RNA-Small-Molecule Interaction: Challenging the "Undruggable" Tag.

Area of Science:

• Drug Discovery and Development
• Molecular Biology
• Medicinal Chemistry

Background:

• RNA, once considered an
• RNA molecules possess complex structures with binding pockets suitable for small-molecule drugs.
• Dysregulation of RNA is implicated in numerous diseases, highlighting therapeutic potential.

Purpose of the Study:

• To highlight the emerging field of small molecule RNA targeting.
• To showcase RNA as a druggable target for therapeutic intervention.
• To emphasize the potential of RNA-oriented knowledge in accelerating drug discovery.

Main Methods:

• Review of RNA biology and its regulatory functions.
• Analysis of RNA structural features enabling small molecule binding.
• Case studies of RNA-small molecule interactions and functional modulators.

Main Results:

• RNA is transitioning from an
• FDA approval of Risdiplam demonstrates the viability of RNA-targeting drugs.
• RNA's structural complexity and functional diversity offer numerous therapeutic opportunities.

Conclusions:

• Small molecule targeting of RNA represents a promising and expanding area in drug discovery.
• Further understanding of RNA biology will accelerate the development of novel RNA-based therapeutics.
• RNA-targeting drugs offer a new paradigm for treating diseases with unmet medical needs.