UNDERSTAND HOW TO BEST DESIGN OLIGONUCLEOTIDE CONJUGATES FROM SIMPLE STARTING BUILDING BLOCKS TO THE FULL OLIGONUCLEOTIDE CONJUGATE

This course will focus on the exciting developments in the field of targeted oligonucleotide conjugates. We discuss why oligonucleotide conjugates are in demand and when they are specifically needed. We discuss standard ligands (GalNac) as well more recently pursued ligand opportunities (lipid, peptide, antibody, small molecule) to target the oligonucleotide of choice (ASO, SSO, siRNA) to the tissue of interest.

This course discusses how to select the right chemistry strategy to conjugate your selected ligand to the correct position on the oligonucleotide of choice, which is the key to maximizing its biological potential. The course will also discuss what to do once you select your conjugation strategy including which “toolbox” items you will need to put the correct linker in place (which allows the conjugate to remain intact). And which tools you will need to facilitate where and when the linker should disengage.