EA202090332A1 - Улучшенная продукция капсида aav в клетках насекомых - Google Patents

Улучшенная продукция капсида aav в клетках насекомых

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Publication number
EA202090332A1
EA202090332A1 EA202090332A EA202090332A EA202090332A1 EA 202090332 A1 EA202090332 A1 EA 202090332A1 EA 202090332 A EA202090332 A EA 202090332A EA 202090332 A EA202090332 A EA 202090332A EA 202090332 A1 EA202090332 A1 EA 202090332A1
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EA
Eurasian Patent Office
Prior art keywords
production
insect cells
aav
protein
adeno
Prior art date
Application number
EA202090332A
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English (en)
Inventor
Давид Йоханнес Франсуа Дю Плесси
Оливье Тер Брейк
Себастиан Менно Босма
Харальд Петер Альберт Петри
Яцек Лубельский
Original Assignee
ЮНИКЬЮРЕ АйПи Б.В.
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Application filed by ЮНИКЬЮРЕ АйПи Б.В. filed Critical ЮНИКЬЮРЕ АйПи Б.В.
Publication of EA202090332A1 publication Critical patent/EA202090332A1/ru

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0601—Invertebrate cells or tissues, e.g. insect cells; Culture media therefor
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vectore
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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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14044—Chimeric viral vector comprising heterologous viral elements for production of another viral vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14051—Methods of production or purification of viral material
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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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • 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
    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Настоящее изобретение относится к продукции аденоассоциированных вирусных векторов в клетках насекомых. Клетки насекомых, таким образом, содержат первую нуклеотидную последовательность, кодирующую капсидные белки аденоассоциированного вируса (AAV), где инициирующий кодон для трансляции капсидного белка VP1 AAV представляет собой AUG. Выше открытой рамки считывания VP1 помещен альтернативный инициирующий кодон вне рамки таким образом, что инициация трансляции белка VP1 является модифицированной, т.е. уменьшенной, для обеспечения продукции VP1:VP2:VP3 в хорошем стехиометрическом соотношении, что приводит к AAV с высокой активностью.
EA202090332A 2017-07-20 2018-07-20 Улучшенная продукция капсида aav в клетках насекомых EA202090332A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP17182429 2017-07-20
PCT/EP2018/069704 WO2019016349A1 (en) 2017-07-20 2018-07-20 PRODUCTION OF ENHANCED VAA CAPSIDS IN INSECT CELLS

Publications (1)

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EA202090332A1 true EA202090332A1 (ru) 2020-04-29

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ID=59383509

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EA202090332A EA202090332A1 (ru) 2017-07-20 2018-07-20 Улучшенная продукция капсида aav в клетках насекомых

Country Status (11)

