JP2021516953A - 合成ｄｎａベクターおよびその使用 - Google Patents

合成ｄｎａベクターおよびその使用 Download PDF

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JP2021516953A
JP2021516953A JP2020545324A JP2020545324A JP2021516953A JP 2021516953 A JP2021516953 A JP 2021516953A JP 2020545324 A JP2020545324 A JP 2020545324A JP 2020545324 A JP2020545324 A JP 2020545324A JP 2021516953 A JP2021516953 A JP 2021516953A
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dna
dna vector
isolated
vector
polymerase
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JP2020545324A
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Japanese (ja)
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JP2021516953A5 (https=
Inventor
ブルース シー． シュネップ
ブルース シー． シュネップ
フィリップ アール． ジョンソン
フィリップ アール． ジョンソン
Original Assignee
インターガラクティック セラピューティクス インコーポレイテッド
インターガラクティック セラピューティクス インコーポレイテッド
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Publication of JP2021516953A publication Critical patent/JP2021516953A/ja
Publication of JP2021516953A5 publication Critical patent/JP2021516953A5/ja
Priority to JP2023222352A priority Critical patent/JP2024041819A/ja
Pending legal-status Critical Current

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    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2020545324A 2018-03-15 2019-03-15 合成ｄｎａベクターおよびその使用 Pending JP2021516953A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023222352A JP2024041819A (ja) 2018-03-15 2023-12-28 合成ｄｎａベクターおよびその使用

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862643336P 2018-03-15 2018-03-15
US62/643,336 2018-03-15
US201862749369P 2018-10-23 2018-10-23
US62/749,369 2018-10-23
PCT/US2019/022511 WO2019178500A1 (en) 2018-03-15 2019-03-15 Synthetic dna vectors and methods of use

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023222352A Division JP2024041819A (ja) 2018-03-15 2023-12-28 合成ｄｎａベクターおよびその使用

Publications (2)

Publication Number Publication Date
JP2021516953A true JP2021516953A (ja) 2021-07-15
JP2021516953A5 JP2021516953A5 (https=) 2022-03-23

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Family Applications (2)

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JP2020545324A Pending JP2021516953A (ja) 2018-03-15 2019-03-15 合成ｄｎａベクターおよびその使用
JP2023222352A Pending JP2024041819A (ja) 2018-03-15 2023-12-28 合成ｄｎａベクターおよびその使用

Family Applications After (1)

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Country Status (10)

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US (2) US20210002667A1 (https=)
EP (1) EP3765023A4 (https=)
JP (2) JP2021516953A (https=)
KR (1) KR20200132893A (https=)
CN (2) CN117757842A (https=)
AU (1) AU2019233901A1 (https=)
CA (1) CA3092088A1 (https=)
IL (1) IL276923A (https=)
MX (1) MX2020009579A (https=)
WO (1) WO2019178500A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024532305A (ja) * 2021-08-24 2024-09-05 賽斯尓▲チン▼生物技術（上海）有限公司 細胞を修飾する方法
JP2025527489A (ja) * 2022-08-16 2025-08-22 アルデブロン，エル．エル．シー． 合成環状核酸を産生する無細胞法

Families Citing this family (13)

* Cited by examiner, † Cited by third party
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US12403305B2 (en) 2016-06-27 2025-09-02 Galvanize Therapeutics, Inc. Immunostimulation in the treatment of viral infection
EP3474760B1 (en) 2016-06-27 2023-01-11 Galvanize Therapeutics, Inc. Generator and a catheter with an electrode for treating a lung passageway
US20200048716A1 (en) * 2017-11-03 2020-02-13 Twister Biotech, Inc Using minivectors to treat ovarian cancer
DK3731771T3 (da) 2017-12-26 2023-08-28 Galvanize Therapeutics Inc V462033dk00
SG11202103917VA (en) 2018-10-16 2021-05-28 Blueallele Llc Methods for targeted insertion of dna in genes
CN118203401A (zh) 2019-04-18 2024-06-18 盖能适治疗股份有限公司 用于治疗异常组织的装置、系统和方法
KR20220128607A (ko) 2019-09-18 2022-09-21 인터갈락틱 테라퓨틱스, 인크. 합성 dna 벡터 및 사용 방법
EP4192572A4 (en) 2020-08-04 2024-08-21 Galvanize Therapeutics, Inc. TRANSFER OF MOLECULES BY PULSED ELECTRIC FIELD TO CELLS IN THE ORGANISM
CN116829691A (zh) 2020-08-04 2023-09-29 盖能适治疗股份有限公司 分子到体内细胞的脉冲电场转移
EP4298214A4 (en) * 2021-02-23 2025-03-19 Aldevron, LLC NUCLEIC ACID VECTORS AND METHODS OF USE
WO2023064934A1 (en) * 2021-10-15 2023-04-20 Intergalactic Therapeutics, Inc. Respiratory delivery of therapeutic agents
EP4522758A4 (en) * 2022-05-13 2026-05-06 Ascidian Therapeutics Inc Abca4 trans-splicing molecules
CA3262218A1 (en) 2022-07-19 2024-01-25 Rampart Bioscience, Inc. CIRCULAR NON-IMMUNOGENEOUS NON-VIRAL DNA VECTORS

