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Oligonucleotide therapeutics have attracted much attention recently. They represent an up-and-coming class of therapeutic options that can lead to major advances in the treatment or cure of diseases, holding great potential for further development in human medicine. Here, we answer some common questions about oligonucleotide therapeutics that you may want to know.
Hear our conversation with Technology Networks on the basics of oligonucleotide therapeutics, their trends and challenges, and what to consider during their development.
Oligonucleotide therapeutics are short DNA or RNA molecules that modulate the expression of target RNA for the treatment of diseases. These oligos are:
This drug family includes antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs) involved in RNA interference (RNAi), small activating RNAs (saRNAs), aptamers, and guide RNAs (gRNAs) involved in CRISPR gene editing technology. As of early 2023, there are 14 such drugs currently commercialized worldwide.* Twelve of these are approved in the United States, the majority of which are ASOs and siRNAs. They treat diseases including muscular dystrophy, polyneuropathy, and hypercholesterolemia, with many more therapeutics currently in development and other diseases being targeted.
* Based on molecular databases, press releases, market reports, clinicaltrials.gov, and expert interviews.
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They both act on target RNA, resulting in RNA cleavage and ultimately preventing protein translation. However, their structure and mechanisms are different. ASOs are single-stranded oligos that recruit RNase H1 to cleave their RNA targets in the nucleus or the cytoplasm. On the other hand, RNAi involves double-stranded siRNAs that recruit the RNA-induced silencing complex (RISC) to cleave their RNA targets in the cytoplasm.
Figure 1. Main differences between ASOs and siRNAs.
The success of mRNA-based SARS-CoV-2 vaccines has brought renewed interest to other nucleic acid drugs, including ASO and RNAi therapeutics. In the next 5 years, the oligo therapeutics market is expected to grow by 25–30%; over 1,000 oligonucleotide molecules are currently in the pipeline.*
As both ASOs and siRNAs are short oligonucleotides, their stability in the body, immunogenicity to the host, specificity to their target sequences, and delivery to target organs have always been challenges.
As the building blocks for oligonucleotide therapeutics, modifications to phosphoramidite structures have been developed to improve performance and mitigate these issues. For example,
* Based on molecular databases, press releases, market reports, clinicaltrials.gov, and expert interviews.
They look for quality assurance, documentation support, capacity, scalability, and batch-to-batch consistency in their products.
We offer Thermo Scientific TheraPure phosphoramidites, which are suitable for oligonucleotide manufacturers for the development of therapeutic applications. Our TheraPure phosphoramidites undergo additional quality control release testing compared to our standard phosphoramidites, helping ensure that impurities and residual solvents are controlled to the stringent levels required by our customers for their oligo therapeutics.
In addition to our on-shelf products, we provide services for custom oligonucleotide chemistry such as customization of existing phosphoramidites and the development of new compounds or oligonucleotide delivery chemistries.
Outside of our phosphoramidite team, Thermo Fisher Scientific offers reagents for oligonucleotide synthesis and analysis tools for synthesized oligonucleotides, as well as the ability to synthesize large-scale oligonucleotides.
See 5 benefits of using TheraPure phosphoramidites in the development of oligonucleotide therapeutics.
A longer version of this article was published in the “Resources and tools for oligonucleotide therapeutics” eBook in collaboration with BioPharm International. Read it and other articles in the eBook
For Research Use Only. Not for use in diagnostic procedures.