Antisense RNA medicines can treat diseases like Huntington’s disease that are caused by excess or defective proteins.
Antisense RNA works by binding to messenger RNA (mRNA) and physically preventing the cell from translating the mRNA into protein. When antisense RNA binds to mRNA, it also signals the cell to destroy the mRNA. These mechanisms are called RNA interference, or RNAi, because they stop the protein product of a particular gene from being produced. Each antisense RNA targets and inhibits one specific mRNA.
Antisense RNAs are quickly degraded inside cells, so antisense RNA medicines can have low potency and efficiency. These problems can be overcome by using Arcturus’ unlocked nucleomonomer agent (UNA) Oligomer™ technology. The enzymes that destroy RNA inside cells cannot recognize UNAs. Therefore, antisense RNAs that contain UNAs are protected from degradation.