JP7562558B2 - プラスミドシステム - Google Patents

プラスミドシステム Download PDF

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Publication number
JP7562558B2
JP7562558B2 JP2021560095A JP2021560095A JP7562558B2 JP 7562558 B2 JP7562558 B2 JP 7562558B2 JP 2021560095 A JP2021560095 A JP 2021560095A JP 2021560095 A JP2021560095 A JP 2021560095A JP 7562558 B2 JP7562558 B2 JP 7562558B2
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JP
Japan
Prior art keywords
plasmid
rep
gene
helper
cap
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JP2021560095A
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English (en)
Japanese (ja)
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JP2022530192A (ja
JP2022530192A5 (enExample
Inventor
ヘーラー、マークス
ゾンターク、フロリアン
コーバー、ルネ
Original Assignee
アセンド アドヴァンスト セラピーズ リミテッド
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Priority claimed from GBGB1905263.8A external-priority patent/GB201905263D0/en
Priority claimed from EP19169121.1A external-priority patent/EP3722434B1/en
Application filed by アセンド アドヴァンスト セラピーズ リミテッド filed Critical アセンド アドヴァンスト セラピーズ リミテッド
Publication of JP2022530192A publication Critical patent/JP2022530192A/ja
Publication of JP2022530192A5 publication Critical patent/JP2022530192A5/ja
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Classifications

    • 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
    • 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
    • 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
    • C12N2750/14122—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
    • 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

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Saccharide Compounds (AREA)
JP2021560095A 2019-04-12 2020-04-14 プラスミドシステム Active JP7562558B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GBGB1905263.8A GB201905263D0 (en) 2019-04-12 2019-04-12 Plasmid system
GB1905263.8 2019-04-12
EP19169121.1 2019-04-12
EP19169121.1A EP3722434B1 (en) 2019-04-12 2019-04-12 Plasmid system
PCT/GB2020/050947 WO2020208379A1 (en) 2019-04-12 2020-04-14 Plasmid system

Publications (3)

Publication Number Publication Date
JP2022530192A JP2022530192A (ja) 2022-06-28
JP2022530192A5 JP2022530192A5 (enExample) 2023-04-17
JP7562558B2 true JP7562558B2 (ja) 2024-10-07

Family

ID=70295566

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021560095A Active JP7562558B2 (ja) 2019-04-12 2020-04-14 プラスミドシステム

Country Status (11)

Country Link
US (2) US20220162642A1 (enExample)
EP (1) EP3953482A1 (enExample)
JP (1) JP7562558B2 (enExample)
KR (1) KR20220019669A (enExample)
CN (1) CN113891942A (enExample)
AU (1) AU2020272557A1 (enExample)
BR (1) BR112021020401A2 (enExample)
CA (1) CA3136545A1 (enExample)
IL (2) IL296544A (enExample)
SG (1) SG11202111149VA (enExample)
WO (1) WO2020208379A1 (enExample)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ762100A (en) 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
DK3722434T3 (da) 2019-04-12 2022-10-24 Freeline Therapeutics Ltd Plasmidsystem
CN114929735A (zh) 2019-11-01 2022-08-19 自由行疗法有限公司 因子viii构建体
JP2024504365A (ja) * 2021-01-22 2024-01-31 バイオジェン・エムエイ・インコーポレイテッド 組換えウイルスベクター力価を増加させる微小管不安定化剤添加剤
BR112023016997A2 (pt) * 2021-02-26 2023-11-07 Logicbio Therapeutics Inc Fabricação e uso de vetores aav recombinantes
EP4410971A4 (en) * 2021-09-30 2025-06-04 FUJIFILM Corporation METHOD FOR PRODUCING AN ADENO-ASSOCIATED VIRUS, CELLS AND EXPRESSION VECTOR
GB202214328D0 (en) 2022-09-29 2022-11-16 Freeline Therapeutics Ltd Method
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
CN118633572A (zh) * 2023-03-06 2024-09-13 北京锦篮基因科技有限公司 神经元核内包涵体病小鼠模型构建方法及应用
WO2024223931A1 (en) * 2023-04-28 2024-10-31 AstraZeneca Ireland Limited Manufacturing and use of recombinant self-complementary aav vectors
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025083617A1 (en) * 2023-10-18 2025-04-24 Nanocell Therapeutics Holdings B.V. Methods and compositions for nucleic construct delivery
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
WO2025194042A1 (en) 2024-03-14 2025-09-18 Affinia Therapeutics Inc. Plasmid system for production of recombinant adenoassociated virus
EP4628576A1 (en) 2024-04-07 2025-10-08 Ascend Advanced Therapies GmbH Microrna molecules for use in methods for improving recombinant aav production
WO2025238067A1 (en) 2024-05-15 2025-11-20 Ascend Advanced Therapies Gmbh Novel vector system for aav production
WO2026064428A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064444A1 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004508041A (ja) 2000-09-08 2004-03-18 メディジーン・アクチェンゲゼルシャフト 組換え型アデノ随伴ウイルス（ｒＡＡＶ）をパッケージングする親細胞、それらの生成方法、およびその使用
JP2004534543A (ja) 2001-07-13 2004-11-18 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン シュードタイプ化アデノ関連ウイルスおよびその使用
US20050080027A1 (en) 2001-11-30 2005-04-14 Markus Horer Optimized production of viral vectors derived from paroviruses in packaging and production cells by hsv infection or treatment with dna methylation inhibitors

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2330826B1 (es) * 2008-06-04 2010-07-26 Proyecto De Biomedicina Cima, S.L. Sistema para empaquetamiento de adenovirus de alta capacidad.
ES2742180T3 (es) * 2009-03-04 2020-02-13 Deutsches Krebsforsch Proteína activadora del ensamblaje (AAP) y su uso para la fabricación de partículas de parvovirus que consisten esencialmente en VP3
ES2857773T5 (es) 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
HK1202434A1 (en) 2011-11-22 2015-10-02 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
EP3384034B1 (en) 2015-12-02 2020-07-08 The Board of Trustees of the Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
CN108048483B (zh) * 2018-01-30 2021-02-02 中国疾病预防控制中心病毒病预防控制所 复制型重组腺病毒HAdV-5载体系统及其应用

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004508041A (ja) 2000-09-08 2004-03-18 メディジーン・アクチェンゲゼルシャフト 組換え型アデノ随伴ウイルス（ｒＡＡＶ）をパッケージングする親細胞、それらの生成方法、およびその使用
JP2004534543A (ja) 2001-07-13 2004-11-18 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン シュードタイプ化アデノ関連ウイルスおよびその使用
US20050080027A1 (en) 2001-11-30 2005-04-14 Markus Horer Optimized production of viral vectors derived from paroviruses in packaging and production cells by hsv infection or treatment with dna methylation inhibitors

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Jpn J Cancer Res，1999年，vol. 90, no. 4，pp. 476-483
Mol Ther，2003年，vol. 7, no. 6，pp. 839-850

Also Published As

Publication number Publication date
JP2022530192A (ja) 2022-06-28
CN113891942A (zh) 2022-01-04
US20260117255A1 (en) 2026-04-30
BR112021020401A2 (pt) 2021-12-07
EP3953482A1 (en) 2022-02-16
WO2020208379A1 (en) 2020-10-15
AU2020272557A1 (en) 2021-12-09
IL287100A (en) 2021-12-01
IL296544A (en) 2022-11-01
US20220162642A1 (en) 2022-05-26
KR20220019669A (ko) 2022-02-17
SG11202111149VA (en) 2021-11-29
CA3136545A1 (en) 2020-10-15

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