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GPI-1046 is a small molecule immunophilin ligand that acts as a non-immunosuppressive inhibitor of FK506-binding proteins (FKBPs). It was initially developed by Eisai. The compound has demonstrated neurotrophic and neuroprotective properties in preclinical models. Mechanistically, it upregulates glutamate transporter 1 (GLT1), particularly in the prefrontal cortex and nucleus accumbens core, which is associated with reduced ethanol intake in animal models. In rodent and primate studies of Parkinson’s disease, GPI‑1046 showed some ability to stimulate recovery of dopaminergic neurons and promote axonal regrowth; however, efficacy was not consistently observed across all models. Additionally, it has been shown to promote nerve regeneration and functional recovery after nerve injury in animal studies[4][5][7].
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