JP7560447B2 - Ａａｖトリプルプラスミドシステム - Google Patents

Ａａｖトリプルプラスミドシステム Download PDF

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JP7560447B2
JP7560447B2 JP2021516750A JP2021516750A JP7560447B2 JP 7560447 B2 JP7560447 B2 JP 7560447B2 JP 2021516750 A JP2021516750 A JP 2021516750A JP 2021516750 A JP2021516750 A JP 2021516750A JP 7560447 B2 JP7560447 B2 JP 7560447B2
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plasmid
nucleic acid
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sequence
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JP2022511348A (ja
JP2022511348A5 (https=
JPWO2020086881A5 (https=
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チョイ，ヴィヴィアン
リー，シン
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バクスアルタ インコーポレイテッド
バクスアルタ ゲーエムベーハー
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    • C—CHEMISTRY; METALLURGY
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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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    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • 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
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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
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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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
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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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/50—Vectors for producing vectors

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  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
JP2021516750A 2018-10-25 2019-10-24 Ａａｖトリプルプラスミドシステム Active JP7560447B2 (ja)

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JP2024161633A JP2025000711A (ja) 2018-10-25 2024-09-19 Ａａｖトリプルプラスミドシステム

Applications Claiming Priority (3)

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US201862750603P 2018-10-25 2018-10-25
US62/750,603 2018-10-25
PCT/US2019/057916 WO2020086881A1 (en) 2018-10-25 2019-10-24 Aav triple-plasmid system

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JP2024161633A Division JP2025000711A (ja) 2018-10-25 2024-09-19 Ａａｖトリプルプラスミドシステム

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JP2022511348A JP2022511348A (ja) 2022-01-31
JP2022511348A5 JP2022511348A5 (https=) 2022-10-19
JPWO2020086881A5 JPWO2020086881A5 (https=) 2022-10-19
JP7560447B2 true JP7560447B2 (ja) 2024-10-02

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JP2024161633A Pending JP2025000711A (ja) 2018-10-25 2024-09-19 Ａａｖトリプルプラスミドシステム

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US (1) US12383587B2 (https=)
EP (1) EP3870148A4 (https=)
JP (2) JP7560447B2 (https=)
KR (1) KR20210086645A (https=)
CN (1) CN112888426A (https=)
AU (1) AU2019363593A1 (https=)
CA (1) CA3112883A1 (https=)
IL (1) IL281909A (https=)
TW (1) TW202029957A (https=)
WO (1) WO2020086881A1 (https=)

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EP4189077A1 (en) 2020-07-30 2023-06-07 Shape Therapeutics Inc. Stable cell lines for inducible production of raav virions
WO2022087321A1 (en) * 2020-10-21 2022-04-28 Duke University Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
IL302578A (en) * 2020-11-04 2023-07-01 Synplogen Co Ltd Production of a virus vector plasmid in Bacillus Subtilis
US11926842B2 (en) * 2021-02-02 2024-03-12 University Of Massachusetts Use of inverted terminal repeats (ITRS) from adeno-associated virus serotypes 8 and RH.39 in gene therapy vectors
US20240360422A1 (en) * 2021-04-21 2024-10-31 Michael T. Leonard Stable production systems for adeno-associated virus production
US12305188B2 (en) 2021-05-27 2025-05-20 Neuracle Genetics Inc. Dual helper plasmid
KR102685652B1 (ko) * 2021-05-27 2024-07-19 주식회사 뉴라클제네틱스 신규한 듀얼 헬퍼 플라스미드
EP4377471A4 (en) * 2021-07-28 2025-10-15 Atsena Therapeutics Inc AAV TRANSFER PLASMIDS
CN114196705B (zh) * 2021-12-07 2024-09-06 中国科学院深圳先进技术研究院 一种重组腺相关病毒包装质粒、重组腺相关病毒及其应用
WO2023102736A1 (zh) * 2021-12-07 2023-06-15 中国科学院深圳先进技术研究院 一种重组腺相关病毒包装质粒、重组腺相关病毒及其应用
KR20250009975A (ko) * 2022-05-12 2025-01-20 아브너진 인코포레이티드 재조합 파르보바이러스 생산을 위한 조성물 및 방법
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CN116064672B (zh) * 2022-09-15 2025-02-14 思鹏生物科技(苏州)有限公司 利用ires基因提升aav包装效率的方法及其功能基因
WO2024098035A2 (en) * 2022-11-04 2024-05-10 National Resilience, Inc. Methods and compositions for preparing recombinant adeno associated viruses and uses thereof
WO2024160232A1 (zh) * 2023-02-01 2024-08-08 纽福斯(苏州)生物科技有限公司 质粒包装系统及其应用
CN116411024A (zh) * 2023-03-10 2023-07-11 上海泰昶生物技术有限公司 用于生产重组腺相关病毒的质粒系统
US20240352483A1 (en) * 2023-04-03 2024-10-24 Decibel Therapeutics, Inc. Modified rep-cap plasmids and uses thereof
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WO2025050347A1 (en) * 2023-09-07 2025-03-13 Chigenovo Co., Ltd. Aav vector backbone optimization and use thereof
CN117683797B (zh) * 2023-12-04 2024-07-05 广州派真生物技术有限公司 一种用于重组腺相关病毒包装的质粒系统及其应用
CN117925663A (zh) * 2023-12-04 2024-04-26 广州派真生物技术有限公司 一种用于重组腺相关病毒包装的质粒系统及其应用
CN117778431B (zh) * 2023-12-04 2024-10-25 广州派真生物技术有限公司 一种用于重组腺相关病毒包装的质粒系统及其应用
CN121780618A (zh) * 2023-12-13 2026-04-03 广州派真生物技术有限公司 一种高产重组腺相关病毒的辅助质粒及应用
CN117660534B (zh) * 2023-12-13 2024-05-07 广州派真生物技术有限公司 一种降低重组腺相关病毒中宿主细胞dna残留的辅助质粒及应用
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WO2025226842A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Expression control by drg-expressed mirnas
WO2025226841A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Gene therapy approach for treating disorders associated with tnnt2
WO2026011008A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Expression control by skeletal muscle-expressed mirnas
WO2026011009A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders
WO2026030242A1 (en) 2024-07-29 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of facioscapulohumeral muscular dystrophy
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WO2018080277A1 (ko) 2016-10-31 2018-05-03 코오롱생명과학 주식회사 통증 완화 또는 치료용 조성물

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EP3870148A4 (en) 2022-11-09
TW202029957A (zh) 2020-08-16
JP2022511348A (ja) 2022-01-31
KR20210086645A (ko) 2021-07-08
WO2020086881A1 (en) 2020-04-30
JP2025000711A (ja) 2025-01-07
US12383587B2 (en) 2025-08-12
US20210275614A1 (en) 2021-09-09
AU2019363593A1 (en) 2021-04-29
EP3870148A1 (en) 2021-09-01
CA3112883A1 (en) 2020-04-30
IL281909A (en) 2021-05-31
CN112888426A (zh) 2021-06-01

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