CL2019003561A1 - Agentes potenciadores para la transfección celular mejorada y/o la producción del vector raav. - Google Patents

Agentes potenciadores para la transfección celular mejorada y/o la producción del vector raav.

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Publication number
CL2019003561A1
CL2019003561A1 CL2019003561A CL2019003561A CL2019003561A1 CL 2019003561 A1 CL2019003561 A1 CL 2019003561A1 CL 2019003561 A CL2019003561 A CL 2019003561A CL 2019003561 A CL2019003561 A CL 2019003561A CL 2019003561 A1 CL2019003561 A1 CL 2019003561A1
Authority
CL
Chile
Prior art keywords
transduced
protein
raav vector
enhancing agents
transcript
Prior art date
Application number
CL2019003561A
Other languages
English (en)
Inventor
Guang Qu
John Fraser Wright
Lin Lu
Jesusa Josue-Almqvist
Original Assignee
Spark Therapeutics Inc
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 Spark Therapeutics Inc filed Critical Spark Therapeutics Inc
Publication of CL2019003561A1 publication Critical patent/CL2019003561A1/es

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    • 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
    • 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
    • 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0603—Embryonic cells ; Embryoid bodies
    • 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

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Molecular Biology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Reproductive Health (AREA)
  • Cell Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

SE PROPORCIONAN COMPOSICIONES Y MÉTODOS PARA TRANSDUCIR / TRANSFECTAR CÉLULAS CON UNA MOLÉCULA, TAL COMO UN ÁCIDO NUCLEICO (POR EJEMPLO, PLÁSMIDO), CON ALTA EFICIENCIA. LAS CÉLULAS TRANSDUCIDAS / TRANSFECTADAS DE ALTA EFICIENCIA PUEDEN, CUANDO SE TRANSDUCEN CON UN ÁCIDO NUCLEICO QUE CODIFICA UNA PROTEÍNA O COMPRENDEN UNA SECUENCIA QUE SE TRANSCRIBE EN UNA TRANSCRIPCIÓN DE INTERÉS, PRODUCIR ALTAS CANTIDADES DE PROTEÍNA Y/O TRANSCRIPCIÓN. LAS CÉLULAS TRANSDUCIDAS / TRANSFECTADAS DE ALTA EFICIENCIA PUEDEN, CUANDO SE TRANSDUCEN CON PLÁSMIDOS QUE COMPRENDEN (I) ÁCIDOS NUCLEICOS QUE CODIFICAN PROTEÍNAS DE EMPAQUETAMIENTO DE AAV Y/O ÁCIDOS NUCLEICOS QUE CODIFICAN PROTEÍNAS AUXILIARES; Y (II) UN TRANSGEN QUE CODIFICA UNA PROTEÍNA O SE TRANSCRIBE EN UNA TRANSCRIPCIÓN DE INTERÉS; PRODUCIR ALTAS CANTIDADES DE VECTOR RECOMBINANTE RAAV.
CL2019003561A 2017-06-07 2019-12-05 Agentes potenciadores para la transfección celular mejorada y/o la producción del vector raav. CL2019003561A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762516432P 2017-06-07 2017-06-07
US201762531626P 2017-07-12 2017-07-12

Publications (1)

Publication Number Publication Date
CL2019003561A1 true CL2019003561A1 (es) 2020-06-12

Family

ID=64566378

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2019003561A CL2019003561A1 (es) 2017-06-07 2019-12-05 Agentes potenciadores para la transfección celular mejorada y/o la producción del vector raav.

Country Status (15)

Country Link
US (1) US20200165632A1 (es)
EP (1) EP3635108A4 (es)
JP (2) JP2020524498A (es)
KR (1) KR20200033840A (es)
CN (1) CN110997912A (es)
AU (1) AU2018281306A1 (es)
BR (1) BR112019025792A2 (es)
CA (1) CA3066358A1 (es)
CL (1) CL2019003561A1 (es)
CO (1) CO2019014704A2 (es)
IL (1) IL271190A (es)
PE (1) PE20200619A1 (es)
PH (1) PH12019502774A1 (es)
SG (1) SG11201911603SA (es)
WO (1) WO2018226887A1 (es)

