Title:
ANTISENSE OLIGONUCLEOTIDE DERIVATIVE FOR ATN1
Document Type and Number:
WIPO Patent Application WO/2022/211129
Kind Code:
A1
Abstract:
As an antisense oligonucleotide derivative that can suppress ATN1 gene expression and treat dentatorubral-pallidoluysian atrophy, the present invention provides a single-stranded oligonucleotide or a pharmaceutically acceptable salt thereof in which a first oligonucleotide and a second oligonucleotide are linked by a third oligonucleotide. The first oligonucleotide and the second oligonucleotide each independently comprise 8–30 nucleosides that are independently selected from deoxyribonucleosides, ribonucleosides, and sugar-modified nucleosides, the first oligonucleotide including a sequence that can hybridize with mRNA or pre-mRNA for atrophin 1 and including at least one sugar-modified nucleoside, and the second oligonucleotide including a sequence that can hybridize with at least a portion of the first oligonucleotide. The third oligonucleotide comprises 1–20 nucleosides that are independently selected from deoxyribonucleosides, ribonucleosides, and sugar-modified nucleosides. The first oligonucleotide and the second oligonucleotide hybridize.
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Inventors:
HASHIMOTO SHINJI (JP)
NAITO AKI (JP)
OHTA TOMOKO (JP)
NAGASHIMA KOSUKE (JP)
YAMADA TAKANORI (JP)
IRIYAMA YUSUKE (JP)
NAITO AKI (JP)
OHTA TOMOKO (JP)
NAGASHIMA KOSUKE (JP)
YAMADA TAKANORI (JP)
IRIYAMA YUSUKE (JP)
Application Number:
PCT/JP2022/017009
Publication Date:
October 06, 2022
Filing Date:
April 01, 2022
Export Citation:
Assignee:
SANWA KAGAKU KENKYUSHO CO (JP)
NISSAN CHEMICAL CORP (JP)
NISSAN CHEMICAL CORP (JP)
International Classes:
C12N15/113; A61K31/7088; A61K31/712; A61K31/7125; A61K31/713; A61K47/54; A61K48/00; A61K49/00; A61P25/00; A61P25/28; A61P43/00; C07H21/04; C12N15/11
Domestic Patent References:
| WO2017131124A1 | 2017-08-03 |
Foreign References:
| US20130198877A1 | 2013-08-01 |
Other References:
JIAXIN HU, JING LIU, K JAYAPRAKASH NARAYANANNAIR, JEREMY G LACKEY, SATYA KUCHIMANCHI, KALLANTHOTTATHIL G RAJEEV, MUTHIAH MANOHARAN: "Allele-Selective Inhibition of Mutant Atrophin-1 Expression by Duplex and Single-Stranded RNAs", BIOCHEMISTRY, AMERICAN CHEMICAL SOCIETY, vol. 53, no. 28, 22 July 2014 (2014-07-22), pages 4510 - 4518, XP055166764, ISSN: 00062960, DOI: 10.1021/bi500610r
MELVIN M. EVERS, PEPERS BARRY A., VAN DEUTEKOM JUDITH C. T., MULDERS SUSAN A. M., DEN DUNNEN JOHAN T., AARTSMA-RUS ANNEMIEKE, VAN : "Targeting Several CAG Expansion Diseases by a Single Antisense Oligonucleotide", PLOS ONE, PUBLIC LIBRARY OF SCIENCE, vol. 6, no. 9, 1 January 2011 (2011-01-01), pages e24308 - e24308, XP055007055, ISSN: 19326203, DOI: 10.1371/journal.pone.0024308
MELVIN M. EVERS, PEPERS BARRY A., VAN DEUTEKOM JUDITH C. T., MULDERS SUSAN A. M., DEN DUNNEN JOHAN T., AARTSMA-RUS ANNEMIEKE, VAN : "Targeting Several CAG Expansion Diseases by a Single Antisense Oligonucleotide", PLOS ONE, PUBLIC LIBRARY OF SCIENCE, vol. 6, no. 9, 1 January 2011 (2011-01-01), pages e24308 - e24308, XP055007055, ISSN: 19326203, DOI: 10.1371/journal.pone.0024308
Attorney, Agent or Firm:
TSUKUNI & ASSOCIATES et al. (JP)
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