Drug Target Strategies
The identification of drug-like small molecule medicines that directly bind to RNA and modulate the biological function of RNA will vastly increase our therapeutic target space. RNA folds into structures that have diverse pockets into which small molecules can selectively bind. At Arrakis Therapeutics, our main focus is to identify small molecules that selectively bind RNA structures in critical regulatory regions of mRNA to modulate the expression of proteins that are otherwise undruggable. Our platform enables the analysis of RNA folding in vitro and in vivo as well as the identification of structures and pockets within an RNA. Drug-like compounds that bind these pockets are identified through high-throughput screening, followed by diverse biophysical assays to confirm and characterize binding such as SEC-MS, SPR and NMR. The biological impact of these compounds is evaluated in cell-based assays. The discovery of compounds that act via RNA binding is a new approach that has the potential to address previously intractable molecular targets and diseases.