JP2024504377A - Ｄｕｘ４の発現を低減するための化合物及び方法 - Google Patents

Ｄｕｘ４の発現を低減するための化合物及び方法 Download PDF

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JP2024504377A
JP2024504377A JP2023544331A JP2023544331A JP2024504377A JP 2024504377 A JP2024504377 A JP 2024504377A JP 2023544331 A JP2023544331 A JP 2023544331A JP 2023544331 A JP2023544331 A JP 2023544331A JP 2024504377 A JP2024504377 A JP 2024504377A
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modified
certain embodiments
oligomeric
seq
modified oligonucleotide
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JP2024504377A5 (enExample
Inventor
バラス，ルーベン・イー
ジャファー・ネジャド，ペイマン
リゴ，フランク
フレアー，スーザン・エム
ブイ，フィン・ホア
シング，プリヤム
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2023544331A 2021-01-22 2022-01-21 Ｄｕｘ４の発現を低減するための化合物及び方法 Pending JP2024504377A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163140674P 2021-01-22 2021-01-22
US63/140,674 2021-01-22
US202163217624P 2021-07-01 2021-07-01
US63/217,624 2021-07-01
US202163231559P 2021-08-10 2021-08-10
US63/231,559 2021-08-10
PCT/US2022/013323 WO2022159712A1 (en) 2021-01-22 2022-01-21 Compounds and methods for reducing dux4 expression

Publications (2)

Publication Number Publication Date
JP2024504377A true JP2024504377A (ja) 2024-01-31
JP2024504377A5 JP2024504377A5 (enExample) 2025-01-24

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JP2023544331A Pending JP2024504377A (ja) 2021-01-22 2022-01-21 Ｄｕｘ４の発現を低減するための化合物及び方法

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Country Link
US (1) US20240336915A1 (enExample)
EP (1) EP4281084A4 (enExample)
JP (1) JP2024504377A (enExample)
WO (1) WO2022159712A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2026122993A3 (en) * 2024-12-05 2026-07-16 Soufflé Therapeutics, Inc. Oligonucleotide-mediated knockdown of dux4

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3830259A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
CN117677698A (zh) * 2021-07-14 2024-03-08 米雷楚来股份有限公司 用于神经肌肉障碍的寡核苷酸及其组合物
WO2023018705A1 (en) * 2021-08-10 2023-02-16 University Of Massachusetts Compositions and methods for sirna treatment of muscular dystrophy
JP2025532128A (ja) * 2022-09-23 2025-09-29 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｍｅｃｐ２発現を低減する化合物及び方法
JP2025532127A (ja) 2022-09-23 2025-09-29 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｍｅｃｐ２発現を低減する化合物及び方法

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140322169A1 (en) * 2011-07-25 2014-10-30 Nationwide Children's Hospital, Inc. Recombinant Virus Products and Methods for Inhibition of Expression of DUX4
WO2017050836A1 (en) * 2015-09-21 2017-03-30 Association Institut De Myologie Antisense oligonucleotides and uses thereof
US20200291398A1 (en) * 2017-09-19 2020-09-17 Children's National Medical Center Gapmers and methods of using the same for the treatment of muscular dystrophy

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3118316A1 (en) * 2010-09-02 2017-01-18 Université de Mons Agents useful in treating facioscapulohumeral muscular dystrophy
US10538763B2 (en) * 2015-01-16 2020-01-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of DUX4
AU2019218987B2 (en) * 2018-02-12 2025-04-24 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
EP3927827A4 (en) * 2019-02-22 2023-04-26 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR REDUCING ATXN3 EXPRESSION
TW202104595A (zh) * 2019-03-29 2021-02-01 日商田邊三菱製藥股份有限公司 用來調節ｄｕｘ４的表現之化合物、方法及醫藥組成物

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140322169A1 (en) * 2011-07-25 2014-10-30 Nationwide Children's Hospital, Inc. Recombinant Virus Products and Methods for Inhibition of Expression of DUX4
WO2017050836A1 (en) * 2015-09-21 2017-03-30 Association Institut De Myologie Antisense oligonucleotides and uses thereof
US20200291398A1 (en) * 2017-09-19 2020-09-17 Children's National Medical Center Gapmers and methods of using the same for the treatment of muscular dystrophy

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
CHISTIAKOV DA. ET AL.: "Strategies to deliver microRNAs as potential therapeutics in the treatment of cardiovascular patholo", DRUG DELIV., vol. vol.19(8), JPN6025050835, 2012, pages 392 - 405, ISSN: 0005906607 *
CHORN G. ET AL.: "Single-stranded microRNA mimics", RNA, vol. vol.18(10), JPN6025050834, 2012, pages 1796 - 1804, ISSN: 0005906606 *
LIM KRQ. ET AL.: "Inhibition of DUX4 expression with antisense LNA gapmers as a therapy for facioscapulohumeral muscul", PROC NATL ACAD SCI U S A, vol. vol.117(28), JPN6025050833, 14 June 2020 (2020-06-14), pages 16509 - 16515, ISSN: 0005906605 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2026122993A3 (en) * 2024-12-05 2026-07-16 Soufflé Therapeutics, Inc. Oligonucleotide-mediated knockdown of dux4

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WO2022159712A1 (en) 2022-07-28
US20240336915A1 (en) 2024-10-10
EP4281084A1 (en) 2023-11-29
EP4281084A4 (en) 2025-05-21

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