JP2023515466A - Ｈｔｔの発現を低下させる方法 - Google Patents

Ｈｔｔの発現を低下させる方法 Download PDF

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Publication number
JP2023515466A
JP2023515466A JP2022549789A JP2022549789A JP2023515466A JP 2023515466 A JP2023515466 A JP 2023515466A JP 2022549789 A JP2022549789 A JP 2022549789A JP 2022549789 A JP2022549789 A JP 2022549789A JP 2023515466 A JP2023515466 A JP 2023515466A
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Japan
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less
weeks
administering
modified oligonucleotide
once
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Pending
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JP2022549789A
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English (en)
Japanese (ja)
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JP2023515466A5 (enExample
Inventor
ベネット，シー・フランク
ノリス，ダニエル・エイ
スミス，アン・ブイ
レーン，ロジャー
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Publication of JP2023515466A publication Critical patent/JP2023515466A/ja
Publication of JP2023515466A5 publication Critical patent/JP2023515466A5/ja
Priority to JP2025281702A priority Critical patent/JP2026075094A/ja
Pending legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • C12N2310/10—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
    • 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
    • C12N2310/321—2'-O-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
    • 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
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Psychology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Steroid Compounds (AREA)
  • Saccharide Compounds (AREA)
JP2022549789A 2020-02-21 2021-02-19 Ｈｔｔの発現を低下させる方法 Pending JP2023515466A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025281702A JP2026075094A (ja) 2020-02-21 2025-12-25 Ｈｔｔの発現を低下させる方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062980010P 2020-02-21 2020-02-21
US62/980,010 2020-02-21
PCT/US2021/018681 WO2021168183A1 (en) 2020-02-21 2021-02-19 Methods for reducing htt expression

Related Child Applications (1)

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JP2025281702A Division JP2026075094A (ja) 2020-02-21 2025-12-25 Ｈｔｔの発現を低下させる方法

Publications (2)

Publication Number Publication Date
JP2023515466A true JP2023515466A (ja) 2023-04-13
JP2023515466A5 JP2023515466A5 (enExample) 2024-02-28

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JP2022549789A Pending JP2023515466A (ja) 2020-02-21 2021-02-19 Ｈｔｔの発現を低下させる方法
JP2025281702A Pending JP2026075094A (ja) 2020-02-21 2025-12-25 Ｈｔｔの発現を低下させる方法

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JP2025281702A Pending JP2026075094A (ja) 2020-02-21 2025-12-25 Ｈｔｔの発現を低下させる方法

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US (2) US20230108340A1 (enExample)
EP (1) EP4107269A4 (enExample)
JP (2) JP2023515466A (enExample)
KR (1) KR20220144837A (enExample)
CN (1) CN115279903A (enExample)
AU (1) AU2021224729A1 (enExample)
BR (1) BR112022016370A2 (enExample)
CA (1) CA3172336A1 (enExample)
IL (1) IL295625A (enExample)
MX (1) MX2022010204A (enExample)
WO (1) WO2021168183A1 (enExample)
ZA (1) ZA202209112B (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025265013A2 (en) 2024-06-20 2025-12-26 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of huntingtin (htt), compositions thereof, and methods of use

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2422819B1 (en) * 2006-01-26 2017-03-01 Ionis Pharmaceuticals, Inc. Compositions and their uses directed to Huntingtin
EP3626823A1 (en) * 2009-09-11 2020-03-25 Ionis Pharmaceuticals, Inc. Modulation of huntingtin expression
WO2015051318A1 (en) * 2013-10-04 2015-04-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the alas1 gene
US20170044538A1 (en) * 2014-04-17 2017-02-16 Biogen Ma Inc. Compositions and Methods for Modulation of SMN2 Splicing in a Subject
CN117965536A (zh) * 2015-07-22 2024-05-03 波涛生命科学有限公司 寡核苷酸组合物及其方法
US11268093B2 (en) * 2017-01-03 2022-03-08 Rula Zain-Luqman Therapeutic method for huntington's disease
KR20220030271A (ko) * 2019-06-28 2022-03-10 더 펜 스테이트 리서어치 파운데이션 헌팅턴병을 치료하기 위한 방법 및 재료

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
"NCT03761849", CLINICALTRIALS.GOV, vol. Record History ver.22,, JPN6025011590, 4 February 2020 (2020-02-04), ISSN: 0005674822 *
BLAIR R.LEAVITT ET AL., NEUROLOGY, vol. Vol.94, No.15 Supplement, JPN6025011589, 2019, pages 950, ISSN: 0005674821 *
OMAR F.F.ODISH ET AL., SCIENTIFIC REPORTS, vol. 8:16090, JPN6025011591, 2018, ISSN: 0005674823 *
SARAH J. TABRIZI ET AL., THE NEW ENGLAND JOURNAL OF MEDICINE, vol. 380, no. 24, JPN6025011588, 2019, pages 2307 - 2316, ISSN: 0005674820 *

Also Published As

Publication number Publication date
IL295625A (en) 2022-10-01
MX2022010204A (es) 2022-09-19
US20230108340A1 (en) 2023-04-06
EP4107269A4 (en) 2024-11-20
ZA202209112B (en) 2024-10-30
KR20220144837A (ko) 2022-10-27
BR112022016370A2 (pt) 2022-10-04
US20260234628A1 (en) 2026-08-13
CA3172336A1 (en) 2021-08-26
EP4107269A1 (en) 2022-12-28
JP2026075094A (ja) 2026-05-07
CN115279903A (zh) 2022-11-01
WO2021168183A1 (en) 2021-08-26
AU2021224729A1 (en) 2022-09-08

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