1996 [2]). This differential expression pattern identifies GM2 as a prominent tumor-associated carbohydrate antigen (TACA) for targeted cancer immunotherapy (Zhang et al., 1997 [3]). In the context of malignancy, GM2 is involved in modulating cell signaling pathways and enhancing cell-to-cell adhesion, which facilitates tumor progression (Hakomori, 2001 [4]). Therapeutic approaches targeting GM2 include the use of conjugate vaccines like GM2-KLH to induce endogenous antibody production and monoclonal antibodies like BIW-8962 to mediate antibody-dependent cellular cytotoxicity (Chapman et al., 2000 [5]). Beyond oncology, GM2 is the primary storage material in Tay-Sachs disease, where genetic mutations in the HEXA gene lead to its pathological accumulation in neurons (Mahuran, 1999 [6]).

Other names
Monosialotetraosylganglioside GM2GalNAc-GM3II3Neu5Ac-Gg3CerGanglioside GM2Tumor-associated carbohydrate antigen GM2
02

Mechanism of action

Induction of humoral immune response and mediation of antibody-dependent cellular cytotoxicity (ADCC) and complement-dependent cytotoxicity (CDC) (Chapman et al., 2000 [5]).

03

Biological functions

Cell-cell recognitionSignal transductionCell adhesionModulation of growth factor receptors
04

Disease associations

MelanomaNeuroblastomaSarcomaSmall cell lung cancerTay-Sachs disease
05

Safety considerations

Potential neurotoxicity due to CNS expressionLow immunogenicity of carbohydrate antigensRisk of autoimmune cross-reactivity
06

Interacting drugs

GM2-KLH vaccine

2 more in the full profile.

07

Biomarkers

GM2 surface expressionAnti-GM2 antibody titer

Beyond the preview

Go deeper on GM2 ganglioside (GM2) (GM2).

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Drug pipeline

Full profile access

Explore the programs pursuing this target and their development progress.

  • Drug candidates
  • Developers
  • Development stage

Clinical trials

Full profile access

Follow the clinical studies evaluating therapies directed at this target.

  • Trial design
  • Status
  • Readouts

Competitive landscape

Full profile access

Compare approaches across drug candidates, modalities, and indications.

  • Programs
  • Modalities
  • Indications

Literature & evidence

Full profile access

Investigate the research and source evidence behind target biology and development.

  • Publications
  • Sources
  • Analysis

Patents

Full profile access

Explore patent activity around therapies and technologies addressing this target.

  • Patents
  • Assignees
  • Technologies

Research & analysis

Full profile access

Connect target biology, drug development, and emerging evidence in your research.

  • Biology
  • Development news
  • Analysis

Bring the full picture into focus.

See how Gosset can support your research on GM2 ganglioside (GM2) (GM2).

Explore the full profile

Gosset Free

Get started with Gosset.

Enter your work email and we’ll be in touch with next steps.

Work email preferred.

Book a call