Citations (5)

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JPH08504102A (ja) * 1992-12-04 1996-05-07 ユニバーシティー オブ ピッツバーグ 組換えウイルスベクター系
JP2011516049A (ja) * 2008-04-02 2011-05-26 フォンダジオーネ テレソン 遺伝子障害を治療する方法
JP2016518142A (ja) * 2013-05-10 2016-06-23 サンガモ バイオサイエンシーズ， インコーポレイテッド ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法および組成物
WO2016183422A1 (en) * 2015-05-14 2016-11-17 St. Jude Children's Research Hospital, Inc. Nucleic acid molecules containing spacers and methods of use thereof
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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US6825012B2 (en) * 1995-02-23 2004-11-30 Gencell S.A. DNA molecules, preparation and use in gene therapy
RU2001135838A (ru) * 1999-05-28 2004-01-20 Камбрекс Байо Сайенс Инк. (Us) Способы очистки ДНК и очищенная ДНК
EP1224313A1 (en) * 1999-10-07 2002-07-24 University of Iowa Research Foundation Adeno-associated viruses and uses thereof
AU2001288839A1 (en) * 2000-09-08 2002-03-22 The General Hospital Corporation Self-rearranging dna vectors
US20070196838A1 (en) * 2000-12-08 2007-08-23 Invitrogen Corporation Methods and compositions for synthesis of nucleic acid molecules using multiple recognition sites
AU2002247014A1 (en) * 2001-01-19 2002-07-30 Genetica, Inc. Methods and reagents for amplification and manipulation of vector and target nucleic acid sequences
US8236548B2 (en) * 2008-07-03 2012-08-07 The Board Of Trustees Of The Leland Stanford Junior University Minicircle DNA vector preparations and methods of making and using the same
US8945885B2 (en) * 2008-07-03 2015-02-03 The Board Of Trustees Of The Leland Stanford Junior University Minicircle DNA vector preparations and methods of making and using the same
EP2439276A1 (de) * 2010-10-05 2012-04-11 Rentschler Biotechnologie GmbH Verfahren zur semi-synthetischen Herstellung hochreiner "MiniCircle" DNA-Vektoren aus Plasmiden
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
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WO2016120186A1 (en) * 2015-01-27 2016-08-04 F. Hoffmann-La Roche Ag Recombinant hbv cccdna, the method to generate thereof and the use thereof
CA3005474A1 (en) * 2015-11-19 2017-05-26 The Trustees Of The University Of Pennsylvania Compositions and methods for correction of heritable ocular disease
KR20220128607A (ko) * 2019-09-18 2022-09-21 인터갈락틱 테라퓨틱스, 인크. 합성 dna 벡터 및 사용 방법

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH08504102A (ja) * 1992-12-04 1996-05-07 ユニバーシティー オブ ピッツバーグ 組換えウイルスベクター系
JP2011516049A (ja) * 2008-04-02 2011-05-26 フォンダジオーネ テレソン 遺伝子障害を治療する方法
JP2016518142A (ja) * 2013-05-10 2016-06-23 サンガモ バイオサイエンシーズ， インコーポレイテッド ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法および組成物
WO2016183422A1 (en) * 2015-05-14 2016-11-17 St. Jude Children's Research Hospital, Inc. Nucleic acid molecules containing spacers and methods of use thereof
WO2017152149A1 (en) * 2016-03-03 2017-09-08 University Of Massachusetts Closed-ended linear duplex dna for non-viral gene transfer

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024532305A (ja) * 2021-08-24 2024-09-05 賽斯尓▲チン▼生物技術（上海）有限公司 細胞を修飾する方法
JP7834384B2 (ja) 2021-08-24 2026-03-24 賽斯尓▲チン▼生物技術（上海）有限公司 細胞を修飾する方法
JP2025527489A (ja) * 2022-08-16 2025-08-22 アルデブロン，エル．エル．シー． 合成環状核酸を産生する無細胞法

Also Published As

Publication number Publication date
CN117757842A (zh) 2024-03-26
AU2019233901A1 (en) 2020-09-17
CA3092088A1 (en) 2019-09-19
CN111954528A (zh) 2020-11-17
JP2024041819A (ja) 2024-03-27
EP3765023A1 (en) 2021-01-20
EP3765023A4 (en) 2022-01-19
IL276923A (en) 2020-10-29
US20210002667A1 (en) 2021-01-07
WO2019178500A1 (en) 2019-09-19
KR20200132893A (ko) 2020-11-25
US20240247284A1 (en) 2024-07-25
MX2020009579A (es) 2020-11-09

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