Trofinetide is a synthetic analog of glycine–proline–glutamate, the N-terminal tripeptide of the insulin-like growth factor 1 protein, and has demonstrated clinical benefit in phase 2 studies in Rett syndrome.
CAT No: R1973
CAS No:853400-76-7
Synonyms/Alias:Trofinetide;853400-76-7;NNZ-2566;DAYBUE;Trofinetide [INN];trofinetida;L-Glutamic acid, glycyl-2-methyl-L-prolyl-;UNII-Z2ME8F52QL;ACP-2566;Z2ME8F52QL;Trofinetide [USAN];glycyl-L-2-methylprolyl-L-glutamic acid;Trofinetide(NNZ2566);Glycyl-2-methyl-L-prolyl-L-glutamic acid;(2S)-2-[[(2S)-1-(2-aminoacetyl)-2-methylpyrrolidine-2-carbonyl]amino]pentanedioic acid;Trofinetide (>90%);((S)-1-glycyl-2-methylpyrrolidine-2-carbonyl)-L-glutamic acid;trofinetidum;Glycyl-2-methyl-L-prolyl-L-glutamic Acid; Glycyl-L-2-methylprolyl-L-glutamic Acid; NNZ 2566;NNZ 2566;Daybue (TN);Trofinetide (USAN/INN);Trofinetide [USAN:INN];TROFINETIDE [WHO-DD];CHEMBL197084;SCHEMBL1170014;GTPL12482;NNZ2566;CHEBI:229599;DTXSID101336041;GLXC-15737;DJB40076;EX-A7815;WHO 10076;AKOS030607026;analogue 4 [PMID: 15837309];AT37725;CS-8154;DB06045;DA-68364;HY-16757;MS-24628;glycyl-alpha-methyl-L-prolyl-L-glutamic acid;D12400;Q27294917;
Chemical Name:(2S)-2-[[(2S)-1-(2-aminoacetyl)-2-methylpyrrolidine-2-carbonyl]amino]pentanedioic acid
Trofinetide is a synthetic peptide derivative based on the terminal tripeptide of insulin-like growth factor 1 (IGF-1), known for its neurotrophic and neuroprotective properties in preclinical studies. As a small peptide, it is structurally engineered to enhance stability and bioavailability compared to its parent compound, making it particularly valuable for research into central nervous system (CNS) function and neurobiological signaling. Its unique design allows for the investigation of peptide-mediated modulation of neuronal pathways, synaptic plasticity, and cellular resilience under various experimental conditions. Trofinetide's biochemical profile has attracted significant interest in neuroscience research, where it serves as a tool for elucidating the molecular underpinnings of neuronal health, peptide transport, and neurochemical adaptation.
Neurobiology research: Trofinetide is widely utilized in studies exploring the mechanisms of neuroprotection, synaptic function, and neuroplasticity. Its structural similarity to IGF-1-derived peptides enables researchers to dissect the downstream signaling cascades involved in neuronal survival, differentiation, and repair. By employing this compound in in vitro and in vivo models, investigators can assess how peptide modulators influence neurotrophic factor signaling, offering insights into the regulation of synaptic connectivity and neuronal network stability.
Peptide transport and metabolism studies: Due to its optimized peptide structure, trofinetide serves as a model substrate for examining peptide transport mechanisms across cellular membranes and biological barriers. Research utilizing this compound can help clarify the role of peptide stability, enzymatic degradation, and receptor-mediated uptake in the pharmacokinetics and biodistribution of peptide-based agents. These studies are essential for advancing the understanding of peptide delivery strategies and improving the design of next-generation neuroactive compounds.
Cellular signaling pathway analysis: Trofinetide is a valuable probe for dissecting intracellular signaling pathways that are modulated by IGF-1-related peptides. Scientists employ it to investigate the activation of key kinases, transcription factors, and cytoskeletal regulators that govern neuronal growth and maintenance. Its application in biochemical assays and cell-based systems enables precise mapping of peptide-induced changes in gene expression and protein phosphorylation, contributing to a deeper comprehension of neuronal adaptation and resilience.
Disease modeling and mechanism elucidation: In the context of disease modeling, trofinetide is instrumental for simulating neurodevelopmental and neurodegenerative conditions in experimental systems. By introducing this peptide into relevant cellular or animal models, researchers can observe its effects on cellular stress responses, synaptic integrity, and neuroinflammatory processes. These studies aid in unraveling the molecular basis of disease phenotypes and identifying potential peptide-responsive biomarkers for further investigation.
Peptide drug development research: The unique properties of trofinetide, including its enhanced metabolic stability and CNS bioactivity, make it an important reference compound for peptide drug development initiatives. It provides a benchmark for evaluating the structure-activity relationships, safety profiles, and pharmacodynamic characteristics of novel peptide analogs. By leveraging trofinetide in comparative studies, research teams can optimize peptide design strategies and advance the preclinical assessment of promising therapeutic candidates, thereby supporting innovation in neuroactive peptide research.
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