JP2023511992A - 修飾ヌクレオチドを用いた閉じた直鎖ｄｎａ - Google Patents

修飾ヌクレオチドを用いた閉じた直鎖ｄｎａ Download PDF

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JP2023511992A
JP2023511992A JP2022545423A JP2022545423A JP2023511992A JP 2023511992 A JP2023511992 A JP 2023511992A JP 2022545423 A JP2022545423 A JP 2022545423A JP 2022545423 A JP2022545423 A JP 2022545423A JP 2023511992 A JP2023511992 A JP 2023511992A
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dna
cldna
nucleotides
interest
sequence
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JP2023511992A5 (https=
JPWO2021152147A5 (https=
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サンタマリナ，フレン オヤルサバル
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Tyris Therapeutics sL
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Tyris Therapeutics sL
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P19/00—Preparation of compounds containing saccharide radicals
    • C12P19/26—Preparation of nitrogen-containing carbohydrates
    • C12P19/28—N-glycosides
    • C12P19/30—Nucleotides
    • C12P19/34—Polynucleotides, e.g. nucleic acids, oligoribonucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6844—Nucleic acid amplification reactions
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/532—Closed or circular

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Analytical Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
JP2022545423A 2020-01-31 2021-01-29 修飾ヌクレオチドを用いた閉じた直鎖ｄｎａ Pending JP2023511992A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025137441A JP2025186257A (ja) 2020-01-31 2025-08-21 修飾ヌクレオチドを用いた閉じた直鎖ｄｎａ

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP20382063 2020-01-31
EP20382063.4 2020-01-31
PCT/EP2021/052204 WO2021152147A1 (en) 2020-01-31 2021-01-29 Closed linear dna with modified nucleotides

Related Child Applications (1)

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JP2025137441A Division JP2025186257A (ja) 2020-01-31 2025-08-21 修飾ヌクレオチドを用いた閉じた直鎖ｄｎａ

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JP2023511992A true JP2023511992A (ja) 2023-03-23
JP2023511992A5 JP2023511992A5 (https=) 2024-02-05
JPWO2021152147A5 JPWO2021152147A5 (https=) 2024-02-05

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JP2022545423A Pending JP2023511992A (ja) 2020-01-31 2021-01-29 修飾ヌクレオチドを用いた閉じた直鎖ｄｎａ
JP2025137441A Pending JP2025186257A (ja) 2020-01-31 2025-08-21 修飾ヌクレオチドを用いた閉じた直鎖ｄｎａ

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Country Link
US (1) US20230323343A1 (https=)
EP (1) EP4097253A1 (https=)
JP (2) JP2023511992A (https=)
KR (1) KR20220133999A (https=)
CN (1) CN115003829A (https=)
AU (1) AU2021213927A1 (https=)
CA (1) CA3164390A1 (https=)
WO (1) WO2021152147A1 (https=)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4083227A1 (en) * 2021-04-29 2022-11-02 4basebio, S.L.U. Linear dna with enhanced resistance against exonucleases
EP4124660A1 (en) * 2021-07-30 2023-02-01 4basebio, S.L.U. Linear dna with enhanced resistance against exonucleases and methods for the production thereof
EP4388118A4 (en) * 2021-08-19 2025-05-21 Twist Bioscience Corporation METHODS AND COMPOSITIONS IN COMPOSITION WITH COVALENTLY CLOSED NUCLEIC ACIDS
CN114075595B (zh) * 2021-11-22 2023-11-17 上海交通大学 甲基化检测组合物、试剂盒及方法
JP2025507359A (ja) * 2022-02-10 2025-03-18 コード バイオセラピューティクス インコーポレイテッド 増強されたｄｎａデンドリマー及びその使用方法
JP2025516638A (ja) * 2022-05-13 2025-05-30 フラッグシップ パイオニアリング イノベーションズ セブン，エルエルシー 二本鎖ｄｎａ組成物及び関連する方法
CN115976138A (zh) * 2022-06-02 2023-04-18 宜明(苏州)细胞生物科技有限公司 新型闭合线性双链dna的体外制备技术
WO2024039652A1 (en) 2022-08-16 2024-02-22 Aldevron, L.L.C. Cell-free method of producing synthetic circular nucleic acid
ES3062031T3 (https=) * 2023-02-01 2026-04-08 4Basebio Uk Ltd
EP4410995A1 (en) * 2023-02-01 2024-08-07 4basebio, S.L.U. Linear dna with enhanced resistance against exonucleases and methods for the production thereof
WO2025034978A1 (en) * 2023-08-09 2025-02-13 Code Biotherapeutics, Inc. Enhanced dna dendrimers and methods of use thereof
WO2025064396A1 (en) * 2023-09-18 2025-03-27 Intellia Therapeutics, Inc. Nuclease resistant double stranded dna product for non-viral delivery to a cell and methods of production thereof
WO2025096807A2 (en) * 2023-10-31 2025-05-08 Flagship Pioneering Innovations Vii, Llc Novel therapeutic dna forms
WO2025202929A1 (en) * 2024-03-29 2025-10-02 Pfizer Inc. Methods for producing nucleic acids
EP4663200A1 (en) * 2024-06-10 2025-12-17 CPTx GmbH Compositions and methods
WO2026012450A1 (zh) * 2024-07-11 2026-01-15 石药集团中奇制药技术（石家庄）有限公司 一种非病毒载体介导的大片段dna定点敲入系统

