JP7598920B2 - 生物製造のための合成遺伝子要素 - Google Patents

生物製造のための合成遺伝子要素 Download PDF

Info

Publication number
JP7598920B2
JP7598920B2 JP2022503956A JP2022503956A JP7598920B2 JP 7598920 B2 JP7598920 B2 JP 7598920B2 JP 2022503956 A JP2022503956 A JP 2022503956A JP 2022503956 A JP2022503956 A JP 2022503956A JP 7598920 B2 JP7598920 B2 JP 7598920B2
Authority
JP
Japan
Prior art keywords
seq
nucleotide sequence
cell
aav
gene
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
JP2022503956A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2021016227A5 (enExample
JP2022541915A (ja
Inventor
ロイド ザ サード ペリー,ウィリアム
トムコウィッツ,ブライアン
Original Assignee
ヤンセン バイオテツク,インコーポレーテツド
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 ヤンセン バイオテツク,インコーポレーテツド filed Critical ヤンセン バイオテツク,インコーポレーテツド
Publication of JP2022541915A publication Critical patent/JP2022541915A/ja
Publication of JPWO2021016227A5 publication Critical patent/JPWO2021016227A5/ja
Priority to JP2024209192A priority Critical patent/JP2025029049A/ja
Application granted granted Critical
Publication of JP7598920B2 publication Critical patent/JP7598920B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10311Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151Methods of production or purification of viral material
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151Methods of production or purification of viral material
    • C12N2750/14152Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/30Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/50Vectors for producing vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/36Vector systems having a special element relevant for transcription being a transcription termination element
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/40Vector systems having a special element relevant for transcription being an insulator
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/42Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2022503956A 2019-07-23 2020-07-21 生物製造のための合成遺伝子要素 Active JP7598920B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024209192A JP2025029049A (ja) 2019-07-23 2024-12-02 生物製造のための合成遺伝子要素

Applications Claiming Priority (17)

Application Number Priority Date Filing Date Title
US201962877524P 2019-07-23 2019-07-23
US201962877516P 2019-07-23 2019-07-23
US201962877508P 2019-07-23 2019-07-23
US201962877532P 2019-07-23 2019-07-23
US201962877540P 2019-07-23 2019-07-23
US201962877551P 2019-07-23 2019-07-23
US201962877577P 2019-07-23 2019-07-23
US201962877561P 2019-07-23 2019-07-23
US62/877,508 2019-07-23
US62/877,551 2019-07-23
US62/877,577 2019-07-23
US62/877,524 2019-07-23
US62/877,516 2019-07-23
US62/877,540 2019-07-23
US62/877,561 2019-07-23
US62/877,532 2019-07-23
PCT/US2020/042854 WO2021016227A1 (en) 2019-07-23 2020-07-21 Synthetic genetic elements for biomanufacture

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024209192A Division JP2025029049A (ja) 2019-07-23 2024-12-02 生物製造のための合成遺伝子要素

Publications (3)

Publication Number Publication Date
JP2022541915A JP2022541915A (ja) 2022-09-28
JPWO2021016227A5 JPWO2021016227A5 (enExample) 2023-07-24
JP7598920B2 true JP7598920B2 (ja) 2024-12-12

Family

ID=72039670

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022503956A Active JP7598920B2 (ja) 2019-07-23 2020-07-21 生物製造のための合成遺伝子要素
JP2024209192A Pending JP2025029049A (ja) 2019-07-23 2024-12-02 生物製造のための合成遺伝子要素

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024209192A Pending JP2025029049A (ja) 2019-07-23 2024-12-02 生物製造のための合成遺伝子要素

Country Status (9)

