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Target-Directed Approaches for Screening Small Molecules against RNA Targets.

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Small molecule targeting of RNA is a promising therapeutic strategy. Recent advances in chemical collections and target-directed approaches are overcoming challenges in RNA drug discovery.

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Area of Science:

  • Biochemistry
  • Medicinal Chemistry
  • Molecular Biology

Background:

  • RNA molecules play crucial roles in cellular functions and disease.
  • RNA presents unique challenges and opportunities as a small-molecule drug target.
  • Historically, targeting RNA with small molecules has faced hurdles in affinity, selectivity, and dynamics.

Purpose of the Study:

  • To review target-directed approaches for identifying RNA-binding compounds.
  • To discuss the current chemical knowledge of small-molecule RNA binders.
  • To highlight the evolution of small molecule-RNA targeting strategies.

Main Methods:

  • Review of recent literature on small molecule-RNA interactions.
  • Analysis of target-directed approaches in RNA drug discovery.
  • Discussion of chemical collections and their role in industrial development.

Main Results:

  • Significant progress has been made in small molecule-RNA targeting over the past 5-10 years.
  • Selective RNA-protein complex stabilizing drugs (e.g., branaplam, risdiplam) are in clinical trials for splicing modulation.
  • Phenotypic screening has led to serendipitous discoveries of RNA-targeting compounds.

Conclusions:

  • Developing RNA as a druggable target necessitates a target engagement-driven approach.
  • Evolving chemical collections is crucial for the industrial development of RNA-targeted therapeutics.
  • Further research into target-directed strategies and chemical knowledge will advance RNA-based drug discovery.