JP2021513355A - 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 - Google Patents

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 Download PDF

Info

Publication number
JP2021513355A
JP2021513355A JP2020543344A JP2020543344A JP2021513355A JP 2021513355 A JP2021513355 A JP 2021513355A JP 2020543344 A JP2020543344 A JP 2020543344A JP 2020543344 A JP2020543344 A JP 2020543344A JP 2021513355 A JP2021513355 A JP 2021513355A
Authority
JP
Japan
Prior art keywords
antibody
itr
cedna vector
cedna
sequence
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
JP2020543344A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021513355A5 (enExample
Inventor
オザン アルカン，
オザン アルカン，
ダグラス アンソニー カー，
ダグラス アンソニー カー，
ロバート マイケル コーティン，
ロバート マイケル コーティン，
デブラ クラット，
デブラ クラット，
リア リウ，
リア リウ，
ナサニエル シルバー，
ナサニエル シルバー，
Original Assignee
ジェネレーション バイオ カンパニー
ジェネレーション バイオ カンパニー
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 ジェネレーション バイオ カンパニー, ジェネレーション バイオ カンパニー filed Critical ジェネレーション バイオ カンパニー
Publication of JP2021513355A publication Critical patent/JP2021513355A/ja
Publication of JP2021513355A5 publication Critical patent/JP2021513355A5/ja
Priority to JP2024141968A priority Critical patent/JP2024167281A/ja
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
    • 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
    • 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
    • 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)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Virology (AREA)
  • Psychiatry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Dermatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2020543344A 2018-02-14 2019-02-14 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 Pending JP2021513355A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024141968A JP2024167281A (ja) 2018-02-14 2024-08-23 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

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201862630670P 2018-02-14 2018-02-14
US201862630676P 2018-02-14 2018-02-14
US62/630,670 2018-02-14
US62/630,676 2018-02-14
US201862680087P 2018-06-04 2018-06-04
US201862680092P 2018-06-04 2018-06-04
US62/680,092 2018-06-04
US62/680,087 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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024141968A Division JP2024167281A (ja) 2018-02-14 2024-08-23 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

Publications (2)

Publication Number Publication Date
JP2021513355A true JP2021513355A (ja) 2021-05-27
JP2021513355A5 JP2021513355A5 (enExample) 2022-02-22

Family

ID=67619630

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020543344A Pending JP2021513355A (ja) 2018-02-14 2019-02-14 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
JP2024141968A Pending JP2024167281A (ja) 2018-02-14 2024-08-23 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

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024141968A Pending JP2024167281A (ja) 2018-02-14 2024-08-23 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

Country Status (14)

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

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023517940A (ja) * 2020-03-11 2023-04-27 イマティクス ユーエス，アイエヌシー． Ｗｐｒｅ変異体コンストラクト、組成物、およびその方法

