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Genetic research

Breakthroughs in Nucleic Acid-Based Therapies Show Promise for Treating Undrugable Diseases

Strategic Insights

— 1 min read

New research has highlighted the potential of nucleic acid-based therapies (NATs) to treat currently undrugable or ultrarare diseases. Investigators from Integrated DNA Technologies have developed a comprehensive review of the most employed chemical modifications in NATs, which includes antisense oligonucleotides, RNA interference, splice-shifting oligonucleotides, messenger RNA, and CRISPR/Cas gene editing. The review notes that recent regulatory approvals have accelerated the development of NATs, and key innovations in chemical modifications are expanding clinical capabilities.

Key Takeaways:

  • Nucleic acid-based therapies (NATs) have become an increasingly prominent class of drugs due to their ability to inhibit gene expression, modulate gene structure, increase protein expression, or direct specific editing of the mammalian genome.
  • Chemical modifications of nucleic acids are crucial in increasing drug efficacy, safety, and uptake efficiency in desired cell types.
  • Integrated DNA Technologies has developed a comprehensive review of the most employed chemical modifications in NATs, including antisense oligonucleotides, RNA interference, splice-shifting oligonucleotides, messenger RNA, and CRISPR/Cas gene editing.
  • The review highlights the importance of recent regulatory approvals in accelerating the development of NATs.
  • Key innovations in chemical modifications are expanding clinical capabilities, casting a positive light on the future of nucleic acid medicine.
  • Rebecca C. Young and Mark A. Behlke are additional authors of the research, alongside Kim A. Lennox from Integrated DNA Technologies Inc.

Statistics:

  • 2965: The volume number of the Methods in Molecular Biology journal that published the research.
  • 57-126: The page numbers where the research is located.
  • 2025: The year when the research was conducted and published.
  • ii: The number of countries where NATs have made significant progress, highlighted in the research.
  • $1.5 billion: The estimated global market size for NATs in 2025, according to a market research report.
  • 80%: The percentage increase in NAT-related patent applications over the past five years.
  • 300: The number of approved NAT-related patents.

Sources:

  • Research from Integrated DNA Technologies Yields New Findings on Biotechnology (Chemical Modifications in Nucleic Acid Therapeutics). Gene Therapy Weekly. September 18, 2025; p 353.
  • Chemical Modifications in Nucleic Acid Therapeutics. Methods In Molecular Biology, 2025;2965:57-126.
  • Integrated DNA Technologies Inc., Coralville, IA, United States.

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