Drug intelligence / Profile preview

psiRNA-VEGF

Development stage
Preclinical
Lead developer
Medical University of Warsaw
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intratumoral, Subcutaneous (in Preclinical Models)
01

Overview

psiRNA-VEGF is a **gene therapy preparation** utilizing a plasmid-based short hairpin RNA (psiRNA) construct specifically designed to silence expression of the *vascular endothelial growth factor* (VEGF) gene. It is formulated with a cationic lipid carrier, enabling efficient non-viral transfection of target cells. Its primary mechanism is to inhibit angiogenesis—the formation of new blood vessels—by RNA interference-mediated suppression of VEGF, thereby reducing the growth, vascularization, and mass of tumors, as demonstrated in preclinical melanoma models. This agent is developed for **anti-tumor and antiangiogenic therapy**, with demonstrated efficacy when administered via local (intratumoral) injection, leading to significant reduction of tumor mass and vascularity. Notably, its antiangiogenic action may be further potentiated when used in combination with psiRNA constructs targeting other angiogenesis-related genes, such as SOX10[2][4].

Other names
psiRNA-VEGF
02

Targets

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