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Expanding the Clinical Applications of Oligonucleotide Therapies: From Rare Diseases to Broader Indications

26 Jun 2025
New Modalities
  • Lessons from approved oligonucleotide therapies (e.g., for SMA and DMD) and their potential in cardiometabolic, inflammatory, and oncology indications

  • Innovations in extrahepatic delivery, crossing the blood-brain barrier, and improving cellular uptake for broader therapeutic applications

  • Addressing clinical trial design, safety profiling, and cost-effectiveness to drive the adoption of oligonucleotide drugs in larger patient populations

Industry Expert
Debasis Patra, Vice President, Head of CMC - Olix Pharmaceuticals