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
Authority
BR
Brazil
Prior art keywords
capsid protein
aav
adeno
macrophages
associated virus
Prior art date
Application number
BR112023001611A
Other languages
English (en)
Portuguese (pt)
Inventor
J M Franklin Robin
Neumann Björn
Segel Michael
Young Adam
Original Assignee
Cambridge Entpr Ltd
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 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
    • 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
    • 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

Landscapes

  • 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)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (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)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Epidemiology (AREA)
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 (https=)
EP (1) EP4188940A1 (https=)
JP (1) JP7841821B2 (https=)
KR (1) KR20230041820A (https=)
CN (1) CN116406427A (https=)
AU (1) AU2021319154A1 (https=)
BR (1) BR112023001611A2 (https=)
CA (1) CA3190289A1 (https=)
GB (1) GB202011871D0 (https=)
WO (1) WO2022023773A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE202022002866U1 (de) 2021-04-01 2024-03-20 The Regents Of The University Of California Genetische Modifikation von Säugetierzellen zur Verleihung von Resistenz gegen CSF1R-Antagonisten
CA3254570A1 (en) * 2022-03-24 2023-09-28 Capsida, Inc. ADENE-ASSOCIATED VIRUS COMPOSITIONS WITH INCREASED CARDIAC ENRICHEMENT
AU2023267474A1 (en) * 2022-05-13 2024-12-05 Children's Medical Research Institute Adeno-associated virus capsids
US20260108560A1 (en) * 2022-07-13 2026-04-23 The Regents Of The University Of California Transplanting stem cell-derived microglia to treat leukodystrophies
CN121311596A (zh) * 2023-04-07 2026-01-09 4D分子治疗有限公司 用于治疗补体病症的aav变体
CN118480097B (zh) * 2023-09-20 2024-11-05 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN117660370B (zh) * 2023-11-08 2025-03-21 中国人民解放军空军军医大学 一种重组腺相关病毒、抗炎药物及其在脓毒性脑病治疗中的应用

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9585971B2 (en) * 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
KR102423442B1 (ko) * 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
JP7221275B2 (ja) * 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Ａａｖを送達するための組成物および方法
US20210277418A1 (en) * 2018-08-03 2021-09-09 Voyager Therapeutics, Inc. Aav variants with enhanced tropism

Also Published As

Publication number Publication date
KR20230041820A (ko) 2023-03-24
WO2022023773A1 (en) 2022-02-03
CA3190289A1 (en) 2022-02-03
GB202011871D0 (en) 2020-09-16
EP4188940A1 (en) 2023-06-07
AU2021319154A1 (en) 2023-03-23
JP7841821B2 (ja) 2026-04-07
JP2023535620A (ja) 2023-08-18
US20230295237A1 (en) 2023-09-21
CN116406427A (zh) 2023-07-07

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