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VTT-001 (also known as VTX-EphB3 inhibitor) is a small molecule inhibitor of the receptor tyrosine kinase EphB3, currently in preclinical development by Violet Therapeutics. Identified through the company's proprietary C-to-C Engine platform, which maps cell-to-cell communication, VTT-001 is designed to treat neurodegenerative and neuroinflammatory diseases by modulating the interactions between microglia and astrocytes. The drug's mechanism of action involves blocking EphB3-mediated pro-inflammatory signaling, thereby reducing neuroinflammation and promoting synaptic health and cognitive function. Violet Therapeutics is advancing VTT-001 for multiple indications, including Alzheimer's disease, Parkinson's disease, Amyotrophic Lateral Sclerosis (ALS), and Huntington's disease, with an Investigational New Drug (IND) application targeted for mid-2026.
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