Advanced Antisense Platform

Antisense molecules exert their pharmacological activities by modulating the function or level of the target RNA via Watson-Crick base-pairing. The exact mechanism of action differs for different antisense platforms.

The small interfering RNAs (siRNA), discovery of which led to a Nobel Prize in 2006, work via RNA interference, a naturally occurring cellular mechanism to silence gene expression and regulate the production of proteins.

Antisense oligonucleotides (ASOs) are more versatile. Depending on the designs, ASOs can decrease, increase or altering the site of splicing of the target RNA to achieve different therapeutic effects.

With our advanced antisense technology, we modulate the expression of the target RNA with programable potency, onset and duration of action for both RNAi and ASO platforms, opening the opportunity to treat diseases in both liver and extra-hepatic organs and for both common and rare disease indications.