PLATFORM
The Power of Our Platform: rSM Toolkit
A robust toolkit to industrialize the discovery of rSMs
Since our launch in 2017, Arrakis has committed to deeply understanding RNA sequence, structure and function. From this commitment, we have developed a comprehensive toolkit to identify RNA substructures within RNA and prosecute the RNA targets:
- 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 a robust RNA-targeted small molecule (rSM) discovery workflow to identify small molecules that selectively and potently bind specific RNA substructures and form the basis of rSM modalities that interrupt RNA structure-function to treat disease.
We have built a proprietary database of hundreds of RNA targets whose structures and ligand-accessible substructures have been determined in dozens of different human cell lines. By integrating these data with available knowledge of biologic function, we prioritize targets for screening with the potential to initiate dozens of new therapeutic programs per year.
rSM structure-based discovery toolkit
Our toolkit industrializes the systematic discovery of rSMs and utilizes 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 selectively and potently bind RNA and determine the best path to impact important biology in disease processes.
| Target Identification |
| Target Validation |
| Screening and Confirmation |
| Hit to Lead |
Sequence to Structure
Structure to Biology
Identification of Chemical Matter
Cellular Activity and Chemical Biology
