CN116323641A - 工程化的aav载体 - Google Patents

工程化的aav载体 Download PDF

Info

Publication number
CN116323641A
CN116323641A CN202180055061.6A CN202180055061A CN116323641A CN 116323641 A CN116323641 A CN 116323641A CN 202180055061 A CN202180055061 A CN 202180055061A CN 116323641 A CN116323641 A CN 116323641A
Authority
CN
China
Prior art keywords
aav
gly
ser
asn
pro
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CN202180055061.6A
Other languages
English (en)
Chinese (zh)
Inventor
斯蒂利亚诺斯·米查拉基斯
莉娜·措贝尔
塞布丽娜·巴布玆卡
马丁·比尔
赫尔曼·阿默尔
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.)
Ludwig Maximilians Universitaet Muenchen LMU
Original Assignee
Ludwig Maximilians Universitaet Muenchen LMU
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
Application filed by Ludwig Maximilians Universitaet Muenchen LMU filed Critical Ludwig Maximilians Universitaet Muenchen LMU
Publication of CN116323641A publication Critical patent/CN116323641A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/0005—Vertebrate antigens
    • A61K39/0011—Cancer antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A61K39/215—Coronaviridae, e.g. avian infectious bronchitis virus
    • 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
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/525—Virus
    • A61K2039/5256—Virus expressing foreign proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/525—Virus
    • A61K2039/5258—Virus-like particles
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/58—Medicinal preparations containing antigens or antibodies raising an immune response against a target which is not the antigen used for immunisation
    • A61K2039/585—Medicinal preparations containing antigens or antibodies raising an immune response against a target which is not the antigen used for immunisation wherein the target is cancer
    • 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
    • C12N2750/14123—Virus like particles [VLP]
    • 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
    • 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect
    • 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
    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/20011—Coronaviridae
    • C12N2770/20022—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
    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/20011—Coronaviridae
    • C12N2770/20034—Use of virus or viral component as vaccine, e.g. live-attenuated or inactivated virus, VLP, viral protein

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Virology (AREA)
  • Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Mycology (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Pulmonology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Superconductors And Manufacturing Methods Therefor (AREA)
  • Manufacture Of Alloys Or Alloy Compounds (AREA)
  • Transition And Organic Metals Composition Catalysts For Addition Polymerization (AREA)
CN202180055061.6A 2020-09-10 2021-09-10 工程化的aav载体 Pending CN116323641A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP20195554.9 2020-09-10
EP20195554 2020-09-10
PCT/EP2021/074987 WO2022053642A1 (en) 2020-09-10 2021-09-10 Engineered aav vectors

Publications (1)

Publication Number Publication Date
CN116323641A true CN116323641A (zh) 2023-06-23

Family

ID=72744535

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180055061.6A Pending CN116323641A (zh) 2020-09-10 2021-09-10 工程化的aav载体

Country Status (7)

Country Link
US (1) US20230365942A1 (https=)
EP (1) EP4211232A1 (https=)
JP (1) JP2023540960A (https=)
CN (1) CN116323641A (https=)
AU (1) AU2021339003A1 (https=)
CA (1) CA3188763A1 (https=)
WO (1) WO2022053642A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025044353A1 (zh) * 2023-09-01 2025-03-06 北京昌平实验室 靶向性基因编辑酶工程化的腺相关病毒及其药物组合物

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4353737A1 (en) * 2022-10-10 2024-04-17 Universitätsklinikum Hamburg-Eppendorf Fusion proteins comprising a cell surface marker specific vhh
AU2024342922A1 (en) 2023-09-15 2026-04-23 Othair Prothena Limited Cell penetrating agents and uses thereof
AU2024342899A1 (en) 2023-09-15 2026-04-23 Prothena Platform Technologies Limited Methods, compositions, and kits including cell-penetrating agents
WO2026052740A1 (en) * 2024-09-04 2026-03-12 Rockberg Johan Sven Erik Modular platform for gene therapy

