BR112023001611A2 - Proteína do capsídeo de vírus adenoassociado (aav), ácido nucleico, dna recombinante, célula hospedeira, partícula viral, animal transgênico, composição farmacêutica, proteína do capsídeo e método de direcionamento para a microglia ou macrófagos cerebrais - Google Patents

Proteína do capsídeo de vírus adenoassociado (aav), ácido nucleico, dna recombinante, célula hospedeira, partícula viral, animal transgênico, composição farmacêutica, proteína do capsídeo e método de direcionamento para a microglia ou macrófagos cerebrais

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Publication number
BR112023001611A2
BR112023001611A2 BR112023001611A BR112023001611A BR112023001611A2 BR 112023001611 A2 BR112023001611 A2 BR 112023001611A2 BR 112023001611 A BR112023001611 A BR 112023001611A BR 112023001611 A BR112023001611 A BR 112023001611A BR 112023001611 A2 BR112023001611 A2 BR 112023001611A2
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BR
Brazil
Prior art keywords
capsid protein
aav
adeno
macrophages
associated virus
Prior art date
Application number
BR112023001611A
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English (en)
Inventor
J M Franklin Robin
Neumann Björn
Segel Michael
Young Adam
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Cambridge Entpr Ltd
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Publication date
Application filed by Cambridge Entpr Ltd filed Critical Cambridge Entpr Ltd
Publication of BR112023001611A2 publication Critical patent/BR112023001611A2/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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
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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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

Abstract

PROTEÍNA DO CAPSÍDEO DE VÍRUS ADENOASSOCIADO (AAV), ÁCIDO NUCLEICO, DNA RECOMBINANTE, CÉLULA HOSPEDEIRA, PARTÍCULA VIRAL, ANIMAL TRANSGÊNICO, COMPOSIÇÃO FARMACÊUTICA, PROTEÍNA DO CAPSÍDEO E MÉTODO DE DIRECIONAMENTO PARA A MICROGLIA OU MACRÓFAGOS CEREBRAIS. A presente invenção refere-se a proteínas do capsídeo de vírus adenoassociado (AAV) que foram modificadas para inserir uma sequência de aminoácidos e/ou métodos de direcionamento para a microglia ou macrófagos cerebrais usando as proteínas do capsídeo de AAV da invenção.
BR112023001611A 2020-07-30 2021-07-30 Proteína do capsídeo de vírus adenoassociado (aav), ácido nucleico, dna recombinante, célula hospedeira, partícula viral, animal transgênico, composição farmacêutica, proteína do capsídeo e método de direcionamento para a microglia ou macrófagos cerebrais BR112023001611A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB2011871.7A GB202011871D0 (en) 2020-07-30 2020-07-30 Composition and method
PCT/GB2021/051985 WO2022023773A1 (en) 2020-07-30 2021-07-30 Composition and method

Publications (1)

Publication Number Publication Date
BR112023001611A2 true BR112023001611A2 (pt) 2023-02-14

Family

ID=72425275

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112023001611A BR112023001611A2 (pt) 2020-07-30 2021-07-30 Proteína do capsídeo de vírus adenoassociado (aav), ácido nucleico, dna recombinante, célula hospedeira, partícula viral, animal transgênico, composição farmacêutica, proteína do capsídeo e método de direcionamento para a microglia ou macrófagos cerebrais

Country Status (10)

Country Link
US (1) US20230295237A1 (pt)
EP (1) EP4188940A1 (pt)
JP (1) JP2023535620A (pt)
KR (1) KR20230041820A (pt)
CN (1) CN116406427A (pt)
AU (1) AU2021319154A1 (pt)
BR (1) BR112023001611A2 (pt)
CA (1) CA3190289A1 (pt)
GB (1) GB202011871D0 (pt)
WO (1) WO2022023773A1 (pt)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023183583A2 (en) * 2022-03-24 2023-09-28 Capsida, Inc. Adeno-associated virus compositions having increased heart enrichment
WO2023215947A1 (en) * 2022-05-13 2023-11-16 Children's Medical Research Institute Adeno-associated virus capsids
WO2024015933A2 (en) * 2022-07-13 2024-01-18 The Regents Of The University Of California Transplanting stem cell-derived microglia to treat leukodystrophies

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3662060A2 (en) * 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3830107A2 (en) * 2018-08-03 2021-06-09 Voyager Therapeutics, Inc. Aav variants with enhanced tropism

Also Published As

Publication number Publication date
US20230295237A1 (en) 2023-09-21
EP4188940A1 (en) 2023-06-07
AU2021319154A1 (en) 2023-03-23
GB202011871D0 (en) 2020-09-16
CA3190289A1 (en) 2022-02-03
WO2022023773A1 (en) 2022-02-03
KR20230041820A (ko) 2023-03-24
CN116406427A (zh) 2023-07-07
JP2023535620A (ja) 2023-08-18

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