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AGT251

Development stage: Preclinical
Lead developer: Gate Neurosciences
Modality: Small Molecules

Overview

AGT251 is a novel small molecule, specifically a tocopherol-omega alkanol chain derivative, identified for its protective effects against muscle atrophy. It functions as an antioxidant and modulates the Notch signaling pathway by counteracting its induction under cytotoxic stress. This leads to a decrease in the expression of Notch1 and Notch3, and subsequently represses the Notch target gene Hes1 and the atrophy/remodeling gene MuRF1. Preclinical studies in murine models of cancer-associated cachexia and amyotrophic lateral sclerosis (ALS) have shown that AGT251 ameliorates body and muscle weight loss, reduces muscular atrophy markers, and improves survival.

Targets

Neurogenic locus notch homolog protein 1 negative regulatory region

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