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ONC-003 is a preclinical small molecule drug candidate being developed by Rapafusyn for the treatment of oncology. It belongs to a novel class of non-degrading molecular glues, which are designed to modulate protein-protein interactions without inducing the degradation of the target protein. Specifically, ONC-003 is engineered to target and modulate the activity of a member of the Solute Carrier (SLC) transporter family. SLC proteins are membrane-embedded transporters that play critical roles in nutrient uptake and metabolic signaling, often being dysregulated in cancer cells. By acting as a molecular glue, ONC-003 aims to stabilize a specific functional state or interaction of the SLC target to achieve a therapeutic effect in cancer. The program is currently in the early preclinical stages, progressing from hit identification toward lead optimization.
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