MA49513B1 - Vecteurs viraux recombinants au tropisme modifié et leurs utilisations pour l'introduction ciblée de matériel génétique dans des cellules humaines - Google Patents

Vecteurs viraux recombinants au tropisme modifié et leurs utilisations pour l'introduction ciblée de matériel génétique dans des cellules humaines

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Publication number
MA49513B1
MA49513B1 MA49513A MA49513A MA49513B1 MA 49513 B1 MA49513 B1 MA 49513B1 MA 49513 A MA49513 A MA 49513A MA 49513 A MA49513 A MA 49513A MA 49513 B1 MA49513 B1 MA 49513B1
Authority
MA
Morocco
Prior art keywords
viral vectors
human cells
genetic material
recombinant viral
targeted introduction
Prior art date
Application number
MA49513A
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English (en)
Other versions
MA49513A (fr
Inventor
Andrew J Murphy
Christos Kyratsous
Leah Sabin
Cheng Wang
Original Assignee
Regeneron Pharma
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Filing date
Publication date
Application filed by Regeneron Pharma filed Critical Regeneron Pharma
Publication of MA49513A publication Critical patent/MA49513A/fr
Publication of MA49513B1 publication Critical patent/MA49513B1/fr

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    • 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
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/08—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses
    • C07K16/081—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses from DNA viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2803—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily
    • C07K16/2809—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily against the T-cell receptor (TcR)-CD3 complex
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2851—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the lectin superfamily, e.g. CD23, CD72
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2869—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against hormone receptors
    • 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
    • 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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/30—Immunoglobulins specific features characterized by aspects of specificity or valency
    • C07K2317/31—Immunoglobulins specific features characterized by aspects of specificity or valency multispecific
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/56—Immunoglobulins specific features characterized by immunoglobulin fragments variable (Fv) region, i.e. VH and/or VL
    • C07K2317/569—Single domain, e.g. dAb, sdAb, VHH, VNAR or nanobody®
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622—Single chain antibody (scFv)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/41—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a Myc-tag
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10022—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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10042—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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
    • C12N2750/14141—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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Immunology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Virology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • Endocrinology (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)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)

Abstract

L'invention concerne des compositions et des procédés de reciblage de protéines de capside/capsides/vecteurs viraux recombinés, par exemple, in vivo, avec une molécule de liaison multispécifique, telle qu'un anticorps bispécifique, qui se lie spécifiquement à un épitope hétérologue présenté par la protéine de capside et à une protéine exprimée sur la cellule d'intérêt pour l'administration ciblée d'un nucléotide d'intérêt.
MA49513A 2017-06-27 2018-06-27 Vecteurs viraux recombinants au tropisme modifié et leurs utilisations pour l'introduction ciblée de matériel génétique dans des cellules humaines MA49513B1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762525704P 2017-06-27 2017-06-27
PCT/US2018/039874 WO2019006043A1 (fr) 2017-06-27 2018-06-27 Vecteurs viraux recombinés à tropisme modifié et utilisations associées pour l'introduction ciblée de matériel génétique dans des cellules humaines

Publications (2)

Publication Number Publication Date
MA49513A MA49513A (fr) 2020-05-06
MA49513B1 true MA49513B1 (fr) 2024-05-31

Family

ID=63080487

Family Applications (1)

Application Number Title Priority Date Filing Date
MA49513A MA49513B1 (fr) 2017-06-27 2018-06-27 Vecteurs viraux recombinants au tropisme modifié et leurs utilisations pour l'introduction ciblée de matériel génétique dans des cellules humaines

Country Status (21)

