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Accelerating ScienceAnalyteGuru / Oligonucleotide / Oligonucleotide Therapeutics: A Beginner’s Guide

Oligonucleotide Therapeutics: A Beginner’s Guide

By Aude Croguennec, Senior Regional Marketing Manager 05.01.2023

What are oligonucleotide therapies?

Oligonucleotide therapies include short, single- or double-stranded DNA, or RNA molecules that bind via Watson-Crick base pairing to enhance or repress the expression of target RNA, to treat or manage a wide range of diseases. They include antisense oligonucleotides (ASOs) RNA interference (RNAi) and aptamers. In general, oligonucleotide therapeutics interfere or inhibit the RNA translational process within the cell and promote degradation of proteins associated with disease.

Oligonucleotide

Why are oligonucleotides important and why now?

You have likely heard about oligonucleotides and mRNA more frequently than in years past … and there is a good reason for that. Therapeutic oligonucleotides comprise a rapidly expanding category of drugs that will change the standard of care for many diseases and actualize personalized medicine.

The rapid growth of this therapeutic area has been made possible by recent advancements that include:

  • Overcoming the previous challenges of controlled immune response
  • Improved product stability
  • Optimizing the delivery system to reach its therapeutic target

What are the challenges with oligonucleotides?

Oligonucleotide therapeutics development and manufacture require confirmation of the correct nucleic acid sequence and comprehensive characterization and quantification of impurities to ensure therapeutic efficacy. In the later stages of development, this must be done in accordance with strict regulatory guidance.

Currently, the guidelines themselves are evolving. The main challenge is presented by the complexity of the oligonucleotide molecules themselves and the modifications that are being applied to them. Chemical modifications have been utilized extensively to improve the binding energy, stability, and tolerability of oligonucleotide therapeutics.

How can Thermo Fisher Scientific support your oligonucleotide therapeutics?

The analysis of oligonucleotide therapeutics requires robust and accurate analytical characterization to confirm identity, and to determine purity, quality, and strength. Methods for accurate characterization that are capable of separating and identifying impurities must be established.

The Thermo Scientific portfolio can support your oligonucleotide workflow from beginning to end with the Thermo Scientific DNAPac HPLC columns, the Vanquish HPLC family, the Orbitrap Exploris mass spectrometer and the BioPharma Finder software.  Whether you need to perform structural characterization, bioanalysis, impurity identification, or product monitoring in quality control, we have a unique and differentiated workflow solution that addresses your needs.

Oligo Worklow.jpg 
HPLC ColumnsVanquish HPLC SystemsMass SpectrometrySoftware & Data Analysis

molecules that bind via Watson-Crick base pairing to enhance or repress the expression of target RNA, to treat or manage a wide range of diseases. They include antisense oligonucleotides (ASOs) RNA interference (RNAi) and aptamers. In general, oligonucleotide therapeutics interfere or inhibit the RNA translational process within the cell and promote degradation of proteins associated with disease.

Why are oligonucleotides important and why now?

You have likely heard about oligonucleotides and mRNA more frequently than in years past … and there is a good reason for that. Therapeutic oligonucleotides comprise a rapidly expanding category of drugs that will change the standard of care for many diseases and actualize personalized medicine.

The rapid growth of this therapeutic area has been made possible by recent advancements that include:

  • Overcoming the previous challenges of controlled immune response
  • Improved product stability
  • Optimizing the delivery system to reach its therapeutic target

What are the challenges with oligonucleotides?

Oligonucleotide therapeutics development and manufacture require confirmation of the correct nucleic acid sequence and comprehensive characterization and quantification of impurities to ensure therapeutic efficacy. In the later stages of development, this must be done in accordance with strict regulatory guidance.

Currently, the guidelines themselves are evolving. The main challenge is presented by the complexity of the oligonucleotide molecules themselves and the modifications that are being applied to them. Chemical modifications have been utilized extensively to improve the binding energy, stability, and tolerability of oligonucleotide therapeutics.

How can Thermo Fisher Scientific support your oligonucleotide therapeutics?

The analysis of oligonucleotide therapeutics requires robust and accurate analytical characterization to confirm identity, and to determine purity, quality, and strength. Methods for accurate characterization that are capable of separating and identifying impurities must be established.

The Thermo Scientific portfolio can support your oligonucleotide workflow from beginning to end with the Thermo Scientific DNAPac HPLC columns, the Vanquish HPLC family, the Orbitrap Exploris mass spectrometer and the BioPharma Finder software.  Whether you need to perform structural characterization, bioanalysis, impurity identification, or product monitoring in quality control, we have a unique and differentiated workflow solution that addresses your needs.

Download your oligonucleotide therapeutic analysis information kit to get you going with speed and confidence with your analysis.

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Aude Croguennec

Aude Croguennec is a Senior Regional Marketing manager with for the EMEA region with Thermo Fisher Scientific, focusing on building content our our customers in mass spectrometry and for omics and biopharma applications.
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