CN113891942A - 质粒系统 - Google Patents

质粒系统 Download PDF

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Publication number
CN113891942A
CN113891942A CN202080039790.8A CN202080039790A CN113891942A CN 113891942 A CN113891942 A CN 113891942A CN 202080039790 A CN202080039790 A CN 202080039790A CN 113891942 A CN113891942 A CN 113891942A
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CN
China
Prior art keywords
plasmid
rep
gene
helper
cap
Prior art date
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Pending
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CN202080039790.8A
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English (en)
Chinese (zh)
Inventor
M·霍莱尔
F·桑塔格
R·科伯
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Denggao Manufacturing Co ltd
Original Assignee
Free Exercise Therapy Co ltd
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Filing date
Publication date
Priority claimed from GBGB1905263.8A external-priority patent/GB201905263D0/en
Priority claimed from EP19169121.1A external-priority patent/EP3722434B1/en
Application filed by Free Exercise Therapy Co ltd filed Critical Free Exercise Therapy Co ltd
Publication of CN113891942A publication Critical patent/CN113891942A/zh
Pending legal-status Critical Current

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    • 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
    • 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
    • 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

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  • 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)
CN202080039790.8A 2019-04-12 2020-04-14 质粒系统 Pending CN113891942A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GB1905263.8 2019-04-12
EP19169121.1 2019-04-12
GBGB1905263.8A GB201905263D0 (en) 2019-04-12 2019-04-12 Plasmid system
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 (1)

Publication Number Publication Date
CN113891942A true CN113891942A (zh) 2022-01-04

Family

ID=70295566

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202080039790.8A Pending CN113891942A (zh) 2019-04-12 2020-04-14 质粒系统

Country Status (11)

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

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202545984A (zh) 2017-08-09 2025-12-01 美商生物化學醫療公司 核酸分子及其用途
WO2020033863A1 (en) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
EP3722434B1 (en) 2019-04-12 2022-07-27 Freeline Therapeutics Limited Plasmid system
EP4051704A2 (en) 2019-11-01 2022-09-07 Freeline Therapeutics Limited Factor viii construct
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
AU2022227005B2 (en) * 2021-02-26 2025-09-11 Logicbio Therapeutics, Inc. Manufacturing and use of recombinant aav vectors
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
WO2024223931A1 (en) * 2023-04-28 2024-10-31 AstraZeneca Ireland Limited Manufacturing and use of recombinant self-complementary aav vectors
TW202525832A (zh) 2023-08-31 2025-07-01 美商戴諾治療公司 衣殼多肽及其使用方法(二)
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
TW202548023A (zh) 2024-03-14 2025-12-16 美商雅菲納治療公司 用於生產重組腺相關病毒的質體系統
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
WO2026064444A1 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
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

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030103939A1 (en) * 2001-07-13 2003-06-05 Engelhardt John F. Pseudotyped adeno-associated viruses and uses thereof
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
CN102112616A (zh) * 2008-06-04 2011-06-29 西马生物医学计划公司 高容量腺病毒的包装系统
CN102341406A (zh) * 2009-03-04 2012-02-01 德国癌症研究中心 组装活化蛋白（aap）及其用于制备基本上由vp3组成的细小病毒颗粒的应用
CN108048483A (zh) * 2018-01-30 2018-05-18 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

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE10044384A1 (de) 2000-09-08 2002-04-18 Medigene Ag Wirtszellen zur Verpackung von rekombinantem Adeno-assoziiertem Virus (rAAV), Verfahren zu ihrer Herstellung und deren Verwendung
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
JP6348064B2 (ja) 2011-11-22 2018-06-27 ザ チルドレンズ ホスピタル オブ フィラデルフィア 効率の高いトランスジーン送達のためのウイルスベクター
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
US10385320B2 (en) 2015-12-02 2019-08-20 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030103939A1 (en) * 2001-07-13 2003-06-05 Engelhardt John F. Pseudotyped adeno-associated viruses and uses thereof
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
CN102112616A (zh) * 2008-06-04 2011-06-29 西马生物医学计划公司 高容量腺病毒的包装系统
CN102341406A (zh) * 2009-03-04 2012-02-01 德国癌症研究中心 组装活化蛋白（aap）及其用于制备基本上由vp3组成的细小病毒颗粒的应用
CN108048483A (zh) * 2018-01-30 2018-05-18 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

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
EMMERLING VV等: "Rational plasmid design and bioprocess optimization to enhance recombinant adeno-associated virus (AAV) productivity in mammalian cells", BIOTECHNOL J, vol. 11, no. 2, 18 August 2015 (2015-08-18), pages 3 *
宁秦洁等: "人体肝脏靶向性新型AAV血清型载体的研发", 华东理工大学学报(自然科学版), vol. 46, no. 3, 30 June 2020 (2020-06-30), pages 404 - 410 *
胡容峰等: "工业药剂学", vol. 1, 31 August 2010, 北京：中国中医药出版社, pages: 450 - 451 *
董小岩: "新型重组AAV5/5载体及包装系统", 生物工程学报, vol. 26, no. 5, 25 May 2010 (2010-05-25), pages 679 - 686 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Also Published As

Publication number Publication date
IL296544A (en) 2022-11-01
KR20220019669A (ko) 2022-02-17
US20220162642A1 (en) 2022-05-26
JP2022530192A (ja) 2022-06-28
BR112021020401A2 (pt) 2021-12-07
EP3953482A1 (en) 2022-02-16
IL287100A (en) 2021-12-01
SG11202111149VA (en) 2021-11-29
CA3136545A1 (en) 2020-10-15
JP7562558B2 (ja) 2024-10-07
WO2020208379A1 (en) 2020-10-15
AU2020272557A1 (en) 2021-12-09

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Applicant before: Free exercise therapy Co.,Ltd.

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CB02 Change of applicant information

Country or region after: Britain

Address after: Cheshire

Applicant after: Asend Advanced Therapy Co.,Ltd.

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