About Us

Arrakis Therapeutics is a biopharmaceutical company pioneering the discovery of small-molecule medicines that directly bind and modify the biological function of RNA to treat disease. This new class of medicines, RNA-targeted small molecules (rSMs), can unlock the therapeutic potential of deeply researched, valuable targets that are not accessible with today’s medicines. Moreover, by interrogating the vast and largely unexplored transcriptome we can identify new disease intervention points that may be exploited by rSMs.

Arrakis is developing a platform integrating leading-edge RNA bioinformatic and structural tools, RNA-specific chemical and biological assays, and traditional medicinal chemistry to produce a pipeline of rSMs. Arrakis’s rSM discovery platform will enable the identification of tractable structural elements with an RNA, the assessment of the biological impact of binding to that element and the design of drug candidates to treat a range of disease including neurology, oncology and rare genetic diseases.

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 is backed by investors with unsurpassed experience in building pioneering biopharmaceutical companies.


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