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
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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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CN202080015962.8A Pending CN113710281A (zh) 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

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US (2) US12416017B2 (https=)
EP (1) EP3927381A1 (https=)
JP (2) JP7637058B2 (https=)
KR (1) KR20210131370A (https=)
CN (1) CN113710281A (https=)
AU (1) AU2020225472A1 (https=)
BR (1) BR112021015817A2 (https=)
CA (1) CA3129672A1 (https=)
CL (1) CL2021002172A1 (https=)
CO (1) CO2021011040A2 (https=)
IL (1) IL285654A (https=)
MX (1) MX2021010134A (https=)
PE (1) PE20211819A1 (https=)
SG (1) SG11202108504YA (https=)
TW (1) TW202045730A (https=)
WO (1) WO2020172490A1 (https=)

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BR112021015817A2 (pt) 2019-02-22 2021-10-13 The Trustees Of The University Of Pennsylvania Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn
GB201913974D0 (en) * 2019-09-27 2019-11-13 King S College London Vector
MX2022004812A (es) * 2019-10-22 2023-02-23 Applied Genetic Tech Corporation Sistemas de virus adeno-asociados (aav) para el tratamiento de enfermedades o trastornos neurodegenerativos asociados a la progranulina.
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
US20240033332A1 (en) * 2020-12-17 2024-02-01 Kyoto University Agent for activating a neuron
EP4473123A4 (en) * 2022-02-01 2026-03-11 Shape Therapeutics Inc STABLE CELL LINES FOR INDUCIBLE VAAR VIRION PRODUCTION
US20250000992A1 (en) * 2023-06-21 2025-01-02 Eli Lilly And Company Compounds for the delivery of granulin across the blood brain barrier
TW202548022A (zh) * 2024-03-03 2025-12-16 美商帕西奇生物公司 用於治療神經退化性病症之重組腺相關病毒

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US12416017B2 (en) 2025-09-16
JP2025081439A (ja) 2025-05-27
AU2020225472A1 (en) 2021-08-26
TW202045730A (zh) 2020-12-16
US20260049335A1 (en) 2026-02-19
MX2021010134A (es) 2021-09-23
CA3129672A1 (en) 2020-08-27
EP3927381A1 (en) 2021-12-29
IL285654A (en) 2021-09-30
BR112021015817A2 (pt) 2021-10-13
CO2021011040A2 (es) 2021-09-09
KR20210131370A (ko) 2021-11-02
WO2020172490A1 (en) 2020-08-27
US20220136008A1 (en) 2022-05-05
JP7637058B2 (ja) 2025-02-27
JP2022523766A (ja) 2022-04-26
CL2021002172A1 (es) 2022-02-11
SG11202108504YA (en) 2021-09-29

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