Arrakis Therapeutics to Target RNA with Small-Molecule Medicines

Waltham-based Arrakis Therapeutics has completed a $38 million Series A financing led by Canaan Partners with participation by Pfizer, Celgene, and biotech industry leader Henri Termeer, among others. Arrakis was founded with a vision to create a proprietary, transformational discovery platform that identifies small-molecule drugs that act directly on disease-causing RNA.  The company was established in 2015 with seed funding provided by Advent Life Sciences and Henri Termeer.

The proceeds from the Series A financing will be used to advance Arrakis’ proprietary discovery platforms, comprised of a high-throughput, comprehensive system of bioinformatics tools, assays and chemical libraries that can identify new RNA targets and create new small-molecule drugs. Using these platforms, Arrakis is developing an internal product pipeline of RNA-targeted small molecules focused on neurology, oncology, and rare genetic disorders. The basis of Arrakis’ unique approach is a proprietary chemical biology technology that interrogates structures of folded RNAs in their native state, within cells, to enable discovery and rational design of potent, selective small-molecule inhibitors of RNA function.

“RNA is the locus of most human biology, yet our current pharmacopeia is largely limited to protein targets,” said Dr. Michael Gilman, newly-named Arrakis Chairman and Chief Executive Officer. “Arrakis intends to re-architect small-molecule drug discovery by redirecting, modifying, and creating tools that enable medicinal chemistry to directly address therapeutically important RNA molecules. Our substantial intellectual property, financial resources, and the extensive experience of our team in discovering and developing novel drugs will enable us to build a pipeline of new small-molecule therapeutics for patients not helped by today’s medicines.”

“The importance of targeting RNA in disease is well recognized,” said Russell C. Petter, Ph.D., founder and CSO of Arrakis Therapeutics. “But until now, targeting RNA with small-molecule compounds has been thought to be too challenging. Nevertheless, there are a number of precedents for conventional small molecules that bind and modulate RNA, including several approved drugs. These molecules were all discovered in functional screens, and only later did we learn that they acted on RNA. Our goal is to intentionally discover RNA-modulating small-molecule medicines. We believe that new developments in informatics, structural biology, and biophysical tools now make that possible.” (Source: Arrakis Therapeutics Website, February 27, 2017)