At Arrakis, we are on an expedition to unlock the biology of RNA to discover new medicines for millions of people. We are discovering ways to target the RNA lifecycle – which is upstream of all biology – to find new points of therapeutic intervention. Our drug discovery tools are inventive yet practical, as we have re‑engineered the pharmaceutical industry’s small-molecule drug discovery toolkit to be directed at RNA. As a result, we have pioneered industrial-scale capabilities to identify RNA-targeted small molecules (rSMs) that directly bind to and modulate the function of RNA to predictably impact the biology of disease processes.
We have a keenly focused vision for developing a pipeline of rSM medicines. We are discovering rSMs that target deeply researched, valuable protein drug targets deemed “undruggable” because they are not accessible with today’s conventional medicines. By discovering rSMs that compromise the function of key mRNAs and pre-mRNAs, our medicines work “upstream” at new intervention points and prevent the expression of these known protein drug targets. Our pipeline programs include several exciting targets, including in cancer, which is rich with opportunity and challenging targets, and in rare genetic diseases, as well as cardiovascular disease. Over time, our approach to discovering rSM medicines can expand into the vast realms of disease where RNA plays a role.
Arrakis brings together a team of trailblazers in drug discovery and company building. Our leaders include scientific experts in biology, chemistry, and RNA structure who work alongside a highly experienced management team with a track record of building successful biotechnology companies and developing novel therapeutics. The company is headquartered in Waltham, MA and backed by investors with deep experience fostering life sciences companies with disruptive technologies.