In today’s oligonucleotide therapeutic development, to make oligonucleotides “druggable” with enhanced PK/PD properties has always been the focus. There are several promising approaches. Chemistry modifications offer the solution with modified oligonucleotides with enhanced characteristics. Conjugation chemistry utilizes peptides, GalNAcs, antibodies, or lipids to improve stability and uptakes. Formulation designs improve the delivery of fragile oligonucleotides, such as lipid nanoparticles (LNP) and Adeno- associated virus vectors (AAV).
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