BR112021026832A2 - Vetores ad35 recombinantes e aprimoramentos da terapia gênica relacionada - Google Patents

Vetores ad35 recombinantes e aprimoramentos da terapia gênica relacionada

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Publication number
BR112021026832A2
BR112021026832A2 BR112021026832A BR112021026832A BR112021026832A2 BR 112021026832 A2 BR112021026832 A2 BR 112021026832A2 BR 112021026832 A BR112021026832 A BR 112021026832A BR 112021026832 A BR112021026832 A BR 112021026832A BR 112021026832 A2 BR112021026832 A2 BR 112021026832A2
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Brazil
Prior art keywords
vectors
helper
dependent
gene therapy
recombinant
Prior art date
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BR112021026832A
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English (en)
Inventor
Andre Lieber
Chang Li
Hans-Peter Kiem
Hongjie Wang
Original Assignee
Hutchinson Fred Cancer Res
Univ Washington
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Application filed by Hutchinson Fred Cancer Res, Univ Washington filed Critical Hutchinson Fred Cancer Res
Publication of BR112021026832A2 publication Critical patent/BR112021026832A2/pt

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    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
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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
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • 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
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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
    • C12N15/87—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
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    • C12N9/14—Hydrolases (3)
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    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10351—Methods of production or purification of viral material
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/50—Vectors for producing vectors
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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    • C12N2800/00—Nucleic acids vectors
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6009—Vectors comprising as targeting moiety peptide derived from defined protein from viruses dsDNA viruses
    • C12N2810/6018—Adenoviridae
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    • C12N2999/00—Further aspects of viruses or vectors not covered by groups C12N2710/00 - C12N2796/00 or C12N2800/00
    • C12N2999/007—Technological advancements, e.g. new system for producing known virus, cre-lox system for production of transgenic animals

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
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  • Public Health (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Veterinary Medicine (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Abstract

vetores ad35 recombinantes e aprimoramentos da terapia gênica relacionada. a presente divulgação fornece, entre outros, vetores adenovirais dependentes de auxiliar sorotipo 35 (ad35). em várias modalidades, vetores ad35 dependentes de auxiliar podem ser usados para distribuir uma carga terapêutica a um sujeito em necessidade. cargas exemplificativas podem codificar proteínas de substituição, anticorpos, cars, tcrs, pequenos rnas e sistemas de edição de genoma. em certas modalidades, um vetor ad35 dependente de auxiliar é manipulado para integração de uma carga em um genoma de célula hospedeira. a presente divulgação inclui ainda métodos de terapia gênica que incluem administrar um vetor ad35 dependente de auxiliar a um sujeito em necessidade.
BR112021026832A 2019-07-02 2020-07-02 Vetores ad35 recombinantes e aprimoramentos da terapia gênica relacionada BR112021026832A2 (pt)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962869907P 2019-07-02 2019-07-02
US201962935507P 2019-11-14 2019-11-14
US202063009385P 2020-04-13 2020-04-13
PCT/US2020/040756 WO2021003432A1 (en) 2019-07-02 2020-07-02 Recombinant ad35 vectors and related gene therapy improvements

Publications (1)

Publication Number Publication Date
BR112021026832A2 true BR112021026832A2 (pt) 2022-05-10

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BR112021026832A BR112021026832A2 (pt) 2019-07-02 2020-07-02 Vetores ad35 recombinantes e aprimoramentos da terapia gênica relacionada

Country Status (12)

Country Link
US (1) US20220257796A1 (pt)
EP (1) EP3994270A1 (pt)
JP (1) JP2022539248A (pt)
KR (1) KR20220038362A (pt)
CN (1) CN114729383A (pt)
AU (1) AU2020298572A1 (pt)
BR (1) BR112021026832A2 (pt)
CA (1) CA3138188A1 (pt)
IL (1) IL289518A (pt)
MX (1) MX2021015433A (pt)
SG (1) SG11202111943UA (pt)
WO (1) WO2021003432A1 (pt)

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CN113755447B (zh) * 2021-09-23 2022-04-29 云舟生物科技(广州)股份有限公司 一种用于生产腺病毒的293a细胞株及其制备与应用
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