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ppp-VEGF is an experimental, multifunctional short interfering RNA (siRNA) molecule designed to treat cancer by simultaneously targeting multiple pathways. It consists of an siRNA sequence specific for Vascular Endothelial Growth Factor (VEGF) modified with a 5'-triphosphate (ppp) moiety. This design enables a triple mechanism of action: first, it silences VEGF expression to inhibit tumor angiogenesis; second, the 5'-triphosphate group acts as a ligand for the cytosolic pattern recognition receptor RIG-I (Retinoic acid-inducible gene I), which triggers an innate immune response and activates antitumor immunity; and third, it directly induces apoptosis in tumor cells. Preclinical studies in murine lung cancer models have demonstrated that ppp-VEGF can effectively suppress tumor growth and overcome the limitations of traditional antiangiogenic agents by combining gene silencing with immunostimulatory activity.
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