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GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
MX2018006682A (es) * 2015-12-01 2018-09-26 Spark Therapeutics Inc Metodos escalables para producir vector viral adenoasociado (aav) recombinante en un sistema de cultivo celular en suspension libre de suero adecuado para su uso clinico.
JP2021533757A (ja) * 2018-08-10 2021-12-09 リジェネクスバイオ インコーポレイテッド 組換えａａｖ生成のためのスケーラブルな方法
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
US11608491B2 (en) * 2019-02-22 2023-03-21 Life Technologies Corporation Suspension system for adeno associated virus production
WO2021041986A1 (en) * 2019-08-28 2021-03-04 University Of Florida Research Foundation, Incorporated Improved production of recombinant aav using embryonated avian eggs
CN111363761A (zh) * 2020-03-17 2020-07-03 苏州吉恒基因科技有限公司 利用阳离子多聚体dna复合物促进aav介导的基因表达方法
WO2022043926A1 (en) * 2020-08-31 2022-03-03 Intas Pharmaceuticals Ltd. Process for preparation of recombinant adeno-associated virus particle
CA3197726A1 (en) 2020-10-15 2022-04-21 Simon Auslaender Nucleic acid constructs for simultaneous gene activation
MX2023004052A (es) 2020-10-15 2023-05-03 Hoffmann La Roche Constructos de acido nucleico para transcripcion de arn asociado a un virus (arn va).
WO2022229853A1 (en) * 2021-04-27 2022-11-03 Novartis Ag Viral vector production system
WO2023053899A1 (ja) 2021-10-01 2023-04-06 株式会社カネカ ウイルスベクター産生誘導剤
WO2023198685A1 (en) 2022-04-13 2023-10-19 F. Hoffmann-La Roche Ag Method for determining aav genomes
WO2023227438A1 (en) 2022-05-23 2023-11-30 F. Hoffmann-La Roche Ag Raman-based method for the differentiation of aav particle serotype and aav particle loading status
WO2023232922A1 (en) 2022-06-03 2023-12-07 F. Hoffmann-La Roche Ag Method for producing recombinant aav particles
WO2024013239A1 (en) 2022-07-14 2024-01-18 F. Hoffmann-La Roche Ag Method for producing recombinant aav particles
WO2024056561A1 (en) 2022-09-12 2024-03-21 F. Hoffmann-La Roche Ag Method for separating full and empty aav particles

Family Cites Families (11)

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ES2567298T3 (es) * 2008-03-14 2016-04-21 Transgene Sa Anticuerpo contra el CSF-1R
US20110212526A1 (en) * 2008-03-18 2011-09-01 National Tsing Hua University Method for producing recombinant adeno-associated virus
JP5697123B2 (ja) * 2009-07-27 2015-04-08 国立大学法人 千葉大学 酸性化ポリエチレンイミンを用いる細胞への核酸導入方法
EP2785741A1 (en) * 2011-12-02 2014-10-08 Cancer Research Technology Limited Antibodies against hgf - receptor and uses
EP3404095B1 (en) * 2012-05-02 2022-07-27 Life Technologies Corporation High yield transient expression in mammalian cells using unique pairing of high density growth and transfection medium and expression enhancers
PL2956165T3 (pl) * 2013-02-14 2020-04-30 The Board Of Trustees Of The University Of Arkansas Kompozycje i sposoby wzmacniania odpowiedzi immunologicznych na Eimeria lub ograniczania infekcji Eimeria
EP3708584A1 (en) * 2013-02-26 2020-09-16 Roche Glycart AG Bispecific t cell activating antigen binding molecules
EP3578190A1 (en) * 2013-03-15 2019-12-11 The Board of Trustees of the University of Arkansas Compositions and methods of enhancing immune responses to enteric pathogens
US11078464B2 (en) * 2013-08-30 2021-08-03 Amgen Inc. High titer recombinant AAV vector production in adherent and suspension cells
EP3110434B1 (en) * 2014-02-24 2018-09-19 Takeda GmbH Uti fusion proteins
EP3322813A1 (en) * 2015-07-13 2018-05-23 Life Technologies Corporation System and method for improved transient protein expression in cho cells

Also Published As

Publication number Publication date
JP2023113706A (ja) 2023-08-16
RU2019143489A3 (es) 2022-04-07
WO2018226887A1 (en) 2018-12-13
SG11201911603SA (en) 2020-01-30
CO2019014704A2 (es) 2020-04-24
CA3066358A1 (en) 2018-12-13
IL271190A (en) 2020-01-30
KR20200033840A (ko) 2020-03-30
BR112019025792A2 (pt) 2020-07-07
RU2019143489A (ru) 2021-07-12
PE20200619A1 (es) 2020-03-11
JP2020524498A (ja) 2020-08-20
CN110997912A (zh) 2020-04-10
AU2018281306A1 (en) 2020-01-16
US20200165632A1 (en) 2020-05-28
EP3635108A4 (en) 2021-03-31
EP3635108A1 (en) 2020-04-15
PH12019502774A1 (en) 2020-09-28

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