JP7610923B2 - Ｃｎｓ障害を治療するための方法及びベクター - Google Patents

Ｃｎｓ障害を治療するための方法及びベクター Download PDF

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JP7610923B2
JP7610923B2 JP2019512292A JP2019512292A JP7610923B2 JP 7610923 B2 JP7610923 B2 JP 7610923B2 JP 2019512292 A JP2019512292 A JP 2019512292A JP 2019512292 A JP2019512292 A JP 2019512292A JP 7610923 B2 JP7610923 B2 JP 7610923B2
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aav
nucleic acid
apoe
vector
aav4
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JP2019529385A5 (enExample
JP2019529385A (ja
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キャサリン エー． ハイ，
ビバリー エル． デイビッドソン，
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Childrens Hospital of Philadelphia CHOP
Spark Therapeutics Inc
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Spark 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
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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
    • C12N15/63—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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/06—Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06004—N-Acetylgalactosamine-6-sulfatase (3.1.6.4)
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    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01031—Beta-glucuronidase (3.2.1.31)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A01K2267/0312—Animal model for Alzheimer's disease
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
    • C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)

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  • Health & Medical Sciences (AREA)
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  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Dermatology (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
JP2019512292A 2016-09-02 2017-09-01 Ｃｎｓ障害を治療するための方法及びベクター Active JP7610923B2 (ja)

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JP2022002163A JP2022050574A (ja) 2016-09-02 2022-01-11 Ｃｎｓ障害を治療するための方法及びベクター

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Application Number Priority Date Filing Date Title
US201662383274P 2016-09-02 2016-09-02
US62/383,274 2016-09-02
PCT/US2017/049959 WO2018045347A1 (en) 2016-09-02 2017-09-01 Methods and vectors for treating cns disorders

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JP2019529385A JP2019529385A (ja) 2019-10-17
JP2019529385A5 JP2019529385A5 (enExample) 2020-09-17
JP7610923B2 true JP7610923B2 (ja) 2025-01-09

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JP2022002163A Pending JP2022050574A (ja) 2016-09-02 2022-01-11 Ｃｎｓ障害を治療するための方法及びベクター

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US (1) US20190192693A1 (enExample)
EP (1) EP3506930A4 (enExample)
JP (2) JP7610923B2 (enExample)
CN (1) CN110121356A (enExample)
AU (2) AU2017318717B2 (enExample)
BR (1) BR112019004353A2 (enExample)
CA (1) CA3035628A1 (enExample)
MX (1) MX2019002518A (enExample)
WO (1) WO2018045347A1 (enExample)

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GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
CA3069880A1 (en) * 2017-07-17 2019-01-24 Spark Therapeutics, Inc. Apheresis methods and uses
IL313246B2 (en) 2018-06-28 2025-10-01 Univ North Carolina Chapel Hill Optimal cln5 expression genes and cassettes and their use
AU2019346447A1 (en) 2018-09-26 2021-04-29 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
KR20210092755A (ko) * 2018-11-14 2021-07-26 리젠엑스바이오 인크. 신경원성 세로이드 리포푸신증에 대한 유전자 요법
KR20210096168A (ko) * 2018-11-28 2021-08-04 프리베일 테라퓨틱스, 인크. 신경퇴행성 질환을 위한 유전자 요법
US12416017B2 (en) 2019-02-22 2025-09-16 The Trustees Of The University Of Pennsylvania Recombinant adeno-associated virus for treatment of GRN-associated adult-onset neurodegeneration
WO2021061909A1 (en) * 2019-09-24 2021-04-01 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Method of targeting neuronal apoe to treat a neurocognitive disorder
CN114761569A (zh) * 2019-10-16 2022-07-15 康奈尔大学 阿尔茨海默氏病的基因疗法
AU2021351720A1 (en) * 2020-10-01 2023-05-11 Sangamo Therapeutics, Inc. Methods of detecting anti-aav antibodies
CN112852881B (zh) * 2021-02-09 2023-08-15 南京贝思奥生物科技有限公司 利用细胞穿膜肽增强腺相关病毒在中枢神经系统转导效率的方法
TW202426479A (zh) * 2022-10-10 2024-07-01 美商星火治療公司 Apoe基因療法

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SG11201706755SA (en) * 2015-02-20 2017-09-28 Univ Iowa Res Found Methods and compositions for treating genetic eye diseases

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WO2012057363A1 (ja) 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
WO2015077473A1 (en) 2013-11-20 2015-05-28 University Of Iowa Research Foundation Methods and compositions for treating amyloid deposits

Non-Patent Citations (1)

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Title
Ａｎｎ． Ｎｅｕｒｏｌ． （２０１４） ｖｏｌ．７６， ｓｕｕｐｌ．１８， ｐ．Ｓ１７６（ＰＬ１－６）

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EP3506930A1 (en) 2019-07-10
BR112019004353A2 (pt) 2019-05-28
AU2017318717B2 (en) 2024-10-24
CA3035628A1 (en) 2018-03-08
AU2017318717A1 (en) 2019-03-21
US20190192693A1 (en) 2019-06-27
MX2019002518A (es) 2019-07-18
JP2019529385A (ja) 2019-10-17
JP2022050574A (ja) 2022-03-30
EP3506930A4 (en) 2020-09-23
AU2024227138A1 (en) 2024-10-31
CN110121356A (zh) 2019-08-13
WO2018045347A1 (en) 2018-03-08

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