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Aukera RAPTOR inhibitor
- Development stage
- Preclinical
- Lead developer
- Aukera Therapeutics
- Modality
- Small Molecules
01
Overview
Aukera Therapeutics is developing a selective small molecule inhibitor of RAPTOR (Regulatory-associated protein of mTOR), a critical component of the mTORC1 complex. By targeting RAPTOR, the drug aims to modulate the mTORC1 signaling pathway, which is frequently dysregulated in various diseases. The lead program is focused on Tuberous Sclerosis Complex (TSC), a genetic disorder characterized by mTORC1 overactivation leading to seizures and neuropsychiatric symptoms. Unlike traditional rapalogs, this inhibitor is designed for high selectivity to minimize off-target effects. The candidate was identified using an AI-driven drug discovery platform and is currently in preclinical development for TSC, with potential future applications in neurodegeneration, metabolic disorders, and aging.
- Other names
- RAPTOR inhibitorunnamed RAPTOR inhibitor
02
Targets
RPTOR (Regulatory-associated protein of mTOR (RAPTOR))
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