JP2024524332A - アルツハイマー病の治療方法 - Google Patents

アルツハイマー病の治療方法 Download PDF

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JP2024524332A
JP2024524332A JP2023579741A JP2023579741A JP2024524332A JP 2024524332 A JP2024524332 A JP 2024524332A JP 2023579741 A JP2023579741 A JP 2023579741A JP 2023579741 A JP2023579741 A JP 2023579741A JP 2024524332 A JP2024524332 A JP 2024524332A
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seq
exon
oligonucleotide
sorla
asos
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JP2024524332A5 (https=
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マイケル アンデルセン，オラフ
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オーフス ウニベルシテット
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    • 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
    • C12N15/1138—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 receptors or cell surface proteins
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • 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
    • C12N2310/11—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
    • C12N2310/30—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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Physics & Mathematics (AREA)
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  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2023579741A 2021-07-02 2022-07-01 アルツハイマー病の治療方法 Pending JP2024524332A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP21183395 2021-07-02
EP21183395.9 2021-07-02
PCT/EP2022/068313 WO2023275376A1 (en) 2021-07-02 2022-07-01 Methods of treating alzheimer's disease

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JP2024524332A true JP2024524332A (ja) 2024-07-05
JP2024524332A5 JP2024524332A5 (https=) 2025-07-08

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JP2023579741A Pending JP2024524332A (ja) 2021-07-02 2022-07-01 アルツハイマー病の治療方法

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US (1) US20240279666A1 (https=)
EP (1) EP4363578A1 (https=)
JP (1) JP2024524332A (https=)
WO (1) WO2023275376A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024178386A1 (en) 2023-02-24 2024-08-29 Aarhus Universitet Methods of treating endosomal trafficking diseases
WO2025262002A2 (en) * 2024-06-17 2025-12-26 Aarhus Universitet Antisense oligonucleotides for treating alzheimer's disease

Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008052016A2 (en) * 2006-10-23 2008-05-02 Columbia University The sortilin-related receptor sorl1 is functionally and genetically associated with alzheimer's disease
JP2012506703A (ja) * 2008-10-24 2012-03-22 エイブイアイ バイオファーマ， インコーポレイテッド Ｄｍｄのための複数のエキソンスキッピング組成物
KR20180130060A (ko) * 2017-05-26 2018-12-06 가천대학교 산학협력단 알츠하이머병 관련 ３０개 유전자를 이용한 유전적 진단방법
WO2019012542A1 (en) * 2017-07-13 2019-01-17 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. DETECTION OF SPECIFIC DNA FROM A FABRIC
WO2019162692A1 (en) * 2018-02-26 2019-08-29 Royal Holloway And Bedford New College Preventing exon 7 and/or exon 8 inclusion into an amyloid precursor protein (app) mrna
WO2020009151A1 (ja) * 2018-07-04 2020-01-09 国立大学法人名古屋大学 タウのスプライシングを制御するアンチセンスオリゴヌクレオチド及びその用途
JP2020534802A (ja) * 2017-08-25 2020-12-03 ストーク セラピューティクス，インク． 状態および疾患の処置のためのアンチセンスオリゴマー
WO2021125311A1 (ja) * 2019-12-19 2021-06-24 日本新薬株式会社 エクソンスキッピングを可能にするアンチセンス核酸

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9994893B2 (en) * 2011-12-13 2018-06-12 Rutgers, The State University Of New Jersey Compositions and methods for functional quality control for human blood-based gene expression products

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008052016A2 (en) * 2006-10-23 2008-05-02 Columbia University The sortilin-related receptor sorl1 is functionally and genetically associated with alzheimer's disease
JP2012506703A (ja) * 2008-10-24 2012-03-22 エイブイアイ バイオファーマ， インコーポレイテッド Ｄｍｄのための複数のエキソンスキッピング組成物
KR20180130060A (ko) * 2017-05-26 2018-12-06 가천대학교 산학협력단 알츠하이머병 관련 ３０개 유전자를 이용한 유전적 진단방법
WO2019012542A1 (en) * 2017-07-13 2019-01-17 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. DETECTION OF SPECIFIC DNA FROM A FABRIC
JP2020534802A (ja) * 2017-08-25 2020-12-03 ストーク セラピューティクス，インク． 状態および疾患の処置のためのアンチセンスオリゴマー
WO2019162692A1 (en) * 2018-02-26 2019-08-29 Royal Holloway And Bedford New College Preventing exon 7 and/or exon 8 inclusion into an amyloid precursor protein (app) mrna
WO2020009151A1 (ja) * 2018-07-04 2020-01-09 国立大学法人名古屋大学 タウのスプライシングを制御するアンチセンスオリゴヌクレオチド及びその用途
WO2021125311A1 (ja) * 2019-12-19 2021-06-24 日本新薬株式会社 エクソンスキッピングを可能にするアンチセンス核酸

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
NUCLEIC ACIDS RES, vol. 48, no. 2, JPN6026009728, 2020, pages 802 - 816, ISSN: 0005818240 *

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WO2023275376A1 (en) 2023-01-05
EP4363578A1 (en) 2024-05-08

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