KR20220011664A - 변형된 바이러스 입자 및 이의 용도 - Google Patents

변형된 바이러스 입자 및 이의 용도 Download PDF

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Publication number
KR20220011664A
KR20220011664A KR1020217041074A KR20217041074A KR20220011664A KR 20220011664 A KR20220011664 A KR 20220011664A KR 1020217041074 A KR1020217041074 A KR 1020217041074A KR 20217041074 A KR20217041074 A KR 20217041074A KR 20220011664 A KR20220011664 A KR 20220011664A
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South Korea
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aav
protein
amino acid
acid sequence
capsid protein
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KR1020217041074A
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English (en)
Korean (ko)
Inventor
리 사빈
크리스토스 키랏수스
스벤 몰러-탱크
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리제너론 파마슈티칼스 인코포레이티드
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Publication of KR20220011664A publication Critical patent/KR20220011664A/ko
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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)
KR1020217041074A 2019-05-24 2020-05-22 변형된 바이러스 입자 및 이의 용도 Pending KR20220011664A (ko)

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

Publications (1)

Publication Number Publication Date
KR20220011664A true KR20220011664A (ko) 2022-01-28

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KR1020217041074A Pending KR20220011664A (ko) 2019-05-24 2020-05-22 변형된 바이러스 입자 및 이의 용도

Country Status (19)

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

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2019227910B2 (en) 2018-02-28 2025-07-17 The Regents Of The University Of California A modular system for gene and protein delivery based on AAV
JP2022533438A (ja) * 2019-05-24 2022-07-22 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 改変ウイルス粒子およびその使用
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
WO2022232575A1 (en) * 2021-04-30 2022-11-03 Duke University Compositions comprising adeno-associated virus chimera capsid proteins and methods of using the same
CA3222914A1 (en) * 2021-06-23 2022-12-29 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
US20250302998A1 (en) 2022-05-09 2025-10-02 Regeneron Pharmaceuticals, Inc. Vectors and methods for in vivo antibody production
EP4536836A1 (en) 2022-06-07 2025-04-16 Regeneron Pharmaceuticals, Inc. Pseudotyped viral particles for targeting tcr-expressing cells
WO2025042462A2 (en) * 2023-08-24 2025-02-27 Ohio State Innovation Foundation Assays and methods for quality control of genome packaging for gene therapy
TW202528546A (zh) * 2023-09-07 2025-07-16 美商再生元醫藥公司 經共價表面修飾之腺相關病毒的生產及純化
TW202540433A (zh) * 2023-11-21 2025-10-16 美商再生元醫藥公司 藉由活體外接合產生共價表面修飾之腺相關病毒及共價表面修飾的腺相關病毒之純化
WO2026037757A1 (en) 2024-08-13 2026-02-19 F. Hoffmann-La Roche Ag Modified aav particles

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2224907A1 (en) * 1995-07-25 1997-02-13 Introgene B.V. Methods and means for targeted gene delivery
WO1998011244A2 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services 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
WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
ATE362542T1 (de) 1998-11-05 2007-06-15 Univ Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
MX359371B (es) * 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
ES2602610T3 (es) 2007-05-31 2017-02-21 Medigene Ag Proteína estructural mutada de un parvovirus
ES2724975T3 (es) 2009-12-10 2019-09-18 Regeneron Pharma Ratones que producen anticuerpos de cadena pesada
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
CA2942289C (en) * 2014-03-10 2024-05-21 Uniqure Ip B.V. Further improved aav vectors produced in insect cells
CA3124228C (en) 2014-03-21 2024-05-14 Regeneron Pharmaceuticals, Inc. Non-human animals that make single domain binding proteins
EP3151866B1 (en) * 2014-06-09 2023-03-08 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
PE20200488A1 (es) 2017-06-27 2020-03-03 Regeneron Pharma Vectores virales recombinantes con tropismo modificado y usos de estos para la introduccion dirigida de material genetico a celulas humanas
LT3645553T (lt) * 2017-06-27 2023-04-25 Regeneron Pharmaceuticals, Inc. Modifikuoto tropizmo rekombinantinės viruso dalelės ir jų panaudojimas genetinės medžiagos tiksliniam įvedimui į žmogaus ląsteles
AU2019227910B2 (en) * 2018-02-28 2025-07-17 The Regents Of The University Of California A modular system for gene and protein delivery based on AAV
JP2022533438A (ja) * 2019-05-24 2022-07-22 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 改変ウイルス粒子およびその使用

Also Published As

Publication number Publication date
PH12021552887A1 (en) 2022-09-05
JP2022533438A (ja) 2022-07-22
AR124119A2 (es) 2023-02-15
JP2025032110A (ja) 2025-03-11
SG11202112917PA (en) 2021-12-30
PE20212357A1 (es) 2021-12-17
CO2021017692A2 (es) 2022-01-17
TW202110869A (zh) 2021-03-16
AR118997A1 (es) 2021-11-17
CL2023000629A1 (es) 2023-10-20
CL2021003096A1 (es) 2022-09-20
AU2020283537A1 (en) 2021-12-16
WO2020242984A9 (en) 2021-01-14
MA56035A (fr) 2022-04-06
BR112021023692A2 (pt) 2022-01-04
MX2021014338A (es) 2022-01-06
JP2025119015A (ja) 2025-08-13
WO2020242984A1 (en) 2020-12-03
CA3140386A1 (en) 2020-12-03
US20220241430A1 (en) 2022-08-04
IL288233A (en) 2022-01-01
CN114989267A (zh) 2022-09-02
EP3976631A1 (en) 2022-04-06
CN113874386A (zh) 2021-12-31

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