For many years, the prospect of harnessing RNA biology to produce new pharmaceuticals has been on the periphery of drug development. Despite the first antisense oligonucleotide (ASO) gaining approval in 1998 – the same year as the discovery of RNA interference (RNAi), which was later awarded a Nobel Prize – wider interest in the class has yet to take off. Back in 2015, there were an equivalent number of RNA therapies under active investigation as gene therapies, although in the years since the gene therapy pipeline has ballooned five-fold to reach approximately 1,700 candidates. The RNA class – encompassing gene expression modifying approaches such as single-stranded ASOs and double-stranded RNAi, as well as messenger RNA (mRNA) vaccines and therapeutics, in addition to a range of alternative non-coding approaches, has merely doubled over the same period.
As of May 2021, Pharmaprojects lists just shy of 200 separate RNA programs in clinical development, with a further 500...
