For over 30 years the pharmaceutical industry has debated the potential of oligonucleotide therapeutics, and these have been hailed by many as the third major development platform after small molecule pharmaceuticals and biologics.

Despite this history and anticipation there has been limited product success, with only three antisense drugs approved for use (Sept 2016) (1). Recent market research has, however, predicted the global antisense and RNAi industry has the potential to be worth $4.58Bm by 2022 (1). This is indicative of the strong candidate pipeline as typified with ClinicalTrials.gov detailing over 140 active clinical programs over various phases of development.

To understand why these numbers are currently being seriously discussed requires consideration of a number of contributory factors, which collectively have “come good” together leading to this increased positivity.  Specifically, these contributory factors include improved chemistries leading to more efficient production, a better understanding of the basic biology of oligonucleotides, more sophisticated and efficient delivery systems and perhaps the biggest measure of success, increased po ...