About Us

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.

The company is located in Waltham, MA and backed by investors with unsurpassed experience in building pioneering biopharmaceutical companies.

Back to Top