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

PubMedSkeletal muscle2026-05-23

Insights into the heterogeneous muscle lipidome of dysferlin-deficient mice: effects of age, muscle type, and sex.

Keenan Stacey N SN, Bayliss Jackie J, Lee Olivia O, Lloyd Erin M EM et al.

Dysferlinopathy is an age-dependent muscular dystrophy caused by loss of the membrane-associated protein dysferlin. Disease severity increases with age and selectively affects specific muscle groups, yet the molecular basis for this vulnerability remains unclear. Since lipid remodeling is a hallmark of dysferlinopathy and aging, we investigated how age, sex, and muscle fiber type interact to shape the muscle lipidome in dysferlin-deficient mice. We performed omics-scale lipid profiling across 738 lipid species and 30 lipid classes in quadriceps and gastrocnemius (fast-twitch muscles exhibiting pronounced pathology), soleus (slow-twitch) and extensor digitorum longus (EDL; fast-twitch, relatively spared) muscles from male and female dysferlin-deficient (BLA/J) and wildtype C57BL/6J (WT) mice aged 3, 10, and 26 months. Normal aging was associated with broad lipid remodeling, however, in the absence of dysferlin markedly amplified this remodeling, leading to elevations in specific triglycerides, diglycerides, cholesterol esters, gangliosides, ceramides, and sphingomyelin compared to WT muscle. Interestingly, we observed minimal sex differences between dysferlin-deficient muscles. Fast-twitch muscles, particularly quadriceps and gastrocnemius, exhibited the most extensive lipid alterations, whereas the slow-twitch soleus muscle showed relative lipid stability even at advanced age. Thus, fast-glycolytic muscles are more susceptible to age- and dysferlin-dependent lipid dysregulation than slow oxidative muscle. The preferential vulnerability of fast-twitch muscles to muscle wasting in dysferlinopathy suggests that fiber-type-dependent lipid handling contributes to selective muscle degeneration. This work defines a comprehensive lipidomic signature of disease progression and provides a framework for understanding how aging, sex, and muscle phenotype interact in muscular dystrophy.

PMID 42174676
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PubMedNeurobiology of disease2026-05-17

GD3 synthase deficiency disrupts Na+/K+-ATPase and plasma membrane Ca2+-ATPase function in mouse brain.

Puljko Borna B, Maček Hrvat Nikolina N, Ilic Katarina K, Ujevic Ana A et al.

GD3 synthase (GD3S) is a key enzyme in the production of gangliosides, sialylated membrane glycosphingolipids with essential physiological roles in mammalian brains. To elucidate the molecular bases of neuropathological findings associated with GD3S deficiency, we performed a multilayered analysis focused on the functionality of ion transporters Na+/K+-ATPase (NKA) and plasma membrane Ca2+-ATPase (PMCA) in the cortex and cerebellum of GD3S-deficient mice (GD3S-/-). We examined global transcriptomes, NKA and PMCA gene and protein expression, the influence of membrane lipid composition on lipid raft integrity, and the activity of both ATPases, pairing them with an exploratory principal component analysis. Transcriptomic data reveal that sets of genes involved in ion transport and membrane dynamics are differentially expressed in the absence of GD3S, whereas qRT-PCR data confirm changes in gene expression of specific NKA and PMCA subunits or isoforms. Altered protein expression and significantly lower activity of both NKA and PMCA were found in the cerebral cortex of GD3S-/- mice. Analysis of membrane cholesterol content revealed segregation of cholesterol into lipid rafts, which may lead to disordered membrane lipid architecture in GD3S deficiency. Additionally, our results confirm that an imbalance in membrane ganglioside composition leads to significant alterations in ion transporters NKA and PMCA activity. Furthermore, we experimentally restored the activity of both ATPases in cortical homogenates by administering exogenous b-series gangliosides, a finding that may aid in developing therapeutic strategies targeting deficits in GD3S and other enzymes of ganglioside biosynthesis.

PMID 42142847
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PubMedJournal of the American Society for Mass Spectrometry2026-04-24

Direct Comparison of MRM-MS and PRM-MS Methods for Quantitative Ganglioside Analysis.

Sanni Akeem A, Koraich Abderrahmane A, Nwaiwu Judith J, Delkhosh Arvin Saffarian AS et al.

Gangliosides are structurally diverse, low-abundance glycosphingolipids central to neuronal signaling and cancer progression; however, their quantitative analysis is hindered by low ionization efficiency, structural heterogeneity, and complex isomeric patterns. Targeted mass spectrometry (MS) represents a powerful approach for resolving these challenges, yet systematic comparisons of multiple reaction monitoring (MRM) and parallel reaction monitoring (PRM) for native gangliosides remain limited. Here, we developed and optimized a targeted LC-MS/MS workflow to directly evaluate PRM on a Q-Exactive HF Orbitrap versus MRM on a TSQ Vantage triple quadrupole. Collision-energy optimization revealed distinct fragmentation behaviors across platforms, identifying optimal normalized collision energies (NCE) for PRM of 28 for GD1a and 25 for GD2, GT1b, GM1, and GQ1b, whereas optimal CE values for MRM were 35 for GD1a, GD2, and GT1b, and 30 for GQ1b. Additionally, PRM enabled multiplexing up to 15 transitions per analyte, improving signal-to-noise up to ∼4-fold and reducing %RSD through postacquisition transition summation. High-energy collision dissociation (HCD) used in PRM generated a richer array of fragment ions, including informative cross-ring cleavages and low-mass diagnostic ions, providing superior structural confidence compared to CID fragmentation in MRM. Notably, PRM uniquely enabled quantification of GM1, which exceeded the mass range of the triple quadrupole instrument. Applied to post-mortem human brain tissue extracts, PRM distinguished GD1a and GD1b isomers with high specificity. These findings establish PRM as a robust, highly sensitive, and structurally informative platform for comprehensive ganglioside profiling in complex biological matrices.

