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.
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.
Connect with us on our Hybrid Road Show to explore oligonucleotide analysis
To help you explore this emerging topic further, this year we've launched an "Oligos on the Road and Beyond" European hybrid roadshow: a series of hybrid events held in key biotech hotspots across Europe. Each event will feature expert guest speakers from the industry to help you learn about the latest trends and techniques in the field, and the opportunity to network with other professionals.
Our first stop of the roadshow will be in Hemel Hempstead, UK, and will be remote via a live broadcast, on 11 May and registrations are open. Sign up today to join us on this journey and stay tuned for our second stop.
Learn more at thermofisher.com/oligonucleotides