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Angiozyme (RPI.4610) is a chemically stabilized, synthetic hammerhead ribozyme designed to inhibit angiogenesis by targeting the mRNA of Vascular Endothelial Growth Factor Receptor 1 (VEGFR-1, also known as FLT-1). As a catalytic RNA molecule, Angiozyme binds to and cleaves the VEGFR-1 mRNA in a sequence-specific manner, leading to a reduction in the expression of the receptor on the cell surface and the subsequent abrogation of VEGF-mediated signaling pathways. Developed by Ribozyme Pharmaceuticals (later Sirna Therapeutics), the drug was investigated in Phase I and Phase II clinical trials for various solid tumors, including metastatic renal cell carcinoma and metastatic breast cancer. Although the drug demonstrated a favorable safety profile and was well-tolerated when administered alone or in combination with chemotherapy, it ultimately failed to show significant clinical efficacy, leading to the discontinuation of its development.
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