PTC299 is a novel, orally administered small-molecule designed to inhibit the production of vascular endothelial growth factor (VEGF) in tumors. Overexpression of VEGF plays a key role in multiple diseases including cancer and macular degeneration. PTC299 was discovered through PTC's GEMS technology by targeting the post-transcriptional processes that regulate VEGF formation, and is currently being developed for the treatment of cancer.
Investigated for use/treatment in cancer/tumors (unspecified) and solid tumors.
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