Celsion Corporation announced the presentation of data related to TheraSilence, the Company's lung-specific RNA delivery technology, at the miRNA World Conference Workshop on miRNA Delivery in Boston, MA.  The data was presented by Dr. Khursheed Anwer, Celsion's executive vice president and chief scientific officer, during a panel presentation, and highlighted formulation characteristics of the TheraSilence delivery platform, preclinical proof-of concept data and data supportive of GEN-2 (formerly referred to as EGEN-002), Celsion's RNA interference (RNAi) therapeutic for the treatment of lung cancer. Celsion acquired the TheraSilence platform and GEN-2 through its acquisition of EGEN, Inc. in June 2014.

"Our TheraSilence delivery platform is designed to overcome the challenges associated with delivery of RNA for the treatment of lung diseases, with a chemically flexible system that allows us to improve the safety, stability, and efficiency of RNA delivery to the lung," stated Dr. Anwer. "The data presented was developed under a research collaboration and shows that intravenous administration of small RNAs formulated with our delivery system produced significant and durable RNA activity specifically in the lung following a single injection, and demonstrate how siRNAs delivered via our TheraSilence platform can specifically target VEGFR-2 to inhibit tumour growth in the lung."

The data from the research collaboration was jointly published in Molecular Therapy. GEN-2 leverages the TheraSilence delivery system and combines two unique molecular targets involved in tumour regulation to inhibit tumour growth and promote direct killing of tumour cells. Among its capabilities as a platform for therapeutic application, GEN-2 also has demonstrated an ability to incorporate short interfering RNA (siRNA) targeting VEGFR-2, a tumour angiogenesis factor, and a microRNA that is involved in tumour inhibition.

The highlights of the presentation are included below, In a mouse lung tumour model, intravenous (IV) delivery of RNA inhibiting VEGFR-2, a tumour angiogenesis factor, with the TheraSilence delivery system resulted in significant knockdown of VEGFR-2 transcript in lungs, reduction in tumour blood vessel density and inhibition of tumour growth.

Studies also demonstrated the ability of anti-miRNA delivered via the TheraSilence system to inhibit miRNA-145, which is associated with the pathogenesis of pulmonary arterial hypertension (PAH). In a rat model of severe PAH, IV administration of antimir-145 formulated with TheraSilence resulted in preferential accumulation of the anti-miRNA in lungs, reduction in miRNA-145 transcript in lungs, and reversal of cardiopulmonary parameters associated with the disease.

Systemically administered RNA complexes using the TheraSilence delivery system demonstrated a good tolerability profile. The presentation also describes ligand modification with the TheraSilence platform to improve cell specificity of RNA delivery in vitro.  

"The results presented highlight the potential of our TheraSilence platform to provide unique treatment options for lung diseases that are not addressable by conventional drugs," Michael H. Tardugno, Celsion's chairman and chief executive officer.  "While we remain focused on advancing our clinical stage programs, ThermoDox and EGEN-001, we see significant value in this delivery platform and in GEN-2 in particular.  Our internal research and collaboration with the University of S. Alabama provide a robust basis to further explore GEN-2's utility and great potential for novel treatments of various lung diseases."