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Inovio Researchers Successfully Demonstrate RNA Drug Delivery via Electroporation

Electroporation delivery platform presents new avenue to enable development of targeted RNA-based therapies for untreatable diseases and conditions
BLUE BELL, Pa., Feb. 22, 2012 /PRNewswire/ -- Inovio Pharmaceuticals, Inc. (NYSE Amex: INO) announced today that its next generation surface skin electroporation technology was successfully used to significantly enhance the delivery of small interfering RNA (siRNA) molecules to skin in animal studies. The data was published in the journal Molecular Therapy - Nucleic Acids in a paper entitled, "Optimized in vivo transfer of small interfering RNA targeting dermal tissue using in vivo surface electroporation."

While Inovio has multiple ongoing human trials demonstrating the efficacy of its electroporation technology to deliver synthetic DNA-based vaccines to both skin and muscle, this is the first time that this technology has been applied to the delivery of siRNA molecules.

Recently, siRNAs have demonstrated their potential as novel therapeutics due to their ability to induce robust, sequence specific gene silencing in cells. Using siRNA to induce RNA interference (RNAi) could become a promising therapeutic approach to treat many currently untreatable disorders, such as some cancers and many viral and genetic diseases. This preclinical study of Inovio's novel delivery method demonstrated positive results using its minimally invasive, low-voltage surface electroporation technology to successfully delivery siRNA to the skin.

The primary point of this study was to establish whether electroporation is able to accomplish effective delivery of RNA in vivo. The successful outcome of this study highlights the far-reaching therapeutic potential for Inovio's electroporation technology. Preclinical and clinical studies have demonstrated that electroporation, as an effective physical delivery method, can improve both the expression and immunogenicity of DNA vaccines by up to 100-fold.

Dr. J. Joseph Kim, President and CEO, said "Perhaps the biggest hurdle in realizing the full potential of RNA-based therapies is the lack of proper and efficient delivery of siRNA molecules. This study supports the idea that Inovio's proprietary electroporation technology can successfully deliver breakthrough RNA therapies with the same efficacy and safety in which we deliver DNA therapies. Most important, our delivery platform could pave the way for the development of targeted RNA-based therapies for diseases and conditions that are now considered untreatable."

Researchers in this study investigated the optimization of electrical parameters for a novel low-voltage electroporation (EP) method to deliver RNA to dermal tissue in vivo. Initially, the electrical parameters were optimized for dermal delivery of plasmid DNA encoding green fluorescent protein (GFP) using this novel surface dermal EP device at as little as 10V voltage parameters. The device was also assessed for the electronic transfer of siRNA into dermal tissue and Inovio researchers observed robust transfection of tagged-siRNA in the skin. The researchers then assessed whether the successful transfer of siRNA led to gene knockdown (silencing) in vivo. Using a reporter gene construct encoding GFP and tagged siRNA targeting the GFP message, researchers demonstrated simultaneous transfection of the siRNA to the skin via EP and the concomitant knockdown of the reporter gene signal. The siRNA delivery was accomplished with no evidence of injection site inflammation or local tissue damage. The minimally invasive low-voltage EP method is able to efficiently deliver functional siRNA molecules to dermal tissue in a tolerable manner.

About Gene Silencing and siRNA Delivery

In our cells, genes produce proteins that determine all body functions. Sometimes these genes produce proteins harmful to the body or cause improper protein accumulation or protein activity. The goal of gene silencing is to reduce or eliminate the production of select proteins that cause or serve the development of diseases at the RNA level. By interfering with protein production, siRNA and RNAi could potentially play a role in controlling or eliminating diseases such as cancers and infections. To achieve this goal, siRNA must be efficiently delivered into cells. Electroporation has a well-established track record of delivering useful drugs and biologics into cells to perform their useful function.

About Inovio Pharmaceuticals, Inc.

Inovio is revolutionizing vaccines to prevent and treat today's cancers and challenging infectious diseases. Its SynCon® vaccines are designed to provide universal cross-strain protection against known as well as newly emergent unmatched strains of pathogens such as influenza. These synthetic vaccines, in combination with Inovio's proprietary electroporation delivery, have been shown in humans to generate best-in-class immune responses with a favorable safety profile. Inovio's clinical programs include Phase II studies for cervical dysplasia, leukemia and hepatitis C virus and Phase I studies for influenza and HIV. Partners and collaborators include the University of Pennsylvania, Merck, ChronTech, National Cancer Institute, U.S. Military HIV Research Program, NIH, HIV Vaccines Trial Network, University of Southampton, US Dept. of Homeland Security and PATH Malaria Vaccine Initiative. More information is available at

This press release contains certain forward-looking statements relating to our business, including our plans to develop electroporation-based drug and gene delivery technologies and synthetic vaccines and our capital resources. Actual events or results may differ from the expectations set forth herein as a result of a number of factors, including uncertainties inherent in pre-clinical studies, clinical trials and product development programs (including, but not limited to, the fact that pre-clinical and clinical results referenced in this release may not be indicative of results achievable in other trials or for other indications, that the studies or trials may not be successful or achieve the results desired, that pre-clinical studies and clinical trials may not commence or be completed in the time periods anticipated, that results from one study may not necessarily be reflected or supported by the results of other similar studies and that results from an animal study may not be indicative of results achievable in human studies), the availability of funding to support continuing research and studies in an effort to prove safety and efficacy of electroporation technology as a delivery mechanism or develop viable synthetic vaccines, the adequacy of our capital resources, the availability or potential availability of alternative therapies or treatments for the conditions targeted by the company or its collaborators, including alternatives that may be more efficacious or cost-effective than any therapy or treatment that the company and its collaborators hope to develop, evaluation of potential opportunities, issues involving product liability, issues involving patents and whether they or licenses to them will provide the company with meaningful protection from others using the covered technologies, whether such proprietary rights are enforceable or defensible or infringe or allegedly infringe on rights of others or can withstand claims of invalidity and whether the company can finance or devote other significant resources that may be necessary to prosecute, protect or defend them, the level of corporate expenditures, assessments of the company's technology by potential corporate or other partners or collaborators, capital market conditions, the impact of government healthcare proposals and other factors set forth in our Annual Report on Form 10-K for the year ended December 31, 2010, our Form 10-Q for the quarter ended September 30, 2011, and other regulatory filings from time to time. There can be no assurance that any product in Inovio's pipeline will be successfully developed or manufactured, that final results of clinical studies will be supportive of regulatory approvals required to market licensed products, or that any of the forward-looking information provided herein will be proven accurate.


Investors: Bernie Hertel, Inovio Pharmaceuticals 858-410-3101
Media: Jeff Richardson, Richardson & Associates 805-491-8313

SOURCE Inovio Pharmaceuticals, Inc.

Posted: February 2012