4. In what medical fields do you see oligonucleotide therapeutics gaining interest?

Chad Christianson (Alturas Analytics)

With the timely approval of two mRNA vaccines, oligonucleotide therapeutics have emerged from obscurity to become a prominent technology in today’s society. Any disease state that requires gene silencing or RNA interference can be treated with oligonucleotide therapies. Vaccines, respiratory diseases, pulmonary diseases, cancer and neurodegenerative diseases are just a fraction of what can be treated with oligonucleotide technology.

 Zamas Lam (QPS)

Oligonucleotides will probably continue with ‘interference and silencing’. Traditionally, these have been in rare and ultra-rare diseases. However, with the positive approval of Inclisiran against hypercholesterolemia, oligo therapeutics are now being developed for more common indications, such as HBV infection, dry eye in Sjögren’s Syndrome, acute kidney injury and others.

 Ken Cook (Thermo Fisher Scientific)

Vaccine production has been a major success already with the COVID-19 response. More mRNA vaccines will be produced for other infectious diseases in the future with a faster response. The other obvious field will be treatments for genetic diseases which may have little or no known treatments. We now know that mRNA can be successfully introduced into cells to produce proteins. This can be utilized to make proteins that are absent due to a genetic fault. siRNA can also be transported to cells in the same way, in this case to inhibit the production of proteins that are produced in amounts in certain disease states that are too high.

Alexey Wolfson (Advirna)

Significant effort is currently aimed at any liver targets (hepatocytes) due to the widespread adoption of GalNac chemistry for oligonucleotide delivery. The next field of interest will be neurological diseases since new conjugate chemistries are showing great promise for brain delivery.

The opinions expressed in this feature are those of the authors and do not necessarily reflect the views of Bioanalysis Zone or Future Science Group.