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TC1

Development stage: Preclinical
Lead developer: Novartis
Modality: Small Molecules
Administration: Intraperitoneal

Overview

TC1 is a novel, brain-penetrant, ATP-competitive catalytic inhibitor of the mechanistic target of rapamycin (mTOR). It effectively inhibits both mTOR complex 1 (mTORC1) and mTOR complex 2 (mTORC2) signaling pathways. Developed by Novartis, TC1 has demonstrated promising therapeutic potential in preclinical studies for Tuberous Sclerosis Complex (TSC), particularly in reducing seizure burden and extending survival in a Tsc2 hypomorphic mouse model, while also restoring species-typical weight gain, suggesting a potentially improved safety profile compared to existing mTORC1 inhibitors like everolimus.

Targets

mTOR (Mammalian target of rapamycin kinase)

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