KR20210127935A - 폐쇄형 DNA(ceDNA) 생산에서의 Rep 단백질 활성의 변형 - Google Patents

폐쇄형 DNA(ceDNA) 생산에서의 Rep 단백질 활성의 변형 Download PDF

Info

Publication number
KR20210127935A
KR20210127935A KR1020217025576A KR20217025576A KR20210127935A KR 20210127935 A KR20210127935 A KR 20210127935A KR 1020217025576 A KR1020217025576 A KR 1020217025576A KR 20217025576 A KR20217025576 A KR 20217025576A KR 20210127935 A KR20210127935 A KR 20210127935A
Authority
KR
South Korea
Prior art keywords
itr
dna
protein
cell
sequence
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.)
Pending
Application number
KR1020217025576A
Other languages
English (en)
Korean (ko)
Inventor
로버트 마이클 코틴
안나 우쳐
아라 칼 말라키안
Original Assignee
제너레이션 바이오 컴퍼니
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 제너레이션 바이오 컴퍼니 filed Critical 제너레이션 바이오 컴퍼니
Publication of KR20210127935A publication Critical patent/KR20210127935A/ko
Pending legal-status Critical Current

Links

Images

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
    • 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
    • C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
    • 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
    • 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
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6009—Vectors comprising as targeting moiety peptide derived from defined protein from viruses dsDNA viruses
    • C12N2810/6018—Adenoviridae
    • 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
    • C12N2820/00—Vectors comprising a special origin of replication system
    • C12N2820/002—Vectors comprising a special origin of replication system inducible or controllable
    • 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
    • C12N2820/00—Vectors comprising a special origin of replication system
    • C12N2820/60—Vectors comprising a special origin of replication system from viruses

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
KR1020217025576A 2019-02-15 2020-02-14 폐쇄형 DNA(ceDNA) 생산에서의 Rep 단백질 활성의 변형 Pending KR20210127935A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962806076P 2019-02-15 2019-02-15
US62/806,076 2019-02-15
PCT/US2020/018332 WO2020168222A1 (en) 2019-02-15 2020-02-14 Modulation of rep protein activity in closed-ended dna (cedna) production

Publications (1)

Publication Number Publication Date
KR20210127935A true KR20210127935A (ko) 2021-10-25

Family

ID=72045641

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217025576A Pending KR20210127935A (ko) 2019-02-15 2020-02-14 폐쇄형 DNA(ceDNA) 생산에서의 Rep 단백질 활성의 변형

Country Status (11)

Country Link
US (1) US20220127625A1 (https=)
EP (1) EP3924491A4 (https=)
JP (2) JP7644014B2 (https=)
KR (1) KR20210127935A (https=)
CN (1) CN113454232B (https=)
AU (1) AU2020221312A1 (https=)
CA (1) CA3129321A1 (https=)
IL (1) IL285415A (https=)
MA (1) MA54958A (https=)
SG (1) SG11202106491VA (https=)
WO (1) WO2020168222A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102737146B1 (ko) * 2023-07-10 2024-12-09 대한민국 신규 발현 벡터 및 이의 용도

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ762100A (en) 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
WO2020251954A1 (en) * 2019-06-10 2020-12-17 Homology Medicines, Inc. Adeno-associated virus compositions for arsa gene transfer and methods of use thereof
JP2023535632A (ja) 2020-07-27 2023-08-18 アンジャリウム バイオサイエンシズ エージー Ｄｎａ分子の組成物、その作製方法、及びその使用方法
EP4214309A4 (en) * 2020-09-16 2025-07-16 Generation Bio Co CLOSED-END DNA VECTORS AND RELATED USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH)
WO2022136616A1 (en) * 2020-12-23 2022-06-30 Vivet Therapeutics Minimal bile acid inducible promoters for gene therapy
EP4308705A4 (en) * 2021-03-16 2025-06-04 Wisconsin Alumni Research Foundation INSULIN GENE THERAPY TO TREAT DIABETES
MX2024002333A (es) * 2021-08-23 2024-03-07 Bioverativ Therapeutics Inc Sistema de expresion de baculovirus.
CN114703203B (zh) * 2022-02-11 2024-08-06 上海渤因生物科技有限公司 杆状病毒载体及其用途
WO2025114932A1 (en) * 2023-11-30 2025-06-05 Sanofi Purification of closed-ended dna molecules
US12509711B1 (en) * 2024-08-09 2025-12-30 Martin Williams System and process for in vivo manufacturing nanostructure-ended double-stranded covalently-closed linear DNA, the resulting molecules and their uses

