Bioanalytics – Chemical
2019 PharmSci 360
This presentation is focused on development of siRNA therapeutics targeting liver-specific diseases. By reduction of undesired gene expression (mRNA knockdown) and consequent lowering of the pathogenic protein, the disease-causing mechanism is interrupted/stopped. Alnylam has designed small interfering RNAs (siRNA) conjugated to a tri-GalNAc ligand which delivers drug specifically to the liver via the asialoglycoprotein receptor (ASGPR). Case studies designed to investigate how alterations of the tri-GalNAc ligand structure and ASGPR expression can affect the PK/PD of the siRNAs will be presented.