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2020001593A (es) 2017-08-09 2020-07-13 Bioverativ Therapeutics Inc Moleculas de acido nucleico y usos de las mismas.
JP2021511047A (ja) 2018-01-19 2021-05-06 ジェネレーション バイオ カンパニー 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
JP7602454B2 (ja) 2018-08-09 2024-12-18 バイオベラティブ セラピューティクス インコーポレイテッド 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用
CN114929205B (zh) * 2019-09-06 2024-09-03 世代生物公司 包括末端封闭式dna和可切割脂质的脂质纳米颗粒组合物及其使用方法
AU2020392243A1 (en) 2019-11-28 2022-06-16 Regenxbio Inc. Microdystrophin gene therapy constructs and uses thereof
WO2021169167A1 (en) * 2020-02-29 2021-09-02 Nanjing GenScript Biotech Co., Ltd. Method for treating coronavirus infections
IL296662A (en) * 2020-03-24 2022-11-01 Generation Bio Co Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
MX2023001109A (es) 2020-07-27 2023-04-24 Anjarium Biosciences Ag Composiciones de moleculas de adn, metodos para elaborar las mismas, y metodos de usos de las mismas.
JP2022108271A (ja) * 2021-01-12 2022-07-25 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
CA3206107A1 (en) * 2021-01-26 2022-08-04 Brian Furmanski Vector constructs for delivery of nucleic acids encoding therapeutic anti-tnf antibodies and methods of using the same
JP2024515788A (ja) * 2021-04-27 2024-04-10 ジェネレーション バイオ カンパニー 治療抗体を発現する非ウイルスｄｎａベクター及びその使用
WO2022236016A1 (en) * 2021-05-07 2022-11-10 Generation Bio Co. Lyophilized non-viral dna vector compositions and uses thereof
US20260167928A1 (en) * 2021-11-18 2026-06-18 The Brigham And Women's Hospital, Inc. Induced proteinopathy models
EP4455298A4 (en) * 2021-12-23 2026-01-14 Takeda Pharmaceuticals Co METHOD FOR PREDICTING THE RATE OF GENE TRANSFER
CN118186005A (zh) * 2022-12-12 2024-06-14 苏州荷光科汇生物科技有限公司 一种适于阿达木单抗的表达盒及其载体与应用
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
CN121127502A (zh) * 2023-05-15 2025-12-12 宜明凯尔生物医药技术(上海)有限公司 用于治疗肺高压的重组融合蛋白
WO2025092951A1 (en) * 2023-11-03 2025-05-08 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant adeno-associated viral vectors for treating fabry disease

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2005517385A (ja) * 2001-08-27 2005-06-16 ジェネンテック・インコーポレーテッド 抗体発現系とその構築法
WO2010097437A1 (en) * 2009-02-25 2010-09-02 Ucb Pharma, S.A. Method for producing antibodies
WO2017152149A1 (en) * 2016-03-03 2017-09-08 University Of Massachusetts Closed-ended linear duplex dna for non-viral gene transfer

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7465583B2 (en) * 2000-06-01 2008-12-16 The University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
JP6091435B2 (ja) * 2011-02-22 2017-03-08 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス（ａａｖ）ベクターを用いたタンパク質の送達
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
EP2970966A4 (en) * 2013-03-15 2016-10-26 Univ North Carolina SYNTHETIC ADENOASSOZED VIRUS INVERTED REPEAT SEQUENCES
KR102234695B1 (ko) * 2014-02-06 2021-04-02 젠자임 코포레이션 황반 변성을 치료하고 예방하기 위한 조성물 및 방법
US10577627B2 (en) * 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
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
EP3679148A4 (en) * 2017-09-08 2021-06-09 Generation Bio Co. LIPID NANOPARTICLE FORMULATIONS FROM NONVIRAL, CAPSID-FREE DNA VECTORS
CA3075168A1 (en) * 2017-09-08 2019-03-14 Generation Bio Co. Modified closed-ended dna (cedna)
KR20200067195A (ko) * 2017-10-18 2020-06-11 리젠엑스바이오 인크. 완전-인간 번역후 변형된 항체 치료제
JP2021505159A (ja) * 2017-12-06 2021-02-18 ジェネレーション バイオ カンパニー 修飾型閉端ｄｎａ（ｃｅｄｎａ）を使用する遺伝子編集
JP2021511047A (ja) * 2018-01-19 2021-05-06 ジェネレーション バイオ カンパニー 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
CA3092459A1 (en) * 2018-03-02 2019-09-06 Generation Bio Co. Closed-ended dna (cedna) vectors for insertion of transgenes at genomic safe harbors (gsh) in humans and murine genomes

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2005517385A (ja) * 2001-08-27 2005-06-16 ジェネンテック・インコーポレーテッド 抗体発現系とその構築法
WO2010097437A1 (en) * 2009-02-25 2010-09-02 Ucb Pharma, S.A. Method for producing antibodies
WO2017152149A1 (en) * 2016-03-03 2017-09-08 University Of Massachusetts Closed-ended linear duplex dna for non-viral gene transfer

