rSM PROGRESS. DESTINATION PATIENTS.
Arrakis is on a path to bring powerful new RNA-targeted small molecule (rSM) therapies to millions of patients.

Therapeutic Potential

Arrakis’ 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 drugged protein targets. We believe our approach will dramatically expand the universe of opportunities for new drug discovery and new treatments for patients.

 

 

 

Destination patients

Derived from blog
Arrakis achieves escape velocity
April 18, 2019

Now, thanks to the remarkable and dedicated effort of the intrepid team at Arrakis and the support of our visionary investors, we are poised to realize the promise of bringing RNA into play for small-molecule drug discovery. We have a burgeoning list of targets that we are truly excited about. Many of these targets are in cancer – targets whose role in disease has been known for decades but have remained out of reach of medicine because their proteins are not druggable. Other important new targets have emerged from genetic and genomic screens for driver and synthetic lethal genes. We’re also interested in targets in other therapeutic areas that are highly validated genetically or clinically, but basically undruggable any other way.

In just two years, Arrakis has built an end-to-end platform for discovering rSMs, RNA-targeted small molecules. We can now begin to make good on our vision of a world in which all RNA biology can be accessed to create new medicines and reach our destination of bringing rSMs to patients.

 

Destination patients

Derived from blog
Arrakis achieves escape velocity
April 18, 2019

Now, thanks to the remarkable and dedicated effort of the intrepid team at Arrakis and the support of our visionary investors, we are poised to realize the promise of bringing RNA into play for small-molecule drug discovery. We have a burgeoning list of targets that we are truly excited about. Many of these targets are in cancer – targets whose role in disease has been known for decades but have remained out of reach of medicine because their proteins are not druggable. Other important new targets have emerged from genetic and genomic screens for driver and synthetic lethal genes. We’re also interested in targets in other therapeutic areas that are highly validated genetically or clinically, but basically undruggable any other way.

In just two years, Arrakis has built an end-to-end platform for discovering rSMs, RNA-targeted small molecules. We can now begin to make good on our vision of a world in which all RNA biology can be accessed to create new medicines and reach our destination of bringing rSMs to patients.

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