Drug Database
GA

gangliosides (Nervomax / gangliosides, Fidia / Neurosido)

✓ Approved

Gramon · therapeutic agent

What is gangliosides?

gangliosides is a therapeutic agent developed by Gramon. It is approved for therapeutic indications via unknown.

Drug Profile

Brand NamesNervomax, gangliosides, Fidia, Neurosido
CompanyGramon
RouteUnknown
StatusApproved
Sources: ClinicalTrials.gov, Gramon

Therapeutic Indications

gangliosides is developed for 4 unique indications across 3 therapeutic areas.

Therapeutic AreaConditionPhase
Nervous system disordersDiabetic neuropathy✓ Approved
Nervous system disordersNeuropathy peripheral✓ Approved
Blood and lymphatic system disordersNeutropenia✓ Approved
Congenital, familial and genetic disordersRetinitis pigmentosa✓ Approved

Related Research Articles

PubMedThe Journal of biological chemistry2026-10-14

Roles of sialoglycans in vertebrate development.

Hildebrandt Herbert H, Münster-Kühnel Anja A

In vertebrates, sialic acids are the terminal sugars of many glycan structures presented by glycolipids, glycoproteins and proteoglycans on the cell surface, in the extracellular matrix, and in bodily fluids. Due to their negative charge, they shape molecular interactions between cells as well as interactions with and within the microenvironment. Strategies to study the numerous and diverse functions of sialoglycans in health and disease range from global sialic acid deletions to the targeting of specific sialoglycan biosynthesis pathways in individual cell types or organs. Some of these approaches reveal fundamental, some rather subtle, or no developmental functions of specific sialoglycans. However, many of the available models aim to investigate selected aspects such as immune cell or tumor development (to name just two prominent examples), and most sialylation pathways remain severely understudied. Despite many excellent reviews on specific pathways, molecular entities, or disease- and organ-specific functions (as, e.g., assembled in this thematic series on sialic acids) a comprehensive current overview of the developmental roles of sialoglycans seems to be missing. This review tries to close this gap, but in light of the expert coverage of sialylated glycosphingolipids (gangliosides) in this series (1), we focus on the insights into the developmental roles of sialoglycans gained by approaches targeting sialylation per se or pathways affecting sialylation of either specifically glycoproteins or glycoproteins and glycolipids alike. In addition to the consequences of manipulations of global sialylation, particular emphasis will be on the developmental roles of polysialic acid in the brain and other organs.

PMID 42874920
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PubMedMolecular biology reports2026-10-09

The sphingolipid code of cancer: divergent roles across tumor types and therapeutic horizons.

Thombre Deepak Ganpatrao DG, Katre Vishal Arun VA, Singh Shailza S, Vaish Vivek V

Sphingolipids have emerged as critical bioactive molecules that orchestrate diverse cellular processes underlying cancer initiation, progression, and therapeutic resistance. Once considered structural membrane lipids, they are now recognized as dynamic regulators of proliferation, apoptosis, angiogenesis, metastasis, and immune evasion. Central to this biology is the ceramide and sphingosine-1-phosphate (S1P) balance, a metabolic switch that dictates cell fate between death and survival. However, accumulating evidence reveals that the "sphingolipid code" is cancer-type specific, shaped by the expression of distinct enzyme isoforms, metabolite profiles, and microenvironmental contexts. This review consolidates current knowledge on sphingolipid alterations across major malignancies, including hematological cancers, colorectal, gastric, pancreatic, breast, ovarian, endometrial, lung, glioblastoma, and melanoma. We have highlighted emerging common mechanistic themes, such as the roles of gangliosides in metastasis and chemoresistance driven by glucosylceramide synthase. Furthermore, we have discussed the therapeutic horizons of targeting sphingolipid metabolism, ranging from sphingosine kinase (SphK1/2) inhibitors and ceramide delivery systems to ganglioside-directed immunotherapy, with emphasis on preclinical and clinical advancements. By integrating sphingolipid biology with precision oncology frameworks, sphingolipids hold a promise as both therapeutic targets and biomarkers. Yet, challenges such as tumor heterogeneity, drug delivery barriers, and toxicity must be overcome to achieve positive clinical translations. Deciphering the sphingolipid code across cancers offers a transformative opportunity to unlock novel strategies for diagnosis, prognosis, and treatment, ultimately bridging basic lipid biology with personalized cancer therapy.

PMID 42853313
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PubMedFrontiers in neurology2026-10-09

Mammalian peripheral nerve specific autoimmunity: proposed mechanisms and redefinition of molecular mimicry.