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012031760A1 (en) * 2010-09-08 2012-03-15 Medigene Ag Parvovirus mutated structural proteins comprising cross - protective b - cell epitopes of a hpv l2 protein as well as products and methods relating thereto
EP3527223A1 (en) * 2018-02-16 2019-08-21 2A Pharma AB Mutated parvovirus structural protein
WO2019211401A1 (en) * 2018-05-03 2019-11-07 Universitätsklinikum Hamburg-Eppendorf Fusion proteins comprising a cell surface marker specific vhh
CN111375055A (zh) * 2020-02-20 2020-07-07 陈宛莎 一种2019-nCoV亚单位疫苗组合物及其免疫方法
CN111411118A (zh) * 2020-03-31 2020-07-14 鲲羽生物科技(江门)有限公司 抗病原高免卵黄抗体及基于aav载体疫苗制备的方法和制剂

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
AU7522498A (en) 1997-04-08 1998-10-30 Tuchenhagen Gmbh Method and device for accelerated fermentation in cylindro-conical fermentation tanks in beer production
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
EP2012122A1 (en) * 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
CN111560354B (zh) * 2020-05-22 2022-07-19 中国人民解放军总医院第五医学中心 重组新型冠状病毒及其制备方法和应用

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012031760A1 (en) * 2010-09-08 2012-03-15 Medigene Ag Parvovirus mutated structural proteins comprising cross - protective b - cell epitopes of a hpv l2 protein as well as products and methods relating thereto
EP3527223A1 (en) * 2018-02-16 2019-08-21 2A Pharma AB Mutated parvovirus structural protein
WO2019211401A1 (en) * 2018-05-03 2019-11-07 Universitätsklinikum Hamburg-Eppendorf Fusion proteins comprising a cell surface marker specific vhh
CN111375055A (zh) * 2020-02-20 2020-07-07 陈宛莎 一种2019-nCoV亚单位疫苗组合物及其免疫方法
CN111411118A (zh) * 2020-03-31 2020-07-14 鲲羽生物科技(江门)有限公司 抗病原高免卵黄抗体及基于aav载体疫苗制备的方法和制剂

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
KENNETH H WARRINGTON JR等: "Adeno-Associated Virus Type 2 VP2 Capsid Protein Is Nonessential and Can Tolerate Large Peptide Insertions at Its N Terminus", 《JOURNAL OF VIROLOGY》, 1 June 2004 (2004-06-01), pages 6595 - 6609 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025044353A1 (zh) * 2023-09-01 2025-03-06 北京昌平实验室 靶向性基因编辑酶工程化的腺相关病毒及其药物组合物

Also Published As

Publication number Publication date
AU2021339003A1 (en) 2023-02-09
CA3188763A1 (en) 2022-03-17
US20230365942A1 (en) 2023-11-16
EP4211232A1 (en) 2023-07-19
JP2023540960A (ja) 2023-09-27
WO2022053642A1 (en) 2022-03-17

Similar Documents

Publication Publication Date Title
Pupo et al. AAV vectors: The Rubik’s cube of human gene therapy
US20250027056A1 (en) Adeno-Associated-Virus Rep Sequences, Vectors and Viruses
CN116323641A (zh) 工程化的aav载体
CN102341406B (zh) 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
ES2857751T3 (es) Métodos para predecir secuencias de virus ancestrales y usos de los mismos
AU2018234695B2 (en) Polyploid adeno-associated virus vectors and methods of making and using the same
JP4842471B2 (ja) 修飾された抗原性を有するアデノ随伴ウイルスの構造タンパク質と、その製造及び使用
CN108137655A (zh) 逃避抗体的病毒载体的方法和组合物
US12226473B2 (en) SARS-COV-2 antigen polypeptide, recombinant adeno-associated virus expressing the polypeptide, and vaccine containing the virus
CN113913464B (zh) 一种表达载体、重组腺相关病毒及其在制备2019新型冠状病毒疫苗中的应用
CN116249771A (zh) 改进的腺相关病毒基因治疗载体
CN114907457A (zh) 一种寨卡/登革疫苗及其应用
EP2542260A1 (en) Recombinant attenuated parvovirus
JP7811576B2 (ja) ＳＡＲＳ－ＣｏＶ－２に対するＡＡＶ５に基づくワクチン
WO2018066999A2 (en) Chimeric enterovirus virus-like particles
JP2022127591A (ja) パルボウイルスの構造タンパク質およびウイルス様粒子ワクチン
AU2013254897B2 (en) Vectors with modified initiation codon for the translation of AAV-Rep78 useful for production of AAV in insect cells

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination