IL281909A - Aav triple-plasmid system - Google Patents

Aav triple-plasmid system

Info

Publication number
IL281909A
IL281909A IL281909A IL28190921A IL281909A IL 281909 A IL281909 A IL 281909A IL 281909 A IL281909 A IL 281909A IL 28190921 A IL28190921 A IL 28190921A IL 281909 A IL281909 A IL 281909A
Authority
IL
Israel
Prior art keywords
adeno
associated virus
plasmid system
triple plasmid
triple
Prior art date
Application number
IL281909A
Other languages
English (en)
Hebrew (he)
Original Assignee
Baxalta GmbH
Baxalta Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
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=IL281909(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Baxalta GmbH, Baxalta Inc filed Critical Baxalta GmbH
Publication of IL281909A publication Critical patent/IL281909A/en

Links

Classifications

    • 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
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • 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)
IL281909A 2018-10-25 2021-03-30 Aav triple-plasmid system IL281909A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862750603P 2018-10-25 2018-10-25
PCT/US2019/057916 WO2020086881A1 (en) 2018-10-25 2019-10-24 Aav triple-plasmid system

Publications (1)

Publication Number Publication Date
IL281909A true IL281909A (en) 2021-05-31

Family

ID=70331899

Family Applications (1)

Application Number Title Priority Date Filing Date
IL281909A IL281909A (en) 2018-10-25 2021-03-30 Aav triple-plasmid system

Country Status (10)

Country Link
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
US10557149B1 (en) * 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
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
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
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
WO2025217308A1 (en) * 2024-04-10 2025-10-16 Solid Biosciences Inc. Method of aav production
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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
WO2026019230A1 (ko) * 2024-07-15 2026-01-22 삼성바이오에피스 주식회사 스터퍼를 포함하는 재조합 aav 생산을 위한 벡터 시스템
WO2026030242A1 (en) 2024-07-29 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of facioscapulohumeral muscular dystrophy
WO2026030244A1 (en) 2024-07-30 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of limb girdle muscular dystrophy 2a
WO2026050402A1 (en) 2024-08-29 2026-03-05 Kate Therapeutics, Inc. Liver de-targeted muscle tropic capsids
CN119662733B (zh) * 2024-12-09 2025-10-28 广州派真生物技术有限公司 一种高产rAAV的质粒系统及应用

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Also Published As

Publication number Publication date
WO2020086881A1 (en) 2020-04-30
CN112888426A (zh) 2021-06-01
EP3870148A1 (en) 2021-09-01
TW202029957A (zh) 2020-08-16
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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