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Advancing Oligonucleotide Therapeutics: From Molecule Design to Precision Delivery

26 Sep 2025
Translational & Clinical Strategy
  • Explore the latest advancements in oligo-based therapeutics including ASOs, siRNA, gRNA, miRNA, aptamers, and mRNA platforms.
  • Examine next-generation delivery systems—from LNPs and AAVs to targeted conjugates—for improved biodistribution and tissue specificity.
  • Address key challenges in stability, immunogenicity, and scalability to optimize clinical translation of oligo therapies.
Industry Expert
Janaiah Kota, Executive Director, Molecular Therapeutics - Ultragenyx