JP2022533438A - 改変ウイルス粒子およびその使用 - Google Patents

改変ウイルス粒子およびその使用 Download PDF

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Publication number
JP2022533438A
JP2022533438A JP2021569402A JP2021569402A JP2022533438A JP 2022533438 A JP2022533438 A JP 2022533438A JP 2021569402 A JP2021569402 A JP 2021569402A JP 2021569402 A JP2021569402 A JP 2021569402A JP 2022533438 A JP2022533438 A JP 2022533438A
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JP
Japan
Prior art keywords
aav
protein
amino acid
capsid protein
acid sequence
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Pending
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JP2021569402A
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English (en)
Japanese (ja)
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JP2022533438A5 (enExample
Inventor
リア サビン，
クリストス カラツオス，
スヴェン モーラー－タンク，
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Regeneron Pharmaceuticals Inc
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Regeneron Pharmaceuticals Inc
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Publication of JP2022533438A publication Critical patent/JP2022533438A/ja
Publication of JP2022533438A5 publication Critical patent/JP2022533438A5/ja
Priority to JP2024196799A priority Critical patent/JP2025032110A/ja
Priority to JP2025085876A priority patent/JP2025119015A/ja
Pending legal-status Critical Current

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    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • C07K14/315—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria from Streptococcus (G), e.g. Enterococci
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C07K2319/00—Fusion polypeptide
    • 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
    • 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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Toxicology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2021569402A 2019-05-24 2020-05-22 改変ウイルス粒子およびその使用 Pending JP2022533438A (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2024196799A JP2025032110A (ja) 2019-05-24 2024-11-11 改変ウイルス粒子およびその使用
JP2025085876A JP2025119015A (ja) 2019-05-24 2025-05-22 改変ウイルス粒子およびその使用

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962852791P 2019-05-24 2019-05-24
US62/852,791 2019-05-24
PCT/US2020/034328 WO2020242984A1 (en) 2019-05-24 2020-05-22 Modified viral particles and uses thereof

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024196799A Division JP2025032110A (ja) 2019-05-24 2024-11-11 改変ウイルス粒子およびその使用

Publications (2)

Publication Number Publication Date
JP2022533438A true JP2022533438A (ja) 2022-07-22
JP2022533438A5 JP2022533438A5 (enExample) 2023-05-30

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Family Applications (3)

Application Number Title Priority Date Filing Date
JP2021569402A Pending JP2022533438A (ja) 2019-05-24 2020-05-22 改変ウイルス粒子およびその使用
JP2024196799A Pending JP2025032110A (ja) 2019-05-24 2024-11-11 改変ウイルス粒子およびその使用
JP2025085876A Pending JP2025119015A (ja) 2019-05-24 2025-05-22 改変ウイルス粒子およびその使用

Family Applications After (2)

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JP2024196799A Pending JP2025032110A (ja) 2019-05-24 2024-11-11 改変ウイルス粒子およびその使用
JP2025085876A Pending JP2025119015A (ja) 2019-05-24 2025-05-22 改変ウイルス粒子およびその使用

Country Status (19)

Country Link
US (1) US20220241430A1 (enExample)
EP (1) EP3976631A1 (enExample)
JP (3) JP2022533438A (enExample)
KR (1) KR20220011664A (enExample)
CN (2) CN113874386A (enExample)
AR (2) AR118997A1 (enExample)
AU (1) AU2020283537A1 (enExample)
BR (1) BR112021023692A2 (enExample)
CA (1) CA3140386A1 (enExample)
CL (2) CL2021003096A1 (enExample)
CO (1) CO2021017692A2 (enExample)
IL (1) IL288233A (enExample)
MA (1) MA56035A (enExample)
MX (1) MX2021014338A (enExample)
PE (1) PE20212357A1 (enExample)
PH (1) PH12021552887A1 (enExample)
SG (1) SG11202112917PA (enExample)
TW (1) TW202110869A (enExample)
WO (1) WO2020242984A1 (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12421525B2 (en) 2018-02-28 2025-09-23 University of Pittsburgh—of the Commonwealth System of Higher Education Conjugates comprising AAVs and CAS9 polypeptides
AU2020283537A1 (en) * 2019-05-24 2021-12-16 Regeneron Pharmaceuticals, Inc. Modified viral particles and uses thereof
US20240124892A1 (en) * 2021-03-02 2024-04-18 University Of Pittsburgh-Of The Commonwealth System Of Higher Education Adeno-associated viruses and methods and materials for making and using adeno-associated viruses
US20250195693A1 (en) * 2021-04-30 2025-06-19 Duke University Compositions Comprising Adeno-Associated Virus Chimera Capsid Proteins and Methods of Using the Same
EP4359550A1 (en) * 2021-06-23 2024-05-01 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
AU2023269134A1 (en) 2022-05-09 2024-12-12 Regeneron Pharmaceuticals, Inc. Vectors and methods for in vivo antibody production
WO2023240124A1 (en) 2022-06-07 2023-12-14 Regeneron Pharmaceuticals, Inc. Pseudotyped viral particles for targeting tcr-expressing cells
EP4766849A2 (en) * 2023-08-24 2026-07-01 Ohio State Innovation Foundation Dosages and quality control methods for genome encapsidation for gene therapy purposes
CN121794393A (zh) * 2023-09-07 2026-04-03 里珍纳龙药品有限公司 经共价表面修饰的腺相关病毒的生产和纯化
WO2025111473A1 (en) * 2023-11-21 2025-05-30 Regeneron Pharmaceuticals, Inc. Covalently surface modified adeno-associated virus production by in vitro conjugation, and purification of covalently surface modified adeno-associated virus
WO2026037757A1 (en) 2024-08-13 2026-02-19 F. Hoffmann-La Roche Ag Modified aav particles
WO2026112457A2 (en) * 2024-11-21 2026-05-28 Regeneron Pharmaceuticals, Inc. Heterodimeric antigen-binding molecules that bind to viral particles and uses thereof

