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Title:
LIGAND COMPOUNDS, CONJUGATES, AND APPLICATIONS THEREOF
Document Type and Number:
WIPO Patent Application WO/2022/155814
Kind Code:
A1
Abstract:
This disclosure features chemical entities (e.g., a compound or a pharmaceutically acceptable salt, and/or hydrate, and/or cocrystal, and/or drug combination of the compound) that comprise one or more ligand moieties for an asialoglycoprotein receptor (ASGPR). Exemplary chemical entities can further comprise an oligonucleotide. Said chemical entities are useful, e.g., in the targeted delivery of oligonucleotides to liver cells (e.g., liver parenchymal cells). The chemical entities are useful e.g., in the treatment of conditions or diseases caused by the expression (e.g., abnormal expression) of one or more genes in liver cells This disclosure also features compositions containing the same as well as methods of using and making the same.

Inventors:
ZHANG BILL BILIANG (CN)
ZHAO HAOTING (CN)
Application Number:
PCT/CN2021/072887
Publication Date:
July 28, 2022
Filing Date:
January 20, 2021
Export Citation:
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Assignee:
ARGORNA PHARMACEUTICALS LTD (CN)
International Classes:
A61K31/7028; A61K31/7115; A61K31/713; A61K47/54; A61K48/00; A61P1/16; C07H13/06; C07H13/10
Domestic Patent References:
WO2019127004A12019-07-04
WO2016152980A12016-09-29
Foreign References:
CN110846320A2020-02-28
Other References:
SHIGEO MATSUDA, KRISTOFER KEISER, JAYAPRAKASH K. NAIR, KLAUS CHARISSE, RAJAR M. MANOHARAN, PHILIP KRETSCHMER, CHANG G. PENG, ALEXA: "siRNA Conjugates Carrying Sequentially Assembled Trivalent N- Acetylgalactosamine Linked Through Nucleosides Elicit Robust Gene Silencing In Vivo in Hepatocytes", ACS CHEMICAL BIOLOGY, vol. 10, no. 5, 15 May 2015 (2015-05-15), pages 1181 - 1187, XP055448305, ISSN: 1554-8929, DOI: 10.1021/cb501028c
See also references of EP 4153186A4
Attorney, Agent or Firm:
LINDA LIU & PARTNERS (CN)
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