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RNA-Targeted Drug Discovery Summit to Address Key Challenges in Small Molecule Development

• Leading biopharmaceutical executives and researchers gather at the 3rd RNA-Targeted Drug Discovery Summit to advance RNA-targeting small molecule therapeutics development and improve patient outcomes.

• The summit aims to tackle critical challenges in RNA drug discovery, including RNA structure determination, target selection optimization, and translation into clinical development.

• Industry leaders from Scripps Research Institute, Anima Biotech, and Arrakis Therapeutics will share insights on enhancing medicinal chemistry approaches and RNA biology understanding for robust therapeutic development.

The landscape of drug discovery is witnessing a paradigm shift as the 3rd RNA-Targeted Drug Discovery Summit emerges as the definitive gathering for experts focused on RNA-targeting small molecule development. This comprehensive event arrives at a crucial time when the field is moving beyond traditional protein-centric approaches to explore the vast potential of RNA-targeted therapeutics.

Advancing RNA-Targeted Drug Development

The summit brings together an elite group of biopharmaceutical directors, VPs, and C-level executives to address fundamental challenges in RNA-targeted drug discovery. Recent scientific breakthroughs in understanding RNA biology and function have opened new possibilities for developing highly selective small molecules, challenging the long-held belief that targeting RNA is an insurmountable task.

Key Scientific Focus Areas

Leading researchers from Storm Therapeutics, Accent Therapeutics, and Saverna Therapeutics will present innovative approaches to targeting RNA through RNA-protein interactions and epitranscriptomics. The summit will feature expertise from prestigious institutions including Yale University and the Whitehead Institute for Biomedical Research, focusing on expanding knowledge of RNA structure and its role in disease progression.

Overcoming Technical Challenges

Scientists from Roche, University of California San Diego, and Novartis will address critical technical hurdles in the field, including:
  • Binding optimization for small molecules targeting RNA
  • Selectivity improvements
  • Solutions for drug-like property challenges

Therapeutic Translation and Validation

PTC Therapeutics and Duke University researchers will share insights on identifying and validating viable RNA target sites for therapeutic intervention. The summit emphasizes translational aspects, focusing on developing practical approaches that can advance from laboratory discoveries to clinical applications.

Industry Collaboration and Innovation

The event highlights the importance of collaborative efforts in advancing RNA-targeted therapeutics. Experts from The Scripps Research Institute, Anima Biotech, and Arrakis Therapeutics will present strategies for optimizing medicinal chemistry insights and enhancing understanding of RNA biology to improve therapeutic translation.
This summit represents a crucial platform for driving forward the field of RNA-targeted small molecule therapeutics, with the ultimate goal of developing innovative treatments that can significantly improve patient outcomes.
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3rd RNA-Targeted Drug Discovery Summit
pharmaphorum.com · May 12, 2025
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