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LAM-004

Development stage: Preclinical
Lead developer: AI Therapeutics
Modality: Small Molecules
Administration: Topical

Overview

**LAM-004** is a proprietary topical formulation of rapamycin (sirolimus), an mTOR inhibitor, developed by AI Therapeutics (now OrphAI Therapeutics) for dermatological applications. It targets facial angiofibroma associated with tuberous sclerosis complex (TSC) and other skin diseases by locally inhibiting mTORC1 signaling to suppress aberrant cell proliferation and smooth muscle cell growth characteristic of these conditions, minimizing systemic exposure and side effects compared to oral rapamycin.[1][11]

Targets

mTOR (Mammalian target of rapamycin kinase)

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