Celsion Corporation, an oncology company focused on developing a portfolio of innovative cancer treatments, including directed chemotherapies, immunotherapies and RNA- or DNA-based therapies, recently announced an abstract publication in the Journal of Controlled Release. The abstract summarizes results from a preclinical trial which confirmed the effective delivery of RNA to lung cells — findings that have implications in the treatment of Severe Occlusive PAH.
For this study, scientists used Celsion’s TheraSilence™ technology platform, which was found to be effective in delivering an inhibitor of microRNA-145 in an animal model of severe occlusive pulmonary arterial hypertension (PAH).
Evidence has shown that an elevated expression of miR-145 is associated with the development and progression of human PAH. Results from this study conducted in collaboration with Dr. William Gerthoffer from the University of Alabama showed that treatment was linked to significant delivery of the inhibitor of miR-145 in the lung, inhibition of the levels of miR-145 levels and with an improvement of pulmonary hypertension.
“There is a clear unmet medical need for novel agents that can treat PAH,” said Dr. William Gerthoffer, Professor and Chair, Biochemistry & Molecular Biology, University of South Alabama. “The lung directed nature of TheraSilence™ platform has provided us with a delivery vector to approach this devastating disease. We have been very encouraged by the positive results seen in this study, which shows that we may be able to significantly improve pulmonary function by inhibiting miR-145 and believe that this approach has tremendous potential as a novel therapeutic.”
Celsion’s TheraSilence™ RNA delivery technology delivers to the lungs synthetically-generated mRNA, inhibitory RNA (RNAi) such as small inhibitory RNAs (siRNAs), microRNAs, microRNA mimics, microRNA inhibitors and associated molecules that serve as regulators of the protein expression at the transcript level by exploiting endogenous cell mechanisms.
In this new study, using the platform in pulmonary arterial hypertension-induced mice to deliver an anti-miR-145 molecule, the researchers were able to normalize vascular remodeling in the lung and restore the compromised cardiac function that results from PAH.
“The data highlights the differentiating and widespread potential of the TheraSilence™ RNA delivery platform to enable lung specific delivery of RNA and related molecules and provide unique treatment options for lung diseases that are not addressable by conventional drugs,” said in the news release Michael H. Tardugno, the Company’s Chairman, President and Chief Executive Officer. “TheraSilence™ has significant potential in a wide variety of indications, including those that fall outside our core focus of oncology. Our strategy is to seek to maximize the value of this platform in the near-term by pursuing collaborations and licensing agreements.”