Efficiently delivering inhibitory RNAs to fight tumor growth
Addressing the primary obstacle to nucleic acid–based therapeutics
TheraSilence technology addresses the primary obstacle to nucleic acid–based therapeutics—efficient delivery to target cells. Specifically, a delivery system must protect the RNAi from nuclease degradation, transfer the molecule across the cellular membranes, and release it so that it can be available to the endogenous RNA silencing machinery. Celsion has developed proprietary, novel structures that are: (1) able to interact with the RNAi molecules, forming protective nanoparticles that can be readily taken up into cells; (2) Chemically flexible and amenable to attachment of tissue-targeted ligands, in vivo stabilizing agents, and other functional moieties which can tailor a formulation for a particular application and delivery modality.
We believe that these features can provide high specificity for RNAi delivery to select tissue, enhance stability, and reduce in vivo toxicity.
Proof-of-concept in lung tumors
In-vivo proof-of-concept studies of our most advanced system have shown the ability to deliver RNAi molecules specifically to pulmonary vasculature following intravenous administration. Using this delivery system we have been able to show in mice that delivery of a siRNA molecule targeting receptor 2 of vascular endothelial growth factor (VEGF), a protein critical for angiogenesis, can significantly inhibit lung tumor growth.
TheraSilence Preclinical Studies
Lung cancer preclinical studies: In a mouse lung tumor model, vascular endothelial growth factor receptor-2 (VEGFR-2) siRNA was formulated with TheraSilence LNPs. Intravenous treatment resulted in a significant decrease in VEGFR-2 transcript and a significant reduction in lung tumors.