IL275878B2 - Closed-ended DNA vectors obtainable by cell-free synthesis and a process for obtaining cedna vectors - Google Patents

Closed-ended DNA vectors obtainable by cell-free synthesis and a process for obtaining cedna vectors

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Publication number
IL275878B2
IL275878B2 IL275878A IL27587820A IL275878B2 IL 275878 B2 IL275878 B2 IL 275878B2 IL 275878 A IL275878 A IL 275878A IL 27587820 A IL27587820 A IL 27587820A IL 275878 B2 IL275878 B2 IL 275878B2
Authority
IL
Israel
Prior art keywords
vectors
obtaining
cell
closed
obtainable
Prior art date
Application number
IL275878A
Other languages
English (en)
Hebrew (he)
Other versions
IL275878B1 (en
IL275878A (en
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 IL275878A publication Critical patent/IL275878A/en
Publication of IL275878B1 publication Critical patent/IL275878B1/en
Publication of IL275878B2 publication Critical patent/IL275878B2/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

Landscapes

  • 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)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
IL275878A 2018-01-19 2019-01-18 Closed-ended DNA vectors obtainable by cell-free synthesis and a process for obtaining cedna vectors IL275878B2 (en)

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 (3)

Publication Number Publication Date
IL275878A IL275878A (en) 2020-08-31
IL275878B1 IL275878B1 (en) 2025-11-01
IL275878B2 true IL275878B2 (en) 2026-03-01

Family

ID=67301565

Family Applications (1)

Application Number Title Priority Date Filing Date
IL275878A IL275878B2 (en) 2018-01-19 2019-01-18 Closed-ended DNA vectors obtainable by cell-free synthesis and a process for obtaining cedna vectors

Country Status (16)

Country Link
US (1) US12442015B2 (https=)
EP (1) EP3740571A4 (https=)
JP (3) JP2021511047A (https=)
KR (2) KR20250159738A (https=)
CN (1) CN111868242A (https=)
AU (1) AU2019210034B2 (https=)
BR (1) BR112020013319A2 (https=)
CA (1) CA3088984A1 (https=)
IL (1) IL275878B2 (https=)
MA (1) MA51626A (https=)
MX (1) MX2020005790A (https=)
MY (1) MY208589A (https=)
PH (1) PH12020550878A1 (https=)
SG (1) SG11202005271TA (https=)
UA (1) UA130154C2 (https=)
WO (1) WO2019143885A1 (https=)

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NZ762100A (en) 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
KR20200120649A (ko) * 2018-02-14 2020-10-21 제너레이션 바이오 컴퍼니 비-바이러스 dna 벡터 및 항체 및 융합 단백질 생산을 위한 이의 용도
EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
KR20210090619A (ko) * 2018-11-09 2021-07-20 제너레이션 바이오 컴퍼니 대칭인 변형된 역말단반복을 포함하는 변형된 폐쇄형 DNA(ceDNA)
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
US20230138409A1 (en) * 2020-03-24 2023-05-04 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
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
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
US20240100132A1 (en) * 2021-01-25 2024-03-28 Prevail Therapeutics, Inc. Modulation of aav-based gene expression
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
JP2024517427A (ja) 2021-04-20 2024-04-22 アンジャリウム バイオサイエンシズ エージー アミロ-α-1,6-グルコシダーゼ、4-α-グルカノトランスフェラーゼをコードするDNA分子の組成物、その作製の方法、及びその使用の方法
US20240216535A1 (en) * 2021-04-27 2024-07-04 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
AU2022264509A1 (en) * 2021-04-27 2023-12-14 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
CA3218126A1 (en) * 2021-05-07 2022-11-10 Matthew MANGANIELLO Lyophilized non-viral dna vector compositions and uses thereof
AU2022422134A1 (en) * 2021-12-23 2024-07-04 Generation Bio Co. Scalable and high-purity cell-free synthesis of closed-ended dna vectors
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
CA3262218A1 (en) 2022-07-19 2024-01-25 Rampart Bioscience, Inc. CIRCULAR NON-IMMUNOGENEOUS 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
KR20250129818A (ko) * 2022-12-01 2025-08-29 제너레이션 바이오 컴퍼니 합성 단일-가닥 dna 분자 및 이를 생산하고 사용하는 방법
EP4665865A1 (en) 2023-02-17 2025-12-24 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
WO2026085495A1 (en) 2024-10-18 2026-04-23 Generation Bio Co. Synthetic partially single-stranded nucleic acid compositions and uses and methods therefor

