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Drugging RNA

Small molecules that target messenger RNA have therapeutic potential, but the field still lacks an unqualified success. Companies differ on how to move the resurgent field forward.

In January, Atomic AI, with a $35 million A round, joined a short but growing list of companies seeking to drug RNA using small molecules. As the name suggests, the company is applying artificial intelligence to predicting RNA structures.

Venture capitalists are not alone in their interest in the RNA-targeted small-molecule approach. Pharma deals have been picking up: Merck & Co., Vertex Pharmaceuticals, Sanofi, Amgen, Celgene, Genentech and Janssen Pharmaceuticals have all recently partnered with biotechs in this space (Table 1). Peter Smith, co-founder and CEO of Remix Therapeutics, which also enjoyed some funding in the last year, calls RNA “the next frontier of small-molecule drug discovery.”


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