CN111818942A - 非病毒dna载体以及其用于产生抗体和融合蛋白的用途 - Google Patents

非病毒dna载体以及其用于产生抗体和融合蛋白的用途 Download PDF

Info

Publication number
CN111818942A
CN111818942A CN201980013028.XA CN201980013028A CN111818942A CN 111818942 A CN111818942 A CN 111818942A CN 201980013028 A CN201980013028 A CN 201980013028A CN 111818942 A CN111818942 A CN 111818942A
Authority
CN
China
Prior art keywords
antibody
cedna
itr
itrs
cedna vector
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CN201980013028.XA
Other languages
English (en)
Chinese (zh)
Inventor
O·阿尔坎
D·A·科尔
R·M·科廷
D·克拉特
L·刘
N·西尔弗
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Generational Biology Co
Original Assignee
Generational Biology 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 Generational Biology Co filed Critical Generational Biology Co
Publication of CN111818942A publication Critical patent/CN111818942A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/22—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors ; against growth regulators
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/24—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
    • C07K16/241—Tumor Necrosis Factors
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/53—DNA (RNA) vaccination
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
    • C12N2710/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14143—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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
    • C12N2710/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14144—Chimeric viral vector comprising heterologous viral elements for production of another viral 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
    • 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
    • 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
    • C12N2750/14144—Chimeric viral vector comprising heterologous viral elements for production of another viral 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Virology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Dermatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN201980013028.XA 2018-02-14 2019-02-14 非病毒dna载体以及其用于产生抗体和融合蛋白的用途 Pending CN111818942A (zh)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201862630676P 2018-02-14 2018-02-14
US201862630670P 2018-02-14 2018-02-14
US62/630,676 2018-02-14
US62/630,670 2018-02-14
US201862680087P 2018-06-04 2018-06-04
US201862680092P 2018-06-04 2018-06-04
US62/680,087 2018-06-04
US62/680,092 2018-06-04
PCT/US2019/018016 WO2019161059A1 (en) 2018-02-14 2019-02-14 Non-viral dna vectors and uses thereof for antibody and fusion protein production

Publications (1)

Publication Number Publication Date
CN111818942A true CN111818942A (zh) 2020-10-23

Family

ID=67619630

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201980013028.XA Pending CN111818942A (zh) 2018-02-14 2019-02-14 非病毒dna载体以及其用于产生抗体和融合蛋白的用途

Country Status (14)

Country Link
US (1) US20220042035A1 (https=)
EP (1) EP3752191A4 (https=)
JP (2) JP2021513355A (https=)
KR (1) KR20200120649A (https=)
CN (1) CN111818942A (https=)
AU (1) AU2019221642A1 (https=)
BR (1) BR112020016288A2 (https=)
CA (1) CA3091250A1 (https=)
IL (1) IL276469A (https=)
MA (1) MA51842A (https=)
MX (1) MX2020008470A (https=)
PH (1) PH12020551039A1 (https=)
SG (1) SG11202006431WA (https=)
WO (1) WO2019161059A1 (https=)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023091708A1 (en) * 2021-11-18 2023-05-25 The Brigham And Women's Hospital, Inc. Induced proteinopathy models
WO2024125530A1 (zh) * 2022-12-12 2024-06-20 苏州荷光科汇生物科技有限公司 一种感染视网膜的aav载体、阿达木单抗及其应用
WO2024234869A1 (en) * 2023-05-15 2024-11-21 Immunecare Biopharmaceuticals (Shanghai) Co., Ltd. Recombinant fusion protein for treatment of pulmonary hypertension

