A study in Cell has uncovered a brain regulatory network involving a circular RNA, a long non-coding RNA and two microRNAs, and could provide some much needed insights in a field where finding links to disease has far outpaced understanding the molecular mechanisms involved.
Non-coding RNAs have drawn the attention of drug developers because of the increasing numbers of molecules being uncovered with apparent translational potential.
Of the 87 new and emerging targets BioCentury identified in abstracts for the 2018 American Association for Cancer Research (AACR) conference, 11 were non-coding RNAs: ten microRNAs (miRNA) and one long non-coding RNA (lncRNA) (see “Hello RNA”).
According to BioCentury’s BCIQ database, at least five companies are developing therapies targeting or delivering miRNAs, and at least one company, HaYa Therapeutics, has active programs targeting lncRNA.
The bulk of progress has been on expression patterns of non-coding RNAs, enabled by improvements in next-generation RNA sequencing that allow even small RNAs to be detected and accurately read, or perturbation experiments. But compared with protein-coding mRNA, the basic biology of the non-coding species lags far behind.
According to Caroline Woo, associate director at RNA therapeutics company Translate Bio Inc., the advances made in mRNA-based drug development are not readily extended to non-coding RNAs. “Their 2-D or 3-D structures are what’s important, not necessarily the sequence. So a lot of the rules we follow for