IL285415A - Modulation of rep protein activity in closed-end DNA production - Google Patents

Modulation of rep protein activity in closed-end DNA production

Info

Publication number
IL285415A
IL285415A IL285415A IL28541521A IL285415A IL 285415 A IL285415 A IL 285415A IL 285415 A IL285415 A IL 285415A IL 28541521 A IL28541521 A IL 28541521A IL 285415 A IL285415 A IL 285415A
Authority
IL
Israel
Prior art keywords
cedna
modulation
closed
production
protein activity
Prior art date
Application number
IL285415A
Other languages
English (en)
Hebrew (he)
Original Assignee
Generation Bio Co
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 Generation Bio Co filed Critical Generation Bio Co
Publication of IL285415A publication Critical patent/IL285415A/en

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    • CCHEMISTRY; METALLURGY
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    • 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
    • C12N15/64General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
    • 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
    • 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
    • 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
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6009Vectors comprising as targeting moiety peptide derived from defined protein from viruses dsDNA viruses
    • C12N2810/6018Adenoviridae
    • 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
    • C12N2820/00Vectors comprising a special origin of replication system
    • C12N2820/002Vectors comprising a special origin of replication system inducible or controllable
    • 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
    • C12N2820/00Vectors comprising a special origin of replication system
    • C12N2820/60Vectors 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)
IL285415A 2019-02-15 2021-08-05 Modulation of rep protein activity in closed-end DNA production IL285415A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962806076P 2019-02-15 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
IL285415A true IL285415A (en) 2021-09-30

Family

ID=72045641

Family Applications (1)

Application Number Title Priority Date Filing Date
IL285415A IL285415A (en) 2019-02-15 2021-08-05 Modulation of rep protein activity in closed-end DNA production

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=)

Families Citing this family (12)

* 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
KR102737146B1 (ko) * 2023-07-10 2024-12-09 대한민국 신규 발현 벡터 및 이의 용도
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

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
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
KR20210127935A (ko) 2021-10-25
CA3129321A1 (en) 2020-08-20
JP7644014B2 (ja) 2025-03-11
SG11202106491VA (en) 2021-07-29

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