Expanding the Clinical Applications of Oligonucleotide Therapies: From Rare Diseases to Broader Indications
26 Jun 2025
New Modalities
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Lessons from approved oligonucleotide therapies (e.g., for SMA and DMD) and their potential in cardiometabolic, inflammatory, and oncology indications
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Innovations in extrahepatic delivery, crossing the blood-brain barrier, and improving cellular uptake for broader therapeutic applications
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Addressing clinical trial design, safety profiling, and cost-effectiveness to drive the adoption of oligonucleotide drugs in larger patient populations