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SAG21K is a potent, brain-penetrant, and orally bioavailable small molecule **agonist of the Hedgehog signaling pathway**. It acts primarily by directly activating the Smoothened (Smo) receptor, leading to downstream induction of Gli1 transcription and Hedgehog target gene expression. SAG21K has been studied in preclinical models for its capacity to stimulate Hedgehog signaling in a tissue-selective manner and exhibits protective effects in disease models such as colitis and nerve injury. Localized administration (e.g., via fibrin hydrogels) enables targeted activation with minimized systemic exposure.
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