MX2020005790A - Vectores de adn de extremo cerrado que pueden obtenerse de síntesis libre de células y un proceso para obtener vectores de ceadn. - Google Patents

Vectores de adn de extremo cerrado que pueden obtenerse de síntesis libre de células y un proceso para obtener vectores de ceadn.

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Publication number
MX2020005790A
MX2020005790A MX2020005790A MX2020005790A MX2020005790A MX 2020005790 A MX2020005790 A MX 2020005790A MX 2020005790 A MX2020005790 A MX 2020005790A MX 2020005790 A MX2020005790 A MX 2020005790A MX 2020005790 A MX2020005790 A MX 2020005790A
Authority
MX
Mexico
Prior art keywords
vectors
methods
closed
dna vectors
cell
Prior art date
Application number
MX2020005790A
Other languages
English (en)
Inventor
Robert Michael Kotin
Ozan Alkan
Douglas Anthony Kerr
Matthew Stanton
Carolyn Pelletier
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 MX2020005790A publication Critical patent/MX2020005790A/es

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    • 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
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/12Viral antigens
    • 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
    • C12N2710/14011Baculoviridae
    • C12N2710/14041Use of virus, viral particle or viral elements as a vector
    • C12N2710/14043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vectore
    • 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
    • C12N2800/00Nucleic acids vectors
    • C12N2800/10Plasmid DNA
    • C12N2800/103Plasmid DNA for invertebrates
    • C12N2800/105Plasmid DNA for invertebrates for insects
    • 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
    • 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
    • C12N2830/48Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Mycology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La solicitud describe métodos para la síntesis sintética y la síntesis libre de células de vectores de ADN, particularmente vectores de ADN de extremo cerrado (por ejemplo, vectores de ceADN) que tienen una estructura lineal y continua para la administración y expresión de un transgén. La presente invención se refiere a un proceso in vitro para la producción de vectores de ADN de extremo cerrado, productos de vectores de ADN correspondientes producidos con los métodos y usos de estos, y oligonucleótidos y kits útiles en el proceso de la invención. Los vectores de ADN producidos mediante los métodos como se describen en la presente están libres de efectos secundarios no deseados debido a contaminantes introducidos durante la producción en líneas celulares, por ejemplo, líneas celulares bacterianas o de insecto. En la presente se proporcionan además métodos y líneas celulares para una expresión génica in vitro, ex vivo e in vivo confiable mediante los vectores de ceADN sintetizados con los métodos de la presente.
MX2020005790A 2018-01-19 2019-01-18 Vectores de adn de extremo cerrado que pueden obtenerse de síntesis libre de células y un proceso para obtener vectores de ceadn. MX2020005790A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862619392P 2018-01-19 2018-01-19
PCT/US2019/014122 WO2019143885A1 (en) 2018-01-19 2019-01-18 Closed-ended dna vectors obtainable from cell-free synthesis and process for obtaining cedna vectors

Publications (1)

Publication Number Publication Date
MX2020005790A true MX2020005790A (es) 2020-10-28

Family

ID=67301565

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020005790A MX2020005790A (es) 2018-01-19 2019-01-18 Vectores de adn de extremo cerrado que pueden obtenerse de síntesis libre de células y un proceso para obtener vectores de ceadn.

Country Status (16)

Country Link
US (1) US12442015B2 (es)
EP (1) EP3740571A4 (es)
JP (3) JP2021511047A (es)
KR (2) KR102879598B1 (es)
CN (1) CN111868242A (es)
AU (1) AU2019210034B2 (es)
BR (1) BR112020013319A2 (es)
CA (1) CA3088984A1 (es)
IL (1) IL275878B2 (es)
MA (1) MA51626A (es)
MX (1) MX2020005790A (es)
MY (1) MY208589A (es)
PH (1) PH12020550878A1 (es)
SG (1) SG11202005271TA (es)
UA (1) UA130154C2 (es)
WO (1) WO2019143885A1 (es)

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MA51842A (fr) * 2018-02-14 2020-12-23 Generation Bio Co Vecteurs d'adn non viraux et utilisations associées pour la production d'anticorps et de protéines de fusion
KR102925832B1 (ko) 2018-08-09 2026-02-11 바이오버라티브 테라퓨틱스 인크. 핵산 분자 및 비바이러스 유전자 치료를 위한 이의 용도
AU2019376663A1 (en) * 2018-11-09 2021-06-24 Generation Bio Co. Modified closed-ended DNA (ceDNA) comprising symmetrical modified inverted terminal repeats
GB201905651D0 (en) 2019-04-24 2019-06-05 Lightbio Ltd Nucleic acid constructs and methods for their manufacture
CA3147414A1 (en) 2019-07-17 2021-01-21 Generation Bio Co. Synthetic production of single-stranded adeno associated viral dna vectors
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AU2021244555A1 (en) * 2020-03-24 2022-11-24 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
WO2022023284A1 (en) * 2020-07-27 2022-02-03 Anjarium Biosciences Ag Compositions of dna molecules, methods of making therefor, and methods of use thereof
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TW202246499A (zh) * 2021-01-25 2022-12-01 美商普維爾治療公司 以腺相關病毒(aav)為基礎的基因表現之調節
CN112980862A (zh) * 2021-02-25 2021-06-18 通用生物系统(安徽)有限公司 一种高纯度微环dna的制备方法
US20240358852A1 (en) 2021-04-20 2024-10-31 Anjarium Biosciences Ag Compositions of dna molecules encoding amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase, methods of making thereof, and methods of use thereof
EP4329884A1 (en) * 2021-04-27 2024-03-06 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
WO2022232289A1 (en) * 2021-04-27 2022-11-03 Generation Bio Co. Non-viral dna vectors expressing therapeutic antibodies and uses thereof
KR20240005903A (ko) * 2021-05-07 2024-01-12 제너레이션 바이오 컴퍼니 백신 전달을 위한 비바이러스성 dna 벡터
US20240261395A1 (en) * 2021-05-07 2024-08-08 Generation Bio Co. Lyophilized non-viral dna vector compositions and uses thereof
CN118489008A (zh) * 2021-12-23 2024-08-13 世代生物公司 封闭端dna载体的可扩展且高纯度无细胞合成
JP2025504404A (ja) 2022-01-14 2025-02-12 アンジャリウム バイオサイエンシズ エージー Viii因子をコードするdna分子の組成物、それを作製する方法、及びその使用方法
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US20260055426A1 (en) 2022-08-19 2026-02-26 Generation Bio Co. CLEAVABLE CLOSED-ENDED DNA (ceDNA) AND METHODS OF USE THEREOF
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Also Published As

Publication number Publication date
KR20200111726A (ko) 2020-09-29
WO2019143885A1 (en) 2019-07-25
IL275878B1 (en) 2025-11-01
BR112020013319A2 (pt) 2020-12-01
MY208589A (en) 2025-05-19
PH12020550878A1 (en) 2021-04-05
EP3740571A1 (en) 2020-11-25
JP2023126487A (ja) 2023-09-07
EP3740571A4 (en) 2021-12-08
CN111868242A (zh) 2020-10-30
IL275878B2 (en) 2026-03-01
KR102879598B1 (ko) 2025-11-03
IL275878A (en) 2020-08-31
CA3088984A1 (en) 2019-07-25
JP2025113389A (ja) 2025-08-01
KR20250159738A (ko) 2025-11-11
MA51626A (fr) 2020-11-25
RU2020127017A (ru) 2022-02-21
SG11202005271TA (en) 2020-07-29
AU2019210034B2 (en) 2025-03-27
US12442015B2 (en) 2025-10-14
AU2019210034A1 (en) 2020-07-09
US20210071197A1 (en) 2021-03-11
JP2021511047A (ja) 2021-05-06
UA130154C2 (uk) 2025-12-03

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