BR112018070250A2 - linhagem celular para proteína recombinante e/ou produção de vetores virais - Google Patents

linhagem celular para proteína recombinante e/ou produção de vetores virais

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Publication number
BR112018070250A2
BR112018070250A2 BR112018070250A BR112018070250A BR112018070250A2 BR 112018070250 A2 BR112018070250 A2 BR 112018070250A2 BR 112018070250 A BR112018070250 A BR 112018070250A BR 112018070250 A BR112018070250 A BR 112018070250A BR 112018070250 A2 BR112018070250 A2 BR 112018070250A2
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BR
Brazil
Prior art keywords
cell line
recombinant protein
viral vector
vector production
vectors
Prior art date
Application number
BR112018070250A
Other languages
English (en)
Inventor
Qu Guang
Zhou Jingmin
Fraser Wright John
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 BR112018070250A2 publication Critical patent/BR112018070250A2/pt

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    • 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
    • C12N5/0684—Cells of the urinary tract or kidneys
    • C12N5/0686—Kidney cells
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • A—HUMAN NECESSITIES
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
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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
    • C12N15/63—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
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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
    • C12N15/63—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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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0012—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7)
    • C12N9/0026—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7) acting on CH-NH groups of donors (1.5)
    • C12N9/0028—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7) acting on CH-NH groups of donors (1.5) with NAD or NADP as acceptor (1.5.1)
    • C12N9/003—Dihydrofolate reductase [DHFR] (1.5.1.3)
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/93—Ligases (6)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y105/00—Oxidoreductases acting on the CH-NH group of donors (1.5)
    • C12Y105/01—Oxidoreductases acting on the CH-NH group of donors (1.5) with NAD+ or NADP+ as acceptor (1.5.1)
    • C12Y105/01003—Dihydrofolate reductase (1.5.1.3)
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    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
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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
    • C12N15/63—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
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • C12N2015/8518—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic expressing industrially exogenous proteins, e.g. for pharmaceutical use, human insulin, blood factors, immunoglobulins, pseudoparticles
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
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    • 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
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    • C12N2750/00011—Details
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12Y—ENZYMES
    • C12Y603/00—Ligases forming carbon-nitrogen bonds (6.3)
    • C12Y603/01—Acid-ammonia (or amine)ligases (amide synthases)(6.3.1)
    • C12Y603/01002—Glutamate-ammonia ligase (6.3.1.2)

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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Urology & Nephrology (AREA)
  • Cell Biology (AREA)
  • Virology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Diabetes (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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  • Peptides Or Proteins (AREA)

Abstract

células e linhas celulares que são divulgadas são capazes de produzir proteínas terapêuticas, anticorpos, vetores e vetores virais tais como vetores lentivirais e vetores virais adeno-associados (aav). as células e/ou linhas celulares podem ter mutações, ou deleções, em um, ou em ambos, os genes endógenos de di-hidrofolato redutase (dhfr -/-) ou de glutamina sintase (gs -/-), de tal modo que a expressão, ou a função, de dhfr e/ou de gs seja substancialmente reduzida ou eliminada.
BR112018070250A 2016-03-30 2017-03-30 linhagem celular para proteína recombinante e/ou produção de vetores virais BR112018070250A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662315480P 2016-03-30 2016-03-30
PCT/US2017/024951 WO2017173043A1 (en) 2016-03-30 2017-03-30 Cell line for recombinant protein and/or viral vector production

Publications (1)

Publication Number Publication Date
BR112018070250A2 true BR112018070250A2 (pt) 2019-01-29

Family

ID=59966430

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112018070250A BR112018070250A2 (pt) 2016-03-30 2017-03-30 linhagem celular para proteína recombinante e/ou produção de vetores virais

Country Status (11)

Country Link
US (1) US20190078099A1 (pt)
EP (1) EP3436576A4 (pt)
JP (2) JP2019509748A (pt)
KR (1) KR102479894B1 (pt)
CN (1) CN109563496B (pt)
AU (2) AU2017244133A1 (pt)
BR (1) BR112018070250A2 (pt)
CA (1) CA3019126A1 (pt)
MX (1) MX2018011928A (pt)
SG (1) SG11201808398PA (pt)
WO (1) WO2017173043A1 (pt)