Citations (4)

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Publication number Priority date Publication date Assignee Title
JP2002516092A (ja) * 1998-05-28 2002-06-04 アメリカ合衆国 Ａａｖ５ベクターおよびその使用
WO2015191508A1 (en) * 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
WO2019006046A2 (en) * 2017-06-27 2019-01-03 Regeneron Pharmaceuticals, Inc. RECOMBINANT VIRAL PARTICLES WITH MODIFIED TROPISM AND USES THEREOF FOR TARGETED INTRODUCTION OF GENETIC MATERIAL INTO HUMAN CELLS
JP2019001805A (ja) * 2013-03-15 2019-01-10 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 二重グリカン結合性ａａｖベクターのための方法および組成物

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US6261554B1 (en) * 1995-07-25 2001-07-17 Introgene B.V. Compositions for targeted gene delivery
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
DE69936104T2 (de) 1998-11-05 2008-01-17 The Trustees Of The University Of Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
CN103589692B (zh) * 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
EP2158211B1 (en) 2007-05-31 2016-08-10 Medigene AG Mutated structural protein of a parvovirus
WO2011072204A1 (en) 2009-12-10 2011-06-16 Regeneron Pharmaceuticals, Inc. Mice that make heavy chain antibodies
GB201002362D0 (en) 2010-02-11 2010-03-31 Isis Innovation Peptide tag systems that spontaneously form an irreversible link to protein partners via isopeptide bonds
WO2015137802A1 (en) * 2014-03-10 2015-09-17 Uniqure Ip B.V. Further improved aav vectors produced in insect cells
AU2015230938C1 (en) 2014-03-21 2021-10-14 Regeneron Pharmaceuticals, Inc. Non-human animals that make single domain binding proteins
US10550405B2 (en) * 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
SI3645551T1 (sl) 2017-06-27 2024-06-28 Regeneron Pharmaceuticals, Inc. S tropizmi spremenjeni rekombinantni virusni vektorji in njihova uporaba za tarčni vnos genetskega materiala v človeške celice
US12421525B2 (en) * 2018-02-28 2025-09-23 University of Pittsburgh—of the Commonwealth System of Higher Education Conjugates comprising AAVs and CAS9 polypeptides
AU2020283537A1 (en) * 2019-05-24 2021-12-16 Regeneron Pharmaceuticals, Inc. Modified viral particles and uses thereof

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002516092A (ja) * 1998-05-28 2002-06-04 アメリカ合衆国 Ａａｖ５ベクターおよびその使用
JP2019001805A (ja) * 2013-03-15 2019-01-10 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 二重グリカン結合性ａａｖベクターのための方法および組成物
WO2015191508A1 (en) * 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
WO2019006046A2 (en) * 2017-06-27 2019-01-03 Regeneron Pharmaceuticals, Inc. RECOMBINANT VIRAL PARTICLES WITH MODIFIED TROPISM AND USES THEREOF FOR TARGETED INTRODUCTION OF GENETIC MATERIAL INTO HUMAN CELLS

Non-Patent Citations (3)

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Title
JOURNAL OF VIROLOGY, vol. 77, no. 12, JPN5022009488, 2003, pages 6799 - 6810, ISSN: 0005501048 *
JOURNAL OF VIROLOGY, vol. 78, no. 12, JPN5022009487, 2004, pages 6509 - 6516, ISSN: 0005501047 *
JOURNAL OF VIROLOGY, vol. 82, no. 12, JPN6024017753, 2008, pages 5887 - 5911, ISSN: 0005501049 *

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Publication number Publication date
SG11202112917PA (en) 2021-12-30
CL2023000629A1 (es) 2023-10-20
WO2020242984A9 (en) 2021-01-14
JP2025032110A (ja) 2025-03-11
TW202110869A (zh) 2021-03-16
MX2021014338A (es) 2022-01-06
US20220241430A1 (en) 2022-08-04
PE20212357A1 (es) 2021-12-17
AR118997A1 (es) 2021-11-17
KR20220011664A (ko) 2022-01-28
IL288233A (en) 2022-01-01
EP3976631A1 (en) 2022-04-06
MA56035A (fr) 2022-04-06
BR112021023692A2 (pt) 2022-01-04
WO2020242984A1 (en) 2020-12-03
JP2025119015A (ja) 2025-08-13
CL2021003096A1 (es) 2022-09-20
CO2021017692A2 (es) 2022-01-17
CA3140386A1 (en) 2020-12-03
PH12021552887A1 (en) 2022-09-05
AU2020283537A1 (en) 2021-12-16
CN113874386A (zh) 2021-12-31
AR124119A2 (es) 2023-02-15
CN114989267A (zh) 2022-09-02

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