Arrakis is on a path to bring powerful new RNA-targeted small molecule (rSM) therapies to millions of patients.

Therapeutic Focus

Targeting RNA offers vast potential for creating medicines with unmatched properties, and Arrakis’ platform can be applied across the universe of diseases.

We are developing our own proprietary pipeline in oncology and genetically validated diseases based upon the compelling benefits that RNA-targeted small molecules would have in these fields of medicine.

  • Oncology. Many compelling cancer targets have remained undrugged because their proteins present poor binding opportunities for small molecules and are located inside the cell and thus inaccessible to biologics. RNA-targeted small molecules can disrupt these pivotal targets by blocking their translation into protein from RNA.
  • Genetically validated diseases. Many genetic diseases result from errors that lead to anomalies in transcription, splicing, or translation—for example, spinal muscular atrophy (SMA). Targeting the flawed protein products directly is often ineffective. In many cases, small molecules that bind RNA offer the prospect of resolving the inborn error by normalizing splicing or by suppressing or promoting translation.

Our strategy anticipates collaborating with pharmaceutical and biotech partners. Together with leaders in key therapeutic areas, we will discover RNA-targeted small molecules to treat important diseases.

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