Elucidation of the Biotransformation Pathways of a Galnac3-conjugated Antisense Oligonucleotide in Rats and Monkeys

• Main Authors: Colby S Shemesh (Author), Rosie Z Yu (Author), Hans J Gaus (Author), Sarah Greenlee (Author), Noah Post (Author), Karsten Schmidt (Author), Michael T Migawa (Author), Punit P Seth (Author), Thomas A Zanardi (Author), Thazha P Prakash (Author), Eric E Swayze (Author), Scott P Henry (Author), Yanfeng Wang (Author)
• Format: Book
• Published: Elsevier, 2016-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Triantennary N-acetyl galactosamine (GalNAc3) is a high-affinity ligand for hepatocyte-specific asialoglycoprotein receptors. Conjugation with GalNAc3 via a trishexylamino (THA)-C6 cluster significantly enhances antisense oligonucleotide (ASO) potency. Herein, the biotransformation, disposition, and...