Therapeutic Potential

Arrakis’s transformative approach to small-molecule drug discovery enables targeting RNA and opens up a vast new domain for discovering new medicines.

Too often, dramatic breakthroughs in our understanding of disease cannot be translated into therapies for patients because the protein target is not druggable with today’s drug discovery toolkit. Unlike conventional approaches that focus on the protein itself, rSMs are designed to modulate the expression level of proteins by impacting processing, transport, stability, or translation of their encoding RNAs. By selectively binding to certain, predictable structures in functionally important regions in the RNA, rSMs can increase or decrease the levels of the target protein to treat disease.

Beyond today’s well-studied and validated protein targets, rSMs have the therapeutic potential to address the entire human transcriptome, the set of all RNAs transcribed from DNA, which is hundreds of times larger than the set of protein drug targets. We believe our approach will dramatically expanding the universe of opportunities for new drug discovery and new treatments for patients.

We are screening thousands of RNA targets to deeply explore chemical and RNA structure space. We are building a unique understanding of the principles of RNA-small molecule interaction to select the most promising target/hit combinations to advance as therapeutic programs.

 

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