Therapeutic Focus

Targeting RNA offers vast potential for treating diseases that cannot be addressed by any other means, and Arrakis’s platforms can be applied across the universe of diseases.

We are developing our own proprietary pipeline in neurology, oncology, and rare genetic disorders based upon the compelling benefits that RNA-targeted small molecules would have in these fields of medicine:

  • Neurology. Pathogenic RNAs have been identified in neurological disorders such as RNA repeat expansion diseases. To abrogate the pathogenicity of these mutant RNAs or any targets in the central nervous system, therapeutics must cross the blood-brain barrier, enter cells, and intervene directly at the target—hurdles reliably surmounted by small molecules.
  • Oncology. Many compelling cancer targets have remained undrugged because their proteins present poor binding opportunities for small molecules and they are intracellular and thus inaccessible to biologics. RNA-targeted small molecules can disrupt these pivotal targets by blocking their translation into protein from RNA.
  • Rare genetic 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 with drugs 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 biotechnology partners. Together with leaders in key therapeutic areas, we will discover RNA-targeted small molecules to treat important diseases.

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