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
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aav
protein
amino acid
acid sequence
capsid protein
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TW109117155A
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English (en)
Chinese (zh)
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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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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • C07K14/315Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria from Streptococcus (G), e.g. Enterococci
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    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14123Virus like particles [VLP]
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151Methods of production or purification of viral material
    • C12N2750/14152Methods 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)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (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) CN114989267A (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)
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Families Citing this family (10)

* 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
KR20220011664A (ko) * 2019-05-24 2022-01-28 리제너론 파마슈티칼스 인코포레이티드 변형된 바이러스 입자 및 이의 용도
WO2022187377A1 (en) * 2021-03-02 2022-09-09 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
CA3256953A1 (en) 2022-05-09 2023-11-16 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
WO2025054526A1 (en) * 2023-09-07 2025-03-13 Regeneron Pharmaceuticals, Inc. Production and purification of covalently surface modified adeno-associated virus
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

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WO1997005266A1 (en) 1995-07-25 1997-02-13 Introgene B.V. Methods and means 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
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
DK1127150T3 (da) 1998-11-05 2007-09-24 Univ Pennsylvania Nucleinsyresekvenser fra det adeno-associerede virus af serotype 1 samt vektorer og værtsceller, der indeholder disse
SG168422A1 (en) * 2001-11-13 2011-02-28 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
ES2602610T3 (es) 2007-05-31 2017-02-21 Medigene Ag Proteína estructural mutada de un parvovirus
MY172472A (en) 2009-12-10 2019-11-26 Regeneron Pharma 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
IL293294B2 (en) * 2013-03-15 2023-04-01 Univ North Carolina Chapel Hill Methods and compounds for ligation of paired aav glycan vectors
ES2990174T3 (es) * 2014-03-10 2024-11-29 Uniqure Ip Bv Vectores de AAV mejorados producidos en células de insecto
BR112016021572A2 (pt) 2014-03-21 2017-10-03 Regeneron Pharma Métodos para preparar um animal não humano geneticamente modificado e para produzir proteínas de ligação, hibridoma, ácido nucleico, célula, e, proteína de ligação
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
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
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
KR20220011664A (ko) * 2019-05-24 2022-01-28 리제너론 파마슈티칼스 인코포레이티드 변형된 바이러스 입자 및 이의 용도

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AR118997A1 (es) 2021-11-17
BR112021023692A2 (pt) 2022-01-04
CL2023000629A1 (es) 2023-10-20
CL2021003096A1 (es) 2022-09-20
EP3976631A1 (en) 2022-04-06
KR20220011664A (ko) 2022-01-28
US20220241430A1 (en) 2022-08-04
CN114989267A (zh) 2022-09-02
WO2020242984A9 (en) 2021-01-14
JP2022533438A (ja) 2022-07-22
CN113874386A (zh) 2021-12-31
CA3140386A1 (en) 2020-12-03
MX2021014338A (es) 2022-01-06
CO2021017692A2 (es) 2022-01-17
PH12021552887A1 (en) 2022-09-05
SG11202112917PA (en) 2021-12-30
WO2020242984A1 (en) 2020-12-03
IL288233A (en) 2022-01-01
MA56035A (fr) 2022-04-06
AR124119A2 (es) 2023-02-15
JP2025032110A (ja) 2025-03-11
PE20212357A1 (es) 2021-12-17
JP2025119015A (ja) 2025-08-13
AU2020283537A1 (en) 2021-12-16

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