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Company Profile Report

RNA, meet small molecules

How Mike Gilman's new company is expanding small molecule targets to RNA

Arrakis Therapeutics Inc.’s unveiling this week marks another notch for RNA, this time not as a modality to treat disease but as a route to expanding the world of druggable targets for small molecules by more than an order of magnitude. The company believes the goal of using small molecules to target RNA, once considered impossible by medicinal chemists, is now close enough in reach to create a viable platform and sustainable pipeline.

On Monday, Arrakis announced the closing of a $38 million series A round led by Canaan Partners, and the appointment of industry veteran Michael Gilman as its chairman and CEO. The company intends to develop therapies for cancer, CNS diseases and rare genetic conditions.

Most approaches to developing therapeutics against RNA employ antisense oligos or RNAi.

However Gilman, who previously founded Padlock Therapeutics Inc. and Stromedix Inc., believes RNA-binding small molecules represent a coveted therapeutic class that opens up a pantheon of new possibilities for traditional drug development.

"The infrastructure in our industry, physical and intellectual,

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