MA56035A - Particules virales modifiées et leurs utilisations - Google Patents

Particules virales modifiées et leurs utilisations

Info

Publication number
MA56035A
MA56035A MA056035A MA56035A MA56035A MA 56035 A MA56035 A MA 56035A MA 056035 A MA056035 A MA 056035A MA 56035 A MA56035 A MA 56035A MA 56035 A MA56035 A MA 56035A
Authority
MA
Morocco
Prior art keywords
viral particles
modified viral
modified
particles
viral
Prior art date
Application number
MA056035A
Other languages
English (en)
French (fr)
Inventor
Christos Kyratsous
Sven Moller-Tank
Leah Sabin
Original Assignee
Regeneron Pharma
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 Regeneron Pharma filed Critical Regeneron Pharma
Publication of MA56035A publication Critical patent/MA56035A/fr

Links

Classifications

    • 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • 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
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

Landscapes

  • 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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (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)
MA056035A 2019-05-24 2020-05-22 Particules virales modifiées et leurs utilisations MA56035A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962852791P 2019-05-24 2019-05-24

Publications (1)

Publication Number Publication Date
MA56035A true MA56035A (fr) 2022-04-06

Family

ID=71094822

Family Applications (1)

Application Number Title Priority Date Filing Date
MA056035A MA56035A (fr) 2019-05-24 2020-05-22 Particules virales modifiées et leurs utilisations

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
US12421525B2 (en) 2018-02-28 2025-09-23 University of Pittsburgh—of the Commonwealth System of Higher Education Conjugates comprising AAVs and CAS9 polypeptides
BR112021023692A2 (pt) * 2019-05-24 2022-01-04 Regeneron Pharma Partícula viral de vírus adeno-associado recombinante, proteína de capsídeo de vírus adeno-associado, molécula de ácido nucleico, célula empacotadora, métodos de produção de uma partícula viral e de distribuição de um nucleotídeo, e, composição farmacêutica
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
US20250195693A1 (en) * 2021-04-30 2025-06-19 Duke University Compositions Comprising Adeno-Associated Virus Chimera Capsid Proteins and Methods of Using the Same
US20240294578A1 (en) * 2021-06-23 2024-09-05 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
WO2023240124A1 (en) 2022-06-07 2023-12-14 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
JPH11510050A (ja) 1995-07-25 1999-09-07 イントロヘーネ ベスローテン フェンノートシャップ 標的遺伝子送達のための方法および手段
CA2265460A1 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secre Tary, 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
EP1082413B1 (en) 1998-05-28 2008-07-23 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav5 vector and uses thereof
CA2349838C (en) 1998-11-05 2011-06-07 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
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
US20100203083A1 (en) 2007-05-31 2010-08-12 Medigene Ag Mutated structural protein of a parvovirus
US8754287B2 (en) 2009-12-10 2014-06-17 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
JP6396988B2 (ja) * 2013-03-15 2018-09-26 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
WO2015137802A1 (en) * 2014-03-10 2015-09-17 Uniqure Ip B.V. Further improved aav vectors produced in insect cells
KR102276752B1 (ko) 2014-03-21 2021-07-13 리제너론 파마슈티칼스 인코포레이티드 단일 도메인 결합 단백질을 생산하는 비-인간 동물
WO2015191508A1 (en) * 2014-06-09 2015-12-17 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
PL3645553T3 (pl) * 2017-06-27 2023-07-17 Regeneron Pharmaceuticals, Inc. Zrekombinowane cząstki wirusowe o zmodyfikowanym tropizmie i ich zastosowania do celowanego wprowadzania materiału genetycznego do komórek ludzkich
MX2020000244A (es) 2017-06-27 2020-09-28 Regeneron Pharma Vectores virales recombinantes con tropismo modificado y usos de estos para la introduccion dirigida de material genetico a celulas humanas.
US12421525B2 (en) * 2018-02-28 2025-09-23 University of Pittsburgh—of the Commonwealth System of Higher Education Conjugates comprising AAVs and CAS9 polypeptides
BR112021023692A2 (pt) * 2019-05-24 2022-01-04 Regeneron Pharma Partícula viral de vírus adeno-associado recombinante, proteína de capsídeo de vírus adeno-associado, molécula de ácido nucleico, célula empacotadora, métodos de produção de uma partícula viral e de distribuição de um nucleotídeo, e, composição farmacêutica

Also Published As

Publication number Publication date
EP3976631A1 (en) 2022-04-06
AR124119A2 (es) 2023-02-15
MX2021014338A (es) 2022-01-06
KR20220011664A (ko) 2022-01-28
US20220241430A1 (en) 2022-08-04
CN113874386A (zh) 2021-12-31
JP2025032110A (ja) 2025-03-11
CL2021003096A1 (es) 2022-09-20
CN114989267A (zh) 2022-09-02
CO2021017692A2 (es) 2022-01-17
CA3140386A1 (en) 2020-12-03
TW202110869A (zh) 2021-03-16
AR118997A1 (es) 2021-11-17
CL2023000629A1 (es) 2023-10-20
PE20212357A1 (es) 2021-12-17
WO2020242984A9 (en) 2021-01-14
JP2025119015A (ja) 2025-08-13
IL288233A (en) 2022-01-01
PH12021552887A1 (en) 2022-09-05
AU2020283537A1 (en) 2021-12-16
BR112021023692A2 (pt) 2022-01-04
WO2020242984A1 (en) 2020-12-03
SG11202112917PA (en) 2021-12-30
JP2022533438A (ja) 2022-07-22

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