In our latest podcast we discuss the bioanalysis of oligonucleotide therapeutics, a growing area in pharmaceutical development.
Min Meng, a leading expert, explains the different types of oligonucleotides (e.g., ASO, sRNA, PMO), highlighting their unique characteristics compared to small molecule and protein therapeutics.
She details and contrasts the various analytical methods used for their detection and quantification, focusing on the advantages and challenges of liquid chromatography-mass spectrometry (LC-MS relative to other technologies).
Finally, the discussion covers the importance of analysing the lipid nanoparticle delivery systems and the factors contributing to the success or failure of oligonucleotide therapies.
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Key Takeaways
Strategic Considerations for Adopting Oligonucleotide Therapies
- Targeted Therapy:
Oligonucleotides offer a targeted approach, allowing for scientific design to target specific genes. - Potential to Cure Diseases:
In theory, oligonucleotide therapy can address any disease caused by a mutant gene, provided the target can be reached. - Higher Success Rates:
Unlike small molecule development, which has a reported 5% success rate (doubling to 10% with good biomarkers), oligonucleotide therapies have shown significantly higher success rates around 60%. - Increased Investment:
The high success rates make them an attractive proposition for pharmaceutical companies and investors. - Potential Failure Causes:
While specific reasons are speculative, potential issues include LNP toxicity or immunogenicity. The use of GalNAc delivery systems (an endogenous sugar carrier) may be a way to mitigate LNP-related challenges.
What You Will Learn
By watching this podcast, you will gain insights into:
💡 Targeted Therapies: How oligonucleotides are designed to address specific genes, offering precision in treatment.
💡 Disease Applications: The potential of oligonucleotide therapies to address genetic diseases, provided the target is accessible.
💡 Higher Success Rates: A closer look at how these therapies achieve success rates significantly higher than small molecules.
💡 Industry Growth: Why increased investment is fuelling innovation and expanding the applications of oligonucleotide therapeutics.
This podcast is a must-watch for anyone working in the pharmaceutical industry, drug development, or interested in the future of medicine. Min’s expertise provides a wealth of knowledge on the complexities and exciting potential of oligonucleotide therapeutics.
Fill in the form to access the full episode and discover how these innovative therapies are paving the way for new treatment options!
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About Resolian
Our footprint includes five sites on four continents. It starts with our headquarters near Philadelphia, Pennsylvania, includes two labs in the UK, a lab in Chongqing, China, and Brisbane, Australia. Our areas of expertise include bioanalysis for all drug modalities, from conventional small molecules, monoclonal antibodies, peptides, oligonucleotides, and complex biologics like ADCs, AOCs, and biospecific antibodies.
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