BR112021020391A2 - Linhagens de células produtoras projetadas e métodos para preparar e usar as mesmas - Google Patents

Linhagens de células produtoras projetadas e métodos para preparar e usar as mesmas

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Publication number
BR112021020391A2
BR112021020391A2 BR112021020391A BR112021020391A BR112021020391A2 BR 112021020391 A2 BR112021020391 A2 BR 112021020391A2 BR 112021020391 A BR112021020391 A BR 112021020391A BR 112021020391 A BR112021020391 A BR 112021020391A BR 112021020391 A2 BR112021020391 A2 BR 112021020391A2
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BR
Brazil
Prior art keywords
cell lines
methods
preparing
producer cell
engineered
Prior art date
Application number
BR112021020391A
Other languages
English (en)
Inventor
R Tiernan Aubrey
Nicholas Richards
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Ultragenyx Pharmaceutical 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 Ultragenyx Pharmaceutical Inc filed Critical Ultragenyx Pharmaceutical Inc
Publication of BR112021020391A2 publication Critical patent/BR112021020391A2/pt

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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
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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
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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
    • C12N15/113—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
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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
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
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    • C12N2310/00—Structure or type of the nucleic acid
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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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • 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
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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  • Life Sciences & Earth Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Plant Pathology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Medicinal Chemistry (AREA)
  • Analytical Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

linhagens de células produtoras projetadas e métodos para preparar e usar as mesmas. este pedido se refere ao empacotamento de vírus adeno-associado recombinante (raav) e/ou linhagens de células produtoras que foram projetadas para reduzir a expressão e/ou atividade de um ou mais genes e/ou proteínas para aumentar os títulos de raav. os métodos de geração de linhagens de células projetadas também foram descritos neste documento.
BR112021020391A 2019-04-12 2020-04-09 Linhagens de células produtoras projetadas e métodos para preparar e usar as mesmas BR112021020391A2 (pt)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962833548P 2019-04-12 2019-04-12
US201962839207P 2019-04-26 2019-04-26
US202062979483P 2020-02-21 2020-02-21
PCT/US2020/027489 WO2020210507A1 (en) 2019-04-12 2020-04-09 Engineered producer cell lines and methods of making and using the same

Publications (1)

Publication Number Publication Date
BR112021020391A2 true BR112021020391A2 (pt) 2022-02-08

Family

ID=70465550

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021020391A BR112021020391A2 (pt) 2019-04-12 2020-04-09 Linhagens de células produtoras projetadas e métodos para preparar e usar as mesmas

Country Status (13)

Country Link
US (1) US12018288B2 (pt)
EP (1) EP3953458A1 (pt)
JP (2) JP7560475B2 (pt)
KR (1) KR20210152514A (pt)
CN (1) CN113966395A (pt)
AU (1) AU2020270960A1 (pt)
BR (1) BR112021020391A2 (pt)
CA (1) CA3136246A1 (pt)
CO (1) CO2021013699A2 (pt)
IL (1) IL287106A (pt)
MX (1) MX2021012489A (pt)
SG (1) SG11202110904PA (pt)
WO (1) WO2020210507A1 (pt)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023172491A1 (en) 2022-03-07 2023-09-14 Ultragenyx Pharmaceutical Inc. Modified batch aav production systems and methods
CA3173178A1 (en) 2020-07-30 2022-02-03 Shape Therapeutics Inc. Stable cell lines for inducible production of raav virions
JP2024531138A (ja) 2021-08-11 2024-08-29 ウルトラジェニックス ファーマシューティカル インコーポレイテッド 筋ジストロフィーを処置するための組成物および方法
WO2023077078A1 (en) 2021-10-29 2023-05-04 Ultragenyx Pharmaceutical Inc. Engineered cell lines for increased production of recombinant adeno-associated virus (raav)

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WO1996017947A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
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Also Published As

Publication number Publication date
CO2021013699A2 (es) 2021-10-29
EP3953458A1 (en) 2022-02-16
US20200325455A1 (en) 2020-10-15
JP2022526646A (ja) 2022-05-25
JP7560475B2 (ja) 2024-10-02
WO2020210507A1 (en) 2020-10-15
CN113966395A (zh) 2022-01-21
SG11202110904PA (en) 2021-10-28
JP2024107295A (ja) 2024-08-08
CA3136246A1 (en) 2020-10-15
MX2021012489A (es) 2021-11-12
TW202104593A (zh) 2021-02-01
AU2020270960A1 (en) 2021-11-04
IL287106A (en) 2021-12-01
US12018288B2 (en) 2024-06-25
KR20210152514A (ko) 2021-12-15

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