How RNAi Works

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Silencing Diseases by Using the Body’s Natural Mechanism of RNAi

RNA interference (RNAi) involves the use of short interfering RNAs (siRNAs) to break down messenger RNA (mRNA), molecules that carry the genetic instructions used to create specific proteins that have particular functions in the body. When mRNA is created from mutated genes, or the body makes too much of a certain mRNA, it affects the production of its corresponding protein, triggering an unwanted effect and/or causing disease.

By temporarily blocking or ‘silencing’ a specific gene’s message, our technology aims to halt or reverse the progress of disease by targeting the underlying disease source rather than the symptoms it causes.

Our mRNAi GOLD™ platform is designed to deliver gene silencing medicine to targeted liver cells in the body by combining siRNA molecules with chemical “address tags” called GaINAc, a naturally occurring sugar that attaches specifically to liver cells. These RNAi molecules can then enter liver cells and ‘silence’ targeted, disease-associated genes expressed in the liver.



Harnesses natural mechanisms present in every cell in the human body



Long-lasting gene silencing possible following a single injection



siRNA designed to bind only to the target gene