Oligonucleotide Therapies New Year New Possibilities

Like everyone, I have been relieved to see the end of a challenging year. It is easy to look back on 2020 and be sad for the many things we missed out on. No doubt we still have challenges ahead, but the new year brings a fresh start, opportunities, and endless possibilities.

As scientists, 2020 was our year — the one where we were actually cool. People wanted to talk science with us and the world waited for us to find a solution to this dreadful pandemic. Thousands of scientists worked tirelessly and they delivered! The pace of change, progress and development has been unprecedented. The advances made will open new avenues across all areas of medicine and it inspires one to think, what’s next?

Is the time now?

Could we be on the cusp of realizing the full potential of gene and oligonucleotide therapies? The ability to modulate the human genome has long been a major objective in medicine. If we think of oligonucleotides in particular, within the past 25 years, the diversity and feasibility of these drugs as diagnostic or therapeutic tools have dramatically increased.

To date, oligonucleotide therapeutics have focused on gene silencing. However, other strategies are being pursued, including gene activation and splice modulation strategies which have the potential to expand therapeutics targets beyond what is generally accessible to conventional pharmaceutical modalities. There are hundreds of oligonucleotide therapies currently in clinical development with several already gaining regulatory approval. Is 2021 the year when the development of these game-changing therapies is accelerated? I certainly hope so.

Solutions for Oligonucleotide Analysis

The development of oligonucleotide therapeutics molecules requires robust, accurate analytical characterization in order to confirm their identity and to determine purity, quality, and strength. Utilizing an unrivaled portfolio of oligonucleotide related products, Thermo Fisher Scientific provides optimized analytical workflows from oligonucleotide product characterization through manufacturing and quality control. As an example, a recent application note presents the capabilities of high-resolution accurate mass (HRAM) and data-dependent tandem mass spectrometry (ddMS2 ) to enable confident identification, mapping, and relative quantitation of oligonucleotides, impurities, and degradation products in a single experiment.

For the first time, software is available to remove the manual interpretation step from your MS analysis. Oligonucleotide Analysis workflows introduced in BioPharma Finder software provide a streamlined process from sequence creation and oligonucleotide mapping to relative quantitation of impurities and result reviewing.

Application Note: Identification and quantitation of oligonucleotides, impurities, and degradation products

With tools like this, what can stop us?!