Development of targeted siRNA delivery technologies by saccharide ligand conjugates

DOI Web Site 21 References
  • Uehara Keiji
    Modality Research Laboratories 1, Research Unit, R&D Division, Kyowa Kirin Co., Ltd.

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  • 糖鎖リガンドによるsiRNAのデリバリー技術の開発

Abstract

Nucleic acid-based medicines such as small interfering RNA（siRNA） and antisense oligonucleotides（ASOs） have great potential to address undruggable targets that use classical small-molecule approaches or traditional biologics. GalNAc-ligand-conjugated oligonucleotides, which targets the asialoglycoprotein receptor（ASGPR） on hepatocytes, is the most validated for targeted delivery of oligonucleotides, have been under evaluation in clinical trials for modulation of various liver-disease-related genes and includes several FDA-approved drugs. Given the success of GalNAc–oligonucleotides, ligand-conjugated oligonucleotides outside the liver are expected to create the novel drugs. However, receptor-mediated targeted delivery outside the liver using ligand-conjugated oligonucleotides remains challenging. We have investigated targeted delivery of siRNA to extrahepatic tissues/cells using glycan conjugated siRNA because we focused on unique binding properties between ligand and receptor result in enhancement of uptake amount of payloads into the cells. In this paper, we introduce a case study.

Journal

  • Drug Delivery System

    Drug Delivery System 38 (4), 313-320, 2023-09-25

    THE JAPAN SOCIETY OF DRUG DELIVERY SYSTEM

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