C&EN White Paper
Oligonucleotide therapeutics: Drug discovery through tightly coupled chemistry and biology
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Oligonucleotide-based therapeutics are set to transform the landscape of treatable human disease. Many traditional pharmacological treatments use small molecules to directly modulate the activity of proteins involved with disease. Yet only about 25% of disease-related proteins have been drugged to date, and the vast space of the human genome provides a variety of biochemical mechanisms to target for treating disease.

Nucleic acid therapies act before protein production, provide instructions for functional proteins, or initiate degradation. While creating oligonucleotide therapeutics is theoretically as straightforward as a sequence of chemical building blocks, designing effective treatments for clinical applications involves understanding both the chemistry and biology of nucleic acids.

Key Objectives:
  • Classes of oligonucleotide therapeutics and their mechanisms of action
  • Chemical modifications for oligonucleotide therapeutics that provide stability and selectivity
  • Biological testing to inform future clinical tests
  • Bioanalytical platforms that address the challenges of quantifying oligonucleotides due to their unique physicochemical properties

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