Summary of siRNA Advantages

The siRNA drug development program exploits the inherent advantages of siNRAs, in general and especially of those that target the liver, over antibodies for the same target.

Among the advantages

• siRNA executes its function by complete base pairing with mRNA, whereas monoclonal antibody drugs (and small molecules) need to recognize the complicated spatial conformation of certain proteins, hence siRNAexercises its effect with high specificity;
• Possibility for longer duration of effect (durable target knock-down (KD)) and thus for substantially reduced drug administration (usually by injection) schedule.
• Due to the amplification effect inherent to the mechanism of action of siRNA, a single siRNA molecule mediates the degradation of multiple mRNA molecules containing the target sequence and the overall process is catalytic. Hence, the siRNA drug will be effective at lower doses and reduced cost per dose.
• Also the synthetic nature of siRNAs significantly simplifies large-scale production and makes siRNAs more economical with reduced cost-of-goods and significantly more affordable cost of therapy.
• The current siRNA drugs have excellent safety profile and are very stable under a wide variety of thermal storage conditions
• Allow streamlined and faster discovery of drug candidates.