Arrakis bolsters RNA-targeting tech with Penn license

Arrakis CEO Michael Gilman.

Arrakis Therapeutics has licensed technology from the University of Pennsylvania that enables the targeting of RNA with small molecules. The deal gives Arrakis rights to families of compounds that bind to three-way junctions found in folded RNA.

Waltham, Massachusetts-based Arrakis emerged earlier this year with a $38 million A round and plans to search for small molecules that bind RNA. That focus puts Arrakis into largely virgin drug discovery territory with huge potential. But to realize that potential it must go after macromolecules previously dismissed as undruggable.

The University of Pennsylvania agreement gives Arrakis another tool to apply to that tough task. 

David Chenoweth, Ph.D., a University of Pennsylvania associate professor and scientific advisor to Arrakis, is behind the IP picked up by the startup. Chenoweth’s work centers on molecules that bind to three-way junctions, nucleic acid structures found in folded RNA. 

Research into these structures dates back more than a decade. But efforts to create small molecule platforms capable of targeting the structures are only just starting to bear meaningful fruit. 

“The lifecycle of RNA is very complex and we are just beginning to understand and predict the different structural components that impact the production of proteins from mRNA,” Chenoweth said. “Our team has just begun to elucidate the role of three-way junctions and how they may open a new avenue for the discovery of small molecules that bind to RNA and intervene in disease processes.”

Chenoweth’s work will slot in alongside Arrakis’ own attempts to drug RNA. The biotech has been plugging away on the idea since 2015 in a belief that ever-expanding knowledge of the structure of RNA, specifically its loops and pockets, is creating opportunities to drug the macromolecule.

If Arrakis succeeds, a vista of therapeutic opportunities will open up.