CN110121356A - 治疗cns疾病的方法和载体 - Google Patents

治疗cns疾病的方法和载体 Download PDF

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Publication number
CN110121356A
CN110121356A CN201780067677.9A CN201780067677A CN110121356A CN 110121356 A CN110121356 A CN 110121356A CN 201780067677 A CN201780067677 A CN 201780067677A CN 110121356 A CN110121356 A CN 110121356A
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CN
China
Prior art keywords
aav
mammal
therapeutic protein
cells
cns
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Pending
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CN201780067677.9A
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English (en)
Chinese (zh)
Inventor
K·A·海
B·L·戴维森
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Childrens Hospital of Philadelphia CHOP
Spark Therapeutics Inc
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Childrens Hospital of Philadelphia CHOP
Spark Therapeutics Inc
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Publication of CN110121356A publication Critical patent/CN110121356A/zh
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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
    • C07K14/775—Apolipopeptides
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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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • 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
    • 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)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Dermatology (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)
CN201780067677.9A 2016-09-02 2017-09-01 治疗cns疾病的方法和载体 Pending CN110121356A (zh)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
CN110121356A true CN110121356A (zh) 2019-08-13

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ID=61309371

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CN201780067677.9A Pending CN110121356A (zh) 2016-09-02 2017-09-01 治疗cns疾病的方法和载体

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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)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112852881A (zh) * 2021-02-09 2021-05-28 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
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

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
IL279131B2 (en) 2018-06-28 2025-06-01 Univ North Carolina Chapel Hill Optimized cln5 genes and expression cassettes and their use
EP3856913A4 (en) 2018-09-26 2022-10-26 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
CA3117982A1 (en) * 2018-11-14 2020-05-22 Bhargavi KONDRAGUNTA Gene therapy for neuronal ceroid lipofuscinoses
WO2020112802A1 (en) * 2018-11-28 2020-06-04 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
KR20210131370A (ko) 2019-02-22 2021-11-02 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Grn-연관 성인-발병 신경퇴화의 치료를 위한 재조합 아데노-연관 바이러스
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
WO2022072888A1 (en) * 2020-10-01 2022-04-07 Sangamo Therapeutics, Inc. Methods of detecting anti-aav antibodies
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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CN104540952A (zh) * 2012-05-18 2015-04-22 衣阿华大学研究基金会 用于治疗淀粉状蛋白沉积的方法和组合物
WO2015077473A1 (en) * 2013-11-20 2015-05-28 University Of Iowa Research Foundation Methods and compositions for treating amyloid deposits
WO2016134375A1 (en) * 2015-02-20 2016-08-25 University Of Iowa Research Foundation Methods and compositions for treating genetic eye diseases

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EP2634253B1 (en) * 2010-10-27 2016-05-11 Jichi Medical University Adeno-associated virus virions for transferring genes into neural cells
RU2018128780A (ru) * 2013-07-26 2018-12-05 Юниверсити Оф Айова Рисерч Фаундейшн Способы и композиции для лечения болезней мозга

Patent Citations (3)

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Publication number Priority date Publication date Assignee Title
CN104540952A (zh) * 2012-05-18 2015-04-22 衣阿华大学研究基金会 用于治疗淀粉状蛋白沉积的方法和组合物
WO2015077473A1 (en) * 2013-11-20 2015-05-28 University Of Iowa Research Foundation Methods and compositions for treating amyloid deposits
WO2016134375A1 (en) * 2015-02-20 2016-08-25 University Of Iowa Research Foundation Methods and compositions for treating genetic eye diseases

Non-Patent Citations (1)

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ALEXANDRA SHARLAND等: "Liver-directed gene expression using recombinant AAV 2/8 vectors -- a tolerogenic strategy for gene delivery?", 《DISCOVERY MEDECINE》 *

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CN112852881A (zh) * 2021-02-09 2021-05-28 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
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

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Publication number Publication date
US20190192693A1 (en) 2019-06-27
JP2019529385A (ja) 2019-10-17
CA3035628A1 (en) 2018-03-08
BR112019004353A2 (pt) 2019-05-28
EP3506930A1 (en) 2019-07-10
MX2019002518A (es) 2019-07-18
WO2018045347A1 (en) 2018-03-08
EP3506930A4 (en) 2020-09-23
AU2017318717B2 (en) 2024-10-24
JP7610923B2 (ja) 2025-01-09
AU2017318717A1 (en) 2019-03-21
AU2024227138A1 (en) 2024-10-31
JP2022050574A (ja) 2022-03-30

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