Vector-based systems (e.g., viral, plasmid, etc.) have been the traditional approach to gene therapy. All vector-based systems require large artificial expression constructs that typically do not resemble natural genes. They contain many foreign sequences from viruses or other sources that drive production of modified genes present in the expression construct. Furthermore, vector-based systems (e.g. viral gene therapies) have been linked to major health problems including secondary malignancies and even death.
On the other hand, small oligonucleotides do not encode for any exogenous genes. Rather, their activity is largely based on principles of complementary base pairing. Over the past decade, oligonucleotides have emerged as an important new class of therapeutic molecules. However, nearly all oligonucleotide-based drugs exhibit only an inhibitory mechanism of action in that they silence gene expression.
Our platform is based on RNAa technology that utilizes short, non-coding oligonucleotides to specifically target and up-regulate therapeutic genes rather than silence them. As a consequence, our pharmaceutical development process benefits from the use of already established medicinal chemistry and drug delivery platforms accelerating drug development. In addition, RNAa has the unique ability to enhance transcription of a targeted gene offering for a more natural alternative to gene therapies, as well as the ability to restore expression of silenced genes previously thought of as undruggable targets.