JP7616995B2 - 神経変性疾患のための遺伝子治療 - Google Patents

神経変性疾患のための遺伝子治療 Download PDF

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Publication number
JP7616995B2
JP7616995B2 JP2021530800A JP2021530800A JP7616995B2 JP 7616995 B2 JP7616995 B2 JP 7616995B2 JP 2021530800 A JP2021530800 A JP 2021530800A JP 2021530800 A JP2021530800 A JP 2021530800A JP 7616995 B2 JP7616995 B2 JP 7616995B2
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nucleic acid
isolated nucleic
seq
vector
raav
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JP2022511453A5 (https=
JPWO2020112802A5 (https=
JP2022511453A (ja
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アベリオビッチ，アサ
ヘックマン，ラウラ
リーン，エルベ
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Prevail Therapeutics Inc
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Prevail Therapeutics Inc
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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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/775—Apolipopeptides
    • 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • 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
    • C12N2320/31—Combination therapy
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hospice & Palliative Care (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2021530800A 2018-11-28 2019-11-26 神経変性疾患のための遺伝子治療 Active JP7616995B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024128634A JP2024177670A (ja) 2018-11-28 2024-08-05 神経変性疾患のための遺伝子治療

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862772230P 2018-11-28 2018-11-28
US62/772,230 2018-11-28
PCT/US2019/063289 WO2020112802A1 (en) 2018-11-28 2019-11-26 Gene therapies for neurodegenerative disease

Related Child Applications (1)

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JP2024128634A Division JP2024177670A (ja) 2018-11-28 2024-08-05 神経変性疾患のための遺伝子治療

Publications (4)

Publication Number Publication Date
JP2022511453A JP2022511453A (ja) 2022-01-31
JP2022511453A5 JP2022511453A5 (https=) 2022-11-21
JPWO2020112802A5 JPWO2020112802A5 (https=) 2022-11-21
JP7616995B2 true JP7616995B2 (ja) 2025-01-17

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JP2021530800A Active JP7616995B2 (ja) 2018-11-28 2019-11-26 神経変性疾患のための遺伝子治療
JP2024128634A Withdrawn JP2024177670A (ja) 2018-11-28 2024-08-05 神経変性疾患のための遺伝子治療

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US (1) US20220010001A1 (https=)
EP (1) EP3887396A4 (https=)
JP (2) JP7616995B2 (https=)
KR (1) KR20210096168A (https=)
CN (1) CN113557243A (https=)
AU (1) AU2019388975A1 (https=)
BR (1) BR112021010234A2 (https=)
CA (1) CA3121211A1 (https=)
IL (1) IL283496B2 (https=)
MX (1) MX2021006253A (https=)
WO (1) WO2020112802A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112020006671A2 (pt) 2017-10-03 2020-12-01 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
KR102709597B1 (ko) 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
EP4045653A1 (en) * 2019-10-16 2022-08-24 Cornell University Gene therapy for alzheimer's disease
EP4232572A4 (en) * 2020-10-22 2024-09-25 Duke University COMPOSITIONS AND PROCEDURES RELATED TO ALZHEIMER’S DISEASE
KR20230112672A (ko) * 2020-11-25 2023-07-27 프리베일 테라퓨틱스, 인크. 신경변성 질환을 위한 유전자 요법
EP4426833A1 (en) * 2021-11-03 2024-09-11 F. Hoffmann-La Roche AG Oligonucleotides for modulating apolipoprotein e4 expression
EP4615981A1 (en) 2022-11-11 2025-09-17 Eli Lilly and Company Synthetic nucleic acids including astrocyte-directed promoter constructs and methods of using the same
CN119220609B (zh) * 2024-09-27 2025-09-12 科辉智药(深圳)新药研究中心有限公司 一种共表达TRIM11和ApoE2的核酸序列构建体及其应用

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070248599A1 (en) 2006-02-21 2007-10-25 Jordan Tang Treatment of alzheimer's disease with inhibitors of apoe binding to apoe receptor
JP2015520161A (ja) 2012-05-18 2015-07-16 ユニバーシティー オブ アイオワ リサーチ ファウンデーション アミロイドの沈着を処置するための方法および組成物
US20150337030A1 (en) 2012-05-31 2015-11-26 The Trustees Of Columbia University In The City Of New York Methods to treat alzheimer's disease using apoe inhibitors
JP2016540977A (ja) 2013-11-20 2016-12-28 ユニバーシティー オブ アイオワ リサーチ ファウンデーション アミロイド沈着を処置するための方法および組成物
WO2018045347A1 (en) 2016-09-02 2018-03-08 Spark Therapeutics, Inc. Methods and vectors for treating cns disorders

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JP3265577B2 (ja) * 1992-10-13 2002-03-11 デューク・ユニバーシティ アポリポ蛋白ｅの４型イソ型の測定法
AU2001253233B2 (en) * 2000-04-06 2006-12-07 Kos Pharmaceuticals, Inc. Compounds and methods for lowering cholesterol levels without inducing hypertriglyceridemia
US20050019927A1 (en) * 2003-07-13 2005-01-27 Markus Hildinger DECREASING GENE EXPRESSION IN A MAMMALIAN SUBJECT IN VIVO VIA AAV-MEDIATED RNAi EXPRESSION CASSETTE TRANSFER
US8435539B2 (en) * 2004-02-13 2013-05-07 Immunomedics, Inc. Delivery system for cytotoxic drugs by bispecific antibody pretargeting
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EP3842514A1 (en) * 2014-09-12 2021-06-30 Whitehead Institute for Biomedical Research Cells expressing apolipoprotein e and uses thereof
KR102719222B1 (ko) * 2017-05-11 2024-10-21 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 신경 세로이드 지질갈색소증에 대한 유전자 요법
EP4045653A1 (en) * 2019-10-16 2022-08-24 Cornell University Gene therapy for alzheimer's disease

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070248599A1 (en) 2006-02-21 2007-10-25 Jordan Tang Treatment of alzheimer's disease with inhibitors of apoe binding to apoe receptor
JP2015520161A (ja) 2012-05-18 2015-07-16 ユニバーシティー オブ アイオワ リサーチ ファウンデーション アミロイドの沈着を処置するための方法および組成物
US20150337030A1 (en) 2012-05-31 2015-11-26 The Trustees Of Columbia University In The City Of New York Methods to treat alzheimer's disease using apoe inhibitors
JP2016540977A (ja) 2013-11-20 2016-12-28 ユニバーシティー オブ アイオワ リサーチ ファウンデーション アミロイド沈着を処置するための方法および組成物
WO2018045347A1 (en) 2016-09-02 2018-03-08 Spark Therapeutics, Inc. Methods and vectors for treating cns disorders

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Publication number Publication date
US20220010001A1 (en) 2022-01-13
CA3121211A1 (en) 2020-06-04
IL283496B1 (en) 2025-05-01
IL283496B2 (en) 2025-09-01
MX2021006253A (es) 2021-09-21
JP2024177670A (ja) 2024-12-20
KR20210096168A (ko) 2021-08-04
BR112021010234A2 (pt) 2021-08-24
AU2019388975A1 (en) 2021-06-24
EP3887396A4 (en) 2022-09-07
IL283496A (en) 2021-07-29
JP2022511453A (ja) 2022-01-31
CN113557243A (zh) 2021-10-26
WO2020112802A1 (en) 2020-06-04
EP3887396A1 (en) 2021-10-06

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