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.
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.
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.
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.
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.
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.
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
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.
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.