Country Link
US (1) US20200140491A1 (fr)
EP (2) EP3645551B1 (fr)
JP (3) JP7372154B2 (fr)
KR (1) KR20200022434A (fr)
CN (2) CN110799524A (fr)
AU (2) AU2018290882B2 (fr)
BR (2) BR122023021213A2 (fr)
CA (1) CA3066947A1 (fr)
CL (2) CL2019003842A1 (fr)
CO (1) CO2019014683A2 (fr)
DK (1) DK3645551T3 (fr)
FI (1) FI3645551T3 (fr)
IL (1) IL271573A (fr)
LT (1) LT3645551T (fr)
MA (1) MA49513B1 (fr)
MX (2) MX2020000244A (fr)
PE (1) PE20200488A1 (fr)
PH (1) PH12019550266A1 (fr)
PT (1) PT3645551T (fr)
SG (1) SG11201911610TA (fr)
WO (1) WO2019006043A1 (fr)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3736330A1 (fr) * 2019-05-08 2020-11-11 European Molecular Biology Laboratory Particules modifiées du virus adéno-associé (aav) pour la thérapie génique
PE20212357A1 (es) 2019-05-24 2021-12-17 Regeneron Pharma Particulas virales modificadas y usos de estas
WO2021230987A1 (fr) 2020-05-13 2021-11-18 Voyager Therapeutics, Inc. Redirection de tropisme de capsides de vaa
MX2023005446A (es) 2020-11-11 2023-07-18 European Molecular Biology Laboratory Particulas virales modificadas para terapia genica.
CA3233698A1 (fr) 2021-11-04 2023-05-11 Regeneron Pharmaceuticals, Inc. Particules virales reciblees sur le muscle squelettique
CN114213505B (zh) * 2021-12-10 2022-09-20 和元生物技术（上海）股份有限公司 一种适用于特异感染u87-mg细胞的腺相关病毒突变体
WO2023164448A1 (fr) * 2022-02-22 2023-08-31 The University Of North Carolina At Chapel Hill Capsides d'aav neurotropes chimériques
WO2023220603A1 (fr) 2022-05-09 2023-11-16 Regeneron Pharmaceuticals, Inc. Vecteurs et procédés de production d'anticorps in vivo
WO2023215947A1 (fr) * 2022-05-13 2023-11-16 Children's Medical Research Institute Capsides de virus adéno-associés
WO2024026494A1 (fr) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Particules virales reciblées vers le récepteur 1 de transferrine
WO2024107765A2 (fr) 2022-11-14 2024-05-23 Regeneron Pharmaceuticals, Inc. Compositions et procédés d'administration médiée par le récepteur 3 du facteur de croissance des fibroblastes à des astrocytes

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WO1997005266A1 (fr) 1995-07-25 1997-02-13 Introgene B.V. Procedes et moyens d'apport cible de genes
WO1999039734A1 (fr) 1998-02-06 1999-08-12 The Uab Research Foundation Vecteur adenoviral contenant un epitope peptidique heterologue dans la boucle hi du bouton de fibre
DE19849643A1 (de) * 1998-10-29 2000-05-04 Deutsches Krebsforsch An das AAV-Kapsid bindender, den Zelltropismus verändernder Antikörper und Verfahren zum gerichteten Gentransfer
DE19933719A1 (de) * 1999-07-19 2001-01-25 Medigene Ag Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
EP1546359A4 (fr) * 2002-09-12 2007-05-09 Molecular Probes Inc Marquage specifique en fonction du site d'etiquettes d'affinite dans les proteines de fusion
US7273835B2 (en) * 2004-08-04 2007-09-25 Honeywell International Inc. Azeotrope-like compositions of difluoromethane
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WO2014193716A2 (fr) * 2013-05-21 2014-12-04 University Of Florida Research Foundation, Inc. Compositions de vecteur raav3 à modification capsidique et procédés d'utilisation dans la thérapie génétique du cancer du foie humain
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Also Published As

Publication number Publication date
CO2019014683A2 (es) 2020-01-17
KR20200022434A (ko) 2020-03-03
LT3645551T (lt) 2024-05-10
WO2019006043A1 (fr) 2019-01-03
JP2020529196A (ja) 2020-10-08
CL2020003343A1 (es) 2021-06-25
PH12019550266A1 (en) 2021-01-11
EP3645551B1 (fr) 2024-03-13
SG11201911610TA (en) 2020-01-30
MX2023009050A (es) 2023-08-10
EP3645551A1 (fr) 2020-05-06
CN110799524A (zh) 2020-02-14
CL2019003842A1 (es) 2020-07-24
FI3645551T3 (fi) 2024-05-03
DK3645551T3 (da) 2024-05-06
AU2018290882B2 (en) 2023-07-27
PE20200488A1 (es) 2020-03-03
AU2018290882A1 (en) 2020-01-16
RU2020101279A (ru) 2021-07-27
JP2024036644A (ja) 2024-03-15
BR112019027854A2 (pt) 2020-07-07
IL271573A (en) 2020-02-27
US20200140491A1 (en) 2020-05-07
AU2023206143A1 (en) 2023-08-10
JP2022183228A (ja) 2022-12-08
MX2020000244A (es) 2020-09-28
PT3645551T (pt) 2024-05-13
CN117051045A (zh) 2023-11-14
RU2020101279A3 (fr) 2022-02-04
BR122023021213A2 (pt) 2024-02-20
EP4372008A2 (fr) 2024-05-22
JP7372154B2 (ja) 2023-10-31
MA49513A (fr) 2020-05-06
CA3066947A1 (fr) 2019-01-03

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