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2596303C (en) * 1997-03-21 2013-09-24 Enzo Therapeutics, Inc. Vectors and viral vectors and packaging cell lines for propagating same
WO2001032899A1 (en) 1999-10-29 2001-05-10 Takara Shuzo Co., Ltd. Gene transfer method
CN103849629B (zh) * 2006-06-21 2017-06-09 尤尼克尔Ip股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体
EP2545165B1 (en) * 2010-03-11 2020-07-29 uniQure IP B.V. Mutated rep encoding sequences for use in aav production
EP2500434A1 (en) * 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
CA2942289C (en) * 2014-03-10 2024-05-21 Uniqure Ip B.V. Further improved aav vectors produced in insect cells
MX2018010633A (es) * 2016-03-03 2019-06-13 Univ Massachusetts Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral.
NZ762100A (en) * 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102737146B1 (ko) * 2023-07-10 2024-12-09 대한민국 신규 발현 벡터 및 이의 용도

Also Published As

Publication number Publication date
CN113454232A (zh) 2021-09-28
EP3924491A4 (en) 2022-12-14
EP3924491A1 (en) 2021-12-22
CN113454232B (zh) 2024-10-29
JP2024083567A (ja) 2024-06-21
IL285415A (en) 2021-09-30
US20220127625A1 (en) 2022-04-28
MA54958A (fr) 2021-12-22
WO2020168222A1 (en) 2020-08-20
AU2020221312A1 (en) 2021-10-07
JP2022520803A (ja) 2022-04-01
CA3129321A1 (en) 2020-08-20
JP7644014B2 (ja) 2025-03-11
SG11202106491VA (en) 2021-07-29

Similar Documents

Publication Publication Date Title
US12442015B2 (en) Closed-ended DNA vectors obtainable from cell-free synthesis and process for obtaining ceDNA vectors
KR20210127935A (ko) 폐쇄형 DNA(ceDNA) 생산에서의 Rep 단백질 활성의 변형
JP2022190081A (ja) 修飾型閉端ｄｎａ（ｃｅｄｎａ）
JP7590963B2 (ja) 対称的な修飾型逆位末端反復配列を含む修飾型閉端ｄｎａ（ｃｅｄｎａ）
US20220228171A1 (en) Compositions and production of nicked closed-ended dna vectors
AU2020315444A1 (en) Synthetic production of single-stranded adeno associated viral DNA vectors
AU2020235121A1 (en) Non-viral DNA vectors and uses thereof for expressing phenylalanine hydroxylase (PAH) therapeutics
KR20230003478A (ko) 비-바이러스성 dna 벡터 및 고셰 치료제 발현을 위한 이의 용도
RU2812850C2 (ru) Модуляция активности rep белка при получении днк с замкнутыми концами (зкднк)
RU2816963C2 (ru) МОДИФИЦИРОВАННАЯ ДНК С ЗАМКНУТЫМИ КОНЦАМИ (зкДНК), СОДЕРЖАЩАЯ СИММЕТРИЧНЫЕ МОДИФИЦИРОВАННЫЕ ИНВЕРТИРОВАННЫЕ КОНЦЕВЫЕ ПОВТОРЫ
RU2829367C2 (ru) ДНК С ЗАМКНУТЫМИ КОНЦАМИ (зкДНК) И ПРИМЕНЕНИЕ В СПОСОБАХ УМЕНЬШЕНИЯ ИММУННОГО ОТВЕТА, СВЯЗАННОГО С ГЕННОЙ ТЕРАПИЕЙ ИЛИ ТЕРАПИЕЙ НУКЛЕИНОВОЙ КИСЛОТОЙ
RU2800026C2 (ru) МОДИФИЦИРОВАННАЯ ДНК С ЗАМКНУТЫМИ КОНЦАМИ (зкДНК)

Legal Events

Date Code Title Description
E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

D21 Rejection of application intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D21-EXM-PE0902 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

E13 Pre-grant limitation requested

Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000