JP7776924B2 - 変異ＦＵＳの発現を選択的に抑制する修飾ｓｉＲＮＡ - Google Patents

変異ＦＵＳの発現を選択的に抑制する修飾ｓｉＲＮＡ

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Publication number
JP7776924B2
JP7776924B2 JP2024506295A JP2024506295A JP7776924B2 JP 7776924 B2 JP7776924 B2 JP 7776924B2 JP 2024506295 A JP2024506295 A JP 2024506295A JP 2024506295 A JP2024506295 A JP 2024506295A JP 7776924 B2 JP7776924 B2 JP 7776924B2
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seq
double
stranded rna
rna consisting
strand
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JPWO2023171587A1 (https=
JPWO2023171587A5 (https=
Inventor
直樹 森田
楓 橋迫
菫 岡田
善信 山本
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Ohara Pharmaceutical Co Ltd
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Ohara Pharmaceutical Co Ltd
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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
    • 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
    • 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
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • 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
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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
    • C12N15/1137—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 against enzymes
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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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
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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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Virology (AREA)
  • Pain & Pain Management (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2024506295A 2022-03-08 2023-03-06 変異ＦＵＳの発現を選択的に抑制する修飾ｓｉＲＮＡ Active JP7776924B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2022035727 2022-03-08
JP2022035727 2022-03-08
PCT/JP2023/008197 WO2023171587A1 (ja) 2022-03-08 2023-03-06 変異ＦＵＳの発現を選択的に抑制する修飾ｓｉＲＮＡ

Publications (3)

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JPWO2023171587A1 JPWO2023171587A1 (https=) 2023-09-14
JPWO2023171587A5 JPWO2023171587A5 (https=) 2024-10-22
JP7776924B2 true JP7776924B2 (ja) 2025-11-27

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JP2024506295A Active JP7776924B2 (ja) 2022-03-08 2023-03-06 変異ＦＵＳの発現を選択的に抑制する修飾ｓｉＲＮＡ

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US (1) US20250101420A1 (https=)
JP (1) JP7776924B2 (https=)
WO (1) WO2023171587A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025163186A1 (en) * 2024-02-01 2025-08-07 Silence Therapeutics Gmbh Conjugated nucleic acids and nucleic acids comprising locked nucleosides and inverted nucleotides for inhibiting gene expression in a cell

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2011528903A (ja) 2008-07-22 2011-12-01 ザ ジェネラル ホスピタル コーポレイション 筋萎縮性側索硬化症および関連する運動ニューロン疾患の診断、処置および予防のための、ｆｕｓ／ｔｌｓベースの化合物および方法
JP2015506905A (ja) 2012-01-17 2015-03-05 国立大学法人京都大学 筋萎縮性側索硬化症の予防および治療薬とそのスクリーニング方法
JP2015520742A (ja) 2012-05-02 2015-07-23 サーナ・セラピューティクス・インコーポレイテッドＳｉｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． Ｔｅｔｒａｇａｌｎａｃを含む新規なコンジュゲートと、オリゴヌクレオチドの送達方法
WO2018185253A1 (en) 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Ligand modified double-stranded nucleic acids
WO2019193189A1 (en) 2018-04-05 2019-10-10 Silence Therapeutics Gmbh siRNAs WITH AT LEAST TWO LIGANDS AT DIFFERENT ENDS
US20200239888A1 (en) 2017-08-08 2020-07-30 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of protein aggregation

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101576602B1 (ko) * 2014-02-11 2015-12-10 한남대학교 산학협력단 퇴행성 신경계 질환 치료제 스크리닝 방법
US20240229025A1 (en) * 2020-09-09 2024-07-11 Ohara Pharmaceutical Co., Ltd. siRNA AND USE THEREOF

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2011528903A (ja) 2008-07-22 2011-12-01 ザ ジェネラル ホスピタル コーポレイション 筋萎縮性側索硬化症および関連する運動ニューロン疾患の診断、処置および予防のための、ｆｕｓ／ｔｌｓベースの化合物および方法
JP2015506905A (ja) 2012-01-17 2015-03-05 国立大学法人京都大学 筋萎縮性側索硬化症の予防および治療薬とそのスクリーニング方法
JP2015520742A (ja) 2012-05-02 2015-07-23 サーナ・セラピューティクス・インコーポレイテッドＳｉｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． Ｔｅｔｒａｇａｌｎａｃを含む新規なコンジュゲートと、オリゴヌクレオチドの送達方法
WO2018185253A1 (en) 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Ligand modified double-stranded nucleic acids
US20200239888A1 (en) 2017-08-08 2020-07-30 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of protein aggregation
WO2019193189A1 (en) 2018-04-05 2019-10-10 Silence Therapeutics Gmbh siRNAs WITH AT LEAST TWO LIGANDS AT DIFFERENT ENDS

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Publication number Publication date
US20250101420A1 (en) 2025-03-27
WO2023171587A1 (ja) 2023-09-14
JPWO2023171587A1 (https=) 2023-09-14

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