Arrakis Therapeutics is a biopharmaceutical company pioneering the discovery of a new class of medicines. Our goal is to interrogate and exploit the vast and largely unexplored transcriptome—the full collection of RNAs transcribed from DNA—to discover small-molecule medicines that target RNA at key disease intervention points. Arrakis is developing a product engine to produce a pipeline of RNA-targeted small-molecule medicines focused in neurology, oncology, and rare genetic disorders. Arrakis has designed two scientific platforms—TRYST™ and PEARL-seq™—to help unlock this unexplored biological space by enabling us to identify tractable RNA targets, design drug molecules, and select candidates for clinical development. A major current focus of our effort is mRNA, especially those that encode therapeutically compelling but “undruggable” proteins, and especially when an effective medicine requires oral bioavailability, blood-brain barrier transport, or frequent dosing. These attributes are difficult to achieve with current RNA-targeted therapies. Arrakis has brought together scientific leaders in RNA structure, chemistry, and biology to work alongside a highly experienced management team with a track record of building transformative biopharmaceutical companies and developing novel therapeutics.