New routes to oligonucleotide manufacturing

Oligonucleotide therapeutics treat diseases at the RNA-level, enabling targets to be reached beyond those generally accessible to conventional pharmaceutical modalities. Since the Nobel Prize winning discovery of small interfering RNA (siRNA), its specificity and selectivity have driven the pharma industry to design 15- to 25-mer oligo analogues as a new class of drugs for a wide range of diseases. Initially, these oligo therapeutics targeted rare diseases, and commercialisation required relatively small amounts of active substance so that the cost of manufacture, although high, did not limit patient access. This has changed in recent years, as an increasing number of the 150+ oligos in clinical trials are showing excellent results for diseases with large patient populations, creating a critical need for sustainable, ton-per-annum-scale oligonucleotide production. 

This project aims at developing new synthetic strategies for oligonucleotide manufacturing with the idea of increasing the global manufacturing capacity while reducing the manufacturing costs and the environmental footprint. 

Ultimately, this research proposal will extend the reach and impact of oligo therapies over a broad range of diseases, transforming healthcare by producing patient and societal benefits and improving quality of care.