JP7511563B2 - Ｃｌｎ３の発現を調節するための化合物及び方法 - Google Patents

Ｃｌｎ３の発現を調節するための化合物及び方法 Download PDF

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Publication number
JP7511563B2
JP7511563B2 JP2021538176A JP2021538176A JP7511563B2 JP 7511563 B2 JP7511563 B2 JP 7511563B2 JP 2021538176 A JP2021538176 A JP 2021538176A JP 2021538176 A JP2021538176 A JP 2021538176A JP 7511563 B2 JP7511563 B2 JP 7511563B2
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modified
cln3
oligomeric compound
modified oligonucleotide
oligonucleotide
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Japanese (ja)
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JP2022500079A5 (https=
JP2022500079A (ja
JPWO2020055917A5 (https=
Inventor
ヘイスティングス，ミシェル・エル
リゴ，フランク
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Inorganic Chemistry (AREA)
JP2021538176A 2018-09-10 2019-09-10 Ｃｌｎ３の発現を調節するための化合物及び方法 Active JP7511563B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862729067P 2018-09-10 2018-09-10
US62/729,067 2018-09-10
US201962891127P 2019-08-23 2019-08-23
US62/891,127 2019-08-23
PCT/US2019/050476 WO2020055917A1 (en) 2018-09-10 2019-09-10 Compounds and methods for modulating cln3 expression

Publications (4)

Publication Number Publication Date
JP2022500079A JP2022500079A (ja) 2022-01-04
JP2022500079A5 JP2022500079A5 (https=) 2022-09-21
JPWO2020055917A5 JPWO2020055917A5 (https=) 2022-09-21
JP7511563B2 true JP7511563B2 (ja) 2024-07-05

Family

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Application Number Title Priority Date Filing Date
JP2021538176A Active JP7511563B2 (ja) 2018-09-10 2019-09-10 Ｃｌｎ３の発現を調節するための化合物及び方法

Country Status (4)

Country Link
US (1) US20220280545A1 (https=)
EP (1) EP3849564A4 (https=)
JP (1) JP7511563B2 (https=)
WO (1) WO2020055917A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EA202091693A1 (ru) 2018-01-12 2021-04-14 Бристол-Маерс Сквибб Компани Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения
BR112020013994A2 (pt) 2018-01-12 2020-12-08 Bristol-Myers Squibb Company Oligonucleotídeos antissenso que direcionam alfa-sinucleína e seus usos
WO2022150369A1 (en) * 2021-01-06 2022-07-14 Exicure Operating Company Compounds for the treatment of batten disease
WO2025091028A1 (en) * 2023-10-27 2025-05-01 The Regents Of The University Of Michigan Compounds and methods for modulating cln3 expression

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012096325A1 (ja) 2011-01-12 2012-07-19 アステラス製薬株式会社 新規ｂｒａｆ融合体の検出法
US20170268000A1 (en) 2013-12-02 2017-09-21 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2305812A3 (en) * 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
US7314750B2 (en) * 2002-11-20 2008-01-01 Affymetrix, Inc. Addressable oligonucleotide array of the rat genome
EP2285961A4 (en) * 2008-05-15 2012-10-17 Topigen Pharmaceuticals Inc OLIGONUCLEOTIDES FOR THE TREATMENT OF INFLAMMATION AND NEOPLASTIC CELL REPRODUCTION
EP4166667A3 (en) * 2013-10-11 2023-08-02 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
AU2015311704B2 (en) * 2014-09-07 2021-12-09 Selecta Biosciences, Inc. Methods and compositions for attenuating gene editing anti-viral transfer vector immune responses
WO2016061263A1 (en) * 2014-10-14 2016-04-21 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof
FI3386518T3 (fi) * 2015-12-07 2025-09-25 Genzyme Corp Menetelmiä ja koostumuksia serpinc1:een liittyvän häiriön hoitamiseksi
WO2022150369A1 (en) * 2021-01-06 2022-07-14 Exicure Operating Company Compounds for the treatment of batten disease

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012096325A1 (ja) 2011-01-12 2012-07-19 アステラス製薬株式会社 新規ｂｒａｆ融合体の検出法
US20170268000A1 (en) 2013-12-02 2017-09-21 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof

Also Published As

Publication number Publication date
EP3849564A1 (en) 2021-07-21
US20220280545A1 (en) 2022-09-08
JP2022500079A (ja) 2022-01-04
EP3849564A4 (en) 2023-08-09
WO2020055917A1 (en) 2020-03-19

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