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Oligonucleotide therapeutics (ONTs) are a promising class of medicine that work by directly interacting with genetic material (DNA or RNA) to influence gene activity. This makes them uniquely suited to treat diseases difficult or impossible to target with conventional drugs. Currently, ONTs are being used to treat conditions like neurodegenerative, kidney, liver, and heart diseases that include some of the most unmet medical needs, such as Parkinson’s, Alzheimer’s and Huntington’s diseases, with ongoing research expanding their potential to areas such as cancer and infectious diseases.  

However, one of the biggest challenges in developing ONTs is how they distribute in the body (pharmacokinetics) and their potential toxicity. A promising way to improve ONTs is by modifying their building blocks, known as nucleosides. While this approach has shown success, the complexity of nucleoside chemistry has slowed progress.  

To address this, a Vancouver-based start-up called Cloudburst Biotech Inc., has developed the Rapid Access to value-added Innovative Nucleosides (RAvIN) platform, a novel chemical process that simplifies nucleoside synthesis. This platform makes creating new nucleoside structures faster, more efficient, and cost-effective. By integrating these improved nucleosides into ONTs, this project aims to enhance their safety and effectiveness. Once optimized, these novel nucleosides can be licensed to pharmaceutical companies to advance ONT-based treatments, bringing new hope to patients with unmet medical needs.