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005014810A1 (ja) * 2003-08-08 2005-02-17 National Institute Of Advanced Industrial Science And Technology ダンベル型ｄｎａの効率的な製造方法
US20070049546A1 (en) * 2004-02-20 2007-03-01 Bernadette Brzezicha Substituted, non-coding nucleic acid molecule for therapeutic and prophylactic stimulation of the immune system in humans and higher animals
JP2016526373A (ja) * 2014-02-18 2016-09-05 モロゲン・アーゲー 非コード免疫調節性共有結合閉鎖ｄｎａ構築物
JP2017512071A (ja) * 2014-04-11 2017-05-18 レッドボールト バイオサイエンシズ エルピー ゲノム適用および治療適用のための、核酸分子のクローン複製および増幅のためのシステムおよび方法
JP2017530714A (ja) * 2014-10-14 2017-10-19 オックスフォード ナノポール テクノロジーズ リミテッド 方法
WO2018213460A1 (en) * 2017-05-16 2018-11-22 Helix Nanotechnologies, Inc. Linear covalently closed vectors and related compositions and methods thereof

Family Cites Families (5)

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US4373071A (en) 1981-04-30 1983-02-08 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
WO2006085664A1 (ja) 2005-02-10 2006-08-17 The University Of Tokyo ポリカチオン荷電性ポリマー及び核酸のキャリヤーとしての使用
ES2359058B1 (es) 2009-07-02 2012-03-27 Consejo Superior De Investigaciones Cient�?Ficas (Csic) Quimera de adn polimerasa del fago ph1 29.
DK3500682T3 (da) * 2016-08-16 2020-08-24 Touchlight Ip Ltd Fremstilling af lukket, lineært dna
EP3714064B8 (en) * 2017-11-21 2024-10-30 4basebio UK Ltd Methods and kits for amplification of double stranded dna

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005014810A1 (ja) * 2003-08-08 2005-02-17 National Institute Of Advanced Industrial Science And Technology ダンベル型ｄｎａの効率的な製造方法
US20070049546A1 (en) * 2004-02-20 2007-03-01 Bernadette Brzezicha Substituted, non-coding nucleic acid molecule for therapeutic and prophylactic stimulation of the immune system in humans and higher animals
JP2016526373A (ja) * 2014-02-18 2016-09-05 モロゲン・アーゲー 非コード免疫調節性共有結合閉鎖ｄｎａ構築物
JP2017512071A (ja) * 2014-04-11 2017-05-18 レッドボールト バイオサイエンシズ エルピー ゲノム適用および治療適用のための、核酸分子のクローン複製および増幅のためのシステムおよび方法
JP2017530714A (ja) * 2014-10-14 2017-10-19 オックスフォード ナノポール テクノロジーズ リミテッド 方法
WO2018213460A1 (en) * 2017-05-16 2018-11-22 Helix Nanotechnologies, Inc. Linear covalently closed vectors and related compositions and methods thereof

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Publication number Publication date
CA3164390A1 (en) 2021-08-05
JP2025186257A (ja) 2025-12-23
KR20220133999A (ko) 2022-10-05
AU2021213927A1 (en) 2022-07-28
EP4097253A1 (en) 2022-12-07
WO2021152147A1 (en) 2021-08-05
CN115003829A (zh) 2022-09-02
US20230323343A1 (en) 2023-10-12

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