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US (2) US11667931B2 (ru)
EP (1) EP3655538A1 (ru)
JP (2) JP2020532286A (ru)
KR (1) KR20200045466A (ru)
CN (1) CN111183225B (ru)
AU (1) AU2018303230A1 (ru)
CA (1) CA3069369A1 (ru)
EA (1) EA202090332A1 (ru)
MX (1) MX2020000663A (ru)
WO (1) WO2019016349A1 (ru)
ZA (1) ZA202000273B (ru)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2020302861A1 (en) * 2019-06-26 2022-02-24 Virovek, Inc. Baculovirus expression system
AU2020398178A1 (en) * 2019-12-04 2022-06-09 Sangamo Therapeutics, Inc. Novel compositions and methods for producing recombinant AAV
CA3169087A1 (en) 2020-04-02 2021-10-07 David Johannes Francois DU PLESSIS Dual bifunctional vectors for aav production
CN115867647A (zh) 2020-04-02 2023-03-28 优尼科生物制药有限公司 新型细胞系
AU2021295718A1 (en) * 2020-06-25 2023-02-02 Ferring Ventures Sa Improved adeno-associated virus gene therapy vectors
CN115427561A (zh) 2021-03-09 2022-12-02 辉大(上海)生物科技有限公司 工程化CRISPR/Cas13系统及其用途
WO2022207899A1 (en) 2021-04-02 2022-10-06 Uniqure Biopharma B.V. Methods for producing single insect cell clones
WO2022253955A2 (en) 2021-06-02 2022-12-08 Uniqure Biopharma B.V. Insect cell production of parvoviral vectors with modified capsid proteins
EP4347849A1 (en) 2021-06-02 2024-04-10 uniQure biopharma B.V. Adeno-associated virus vectors modified to bind high-density lipoprotein
CN117836010A (zh) 2021-06-21 2024-04-05 优尼科生物制药有限公司 改进的裂解程序
WO2023283962A1 (en) 2021-07-16 2023-01-19 Huigene Therapeutics Co., Ltd. Modified aav capsid for gene therapy and methods thereof
WO2023025920A1 (en) 2021-08-26 2023-03-02 Uniqure Biopharma B.V. Insect cell-produced high potency aav vectors with cns-tropism
CN113897396B (zh) * 2021-09-18 2022-08-30 劲帆生物医药科技（武汉）有限公司 一种用于在昆虫细胞中表达包含重叠开放阅读框的基因的表达盒及其应用
CN114150021B (zh) * 2021-11-26 2022-10-28 劲帆生物医药科技（武汉）有限公司 一种包含重叠开放阅读框的基因的表达盒及其在昆虫细胞中的应用
WO2023198745A1 (en) 2022-04-12 2023-10-19 Uniqure Biopharma B.V. Nucleic acid regulation of apoe
WO2023198663A1 (en) 2022-04-12 2023-10-19 Uniqure Biopharma B.V. Nucleic acid regulation of snca
WO2023198702A1 (en) 2022-04-12 2023-10-19 Uniqure Biopharma B.V. Nucleic acid regulation of c9orf72
WO2023198662A1 (en) 2022-04-12 2023-10-19 Uniqure Biopharma B.V. Novel systems for nucleic acid regulation

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4745051A (en) 1983-05-27 1988-05-17 The Texas A&M University System Method for producing a recombinant baculovirus expression vector
ATE404217T1 (de) 1999-06-24 2008-08-15 Univ British Columbia Therapie mit lipoproteinlipase (lpl) variant
US7271002B2 (en) 2001-11-09 2007-09-18 United States Of America, Represented By The Secretary, Department Of Health And Human Services Production of adeno-associated virus in insect cells
US6723551B2 (en) * 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
AU2003212708A1 (en) 2002-03-05 2003-09-16 Stichting Voor De Technische Wetenschappen Baculovirus expression system
AU2006304997B2 (en) 2005-10-20 2012-03-01 Uniqure Ip B.V. Improved AAV vectors produced in insect cells
CN101405033A (zh) * 2006-01-20 2009-04-08 北卡罗来纳大学教堂山分校 增强感染性细小病毒载体在昆虫细胞中的生产
ES2785223T3 (es) * 2006-06-21 2020-10-06 Uniqure Ip Bv Células de insecto para la producción de vectores de AAV
EP2297185A1 (en) 2008-06-17 2011-03-23 Amsterdam Molecular Therapeutics (AMT) B.V. Parvoviral capsid with incorporated gly-ala repeat region
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
CN106459984B (zh) * 2014-03-10 2021-09-07 优尼科Ip有限公司 昆虫细胞中产生的进一步改善的aav载体

Also Published As

Publication number Publication date
US11667931B2 (en) 2023-06-06
JP2023093577A (ja) 2023-07-04
CN111183225B (zh) 2023-11-17
MX2020000663A (es) 2020-07-29
KR20200045466A (ko) 2020-05-04
US20240093231A1 (en) 2024-03-21
US20200248206A1 (en) 2020-08-06
CN111183225A (zh) 2020-05-19
WO2019016349A1 (en) 2019-01-24
EP3655538A1 (en) 2020-05-27
ZA202000273B (en) 2021-02-24
JP2020532286A (ja) 2020-11-12
AU2018303230A1 (en) 2020-01-30
CA3069369A1 (en) 2019-01-24

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