RNA delivery coming of age?

Data supportive of GEN-2 presented at the miRNA World Conference Workshop on miRNA delivery

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LAWRENCEVILLE, N.J.—Celsion Corp. has presented data related to TheraSilence, the company’s lung-specific RNA delivery technology, at the miRNA World Conference Workshop on miRNA Delivery in Boston. The data were 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 Anwer.
GEN-2 leverages the TheraSilence delivery system and combines two unique molecular targets involved in tumor regulation to inhibit tumor growth and promote direct killing of tumor 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 tumor angiogenesis factor and a microRNA that is involved in tumor inhibition.
The Celsion presentation pointed out that in a mouse lung tumor model, intravenous delivery of RNA inhibiting VEGFR-2 with the TheraSilence delivery system resulted in significant knockdown of VEGFR-2 transcript in lungs, reduction in tumor blood vessel density and inhibition of tumor growth.
“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 demonstrates how siRNAs delivered via our TheraSilence platform can specifically target VEGFR-2 to inhibit tumor growth in the lung,” Anwer noted.
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 reportedly demonstrated a good tolerability profile. The presentation also described ligand modification with the TheraSilence platform to improve cell specificity of RNA delivery in vitro.
“The results presented today highlight the potential of our TheraSilence platform to provide unique treatment options for lung diseases that are not addressable by conventional drugs,” said Michael H. Tardugno, Celsion’s chairman and CEO, at the time of the presentation in Boston. “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 South Alabama provide a robust basis to further explore GEN-2’s utility and great potential for novel treatments of various lung diseases.”
Celsion is a fully integrated oncology company focused on developing a portfolio of innovative cancer treatments, including directed chemotherapies, immunotherapies and RNA- or DNA-based therapies. The company’s lead program is ThermoDox, a proprietary heat-activated liposomal encapsulation of doxorubicin, currently in Phase 3 development for the treatment of primary liver cancer. The pipeline also includes EGEN-001, a DNA-based immunotherapy for the localized treatment of ovarian and brain cancers. Celsion has three platform technologies for the development of novel nucleic acid-based immunotherapies and other anticancer DNA or RNA therapies, including TheraPlas, TheraSilence and RAST.

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