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AE116 is a selective small molecule inhibitor of mechanistic target of rapamycin complex 1 (mTORC1), a key regulator of cellular metabolism, growth, and aging pathways. Developed by Aeovian Pharmaceuticals through a collaboration with Evotec, it is designed to treat rare and aging-related diseases by modulating mTORC1 activity without broadly inhibiting mTORC2, potentially offering improved safety over non-selective rapalogs like rapamycin. As of 2019, it was advancing through IND-enabling studies for an undisclosed rare disease, with preclinical data supporting its potential in central nervous system diseases.[3][5][9][11]
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