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OZC-004 is a targeted protein degrader being developed by ONCOZEN for the treatment of mutant gastrointestinal stromal tumors (GIST). It utilizes a novel modality known as Chaperone-Mediated Protein Degradation (CMPD) to selectively degrade the c-KIT protein. Unlike traditional Proteolysis-Targeting Chimeras (PROTACs) that rely on the ubiquitin-proteasome system, CMPD leverages the cell's chaperone-mediated autophagy or similar chaperone-assisted pathways to eliminate pathogenic proteins. OZC-004 is specifically designed to address resistance mutations in c-KIT that often arise during treatment with standard tyrosine kinase inhibitors (TKIs) in GIST patients. As of 2024, the program is in the lead optimization stage.
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