TW202110869A - 修飾之病毒顆粒及其用途 - Google Patents

修飾之病毒顆粒及其用途 Download PDF

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Publication number
TW202110869A
TW202110869A TW109117155A TW109117155A TW202110869A TW 202110869 A TW202110869 A TW 202110869A TW 109117155 A TW109117155 A TW 109117155A TW 109117155 A TW109117155 A TW 109117155A TW 202110869 A TW202110869 A TW 202110869A
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Taiwan
Prior art keywords
aav
protein
amino acid
acid sequence
capsid protein
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TW109117155A
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English (en)
Chinese (zh)
Inventor
利亞 薩賓
克里斯托斯 基拉索斯
坦克 斯文 莫勒
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美商再生元醫藥公司
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Publication of TW202110869A publication Critical patent/TW202110869A/zh

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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)
TW109117155A 2019-05-24 2020-05-22 修飾之病毒顆粒及其用途 TW202110869A (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962852791P 2019-05-24 2019-05-24
US62/852,791 2019-05-24

Publications (1)

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TW202110869A true TW202110869A (zh) 2021-03-16

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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

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
DE69939169D1 (de) 1998-05-28 2008-09-04 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
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
ES2926774T3 (es) * 2013-03-15 2022-10-28 Univ North Carolina Chapel Hill Métodos y composiciones para vectores AAV de doble unión de glicano
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
US10577627B2 (en) * 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
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
EP4706687A3 (en) 2017-06-27 2026-07-15 Regeneron Pharmaceuticals, Inc. Tropism-modified recombinant viral particles and uses thereof for the targeted introduction of genetic material into human cells
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

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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
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
JP2022533438A (ja) 2022-07-22
CN113874386A (zh) 2021-12-31
AR124119A2 (es) 2023-02-15
CN114989267A (zh) 2022-09-02

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