Ubogu Eroboghene E EE

Molecular mimicry is considered essential for peripheral nerve-specific autoimmunity: a process by which antibodies, systemically generated primarily as a consequence of infection with bacteria or viruses possessing epitopes that are similarly expressed by peripheral axon complex gangliosides or peripheral nerve myelin proteins, are hypothesized to induce peripheral nerve injury in genetically susceptible individuals. Human in vitro studies have proposed that recently activated antigen-specific effector memory CD4+ T-cells initiate tissue specific autoimmunity by T-cell receptor-driven transmigration following local microvasculature activation. However, how do these rare T-cells find their way from secondary lymphoid organs where they are generated to restrictively migrate to peripheral nerves and nerve roots to initiate an autoimmune response? Using severe murine experimental autoimmune neuritis as an animal model of peripheral nerve-specific autoimmunity, we elucidated that Major Histocompatibility Complex (MHC) class II expression on endoneurial endothelial cells is essential for disease onset using conditional knockout transgenic mice. This implies that specific BNB antigen-expressing MHC Class II molecules could serve as a directional cue for antigen-specific effector memory CD4+ T-cell transmigration: an active, molecular-dependent, coordinated multi-step mechanistic paradigm, to initiate tissue-specific autoimmunity and drive subsequent monocyte and B-cell infiltration, resulting in peripheral nerve demyelination and axonal degeneration. Based on these initial observations, I propose to redefine molecular mimicry as follows: The process by which recently activated antigen-specific effector memory CD4+ T-cells engage with MHC class II-expressing microvascular endothelial cells that present the same processed antigen peptide sequences during immunosurveillance to trigger their transmigration and initiate tissue-specific autoimmunity.

PMID 42851660
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PubMedCell reports2026-10-06

Genome-wide CRISPR screening identified hemojuvelin as a non-neuronal receptor for the botulinum neurotoxin serotypes A and C.

Ma Wantong W, Liu Bo B, Gao Jingjing J, Nie Rong R et al.

Botulinum neurotoxins (BoNTs) enter cells primarily through neuronal receptors and gangliosides. However, the mechanism that enables their specific accumulation at neuromuscular junctions (NMJs) remains poorly understood. In this study, a genome-wide CRISPR-Cas9 screen identifies hemojuvelin (HJV), a membrane protein abundant in skeletal muscle, as a non-neuronal receptor for BoNT/A and BoNT/C. Knockout and rescue experiments show that HJV mediates toxin binding by a direct interaction between its C-terminal region (residues 155-393) and the toxin WY loop within the receptor-binding domain (HCR). In mice, HJV knockout markedly reduces toxin enrichment on the sarcolemma and significantly attenuates toxin sensitivity such as digital paralysis and lethality. Thus, HJV captures these toxins on the muscle sarcolemma, establishing a critical prerequisite for efficient recognition and internalization by neuronal receptors at NMJs. These findings further refine the entry mechanism of BoNTs in vivo and offer actionable insights for treating BoNT intoxication and for optimizing their clinical applications.

PMID 42837260
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PubMedFrontiers in molecular biosciences2026-10-03

Lipid signature in X-ALD: a comparison between phenotypes.

Di Veroli Alessandra A, Petrillo Sara S, Torda Caterina C, Loia Federica F et al.

X-linked adrenoleukodystrophy (X-ALD) has highly variable phenotypes with no known genotype/phenotype correlation or method for predicting the course of the disease. Screening for X-ALD allows for early, potentially lifesaving treatment of adrenal insufficiency and cerebral demyelination, possibly useful to detect pre-clinical differences in patients and extend treatments to pre-symptomatic subjects. In this study, we analyzed the lipid signature in fibroblasts of patients with Adrenomyeloneuropathy (AMN), the late onset and slowly progressive form of X-ALD, and in patients with Cerebral Adrenoleukodystrophy (CALD), the cerebral inflammatory demyelinating form of early childhood, in order to identify specific lipid molecules as potential early predictors for CALD. We analyzed lipidomic profiles in fibroblasts obtained from n = 4 clinically affected (and genetically proven) X-ALD patients (two CALD and two AMN) and n = 4 age-matched controls, using a Dionex UltiMate 3000 UHPLC system, coupled to a Q-Exactive mass spectrometer. Our results highlight alterations in lipid metabolism both in AMN and in CALD fibroblasts relative to controls, further confirming the role of LPC 26:0 as a potential biomarker of X-ALD. However, the extent and pattern of lipid remodeling differ substantially between the two phenotypes, with a contribution of neutral lipids in CALD patients, along with a selective involvement of gangliosides. Although both AMN and CALD fibroblasts share some disease-associated lipid alterations, CALD samples exhibited a broader and more pronounced perturbation in multiple lipid classes, particularly in LPCs and steryl esters. The identification of phenotype-associated lipid signatures may represent a step forward toward precision medicine in X-ALD, in order to improve patient phenotypic evaluation.

PMID 42827457
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PubMedFEBS letters2026-09-15

Leucine-rich glioma inactivated 1 (LGI1) is a ganglioside-binding protein.

Debreux Kévin K, Lévêque Christian C, Azzaz Fodil F, Sangiardi Marion M et al.

In Lgi1-/- mice, increased neuronal excitability is accompanied by a marked decrease in Kv1 channels, but the mechanism linking LGI1 loss to reduced ion channel expression remains unknown. We show that LGI1 contains multiple conserved canonical ganglioside-binding domains (GBDs) and that GT1b copurifies with LGI1 antibodies from native rat brain extracts. Recombinant LGI1 bound ganglioside-containing liposomes, and the ganglioside-binding capacity of surface-exposed GBD peptide sequences was confirmed experimentally. These findings suggest LGI1 interacts with gangliosides and may help organize lipid membrane platforms that accommodate functional protein complexes. We hypothesize that loss of LGI1 destabilizes these platforms, contributing to the reduced ion channel expression observed in Lgi1-/- mice.

PMID 42742099
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