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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JP2021516953A5 (enExample
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
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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
Priority to JP2026050925A priority patent/JP2026102885A/ja
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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)
  • Zoology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (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 (2)

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
JP2026050925A JP2026102885A (ja) 2018-03-15 2026-03-25 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 (enExample) 2022-03-23

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Family Applications (3)

Application Number Title Priority Date Filing Date
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
JP2026050925A Pending JP2026102885A (ja) 2018-03-15 2026-03-25 合成ｄｎａベクターおよびその使用

Family Applications After (2)

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JP2023222352A Pending JP2024041819A (ja) 2018-03-15 2023-12-28 合成ｄｎａベクターおよびその使用
JP2026050925A Pending JP2026102885A (ja) 2018-03-15 2026-03-25 合成ｄｎａベクターおよびその使用

Country Status (10)

Country Link
US (2) US20210002667A1 (enExample)
EP (1) EP3765023A4 (enExample)
JP (3) JP2021516953A (enExample)
KR (1) KR20200132893A (enExample)
CN (2) CN117757842A (enExample)
AU (1) AU2019233901A1 (enExample)
CA (1) CA3092088A1 (enExample)
IL (1) IL276923A (enExample)
MX (1) MX2020009579A (enExample)
WO (1) WO2019178500A1 (enExample)

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

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US12403305B2 (en) 2016-06-27 2025-09-02 Galvanize Therapeutics, Inc. Immunostimulation in the treatment of viral infection
DK3474760T3 (da) 2016-06-27 2023-03-20 Galvanize Therapeutics Inc Generator og et kateter med en elektrode til at behandle en lungepassage
US20200048716A1 (en) * 2017-11-03 2020-02-13 Twister Biotech, Inc Using minivectors to treat ovarian cancer
EP4552592A3 (en) 2017-12-26 2025-06-18 Galvanize Therapeutics, Inc. Optimization of energy delivery for various applications
IL326835A (en) * 2018-10-16 2026-04-01 Blueallele Llc Methods for targeted insertion of dna genes
CN114007535A (zh) 2019-04-18 2022-02-01 盖乐世公司 用于治疗异常组织的装置、系统和方法
WO2021055760A1 (en) * 2019-09-18 2021-03-25 Intergalactic Therapeutics, Inc. Synthetic dna vectors and methods of use
CN115348844A (zh) 2020-04-08 2022-11-15 盖能适治疗股份有限公司 脉冲电场波形操作和使用
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
EP4192572A4 (en) 2020-08-04 2024-08-21 Galvanize Therapeutics, Inc. TRANSFER OF MOLECULES BY PULSED ELECTRIC FIELD TO CELLS IN THE ORGANISM
JP2024509084A (ja) * 2021-02-23 2024-02-29 インターギャラクティック セラピューティクス インコーポレイテッド 核酸ベクター及び使用方法
AU2022367527A1 (en) * 2021-10-15 2024-05-02 Aldevron, Llc Respiratory delivery of therapeutic agents
KR20250011939A (ko) * 2022-05-13 2025-01-22 아시디엔 테라퓨틱스, 인크. Abca4 트랜스-스플라이싱 분자
JP2025525583A (ja) 2022-07-19 2025-08-05 ランパート バイオサイエンス インコーポレイテッド 非免疫原性環状非ウイルス性ｄｎａベクター

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
CN1264854C (zh) * 1999-05-28 2006-07-19 坎布雷科斯生物科学巴尔的摩公司 Dna纯化方法和纯化的dna
EP1224313A1 (en) * 1999-10-07 2002-07-24 University of Iowa Research Foundation Adeno-associated viruses and uses thereof
EP1317560A4 (en) * 2000-09-08 2005-10-05 Gen Hospital Corp DNA VECTORS WHICH COULD REENGANIZE
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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
CN102083975A (zh) * 2008-07-03 2011-06-01 利兰·斯坦福青年大学托管委员会 小环dna载体制剂及其制备方法和使用方法
EP2439276A1 (de) * 2010-10-05 2012-04-11 Rentschler Biotechnologie GmbH Verfahren zur semi-synthetischen Herstellung hochreiner "MiniCircle" DNA-Vektoren aus Plasmiden
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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

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
EP3765023A1 (en) 2021-01-20
AU2019233901A1 (en) 2020-09-17
JP2026102885A (ja) 2026-06-23
EP3765023A4 (en) 2022-01-19
US20210002667A1 (en) 2021-01-07
IL276923A (en) 2020-10-29
MX2020009579A (es) 2020-11-09
WO2019178500A1 (en) 2019-09-19
CN111954528A (zh) 2020-11-17
CN117757842A (zh) 2024-03-26
KR20200132893A (ko) 2020-11-25
CA3092088A1 (en) 2019-09-19
JP2024041819A (ja) 2024-03-27
US20240247284A1 (en) 2024-07-25

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