BR112021020401A2 - Sistema de dois plasmídeos, plasmídeo auxiliar, plasmídeo vetor, usos do sistema de dois plasmídeos, métodos para controlar ou maximizar a razão de partículas completas para totais produzidas durante a produção de aav recombinante e para aumentar, otimizar ou maximizar o rendimento de aav recombinante - Google Patents

Sistema de dois plasmídeos, plasmídeo auxiliar, plasmídeo vetor, usos do sistema de dois plasmídeos, métodos para controlar ou maximizar a razão de partículas completas para totais produzidas durante a produção de aav recombinante e para aumentar, otimizar ou maximizar o rendimento de aav recombinante

Info

Publication number
BR112021020401A2
BR112021020401A2 BR112021020401A BR112021020401A BR112021020401A2 BR 112021020401 A2 BR112021020401 A2 BR 112021020401A2 BR 112021020401 A BR112021020401 A BR 112021020401A BR 112021020401 A BR112021020401 A BR 112021020401A BR 112021020401 A2 BR112021020401 A2 BR 112021020401A2
Authority
BR
Brazil
Prior art keywords
plasmid
maximizing
recombinant aav
methods
optimizing
Prior art date
Application number
BR112021020401A
Other languages
English (en)
Inventor
Florian Sonntag
Markus Hörer
Renée Kober
Original Assignee
Freeline Therapeutics 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
Priority claimed from GBGB1905263.8A external-priority patent/GB201905263D0/en
Priority claimed from EP19169121.1A external-priority patent/EP3722434B1/en
Application filed by Freeline Therapeutics Ltd filed Critical Freeline Therapeutics Ltd
Publication of BR112021020401A2 publication Critical patent/BR112021020401A2/pt

Links

Classifications

    • 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
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Saccharide Compounds (AREA)

Abstract

sistema de dois plasmídeos, usos do sistema de dois plasmídeos, métodos para controlar ou maximizar a razão de partículas completas para totais produzidas durante a produção de aav recombinante e para aumentar, otimizar ou maximizar o rendimento de aav recombinante. a presente invenção se refere a sistemas de dois plasmídeos, plasmídeos auxiliares e/ou plasmídeos vetor para a produção de vetores de aav recombinantes (raav). a invenção se refere ainda a métodos usando, ou usos dos sistemas de dois plasmídeos, plasmídeos auxiliares e/ou plasmídeos vetor da invenção.
BR112021020401A 2019-04-12 2020-04-14 Sistema de dois plasmídeos, plasmídeo auxiliar, plasmídeo vetor, usos do sistema de dois plasmídeos, métodos para controlar ou maximizar a razão de partículas completas para totais produzidas durante a produção de aav recombinante e para aumentar, otimizar ou maximizar o rendimento de aav recombinante BR112021020401A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1905263.8A GB201905263D0 (en) 2019-04-12 2019-04-12 Plasmid system
EP19169121.1A EP3722434B1 (en) 2019-04-12 2019-04-12 Plasmid system
PCT/GB2020/050947 WO2020208379A1 (en) 2019-04-12 2020-04-14 Plasmid system

Publications (1)

Publication Number Publication Date
BR112021020401A2 true BR112021020401A2 (pt) 2021-12-07

Family

ID=70295566

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021020401A BR112021020401A2 (pt) 2019-04-12 2020-04-14 Sistema de dois plasmídeos, plasmídeo auxiliar, plasmídeo vetor, usos do sistema de dois plasmídeos, métodos para controlar ou maximizar a razão de partículas completas para totais produzidas durante a produção de aav recombinante e para aumentar, otimizar ou maximizar o rendimento de aav recombinante

Country Status (11)

Country Link
US (1) US20220162642A1 (pt)
EP (1) EP3953482A1 (pt)
JP (1) JP2022530192A (pt)
KR (1) KR20220019669A (pt)
CN (1) CN113891942A (pt)
AU (1) AU2020272557A1 (pt)
BR (1) BR112021020401A2 (pt)
CA (1) CA3136545A1 (pt)
IL (2) IL296544A (pt)
SG (1) SG11202111149VA (pt)
WO (1) WO2020208379A1 (pt)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT3722434T (pt) 2019-04-12 2022-10-18 Freeline Therapeutics Ltd Sistema plasmídico
EP4051703A1 (en) 2019-11-01 2022-09-07 Freeline Therapeutics Limited Factor viii polypeptide
BR112023016997A2 (pt) * 2021-02-26 2023-11-07 Logicbio Therapeutics Inc Fabricação e uso de vetores aav recombinantes
WO2023054671A1 (ja) 2021-09-30 2023-04-06 富士フイルム株式会社 アデノ随伴ウイルスの製造方法、細胞および発現ベクター
GB202214328D0 (en) 2022-09-29 2022-11-16 Freeline Therapeutics Ltd Method

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2453405A1 (en) * 2001-07-13 2003-01-23 University Of Iowa Research Foundation Pseudotyped adeno-associated viruses and uses thereof
AU2002352195A1 (en) * 2001-11-30 2003-06-10 Medigene Aktiengesellschaft Optimized production of viral vectors derived from paroviruses in packaging and production cells by hsv infection or treatment with dna methylation inhibitors
ES2330826B1 (es) * 2008-06-04 2010-07-26 Proyecto De Biomedicina Cima, S.L. Sistema para empaquetamiento de adenovirus de alta capacidad.
MX2011009193A (es) * 2009-03-04 2011-12-16 Deutsches Krebsforsch Proteina activadora del ensamblaje (aap) y su uso para la fabricacion de particulas de parvovirus que consisten esencialmente de vp3.
ES2857773T3 (es) 2011-08-24 2021-09-29 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
EP2782596A4 (en) 2011-11-22 2015-07-29 Philadelphia Children Hospital VIRAL VECTORS FOR HIGHLY EFFECTIVE TRANSGEN DELIVERY
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
US10385320B2 (en) 2015-12-02 2019-08-20 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
CN108048483B (zh) * 2018-01-30 2021-02-02 中国疾病预防控制中心病毒病预防控制所 复制型重组腺病毒HAdV-5载体系统及其应用

Also Published As

Publication number Publication date
CN113891942A (zh) 2022-01-04
IL296544A (en) 2022-11-01
EP3953482A1 (en) 2022-02-16
IL287100A (en) 2021-12-01
SG11202111149VA (en) 2021-11-29
CA3136545A1 (en) 2020-10-15
WO2020208379A1 (en) 2020-10-15
AU2020272557A1 (en) 2021-12-09
US20220162642A1 (en) 2022-05-26
JP2022530192A (ja) 2022-06-28
KR20220019669A (ko) 2022-02-17

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Owner name: ASCEND GENE AND CELL THERAPIES LTD (GB)