This week, New Jersey based biopharmaceutical company Celsion, whose mission is the development of innovative, targeted therapies that address unmet medical needs in oncology, announced the publication of the abstract for their pre-clinical study, entitled, “Lipid nano particle delivery of a microRNA-145 inhibitor improves experimental pulmonary hypertension.” The study investigated Celsion’s TheraSilence technology platform for safety and efficacy in delivering an inhibitor of microRNA-145 (miR-145) in a well-established model of severe occlusive pulmonary arterial hypertension (PAH). The abstract was published in the latest edition of the Journal of Controlled Release.
The pre-clinical study was conducted in the laboratory of Dr. William Gerthoffer, PhD, Professor and Chair, Biochemistry & Molecular Biology, University of South Alabama. Dr. Gerthoffer’s laboratory research aims are focused on understanding the regulation of vascular and airway smooth muscle contraction and regulation of smooth muscle phenotype, as well as the role of microRNAs in determining smooth muscle cell functions as it relates to diseases of the lungs.
In this study, Dr. Gerthoffer and his team conducted experiments using laboratory rats with induced PAH to test the distribution, toxicity, and efficacy of TheraSilence mediated delivery of antimiR-145.
The primary study finding showed that AntimiR-145 reduced the severity of pulmonary hypertension, and reduced the degree of cardiac dysfunction. Other findings included:
- Using TheraSilence resulted in substantial distribution of antimiR-145 in lungs as well as liver, kidney, and spleen.
- There were no toxic effects observed during the experiments.
In a company press release about the study findings, Dr. Gerthoffer stated “There is a clear unmet medical need for novel agents that can treat PAH. The lung directed nature of TheraSilence(TM) 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.”
His sentiments were shared by Michael H. Tardugno, President and CEO of Celsion: “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. TheraSilence(TM) 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.”
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 III development for the treatment of primary liver cancer and in Phase II development for the treatment of recurrent chest wall breast cancer. The pipeline also includes GEN-1, 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 anti-cancer DNA or RNA therapies, including TheraPlas(TM), TheraSilence(TM) and RAST (TM).