CA3129321A1 - Modulation of rep protein activity in closed-ended dna (cedna) production - Google Patents

Modulation of rep protein activity in closed-ended dna (cedna) production Download PDF

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Publication number
CA3129321A1
CA3129321A1 CA3129321A CA3129321A CA3129321A1 CA 3129321 A1 CA3129321 A1 CA 3129321A1 CA 3129321 A CA3129321 A CA 3129321A CA 3129321 A CA3129321 A CA 3129321A CA 3129321 A1 CA3129321 A1 CA 3129321A1
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Canada
Prior art keywords
itr
rep
protein
dna
cell
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Pending
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CA3129321A
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English (en)
French (fr)
Inventor
Robert Michael Kotin
Anna UCHER
Ara Karl MALAKIAN
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Generation Bio Co
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Generation Bio Co
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Application filed by Generation Bio Co filed Critical Generation Bio Co
Publication of CA3129321A1 publication Critical patent/CA3129321A1/en
Pending legal-status Critical Current

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    • 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

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  • 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)
CA3129321A 2019-02-15 2020-02-14 Modulation of rep protein activity in closed-ended dna (cedna) production Pending CA3129321A1 (en)

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
CA3129321A1 true CA3129321A1 (en) 2020-08-20

Family

ID=72045641

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3129321A Pending CA3129321A1 (en) 2019-02-15 2020-02-14 Modulation of rep protein activity in closed-ended dna (cedna) 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
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
KR20210127935A (ko) 2021-10-25
JP7644014B2 (ja) 2025-03-11
SG11202106491VA (en) 2021-07-29

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