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CC-2055 is a novel, brain-penetrant, and selective small molecule cyclophilin D (CypD) antagonist developed by Cypralis. It is designed to inhibit calcium-mediated mitochondrial permeability transition pore (mPTP) opening by binding to CypD, thereby preventing mitochondrial dysfunction and apoptotic cell death. Preclinical studies in mouse models of epilepsy (such as the pilocarpine model) have demonstrated that CC-2055 exerts anti-seizure effects, reduces the frequency of spontaneous recurrent seizures, restores parvalbumin-positive (PV+) GABAergic interneurons in the CA1 hippocampus, and normalizes the resting membrane potential of CA1 pyramidal cells.
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