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202545984A (zh) 2017-08-09 2025-12-01 美商生物化學醫療公司 核酸分子及其用途
SG11202005271TA (en) * 2018-01-19 2020-07-29 Generation Bio Co Closed-ended dna vectors obtainable from cell-free synthesis and process for obtaining cedna vectors
WO2020033863A1 (en) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
JP7700101B2 (ja) * 2019-09-06 2025-06-30 ジェネレーション バイオ カンパニー 閉端ｄｎａおよび切断可能脂質を含む脂質ナノ粒子組成物ならびにそれらの使用方法
WO2021169167A1 (en) * 2020-02-29 2021-09-02 Nanjing GenScript Biotech Co., Ltd. Method for treating coronavirus infections
DE102020111571A1 (de) * 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
AU2021244559A1 (en) * 2020-03-24 2022-11-17 Generation Bio Co. Non-viral DNA vectors and uses thereof for expressing Gaucher therapeutics
CN116234917A (zh) 2020-07-27 2023-06-06 安杰瑞姆生物科学公司 Dna分子组合物及其制备方法和使用方法
WO2022153957A1 (ja) * 2021-01-12 2022-07-21 Ｊｃｒファーマ株式会社 リガンドと生理活性を有する蛋白質の融合蛋白質をコードする遺伝子が組み込まれた核酸分子
US20240092885A1 (en) * 2021-01-26 2024-03-21 Kriya Therapeutics, Inc. Vector constructs for delivery of nucleic acids encoding therapeutic anti-tnf antibodies and methods of using the same
CN117881786A (zh) * 2021-04-27 2024-04-12 世代生物公司 表达治疗性抗体的非病毒dna载体及其用途
WO2022236016A1 (en) * 2021-05-07 2022-11-10 Generation Bio Co. Lyophilized non-viral dna vector 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
CN116549632B (zh) * 2023-03-28 2024-09-10 中国人民解放军军事科学院军事医学研究院 抗Lingo-1抗体Opicinumab的新用途及针对新用途的药物
WO2025092951A1 (en) * 2023-11-03 2025-05-08 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant adeno-associated viral vectors for treating fabry disease

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US20150010578A1 (en) * 2011-02-22 2015-01-08 California Institute Of Technology Delivery of proteins using adeno-associated virus (aav) vectors
US20170007719A1 (en) * 2014-02-06 2017-01-12 Genzyme Corporation Compositions and methods for treating and preventing macular degeneration
US20170130245A1 (en) * 2014-06-09 2017-05-11 Voyager Therapeutics, Inc. Chimeric capsids

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ522840A (en) * 2000-06-01 2004-12-24 Univ North Carolina A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together
KR100968664B1 (ko) * 2001-08-27 2010-07-06 제넨테크, 인크. 항체 발현 및 조립을 위한 시스템
GB0903207D0 (en) * 2009-02-25 2009-04-08 Ucb Pharma Sa Method for expressing multimeric proteins
US9447433B2 (en) * 2013-03-15 2016-09-20 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
US11549125B2 (en) * 2017-08-09 2023-01-10 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Closed-ended, linear, duplex adenoassociated virus DNA, and uses thereof
AU2018330208B2 (en) * 2017-09-08 2025-04-17 Generation Bio Co. Lipid nanoparticle formulations of non-viral, capsid-free DNA vectors
JP2020532981A (ja) * 2017-09-08 2020-11-19 ジェネレーション バイオ カンパニー 修飾型閉端ｄｎａ（ｃｅｄｎａ）
BR112020007405A2 (pt) * 2017-10-18 2020-12-08 Regenxbio Inc. Produtos terapêuticos de anticorpo pós-translacionalmente modificados totalmente humanos
KR20200093635A (ko) * 2017-12-06 2020-08-05 제너레이션 바이오 컴퍼니 변형된 폐쇄된 말단 dna (cedna)를 사용한 유전자 편집
SG11202005271TA (en) * 2018-01-19 2020-07-29 Generation Bio Co Closed-ended dna vectors obtainable from cell-free synthesis and process for obtaining cedna vectors
US20210054405A1 (en) * 2018-03-02 2021-02-25 Generation Bio Co. Closed-ended dna (cedna) vectors for insertion of transgenes at genomic safe harbors (gsh) in humans and murine genomes

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150010578A1 (en) * 2011-02-22 2015-01-08 California Institute Of Technology Delivery of proteins using adeno-associated virus (aav) vectors
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
US20170007719A1 (en) * 2014-02-06 2017-01-12 Genzyme Corporation Compositions and methods for treating and preventing macular degeneration
US20170130245A1 (en) * 2014-06-09 2017-05-11 Voyager Therapeutics, Inc. Chimeric capsids

