rSM Platform

We start with the knowledge that RNA has a three-dimensional structure with predictable substructures that influence the lifecycle of RNA – from transcription, processing, transport and translation to degradation. To identify these substructures and prosecute the RNA targets, Arrakis has developed a comprehensive drug discovery toolkit in two realms:

• Where possible, we have adapted the conventional tools of small molecule drug discovery to address RNA targets, rather than traditional proteins.
• Where needed, we have invented new tools to establish a broad structure-based drug design platform for RNA.

The result is an end-to-end RNA-targeted small molecule (rSM) drug discovery platform to identify small molecules that bind to and modulate RNA to predictably impact the RNA lifecycle and the downstream disease-related biology.

The value of the rSM Platform is embodied in our proprietary database of hundreds of RNA targets whose structures and ligand-accessible substructures have been determined in dozens of different human cell lines. Each target is annotated with in-house biological data and prioritized for screening with the potential to initiate dozens of new therapeutic programs per year.

Our End-to-End Platform

Our proprietary platform for the systematic discovery and design of rSMs integrates leading‐edge RNA bioinformatics and chemical biology tools, RNA‐specific chemical and biological assays, and RNA-directed medicinal chemistry. By leveraging the best existing tools with Arrakis’ exclusive technologies, we can, for the first time on an industrial scale, identify small molecules that modulate RNA function and predictably impact important biology in disease processes.