Arrakis Therapeutics Biological methodology to identify RNA – targeted small molecules and Technologies behind the research

About Arrakis Therapeutics Mission

Arrakis Therapeutics is a biotechnology company co-founded in 2015 by Chief Executive Officer – Michael Gilman who is a scientist, general manager, biotech executive, and serial entrepreneur.  The aim of Arrakis therapeutics which is based at Waltham, Massachusetts it to develop drugs for neurological disorders and other diseases.

Arrakis Therapeutics Mission

Ribonucleic acid (RNA) is a polymeric molecule fundamental in various biological roles such as coding, decoding, regulation, and expression of genes.  This team is 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 said to be the demanding of all-natural science to find new points of therapeutic intervention. Arrakis re-engineered the pharmaceutical industry’s small-molecule drug discovery toolkit which is directed at RNA. And thus drug discovery tools are said to be original and practical. As a result, we have to lead the way industrial-scale capabilities to identify RNA-targeted small molecules (rSMs) that directly bind to and modulate the function of RNA to unavoidably force the biology of disease course of action.


Arrakis has a simple but powerful vision towards extending small-molecule medicines into the new kingdom of biology by unlocking medicine where it’s building the existing complicated drug discovery toolkit which is developed for protein targets.  They are initiating new areas, with an expedition led by a team of skilled scientists and drug development leaders who are consistently breached barriers and blazed new trails in search of solutions. They are now suspended to comprehend the promise of bringing RNA into play for small-molecule drug discovery.

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They believe that by targeting RNA biology in new ways will have the potential to impact millions of patients.  This biology of healthy and disease-affected cells is often mediated by RNA structures, which offers nearly unlimited potential as a target for small-molecule chemical probes and lead medicines.  This point of view discusses molecular recognition of RNA by small molecules and highlights key enabling technologies and properties of bioactive interactions.

Research method

  • In developing the small molecules interacting with RNA (SMIRNAs) to capture their major prospect at the connection of chemistry, biology, and medicine the following methodology is used.
  • By design, bioactive compounds that modulate RNA targets and companion methods that validate their direct effects in cells and pre-clinical models.
  • The demonstration of direct target engagement in the complex cellular milieu, along with methods to establish modes of action, is required to push this field forward.
  • By describing frameworks for accelerated advancements in the burgeoning area, the key new technologies for the development of SMIRNAs, and milestones that have led to broader acceptance of RNA as a small molecule druggable target.
  • Sequence-based design of a molecule attached to a metal atom by coordinate bonding called ligands targeting RNA has established rules for disturbing RNA targets and provides a potentially general platform for the innovation of bioactive small molecules.

These growing technologies suggest that the time is right to provide small molecule chemical probes to target functionally relevant RNAs throughout the human transcriptome.

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