A quick scan of recent advances in the scientific community will show that genomic data and new techniques in next-gen sequencing have been gaining importance in drug discovery innovation. Identifying potential leads for various diseases in the drug discovery pipeline have traditionally included high throughput screening or computational approaches including structure-based docking algorithms.


The Inforna method of identifying potential therapies that target specific RNAs. Image courtesy of Nature Chemical Biology.1

However, researchers at The Scripps Research Institute have developed a new method of searching potential leads that they have termed Inforna. This method exploits the cellular function of RNAs. While previous studies have found challenges in developing compounds to target RNAs, Inforna has been able to combine advances in the knowledge of RNA structure and the identification of RNA-small molecule interactions, and score these interactions to determine the efficacy of the compound.1

Inforna’s high hit rate and efficacy in finding good small-molecule RNA targets can potentially become the next hot target in pharmaceutical research. This also allows for very specific targeting of particular RNAs in the cell, which can allow for more directed targeting of disease cells. For example, cancerous cells are often treated indiscriminately with healthy cells because the drugs targeting these cells cannot differentiate between them. Inforna’s technology can potentially discover specific drug targets that only target RNA products of specific disease associated genes. This is especially exciting as new technologies in genome sequencing such as Illumina’s $1000 genome are announced.

Innovations in drug discovery are needed in the current pharmaceutical climate, and so any new technique is always welcome in the field. Not only is Inforna effective in discovering a therapeutic small molecule for the treatment of a specific RNA sequence, the researchers who invented the method show that Inforna actually can target a specific RNA that is known to cause cancer. As more genomic information is discovered on various diseases, biotech companies can utilize the Inforna method in their drug development research and even look into RNA research services that are offered by Assay Depot. The promise of finding specific target drugs is always welcome in the pharmaceutical industry!



  1. Velagapudi SP, Gallo SM, Disney MD. Sequence-based design of bioactive small molecules that target precursor microRNAs. Nature Chemical Biology (2014) doi: 10.1038/nchembio.1452.
  2. Clark, Liat. Data mine your DNA to develop better drugs. http://www.wired.co.uk/news/archive/2014-02/10/new-drug-discovery-technique-cancer