Country Link
US (2) US12049638B2 (enExample)
EP (1) EP4003430A1 (enExample)
JP (2) JP7598920B2 (enExample)
CN (2) CN114514319B (enExample)
AU (1) AU2020316034A1 (enExample)
CA (1) CA3148079A1 (enExample)
IL (1) IL289968A (enExample)
TW (1) TW202116794A (enExample)
WO (1) WO2021016227A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4189077A1 (en) 2020-07-30 2023-06-07 Shape Therapeutics Inc. Stable cell lines for inducible production of raav virions
US20250129127A1 (en) * 2022-01-28 2025-04-24 Shape Therapeutics Inc. Systems for Amplification of AAV Rep and Cap Proteins
JP2025512585A (ja) * 2022-04-25 2025-04-17 ヤンセン・リサーチ・アンド・ディベロップメント・エルエルシー アデノ随伴ウイルスパッケージングベクター及びそれを使用する方法
WO2025054007A1 (en) * 2023-09-06 2025-03-13 Shape Therapeutics Inc. Genetic elements to increase recombinant aav production

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008528056A (ja) 2005-02-02 2008-07-31 イントレクソン コーポレイション 部位特異的セリンリコンビナーゼ及びそれらの使用方法
WO2018158141A1 (en) 2017-03-03 2018-09-07 Ge Healthcare Bio-Sciences Ab Method for protein expression

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7348178B1 (en) * 1994-03-14 2008-03-25 New York University Medical Center Recombinant adenoviral vector system
US6413776B1 (en) 1998-06-12 2002-07-02 Galapagos Geonomics N.V. High throughput screening of gene function using adenoviral libraries for functional genomics applications
WO2003084977A1 (en) * 2002-04-04 2003-10-16 Xiao Xiao Gene expression control system and its use in recombinant virus packaging cell lines
ES2385679T3 (es) 2006-08-24 2012-07-30 Virovek, Inc. Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos
GB201609597D0 (en) * 2016-06-01 2016-07-13 Univ Sheffield Therapy
GB201706090D0 (en) * 2017-04-18 2017-05-31 Glaxosmithkline Ip Dev Ltd Methods for adeno-associated viral vector production

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008528056A (ja) 2005-02-02 2008-07-31 イントレクソン コーポレイション 部位特異的セリンリコンビナーゼ及びそれらの使用方法
WO2018158141A1 (en) 2017-03-03 2018-09-07 Ge Healthcare Bio-Sciences Ab Method for protein expression

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
J Virol,2002年,vol. 76,pp. 13015-13027
Methods Mol Biol,2015年,vol. 1239,pp. 29-38

Also Published As

Publication number Publication date
AU2020316034A1 (en) 2022-02-10
WO2021016227A8 (en) 2021-09-02
CA3148079A1 (en) 2021-01-28
US12049638B2 (en) 2024-07-30
CN120700001A (zh) 2025-09-26
WO2021016227A1 (en) 2021-01-28
US20210032657A1 (en) 2021-02-04
CN114514319B (zh) 2025-06-24
US20240327869A1 (en) 2024-10-03
TW202116794A (zh) 2021-05-01
IL289968A (en) 2022-03-01
CN114514319A (zh) 2022-05-17
EP4003430A1 (en) 2022-06-01
JP2022541915A (ja) 2022-09-28
JP2025029049A (ja) 2025-03-05

Similar Documents

Publication Publication Date Title
JP7463358B2 (ja) アデノ随伴ウイルスベクタープロデューサー細胞株
JP2023081975A (ja) アデノ随伴ウイルスベクターの産生方法
US20240327869A1 (en) Synthetic genetic elements for biomanufacture
JP7384457B2 (ja) ウイルスベクターを調製するための組成物および方法
JP2024032913A (ja) ウイルスベクター産生系
JP2023546113A (ja) 同時遺伝子活性化のための核酸構築物
GB2566572A (en) Methods for adeno-associated viral vector production
JP2025523458A (ja) アデノウイルスに基づく核酸およびその方法
US6586208B2 (en) Use of suppressor tRNA's to regulate cytotoxicity during the production of recombinant gene products
JP2023546116A (ja) Va rna転写のための核酸構築物
Krüger‐Haag et al. Evaluation of life cycle defective adenovirus mutants for production of adeno‐associated virus vectors
HK40073527A (en) Synthetic genetic elements for biomanufacture

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230713

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230713

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20240717

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240723

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241018

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20241105

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20241202

R150 Certificate of patent or registration of utility model

Ref document number: 7598920

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150