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TSC2 small molecule
- Development stage
- Preclinical
- Lead developer
- Quiver Bioscience
- Modality
- Small Molecules
01
Overview
Quiver Bioscience is developing a small molecule modulator targeting the TSC2 (tuberous sclerosis complex-2) protein for the treatment of tuberous sclerosis complex (TSC). TSC is a genetic disorder caused by mutations in the TSC1 or TSC2 genes, which lead to the overactivation of the mTORC1 pathway. This drug candidate was identified through phenotypic screening using human neuronal models to find compounds capable of rescuing functional disease parameters. Unlike traditional mTOR inhibitors, this small molecule specifically targets the TSC2 protein or its associated complex to restore normal signaling and cellular function. It is currently in the discovery phase of development.
- Other names
- TSC2 Small MoleculeTSC-2 Small MoleculeTSC 2 Small Molecule
02
Targets
TSC2 (Tuberous sclerosis complex 2 protein)
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