TW202029957A - Aav三質體系統 - Google Patents

Aav三質體系統 Download PDF

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Publication number
TW202029957A
TW202029957A TW108138732A TW108138732A TW202029957A TW 202029957 A TW202029957 A TW 202029957A TW 108138732 A TW108138732 A TW 108138732A TW 108138732 A TW108138732 A TW 108138732A TW 202029957 A TW202029957 A TW 202029957A
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plastid
nucleic acid
seq
sequence
itr
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TW108138732A
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Chinese (zh)
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薇薇安 崔
興 李
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美商百克莎塔股份有限公司
瑞士商百克莎塔有限公司
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=70331899&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=TW202029957(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by 美商百克莎塔股份有限公司, 瑞士商百克莎塔有限公司 filed Critical 美商百克莎塔股份有限公司
Publication of TW202029957A publication Critical patent/TW202029957A/zh

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • 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
    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • 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
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/40—Systems of functionally co-operating vectors
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/50—Vectors for producing vectors

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (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)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
TW108138732A 2018-10-25 2019-10-25 Aav三質體系統 TW202029957A (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862750603P 2018-10-25 2018-10-25
US62/750,603 2018-10-25

Publications (1)

Publication Number Publication Date
TW202029957A true TW202029957A (zh) 2020-08-16

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ID=70331899

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TW108138732A TW202029957A (zh) 2018-10-25 2019-10-25 Aav三質體系統

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

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US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
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AU2021306697A1 (en) * 2020-07-07 2023-02-09 The Board Of Regents Of The University Of Texas System Adeno-associated virus vector for dwarf open reading frame
JP2023536148A (ja) 2020-07-30 2023-08-23 シェイプ セラピューティクス インク． Ｒａａｖウイルス粒子の誘導産生のための安定な細胞株
EP4232152A4 (en) * 2020-10-21 2025-04-23 Duke University Dual AAV vector-mediated deletion of a large mutation hotspot for the treatment of Duchenne muscular dystrophy
US20240392314A1 (en) * 2020-11-04 2024-11-28 Synplogen Co., Ltd. Production of virus vector plasmid in bacillus subtilis
EP4288553A4 (en) * 2021-02-02 2025-06-25 University of Massachusetts INVERTED TERMINAL REPEAT SEQUENCES OF AAV SEROTYPES 8 AND RH.39 IN GENE THERAPY VECTORS
MX2023012518A (es) * 2021-04-21 2024-02-08 Asimov Inc Sistemas de producción estables para la producción de virus adenoasociado.
AU2022282057B2 (en) * 2021-05-27 2025-01-23 Elisigen Inc. Novel dual helper plasmid
US12305188B2 (en) 2021-05-27 2025-05-20 Neuracle Genetics Inc. Dual helper plasmid
WO2022272297A1 (en) * 2021-06-25 2022-12-29 Oxford Biomedica Solutions Llc Adeno-associated virus packaging systems
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
CN119487183A (zh) * 2022-05-12 2025-02-18 艾诺健基因治疗股份有限公司 用于生产重组细小病毒的组合物和方法
CN119866377A (zh) * 2022-08-31 2025-04-22 镇江蓬勃生物科技有限公司 能够提高腺相关病毒滴度的腺相关病毒结构质粒
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
EP4611784A2 (en) * 2022-11-04 2025-09-10 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
WO2024211394A1 (en) * 2023-04-03 2024-10-10 Decibel Therapeutics, Inc. Modified rep-cap plasmids and uses thereof
WO2024238742A2 (en) * 2023-05-16 2024-11-21 Oxford Biomedica (Us) Llc Compositions and methods for increasing aav productivity
WO2025050347A1 (en) * 2023-09-07 2025-03-13 Chigenovo Co., Ltd. Aav vector backbone optimization and use thereof
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
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
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
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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
TW202603165A (zh) 2024-03-04 2026-01-16 美商凱特治療公司 用於治療杜興氏肌肉失養症之腺相關病毒組成物
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WO2020086881A1 (en) 2020-04-30
CN112888426A (zh) 2021-06-01
IL281909A (en) 2021-05-31
EP3870148A1 (en) 2021-09-01
KR20210086645A (ko) 2021-07-08
EP3870148A4 (en) 2022-11-09
US20210275614A1 (en) 2021-09-09
AU2019363593A1 (en) 2021-04-29
JP2022511348A (ja) 2022-01-31
CA3112883A1 (en) 2020-04-30
JP7560447B2 (ja) 2024-10-02
US12383587B2 (en) 2025-08-12
JP2025000711A (ja) 2025-01-07

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