DIA/FDA Oligonucleotide-Based Therapeutics Conference

Single and double stranded oligonucleotide therapeutics share the challenge of limited uptake into many cell types of therapeutic interest. This restricted cellular uptake is one of the major hurdles to solve before oligonucleotides can be utilized to their full potential. One way to increase productive uptake is to hitchhike on receptors that internalize ligands, best exemplified by GalNAc-conjugated oligonucleotides to increase uptake into hepatocytes via the asioalyglycoprotein receptor. This session will cover new DMPK understanding of GalNAc conjugated siRNA and antisense oligonucleotides like parameters driving potency, bioavailability, and intracellular trafficking as well as aspects of receptor saturation studied in preclinical models and the clinic. The session will also cover novel strategies of targeted delivery of miR-29 mimics to the lung as well as delivery to muscle using antibody-conjugates targeting the transferrin receptor.