BR112020023292A2 - vírus adeno-associado (aav), composição, método para a entrega de um transgene a uma célula, uso do aav, método para o tratamento de um indivíduo, vetor, célula, método para a produção de um vírus adeno-associado (aav), vírus adeno-associado (aav) - Google Patents

vírus adeno-associado (aav), composição, método para a entrega de um transgene a uma célula, uso do aav, método para o tratamento de um indivíduo, vetor, célula, método para a produção de um vírus adeno-associado (aav), vírus adeno-associado (aav) Download PDF

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Publication number
BR112020023292A2
BR112020023292A2 BR112020023292-0A BR112020023292A BR112020023292A2 BR 112020023292 A2 BR112020023292 A2 BR 112020023292A2 BR 112020023292 A BR112020023292 A BR 112020023292A BR 112020023292 A2 BR112020023292 A2 BR 112020023292A2
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Prior art keywords
aav
seq
cell
fact
capsid
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BR112020023292-0A
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English (en)
Portuguese (pt)
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Peter Cameron Colosi
Silvia Ramirez
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Biomarin Pharmaceutical Inc.
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Publication of BR112020023292A2 publication Critical patent/BR112020023292A2/pt

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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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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    • 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
    • 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
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    • 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
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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
    • C12N2750/14151—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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  • Bioinformatics & Cheminformatics (AREA)
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  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
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  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Mycology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
BR112020023292-0A 2018-05-14 2019-05-14 vírus adeno-associado (aav), composição, método para a entrega de um transgene a uma célula, uso do aav, método para o tratamento de um indivíduo, vetor, célula, método para a produção de um vírus adeno-associado (aav), vírus adeno-associado (aav) BR112020023292A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862671265P 2018-05-14 2018-05-14
US62/671,265 2018-05-14
PCT/US2019/032097 WO2019222136A2 (en) 2018-05-14 2019-05-14 Novel liver targeting adeno-associated viral vectors

Publications (1)

Publication Number Publication Date
BR112020023292A2 true BR112020023292A2 (pt) 2021-02-23

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BR112020023292-0A BR112020023292A2 (pt) 2018-05-14 2019-05-14 vírus adeno-associado (aav), composição, método para a entrega de um transgene a uma célula, uso do aav, método para o tratamento de um indivíduo, vetor, célula, método para a produção de um vírus adeno-associado (aav), vírus adeno-associado (aav)

Country Status (16)

Country Link
US (1) US11821008B2 (https=)
EP (1) EP3794016A2 (https=)
JP (1) JP2021522842A (https=)
KR (1) KR20210010496A (https=)
CN (1) CN112424217A (https=)
AR (1) AR115097A1 (https=)
AU (1) AU2019268302A1 (https=)
BR (1) BR112020023292A2 (https=)
CA (1) CA3100001A1 (https=)
CL (2) CL2020002960A1 (https=)
MX (1) MX2020012170A (https=)
PE (1) PE20210113A1 (https=)
PH (1) PH12020551938A1 (https=)
SG (1) SG11202011145UA (https=)
TW (1) TW202005978A (https=)
WO (1) WO2019222136A2 (https=)

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TW202104592A (zh) 2019-05-14 2021-02-01 美商拜奧馬林製藥公司 重複投予基因療法載體之方法
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EP4396203A1 (en) 2021-09-03 2024-07-10 BioMarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
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CN120380156A (zh) 2022-09-22 2025-07-25 迪纳柯公司 用aav基因治疗载体治疗心肌病
TW202525313A (zh) 2023-09-06 2025-07-01 瑞士商迪納柯公司 用ａａｖ基因治療載體及治療調配物治療肥厚性心肌病之方法
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