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p70 S6 kinase inhibitor is a preclinical-stage small molecule program being developed by Epigen Biosciences (in collaboration with the University of California, San Diego) for the treatment of Fragile X Syndrome (FXS). The program targets p70 S6 kinase (p70S6K), a downstream effector of both mTORC1 and ERK signaling pathways that plays a critical role in regulating translation. Dysregulated protein synthesis is a core molecular abnormality in FXS, and inhibition of p70S6K aims to normalize synaptic translation and correct behavioral and cognitive deficits. Lead candidates in this program include EPGN-2036 and EPGN-1370.
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