MX2023009050A - Vectores virales recombinantes con tropismo modificado y usos de estos para la introduccion dirigida de material genetico a celulas humanas. - Google Patents

Vectores virales recombinantes con tropismo modificado y usos de estos para la introduccion dirigida de material genetico a celulas humanas.

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Publication number
MX2023009050A
MX2023009050A MX2023009050A MX2023009050A MX2023009050A MX 2023009050 A MX2023009050 A MX 2023009050A MX 2023009050 A MX2023009050 A MX 2023009050A MX 2023009050 A MX2023009050 A MX 2023009050A MX 2023009050 A MX2023009050 A MX 2023009050A
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MX
Mexico
Prior art keywords
recombinant viral
tropism
viral vectors
human cells
genetic material
Prior art date
Application number
MX2023009050A
Other languages
English (en)
Inventor
Christos Kyratsous
Andrew J Murphy
Leah Sabin
Cheng Wang
Original Assignee
Regeneron Pharma
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Publication date
Application filed by Regeneron Pharma filed Critical Regeneron Pharma
Publication of MX2023009050A publication Critical patent/MX2023009050A/es

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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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • 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
    • 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
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    • 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
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    • 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
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    • 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
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    • 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
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    • 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
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    • 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)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Virology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (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)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

En la presente descripción se proporcionan composiciones y métodos para redireccionar proteínas de la cápside viral/cápsides/vectores recombinantes, por ejemplo in vivo, con una molécula de unión multiespecífica, tal como un anticuerpo biespecífico, que se une específicamente a un epítopo heterólogo presentado por la proteína de la cápside y una proteína expresada en la célula de interés para el suministro dirigido de un nucleótido de interés.
MX2023009050A 2017-06-27 2020-01-08 Vectores virales recombinantes con tropismo modificado y usos de estos para la introduccion dirigida de material genetico a celulas humanas. MX2023009050A (es)

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US201762525704P 2017-06-27 2017-06-27

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MX2023009050A true MX2023009050A (es) 2023-08-10

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MX2020000244A MX2020000244A (es) 2017-06-27 2018-06-27 Vectores virales recombinantes con tropismo modificado y usos de estos para la introduccion dirigida de material genetico a celulas humanas.
MX2023009050A MX2023009050A (es) 2017-06-27 2020-01-08 Vectores virales recombinantes con tropismo modificado y usos de estos para la introduccion dirigida de material genetico a celulas humanas.

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US (1) US20200140491A1 (es)
EP (2) EP3645551B1 (es)
JP (3) JP7372154B2 (es)
CN (2) CN110799524A (es)
AU (2) AU2018290882B2 (es)
BR (2) BR112019027854A2 (es)
CA (1) CA3066947A1 (es)
CL (2) CL2019003842A1 (es)
CO (1) CO2019014683A2 (es)
DK (1) DK3645551T5 (es)
ES (1) ES2978295T3 (es)
FI (1) FI3645551T3 (es)
HR (1) HRP20240773T1 (es)
HU (1) HUE066398T2 (es)
IL (1) IL271573A (es)
LT (1) LT3645551T (es)
MA (1) MA49513B1 (es)
MD (1) MD3645551T2 (es)
MX (2) MX2020000244A (es)
PE (1) PE20200488A1 (es)
PH (1) PH12019550266A1 (es)
PL (1) PL3645551T3 (es)
PT (1) PT3645551T (es)
RS (1) RS65569B1 (es)
SG (1) SG11201911610TA (es)
SI (1) SI3645551T1 (es)
WO (1) WO2019006043A1 (es)

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WO2020242984A1 (en) 2019-05-24 2020-12-03 Regeneron Pharmaceuticals, Inc. Modified viral particles and uses thereof
BR112022023106A2 (pt) 2020-05-13 2023-01-17 Voyager Therapeutics Inc Redirecionamento de tropismo de capsídeos de aav
CN116648448A (zh) 2020-11-11 2023-08-25 欧洲分子生物学实验室 用于基因治疗的修饰病毒粒子
CA3233698A1 (en) 2021-11-04 2023-05-11 Regeneron Pharmaceuticals, Inc. Viral particles retargeted to skeletal muscle
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 (en) * 2022-02-22 2023-08-31 The University Of North Carolina At Chapel Hill Chimeric neurotropic aav capsids
WO2023220603A1 (en) 2022-05-09 2023-11-16 Regeneron Pharmaceuticals, Inc. Vectors and methods for in vivo antibody production
WO2023215947A1 (en) * 2022-05-13 2023-11-16 Children's Medical Research Institute Adeno-associated virus capsids
WO2024026494A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Viral particles retargeted to transferrin receptor 1
WO2024107765A2 (en) 2022-11-14 2024-05-23 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
WO2024130175A2 (en) * 2022-12-16 2024-06-20 Regeneron Pharmaceuticals, Inc. Antigen-binding molecules that bind to aav particles and uses
WO2024131982A1 (zh) * 2022-12-23 2024-06-27 北京大学宁波海洋药物研究院 衣壳蛋白-抗体偶联物、修饰的病毒、药物组合和用途
WO2024173248A1 (en) 2023-02-13 2024-08-22 Regeneron Pharmaceuticals, Inc. Treatment of muscle related disorders with anti-human cacng1 antibodies

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HUE066398T2 (hu) 2024-07-28
AU2023206143A1 (en) 2023-08-10
ES2978295T3 (es) 2024-09-10
EP3645551A1 (en) 2020-05-06
BR122023021213A2 (pt) 2024-02-20
FI3645551T3 (fi) 2024-05-03
DK3645551T5 (da) 2024-08-26
SI3645551T1 (sl) 2024-06-28
EP4372008A2 (en) 2024-05-22
JP2022183228A (ja) 2022-12-08
JP2020529196A (ja) 2020-10-08
CL2020003343A1 (es) 2021-06-25
PH12019550266A1 (en) 2021-01-11
CO2019014683A2 (es) 2020-01-17
RU2020101279A3 (es) 2022-02-04
RS65569B1 (sr) 2024-06-28
AU2018290882B2 (en) 2023-07-27
EP3645551B1 (en) 2024-03-13
LT3645551T (lt) 2024-05-10
CA3066947A1 (en) 2019-01-03
WO2019006043A1 (en) 2019-01-03
IL271573A (en) 2020-02-27
MA49513B1 (fr) 2024-05-31
BR112019027854A2 (pt) 2020-07-07
PE20200488A1 (es) 2020-03-03
JP7372154B2 (ja) 2023-10-31
JP2024036644A (ja) 2024-03-15
PL3645551T3 (pl) 2024-07-01
US20200140491A1 (en) 2020-05-07
RU2020101279A (ru) 2021-07-27
SG11201911610TA (en) 2020-01-30
MA49513A (fr) 2020-05-06
AU2018290882A1 (en) 2020-01-16
DK3645551T3 (da) 2024-05-06
EP4372008A3 (en) 2024-07-31
HRP20240773T1 (hr) 2024-09-13
CN117051045A (zh) 2023-11-14
PT3645551T (pt) 2024-05-13
CL2019003842A1 (es) 2020-07-24
KR20200022434A (ko) 2020-03-03
MX2020000244A (es) 2020-09-28
CN110799524A (zh) 2020-02-14
MD3645551T2 (ro) 2024-08-31

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