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020164783A1 (en) * 1997-10-21 2002-11-07 Feldhaus Andrew L. Transcriptionally-activated AAV inverted terminal repeats (ITRs) for use with recombinant AAV vectors
US20100105110A1 (en) * 2008-10-28 2010-04-29 Xavier Danthinne Method of Adenoviral Vector Synthesis
US20140107186A1 (en) * 2011-03-11 2014-04-17 Association Institut De Myologie Capsid-free aav vectors, compositions, and methods for vector production and gene delivery
US20140271551A1 (en) * 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Synthetic Adeno-Associated Virus Inverted Terminal Repeats
WO2017152149A1 (en) * 2016-03-03 2017-09-08 University Of Massachusetts Closed-ended linear duplex dna for non-viral gene transfer

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US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
CA2467647A1 (en) 2001-11-30 2003-06-05 Innogenetics N.V. Therapeutic use of antibodies and fragments thereof binding primate ifn-gamma
US8071332B2 (en) 2007-08-10 2011-12-06 Toto Ltd. Recombinant mammal cells, method of producing thereof, and method of producing proteins of interest
JP7026248B2 (ja) 2017-11-21 2022-02-25 4ベースバイオ ソシエダー リミターダ 二本鎖dnaを増幅するための方法およびキット
KR20200120649A (ko) * 2018-02-14 2020-10-21 제너레이션 바이오 컴퍼니 비-바이러스 dna 벡터 및 항체 및 융합 단백질 생산을 위한 이의 용도
JP2022518504A (ja) * 2019-01-24 2022-03-15 ジェネレーション バイオ カンパニー 閉端dna(cedna)ならびに遺伝子療法または核酸療法に関連する免疫応答を低減させる方法における使用
CA3147414A1 (en) * 2019-07-17 2021-01-21 Generation Bio Co. Synthetic production of single-stranded adeno associated viral dna vectors
CA3146966A1 (en) * 2019-07-17 2021-01-21 Generation Bio Co. Compositions and production of nicked closed-ended dna vectors

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020164783A1 (en) * 1997-10-21 2002-11-07 Feldhaus Andrew L. Transcriptionally-activated AAV inverted terminal repeats (ITRs) for use with recombinant AAV vectors
US20100105110A1 (en) * 2008-10-28 2010-04-29 Xavier Danthinne Method of Adenoviral Vector Synthesis
US20140107186A1 (en) * 2011-03-11 2014-04-17 Association Institut De Myologie Capsid-free aav vectors, compositions, and methods for vector production and gene delivery
US20140271551A1 (en) * 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Synthetic Adeno-Associated Virus Inverted Terminal Repeats
WO2017152149A1 (en) * 2016-03-03 2017-09-08 University Of Massachusetts Closed-ended linear duplex dna for non-viral gene transfer

Also Published As

Publication number Publication date
UA130154C2 (uk) 2025-12-03
MY208589A (en) 2025-05-19
EP3740571A4 (en) 2021-12-08
PH12020550878A1 (en) 2021-04-05
KR102879598B1 (ko) 2025-11-03
RU2020127017A (ru) 2022-02-21
MA51626A (fr) 2020-11-25
JP2025113389A (ja) 2025-08-01
BR112020013319A2 (pt) 2020-12-01
US20210071197A1 (en) 2021-03-11
IL275878B1 (en) 2025-11-01
WO2019143885A1 (en) 2019-07-25
KR20200111726A (ko) 2020-09-29
MX2020005790A (es) 2020-10-28
EP3740571A1 (en) 2020-11-25
JP2023126487A (ja) 2023-09-07
CN111868242A (zh) 2020-10-30
JP2021511047A (ja) 2021-05-06
IL275878A (en) 2020-08-31
KR20250159738A (ko) 2025-11-11
AU2019210034A1 (en) 2020-07-09
US12442015B2 (en) 2025-10-14
AU2019210034B2 (en) 2025-03-27
SG11202005271TA (en) 2020-07-29
CA3088984A1 (en) 2019-07-25

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