JP2024505226A - ハンチンチンを調節するための化合物及び方法 - Google Patents

ハンチンチンを調節するための化合物及び方法 Download PDF

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Publication number
JP2024505226A
JP2024505226A JP2023546047A JP2023546047A JP2024505226A JP 2024505226 A JP2024505226 A JP 2024505226A JP 2023546047 A JP2023546047 A JP 2023546047A JP 2023546047 A JP2023546047 A JP 2023546047A JP 2024505226 A JP2024505226 A JP 2024505226A
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modified
oligomeric compound
modified oligonucleotide
certain embodiments
nucleosides
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JP2024505226A5 (https=
Inventor
フィッツシモンズ，ベサニー
フレアー，スーザン・エム
コルダシエビッチ，ホリー
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • 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
    • 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
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
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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
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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
    • 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
    • C12N2310/33—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 ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
    • 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
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
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  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Liquid Crystal Substances (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
JP2023546047A 2021-01-29 2022-01-28 ハンチンチンを調節するための化合物及び方法 Pending JP2024505226A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163143686P 2021-01-29 2021-01-29
US63/143,686 2021-01-29
PCT/US2022/014231 WO2022165122A1 (en) 2021-01-29 2022-01-28 Compounds and methods for modulating huntingtin

Publications (2)

Publication Number Publication Date
JP2024505226A true JP2024505226A (ja) 2024-02-05
JP2024505226A5 JP2024505226A5 (https=) 2025-02-05

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ID=82653847

Family Applications (1)

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JP2023546047A Pending JP2024505226A (ja) 2021-01-29 2022-01-28 ハンチンチンを調節するための化合物及び方法

Country Status (11)

Country Link
US (1) US20240376469A1 (https=)
EP (1) EP4284504A4 (https=)
JP (1) JP2024505226A (https=)
KR (1) KR20230137958A (https=)
CN (1) CN116940683A (https=)
AU (1) AU2022213384A1 (https=)
CA (1) CA3210076A1 (https=)
IL (1) IL304218A (https=)
MX (1) MX2023008922A (https=)
TW (1) TW202241462A (https=)
WO (1) WO2022165122A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4423272A2 (en) * 2021-10-29 2024-09-04 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
IL326478A (en) * 2023-08-16 2026-04-01 Servier Lab Oligonucleotides for modulation of KCNT1 expression
EP4527927A1 (en) * 2023-09-22 2025-03-26 Medizinische Hochschule Hannover Oligonucleotide-based strategies to combat respiratory viruses

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005105995A2 (en) * 2004-04-14 2005-11-10 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2013022984A1 (en) * 2011-08-11 2013-02-14 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
JP2013518604A (ja) * 2010-02-08 2013-05-23 アイシス ファーマシューティカルズ， インコーポレーテッド 対立遺伝子多様体の選択的低減
JP2017529082A (ja) * 2014-09-18 2017-10-05 ザ・ユニバーシティ・オブ・ブリティッシュ・コロンビア ハンチントン病ハプロタイプのための対立遺伝子特異的な療法
US20190195863A1 (en) * 2016-02-24 2019-06-27 The Rockefeller University Embryonic Cell-Based Therapeutic Candidate Screening Systems, Models for Huntington's Disease and Uses Thereof

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ714887A (en) * 2009-09-11 2019-07-26 Ionis Pharmaceuticals Inc Modulation of huntingtin expression
CA2789038A1 (en) * 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
US12281305B2 (en) * 2018-11-21 2025-04-22 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing prion expression

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005105995A2 (en) * 2004-04-14 2005-11-10 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA)
JP2013518604A (ja) * 2010-02-08 2013-05-23 アイシス ファーマシューティカルズ， インコーポレーテッド 対立遺伝子多様体の選択的低減
WO2013022984A1 (en) * 2011-08-11 2013-02-14 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
JP2017529082A (ja) * 2014-09-18 2017-10-05 ザ・ユニバーシティ・オブ・ブリティッシュ・コロンビア ハンチントン病ハプロタイプのための対立遺伝子特異的な療法
US20190195863A1 (en) * 2016-02-24 2019-06-27 The Rockefeller University Embryonic Cell-Based Therapeutic Candidate Screening Systems, Models for Huntington's Disease and Uses Thereof

Also Published As

Publication number Publication date
EP4284504A4 (en) 2025-08-20
EP4284504A1 (en) 2023-12-06
CA3210076A1 (en) 2022-08-04
AU2022213384A1 (en) 2023-07-20
US20240376469A1 (en) 2024-11-14
WO2022165122A1 (en) 2022-08-04
KR20230137958A (ko) 2023-10-05
CN116940683A (zh) 2023-10-24
MX2023008922A (es) 2023-08-10
IL304218A (en) 2023-09-01
TW202241462A (zh) 2022-11-01

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