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
INT. J. RETIN. VITR., vol. Vol. 2:3, JPN6023013132, 2016, pages 1 - 9, ISSN: 0005027976 *
MICROBIOL. SPECTR., vol. Vol. 2(4), JPN6024015509, 2014, pages 1 - 11, ISSN: 0005509353 *
PREP. BIOCHEM. BIOTECHNOL., vol. 48, JPN6023040904, February 2018 (2018-02-01), pages 160 - 164, ISSN: 0005309229 *

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023517940A (ja) * 2020-03-11 2023-04-27 イマティクス ユーエス，アイエヌシー． Ｗｐｒｅ変異体コンストラクト、組成物、およびその方法
JP7739319B2 (ja) 2020-03-11 2025-09-16 イマティクス ユーエス，アイエヌシー． Ｗｐｒｅ変異体コンストラクト、組成物、およびその方法
JP2025182221A (ja) * 2020-03-11 2025-12-12 イマティクス ユーエス，アイエヌシー． Ｗｐｒｅ変異体コンストラクト、組成物、およびその方法
JP7884129B2 (ja) 2020-03-11 2026-07-02 イマティクス ユーエス，アイエヌシー． Ｗｐｒｅ変異体コンストラクト、組成物、およびその方法

Also Published As

Publication number Publication date
MX2020008470A (es) 2020-09-25
EP3752191A1 (en) 2020-12-23
PH12020551039A1 (en) 2021-08-23
AU2019221642A1 (en) 2020-07-09
EP3752191A4 (en) 2021-12-22
JP2024167281A (ja) 2024-12-03
WO2019161059A1 (en) 2019-08-22
RU2020130010A (ru) 2022-03-14
US20220042035A1 (en) 2022-02-10
CN111818942A (zh) 2020-10-23
SG11202006431WA (en) 2020-08-28
CA3091250A1 (en) 2019-08-22
BR112020016288A2 (pt) 2020-12-15
IL276469A (en) 2020-09-30
KR20200120649A (ko) 2020-10-21
MA51842A (fr) 2020-12-23

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
JP2022190081A (ja) 修飾型閉端ｄｎａ（ｃｅｄｎａ）
JP6983511B2 (ja) 脳中の転移乳癌および他の癌を処置するための方法および組成物
JP2021511047A (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
JP2022524434A (ja) Ｆｖｉｉｉ治療薬を発現するための非ウイルス性ｄｎａベクターおよびその使用
JP2022520803A (ja) 閉端ＤＮＡ（ｃｅＤＮＡ）の産生におけるＲＥＰタンパク質活性の調節
EP3452103A1 (en) Compositions for treatment of wet age-related macular degeneration
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）
JP2022525302A (ja) フェニルアラニンヒドロキシラーゼ（ｐａｈ）治療薬を発現するための非ウイルス性ｄｎａベクターおよびその使用
JP7842082B2 (ja) 眼疾患を処置するための組成物および方法
KR20230131246A (ko) 파브리병을 치료하기 위한 조성물 및 방법
JP2023520764A (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
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
KR20230117179A (ko) 청신경초종 연관 증상을 치료하기 위한 항-vegf 항체작제물 및 관련된 방법
CA3228666A1 (en) Compositions and methods for transgene expression
CN116568283A (zh) 用于治疗前庭神经鞘瘤相关症状的抗vegf抗体构建体和相关方法
US20240216535A1 (en) Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
US20250255985A1 (en) Parvovirus itr-based gene delivery vector system
US20240226324A1 (en) Non-viral dna vectors expressing therapeutic antibodies and uses thereof
RU2800914C2 (ru) Невирусные днк-векторы и их применение для продуцирования антител и слитых белков
RU2800914C9 (ru) Невирусные днк-векторы и их применение для продуцирования антител и слитых белков
HK40090167A (en) Construction and application of an in vivo anti-vegf antibody expression system
HK40091511A (en) Construction and application of anti-vegf antibody in-vivo expression system
JP2024527344A (ja) 抗体応答を抗原に適合させるための抗体の利用
NZ787256A (en) Compositions For Treatment of Wet Age-Related Macular Degeneration

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220214

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220214

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230404

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230704

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20231003

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240104

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20240423

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240823

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20241007