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SG11201703419UA (en) 2014-11-14 2017-05-30 Voyager Therapeutics Inc Modulatory polynucleotides
CN107109407A (zh) 2014-11-14 2017-08-29 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
CN110214187B (zh) 2016-05-18 2024-01-30 沃雅戈治疗公司 调节性多核苷酸
EP4119133A1 (en) 2017-05-24 2023-01-18 Thoeris GmbH Glutamine synthetase protein conjugate
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
MX2019014578A (es) * 2017-06-21 2020-12-01 Timothy A Bertram Celulas renales bioactivas inmunoprivilegiadas para el tratamiento de enfermedad renal.
CA3079416A1 (en) 2017-10-20 2019-04-25 Research Institute At Nationwide Children's Hospital Methods and materials for nt-3 gene therapy
EP3821027A1 (en) * 2018-07-13 2021-05-19 Enzene Biosciences Ltd. Double knock-out cho cell line method of its generation and producing therapeutic proteins therefrom
TW202028472A (zh) * 2018-10-01 2020-08-01 美商奧崔基尼克斯製藥公司 用於治療丙酸血症之基因療法
CN117425731A (zh) * 2020-12-25 2024-01-19 Ａｇｃ株式会社 提高了载体产生能力的病毒载体产生细胞及其制造方法及其选择方法
EP4301861A1 (en) * 2021-03-03 2024-01-10 Shape Therapeutics Inc. Auxotrophic cells for virus production and compositions and methods of making
WO2023212298A1 (en) 2022-04-29 2023-11-02 Broadwing Bio Llc Bispecific antibodies and methods of treating ocular disease
WO2023212294A1 (en) 2022-04-29 2023-11-02 Broadwing Bio Llc Angiopoietin-related protein 7-specific antibodies and uses thereof
WO2023212293A1 (en) 2022-04-29 2023-11-02 Broadwing Bio Llc Complement factor h related 4-specific antibodies and uses thereof

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Publication number Priority date Publication date Assignee Title
US20080001001A1 (en) * 2006-04-17 2008-01-03 Pevnick Stephen H Pneumatic Activated Fountain
CA2650414A1 (en) * 2006-05-19 2007-11-29 Sangamo Biosciences, Inc. Methods and compositions for inactivation of dihydrofolate reductase
AU2009311697B2 (en) * 2008-10-29 2014-12-18 Sangamo Therapeutics, Inc. Methods and compositions for inactivating glutamine synthetase gene expression
AU2015201300A1 (en) * 2008-10-29 2015-04-02 Sangamo Therapeutics, Inc. Methods and Compositions For Inactivating Glutamine Synthetase Gene Expression
DK3489366T3 (da) * 2011-06-01 2020-02-24 Prec Biosciences Inc Fremgangsmåder og produkter til produktion af manipulerede mammale cellelinier med forstærkede transgener
CN104520428B (zh) * 2012-02-17 2018-09-21 费城儿童医院 将基因转移到细胞、器官和组织的aav载体组合物和方法
PT2970920T (pt) * 2013-03-15 2018-07-30 Childrens Hospital Philadelphia Processo de fabrico escalável para produzir vetores lentivirais recombinantes em sistema de cultura de células de suspensão livres de soro
PE20160188A1 (es) * 2013-07-22 2016-04-27 Philadelphia Children Hospital Variantes aav y composiciones, metodos y usos para transferencia de gen a celulas, organos y tejidos
KR20170081784A (ko) * 2016-01-04 2017-07-13 한국과학기술원 Gs 유전자가 결핍된 신규한 hek293 세포주 및 상기 형질전환된 hek293 숙주세포를 이용한 목적 단백질의 생산 방법

Also Published As

Publication number Publication date
US20190078099A1 (en) 2019-03-14
KR102479894B1 (ko) 2022-12-21
SG11201808398PA (en) 2018-10-30
KR20190008197A (ko) 2019-01-23
JP2022101642A (ja) 2022-07-06
MX2018011928A (es) 2019-03-28
JP2019509748A (ja) 2019-04-11
CN109563496A (zh) 2019-04-02
CA3019126A1 (en) 2017-10-05
CN109563496B (zh) 2023-08-29
WO2017173043A1 (en) 2017-10-05
AU2017244133A1 (en) 2018-10-18
EP3436576A1 (en) 2019-02-06
EP3436576A4 (en) 2019-10-30
AU2023204029A1 (en) 2023-07-13

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