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Oligonucleotides for Drug Discovery
Multiple therapeutics using small oligonucleotides have been designed since the first antisense drugs of the 1990s. Although only four therapies have been approved by the FDA to date, recent and expected approvals from regulators in the U.S. may give the field the boost it needs to launch this class of molecules as the third major drug development platform after small molecules and biologics.
Unmodified oligonucleotides are polyanionic macromolecules with poor drug-like properties. Over the past two decades, medicinal chemists have identified a number of chemical modification and conjugation strategies which can improve the nuclease stability, RNA-binding affinity, and pharmacokinetic properties of oligonucleotides for in vivo applications.
Since 1989 Microsynth has acquired strong skills in synthesizing oligonucleotides with drug-like properties from screening up to pilot scale.
Features and Benefits
- Experienced in the incorporation of numerous modifications and in the conjugation of small molecules to oligonucleotides
- Coverage of essentially all relevant backbone modifications like:
- MOE (2’-methoxyethyl)
- LNA (locked nucleic acid)
- S-cEt (constrained ethyl, on request)
- Phosphorothioate (PTO)
- Gapmers or other chimeric structures are routinely synthesized.
- Modifications for delivery to specific cell types (e.g. GalNAc)
- From screening scale (mg) to pilot-scale manufacturing (10 g)
- Comprehensive batch records
- Tech transfer possible at any development phase
- Dedicated expert contact person for consultation on all aspects of manufacturing and product testing
- Regular status updates
- Customized manufacturing processes (synthesis scale, purification, formulation, QC) as well as documentation across all phases of development
- Reference material manufacturing
- Antisense oligonucleotides