AU2021213927A1 - Closed linear DNA with modified nucleotides - Google Patents

Closed linear DNA with modified nucleotides Download PDF

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Publication number
AU2021213927A1
AU2021213927A1 AU2021213927A AU2021213927A AU2021213927A1 AU 2021213927 A1 AU2021213927 A1 AU 2021213927A1 AU 2021213927 A AU2021213927 A AU 2021213927A AU 2021213927 A AU2021213927 A AU 2021213927A AU 2021213927 A1 AU2021213927 A1 AU 2021213927A1
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AU
Australia
Prior art keywords
dna
cldna
nucleotide
interest
sequence
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Pending
Application number
AU2021213927A
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English (en)
Inventor
Santamarina Julen Oyarzabal
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.)
Tyris Therapeutics SL
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Tyris Therapeutics S L
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Publication date
Application filed by Tyris Therapeutics S L filed Critical Tyris Therapeutics S L
Publication of AU2021213927A1 publication Critical patent/AU2021213927A1/en
Pending legal-status Critical Current

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    • 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
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • C12N15/10—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
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • 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
    • C12N2310/30—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
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C12N2310/531—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)
AU2021213927A 2020-01-31 2021-01-29 Closed linear DNA with modified nucleotides Pending AU2021213927A1 (en)

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

Publications (1)

Publication Number Publication Date
AU2021213927A1 true AU2021213927A1 (en) 2022-07-28

Family

ID=69713977

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021213927A Pending AU2021213927A1 (en) 2020-01-31 2021-01-29 Closed linear DNA with modified nucleotides

Country Status (8)

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

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4373071A (en) 1981-04-30 1983-02-08 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
JP4517061B2 (ja) * 2003-08-08 2010-08-04 独立行政法人産業技術総合研究所 ダンベル型ｄｎａの効率的な製造方法
CN1918293A (zh) * 2004-02-20 2007-02-21 莫洛根股份公司 用于对人及高等动物进行治疗性和预防性免疫刺激的取代的非编码核酸分子
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.
GB2523187A (en) * 2014-02-18 2015-08-19 Mologen Ag Covalently closed non-coding immunomodulatory DNA construct
US20170067097A1 (en) * 2014-04-11 2017-03-09 Redvault Biosciences, Lp Systems and methods for clonal replication and amplification of nucleic acid molecules for genomic and therapeutic applications
GB201418159D0 (en) * 2014-10-14 2014-11-26 Oxford Nanopore Tech Ltd Method
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US11473090B2 (en) * 2017-05-16 2022-10-18 Helix Nanotechnologies, Inc. Linear covalently closed vectors and related compositions and methods thereof
EP3714064B8 (en) * 2017-11-21 2024-10-30 4basebio UK Ltd Methods and kits for amplification of double stranded dna

Also Published As

Publication number Publication date
CA3164390A1 (en) 2021-08-05
JP2025186257A (ja) 2025-12-23
JP2023511992A (ja) 2023-03-23
KR20220133999A (ko) 2022-10-05
EP4097253A1 (en) 2022-12-07
WO2021152147A1 (en) 2021-08-05
CN115003829A (zh) 2022-09-02
US20230323343A1 (en) 2023-10-12

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