CN113710281A - 用于治疗grn相关成人发作性神经退行性病变的重组腺相关病毒 - Google Patents

用于治疗grn相关成人发作性神经退行性病变的重组腺相关病毒 Download PDF

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CN113710281A
CN113710281A CN202080015962.8A CN202080015962A CN113710281A CN 113710281 A CN113710281 A CN 113710281A CN 202080015962 A CN202080015962 A CN 202080015962A CN 113710281 A CN113710281 A CN 113710281A
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aav
raav
human
vector genome
grn
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C·欣德勒
N·米勒
J·M·威尔逊
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University of Pennsylvania Penn
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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
    • 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
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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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    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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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    • C12N2750/00011—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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    • 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
    • C12N2750/14171—Demonstrated in vivo effect

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CN202080015962.8A 2019-02-22 2020-02-21 用于治疗grn相关成人发作性神经退行性病变的重组腺相关病毒 Pending CN113710281A (zh)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962809329P 2019-02-22 2019-02-22
US62/809329 2019-02-22
US201962923812P 2019-10-21 2019-10-21
US62/923812 2019-10-21
US202062969108P 2020-02-02 2020-02-02
US62/969108 2020-02-02
PCT/US2020/019149 WO2020172490A1 (en) 2019-02-22 2020-02-21 Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration

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CN113710281A true CN113710281A (zh) 2021-11-26

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US (1) US12416017B2 (enExample)
EP (1) EP3927381A1 (enExample)
JP (2) JP7637058B2 (enExample)
KR (1) KR20210131370A (enExample)
CN (1) CN113710281A (enExample)
AU (1) AU2020225472A1 (enExample)
BR (1) BR112021015817A2 (enExample)
CA (1) CA3129672A1 (enExample)
CL (1) CL2021002172A1 (enExample)
CO (1) CO2021011040A2 (enExample)
IL (1) IL285654A (enExample)
MX (1) MX2021010134A (enExample)
PE (1) PE20211819A1 (enExample)
SG (1) SG11202108504YA (enExample)
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Cited By (1)

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WO2024245120A1 (en) * 2023-05-26 2024-12-05 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant aav vectors for treating proteinopathies in central nervous system

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JP2022523913A (ja) * 2019-02-01 2022-04-27 アブロバイオ，インコーポレーテッド 神経認知障害を処置するための組成物及び方法
SG11202108504YA (en) 2019-02-22 2021-09-29 Univ Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
GB201913974D0 (en) * 2019-09-27 2019-11-13 King S College London Vector
CN115715327A (zh) * 2019-10-22 2023-02-24 应用遗传科技公司 用于治疗颗粒蛋白前体相关的神经退行性疾病或病症的腺伴随病毒(aav)系统
EP4204014A1 (en) * 2020-08-26 2023-07-05 The Trustees of The University of Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
WO2022131322A1 (ja) * 2020-12-17 2022-06-23 国立大学法人京都大学 神経細胞賦活剤
EP4473123A2 (en) * 2022-02-01 2024-12-11 Shape Therapeutics Inc. Stable cell lines for inducible production of raav virions
AU2024311750A1 (en) * 2023-06-21 2025-12-18 Eli Lilly And Company Compounds for the delivery of granulin across the blood brain barrier
WO2025188625A1 (en) * 2024-03-03 2025-09-12 Passage Bio, Inc. Recombinant adeno-associated virus for treatment of neurodegenerative disorders

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PE20211819A1 (es) 2021-09-14
MX2021010134A (es) 2021-09-23
KR20210131370A (ko) 2021-11-02
BR112021015817A2 (pt) 2021-10-13
IL285654A (en) 2021-09-30
JP2025081439A (ja) 2025-05-27
CO2021011040A2 (es) 2021-09-09
AU2020225472A1 (en) 2021-08-26
US12416017B2 (en) 2025-09-16
US20220136008A1 (en) 2022-05-05
JP2022523766A (ja) 2022-04-26
TW202045730A (zh) 2020-12-16
WO2020172490A1 (en) 2020-08-27
JP7637058B2 (ja) 2025-02-27
CL2021002172A1 (es) 2022-02-11
CA3129672A1 (en) 2020-08-27
EP3927381A1 (en) 2021-12-29

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