PMID 42028805
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PubMedIbrain2026-04-23

Potential neuroprotective and therapeutic agents and their mechanisms for irradiation-induced brain injury.

Richard Seidu A SA, Abem Vivian Kapio VK, Roy Sagor Kumar SK

Cranial-irradiation is associated with tissue damage resulting in neurocognitive impediments that adversely influence patient quality of life. Administration of radiation directly to the tumor may extend to the neighboring healthy tissues, which may induce acute to persistent oxidative stress, lessening neurogenesis, neuroinflammation, as well as vascular alterations, leading to neurocognitive sequelae as a result of decline in neuronal structural complexity as well as synaptic connections. Almost all the medications indicated in the treatment of irradiation-triggered brain injury work via signaling pathways that are associated with lessening chronic oxidative stress, which is considered a consequence of the inflammatory response, reduction of edema, as well as microglia activation. Some agents have both preventative as well as therapeutic potential via the amalgamation of both neuroprotective and therapeutic mechanisms above. Thus, in this review, agents such as baicalein, troxerutin, epigallocatechin gallate, quercetin, melatonin, valproic acid, lithium, neurosteroid progesterone as well as minocycline have been implicated as neuroprotective agents for irradiation-induced neurological deficits. Also, agents such as glucocorticoids, methylphenidate, vitamin E, bisdemethoxycurcumin, phosphodiesterases, edaravone, pioglitazone and fenofibrate, glutamate antagonists, human urinary kallidinogenase, bevacizumab, as well as hyperbaric oxygen have been implicated as therapeutic agents for irradiation-induced neurological deficits. Furthermore, agents such as angiotensin-converting enzyme, 3-N-butyl-phthalide, stem cell therapy, sphingosine-1-phosphate, gangliosides, and neurotrophins have been implicated as combined potential neuroprotective and therapeutic agents for irradiation-induced neurological deficits. The aim of this review is to elucidate the potential neuroprotective and therapeutic agents above and their mechanisms for irradiation-induced neurological deficits after brain irradiation.

PMID 42023248
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PubMedInternational journal of molecular sciences2026-04-14

Forgotten Gangliosides: O-Acetylated and Lactone Gangliosides.

Mauri Laura L, Vaghi Stefano S, Sonnino Sandro S

Gangliosides containing O-acetyl-sialic acid or a sialic acid forming a lactone ring, classified as alkali-labile gangliosides (ALGs), were the focus of extensive research until the late 1990s. Their identification and isolation for structural characterization require strict avoidance of alkaline treatments, a common step in standard ganglioside purification protocols used to eliminate glycerophospholipid contamination. After 2000, scientific interest in alkali-labile gangliosides declined significantly, resulting in a dearth of new information and few updates on their biological significance. However, in recent years, new insights have emerged regarding the potential of anti-acetylated ganglioside antibodies for specific tumor treatments and how ganglioside structures can modulate the activity of membrane receptors through specific interactions. Further knowledge of alkali-labile gangliosides could be very useful for better understanding their significance in both normal and tumor cells. In this manuscript, we intend to revisit the existing knowledge on alkali-labile gangliosides and form hypotheses regarding their possible roles in cells.

PMID 41977370
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PubMedMolecular neurobiology2026-04-09

Suppression of Inflammatory Responses Through GD3 Synthase (GD3S) Silencing Mediated by TLR4 Signaling: In Vitro and In Silico Analysis.

Park Jun-Young JY, Fukushi Abekura A, Kim Hee-Do HD, Kuo Han-His HH et al.

Glycosphingolipids (GSLs) regulate cell proliferation by modulating transmembrane signaling pathways. Gangliosides, as essential constituents of GSLs located on the cell membrane surface, contribute to diverse biological processes. Among them, ganglioside GD3 is found in neuronal precursors, TrkA receptor-expressing cells, and various neuronal populations, where it influences cell-cell interactions and metabolic activities. However, its immunity-related function and signaling mechanism remain unclear. In this study, we investigated THP-1-derived macrophages transfected with shGD3 synthesis (shGD3S) and observed reduced mRNA and protein expression of COX-2, iNOS, Myd88, TLR4, and pro-inflammatory cytokines. Furthermore, activation of the MAPK and NF-κB signaling pathways was diminished in shGD3S-transfected macrophages. We also examined the direct interaction between GD3 and TLR4, confirming that GD3 binds directly to TLR4. Using molecular docking simulations and fragment molecular orbital (FMO) calculations, we analyzed the binding affinities and hotspot residues of GD3, GM3, and LPS with the TLR4-MD2 complex. GD3 exhibited a higher binding affinity than GM3 and similar interaction energy to LPS, with Asp44 and Tyr82 identified as key hotspot residues. Ganglioside derivatives interacted with different TLR4 chains, with LPS and GM3 located near Arg90 and between fatty acid chains, while the linear carbohydrate moiety clustered around Phe121. These findings indicate that shGD3S-transfected macrophages suppress LPS-induced inflammatory responses by attenuating NF-κB, MAPK, and TLR4 signaling. Thus, sialyl-glycolipid GD3 appears to promote inflammatory responses in THP-1-derived macrophages.

PMID 41954870
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