KATHRYN L. ACKLEY
Member of the Scientific Advisory Board – Chimica Oggi – Chemistry Today

Abstract

Oligonucleotide therapeutics have developed into a viable drug platform capable of delivering first-in-class therapeutics to patients. A virtual panel of experts from across the oligonucleotide industry answer questions about the state of the industry and what the future holds for the field of oligonucleotide drug development.

Nearly four decades ago when I was a student, I read an article about scientists employed by pharmaceutical companies to travel the globe collecting plant and soil samples. Thousands of samples were collected and taken back to company labs to be screened for compounds with medicinal properties. The next blockbuster drug could be waiting to be discovered in a vial full of soil taken from the rainforest floor. The whole process seemed so romantic and exciting to my teenage brain. My adult brain sees that process for what it is, an expensive and inefficient way to discover new drugs.

Oligonucleotide based therapeutics (oligos) are attractive to drug developers because they offer an efficient and rational way to design drugs. Oligos also have well understood mechanisms of action. Most of these drugs derive their therapeutic properties from the regulation of gene expression. Some oligo drugs incorporate features that impart immunostimulatory properties, which is why oligos can be potent vaccine adjuvants. Oligos are produced using well established solid phase synthesis methodologies that can be applied to multiple oligo sequences.

The potential o ...