Overcoming the challenges of metabolite identification and profiling for developing oligonucleotides

[Image courtesy of WuXi AppTec]

The use of oligonucleotides (i.e., “oligos”) was first proposed in the late 1970s, but has not achieved widespread popularity until recently. Developments in delivery method and target specificity for oligos have led to oligos’ resurgence in popularity. These therapies are particularly of interest because their mechanism of action allows for targeting virtually any nucleic acid sequence including previously “undruggable” disease targets. Therefore, the development of oligo drugs can help to address unmet medical needs.

Ten oligo-based drugs have been approved since 2018, with 16 approved drugs in total. Additionally, more than 100 phase II or phase III clinical trials are currently underway. However, many unique challenges remain for oligo drugs. The novelty, complexity and evolving regulatory guidance of these therapies can pose greater technical challenges than conve…

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