Inovio's pre-clinical trial shows successful delivery of RNA drug via electroporation

Inovio Pharmaceuticals (AMEX:INO) said Wednesday that its surface skin electroporation technology was used successfully to signficantly enhance the delivery of small, interfering RNA (siRNA) molecules to the skin in animal studies.

The data was published in the journal Molecular Therapy - Nucleic Acids.

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

siRNAs have shown therapeutic potential in their ability to induce sequence-specific gene silencing in cells. In cells, genes produce proteins that determine all body functions, and can sometimes produce proteins harmful to the body.

The goal of gene silencing is to reduce or eliminate the production of select proteins that cause 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, siRNA must be efficiently delivered into cells. Using siRNA to induce RNA interference (RNAi) could become a promising approach to treat many currently-untreatable disorders, such as some cancers and viral and genetic diseases.

The company said its preclinical study of the eletroporation delivery method showed positive results in the successfully delivery of siRNA to the skin, highlighting the potential for Inovio's technology.

Both previous preclinical and clinical studies have showed that electroporation can improve both the expression and immunogenicity of DNA vaccines by up to 100-fold.

“Perhaps the biggest hurdle in realizing the full potential of RNA-based therapies is the lack of proper and efficient delivery of siRNA molecules," said president and CEO of Inovio, Dr. J. Joseph Kim.

"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 the preclinical study investigated the optimization of electrical parameters for a low-voltage electroporation (EP) method to deliver RNA to dermal tissue in vivo.

The study then assessed whether the successful transfer of siRNA led to gene knockdown (silencing) in vivo.

Inovio said the siRNA delivery was done with no evidence of injection site inflammation or local tissue damage.

The company's SynCon vaccines are designed to provide broad 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 to generate positive immune responses, along with a favourable safety profile.

Inovio's clinical programs include phase II studies for cervical dysplasia (pre-cancerous lesions), leukemia and the hepatitis C virus, as well as phase I studies for influenza and HIV.