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
HADI BAYAT等: "Evaluation of different vector design strategies for the expression of recombinant monoclonal antibody in CHO cells_ Preparative Biochemistry & Biotechnology", PREPARATIVE BIOCHEMISTRY & BIOTECHNOLOGY, vol. 48, no. 2, 9 February 2018 (2018-02-09), pages 160 - 164 *
LINA LI等: "Production and Characterization of Novel Recombinant Adeno-Associated Virus Replicative-Form Genomes: A Eukaryotic Source of DNA for Gene Transfer", PLOS ONE, vol. 8, no. 8, pages 1 - 14, XP055416248, DOI: 10.1371/journal.pone.0069879 *
QINGZHANG ZHOU等: "Deletion of the B-B’ and C-C’ regions of inverted terminal repeats reduces rAAV productivity but increases transgene expression", SCIENTIFIC REPORTS, vol. 1, pages 1 - 13 *
QINGZHANG ZHOU等: "Deletion of the B-B’ and C-C’ regions of inverted terminal repeats reduces rAAV productivity but increases transgene expression", SCIENTIFIC REPORTS, vol. 7, 14 July 2017 (2017-07-14), pages 1 - 13 *
ZIYING YAN等: "Inverted Terminal Repeat Sequences Are Important for Intermolecular Recombination and Circularization of Adeno-Associated Virus Genomes", JOURNAL OF VIROLOGY, vol. 79, no. 1, pages 364 - 379, XP002399264 *

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023091708A1 (en) * 2021-11-18 2023-05-25 The Brigham And Women's Hospital, Inc. Induced proteinopathy models
WO2024125530A1 (zh) * 2022-12-12 2024-06-20 苏州荷光科汇生物科技有限公司 一种感染视网膜的aav载体、阿达木单抗及其应用
WO2024234869A1 (en) * 2023-05-15 2024-11-21 Immunecare Biopharmaceuticals (Shanghai) Co., Ltd. Recombinant fusion protein for treatment of pulmonary hypertension

Also Published As

Publication number Publication date
MA51842A (fr) 2020-12-23
PH12020551039A1 (en) 2021-08-23
JP2021513355A (ja) 2021-05-27
EP3752191A4 (en) 2021-12-22
US20220042035A1 (en) 2022-02-10
EP3752191A1 (en) 2020-12-23
KR20200120649A (ko) 2020-10-21
WO2019161059A1 (en) 2019-08-22
BR112020016288A2 (pt) 2020-12-15
MX2020008470A (es) 2020-09-25
JP2024167281A (ja) 2024-12-03
RU2020130010A (ru) 2022-03-14
SG11202006431WA (en) 2020-08-28
CA3091250A1 (en) 2019-08-22
IL276469A (en) 2020-09-30
AU2019221642A1 (en) 2020-07-09

Similar Documents

Publication Publication Date Title
JP2024167281A (ja) 非ウイルス性ｄｎａベクター、ならびに抗体および融合タンパク質の産生のためのその使用
JP2023126487A (ja) 無細胞合成から得ることができる閉端ＤＮＡベクターおよびｃｅＤＮＡベクターを得るためのプロセス
JP2022190081A (ja) 修飾型閉端ｄｎａ（ｃｅｄｎａ）
JP2023002828A (ja) 非ウイルス性カプシド不含ｄｎａベクターの脂質ナノ粒子製剤
CN113454232B (zh) 封闭端dna（cedna）产生中对rep蛋白活性的调节
JP2022506771A (ja) 対称的な修飾型逆位末端反復配列を含む修飾型閉端ｄｎａ（ｃｅｄｎａ）
US20240382432A1 (en) Single chain variable fragment (scfv) modified lipid nanoparticle compositions and uses thereof
CN113874513A (zh) 非病毒dna载体及其用于表达fviii治疗剂的用途
JP2026065641A (ja) 第ｉｘ因子治療薬を発現するための非ウイルス性ｄｎａベクター及びその使用
CN117295530A (zh) 非病毒dna载体及其用于表达pfic治疗剂的用途
JP2023520763A (ja) ゴーシェ治療薬を発現するための非ウイルス性ｄｎａベクター及びその使用
US20250255985A1 (en) Parvovirus itr-based gene delivery vector system
US20240216535A1 (en) Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
US20240226324A1 (en) Non-viral dna vectors expressing therapeutic antibodies and uses thereof
RU2800914C9 (ru) Невирусные днк-векторы и их применение для продуцирования антител и слитых белков
RU2800914C2 (ru) Невирусные днк-векторы и их применение для продуцирования антител и слитых белков
RU2816963C2 (ru) МОДИФИЦИРОВАННАЯ ДНК С ЗАМКНУТЫМИ КОНЦАМИ (зкДНК), СОДЕРЖАЩАЯ СИММЕТРИЧНЫЕ МОДИФИЦИРОВАННЫЕ ИНВЕРТИРОВАННЫЕ КОНЦЕВЫЕ ПОВТОРЫ

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination