CA3088984A1 - Closed-ended dna vectors obtainable from cell-free synthesis and process for obtaining cedna vectors - Google Patents

Closed-ended dna vectors obtainable from cell-free synthesis and process for obtaining cedna vectors Download PDF

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Publication number
CA3088984A1
CA3088984A1 CA3088984A CA3088984A CA3088984A1 CA 3088984 A1 CA3088984 A1 CA 3088984A1 CA 3088984 A CA3088984 A CA 3088984A CA 3088984 A CA3088984 A CA 3088984A CA 3088984 A1 CA3088984 A1 CA 3088984A1
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Canada
Prior art keywords
itr
sequence
sense
expression cassette
stranded
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Pending
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CA3088984A
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English (en)
French (fr)
Inventor
Ozan ALKAN
Robert Michael Kotin
Matthew Stanton
Douglas Anthony KERR
Carolyn PELLETIER
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Generation Bio Co
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Generation Bio Co
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Publication of CA3088984A1 publication Critical patent/CA3088984A1/en
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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
    • 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
    • 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
    • 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)
CA3088984A 2018-01-19 2019-01-18 Closed-ended dna vectors obtainable from cell-free synthesis and process for obtaining cedna vectors Pending CA3088984A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862619392P 2018-01-19 2018-01-19
US62/619,392 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
CA3088984A1 true CA3088984A1 (en) 2019-07-25

Family

ID=67301565

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3088984A Pending CA3088984A1 (en) 2018-01-19 2019-01-18 Closed-ended dna vectors obtainable from cell-free synthesis and process for obtaining cedna vectors

Country Status (16)

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

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BR112020002394A2 (pt) 2017-08-09 2020-07-28 Bioverativ Therapeutics Inc. moléculas de ácidos nucleicos e usos das mesmas
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
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
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
US20220243201A1 (en) * 2020-08-23 2022-08-04 Bioverativ Therapeutics Inc. Engineered itr sequences and methods of use
EP4214309A4 (en) * 2020-09-16 2025-07-16 Generation Bio Co CLOSED-END DNA VECTORS AND RELATED USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH)
GB202014751D0 (en) 2020-09-18 2020-11-04 Lightbio Ltd Targeting vector
GB202014772D0 (en) 2020-09-18 2020-11-04 Lightbio Ltd Nucleic acid construct
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
GB202204112D0 (en) 2022-03-23 2022-05-04 Lightbio Ltd Linear construct
EP4293101A1 (en) 2022-06-14 2023-12-20 Asklepios Biopharmaceutical, Inc. Reactor with temperature control and method of using the same
EP4558637A2 (en) 2022-07-19 2025-05-28 Rampart Bioscience, Inc. Non-immunogenic circular, non-viral dna vectors
US20260055426A1 (en) 2022-08-19 2026-02-26 Generation Bio Co. CLEAVABLE CLOSED-ENDED DNA (ceDNA) AND METHODS OF USE THEREOF
WO2024119017A1 (en) * 2022-12-01 2024-06-06 Generation Bio Co. Synthetic single stranded nucleic acid compositions and methods thereof
WO2024170778A1 (en) 2023-02-17 2024-08-22 Anjarium Biosciences Ag Methods of making dna molecules and compositions and uses thereof
US11993783B1 (en) 2023-03-27 2024-05-28 Genecraft Inc. Nucleic acid molecule comprising asymmetrically modified ITR for improving expression rate of inserted gene, and 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
MX2020005790A (es) 2020-10-28
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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