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Title:
HETERODUPLEX NUCLEIC ACID MOLECULES AND USES THEREOF
Document Type and Number:
WIPO Patent Application WO/2019/136180
Kind Code:
A3
Abstract:
Disclosed herein are heteroduplex nucleic acid molecules, heteroduplex nucleic acid conjugates, and pharmaceutical compositions for modulating a protein expression. Also described herein include methods of treating a disease or indication which utilize a heteroduplex nucleic acid molecule, a heteroduplex nucleic acid conjugate, or a pharmaceutical composition that comprises a heteroduplex nucleic acid molecule.

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Inventors:
GEALL ANDREW JOHN (US)
DOPPALAPUDI VENKATA RAMANA (US)
JOHNS RACHEL ELIZABETH (US)
BURKE ROB (US)
CHU DAVID SAI-HO (US)
COCHRAN MICHAEL CARAMIAN (US)
HOOD MICHAEL DAVID (US)
HUANG HANHUA (US)
Application Number:
PCT/US2019/012223
Publication Date:
August 29, 2019
Filing Date:
January 03, 2019
Export Citation:
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Assignee:
AVIDITY BIOSCIENCES LLC (US)
International Classes:
A61K31/713; A61K31/712; A61K31/7125; C07H21/02; C12N15/113
Foreign References:
US20170281795A12017-10-05
US20160186185A12016-06-30
US20160376577A12016-12-29
US9809817B22017-11-07
EP0532423A11993-03-17
US20170051286A12017-02-23
Other References:
ELKAYAM, E ET AL.: "siRNA carrying an (E)-vinylphosphonate moiety at the 5' end of the guide strand augments gene silencing by enhanced binding to human Argonaute-2", NUCLEIC ACIDS RESEARCH, vol. 45, no. 6, 28 November 2016 (2016-11-28), pages 3528 - 3536, XP055448298
MILLER, CM ET AL.: "Stabilin-Mediated Cellular Internalization of Phosphorothioate-Modified Antisense Oligonucleotides (ASOs", 2016, pages 1 - 3, XP055632390, Retrieved from the Internet [retrieved on 20190423]
AUGUSTYNS, K ET AL.: "Incorporation of hexose nucleoside analogues into oligonucleotides: synthesis, base-pairing properties and enzymatic stability", NUCLEIC ACIDS RESEARCH, vol. 20, no. 18, 25 September 1992 (1992-09-25), pages 4711 - 4716, XP002024092
COLLIS, AEC: "The synthesis of vinylphosphonate-linked RNA", PHD THESIS, February 2008 (2008-02-01), pages 1 - 262, XP055632400, Retrieved from the Internet [retrieved on 20190423]
Attorney, Agent or Firm:
ZHANG, Adrianna P. (US)
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