KR20220133999A - 변형된 뉴클레오티드들을 갖는 닫힌 선형 dna - Google Patents

변형된 뉴클레오티드들을 갖는 닫힌 선형 dna Download PDF

Info

Publication number
KR20220133999A
KR20220133999A KR1020227030065A KR20227030065A KR20220133999A KR 20220133999 A KR20220133999 A KR 20220133999A KR 1020227030065 A KR1020227030065 A KR 1020227030065A KR 20227030065 A KR20227030065 A KR 20227030065A KR 20220133999 A KR20220133999 A KR 20220133999A
Authority
KR
South Korea
Prior art keywords
dna
cldna
nucleotides
sequence
interest
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
KR1020227030065A
Other languages
English (en)
Korean (ko)
Inventor
줄렌 오야르자발 산타마리나
Original Assignee
타이리스 테라뷰틱스, 에스.엘.
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 타이리스 테라뷰틱스, 에스.엘. filed Critical 타이리스 테라뷰틱스, 에스.엘.
Publication of KR20220133999A publication Critical patent/KR20220133999A/ko
Pending legal-status Critical Current

Links

Images

Classifications

    • 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

Landscapes

  • 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)
KR1020227030065A 2020-01-31 2021-01-29 변형된 뉴클레오티드들을 갖는 닫힌 선형 dna Pending KR20220133999A (ko)

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
KR20220133999A true KR20220133999A (ko) 2022-10-05

Family

ID=69713977

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020227030065A Pending KR20220133999A (ko) 2020-01-31 2021-01-29 변형된 뉴클레오티드들을 갖는 닫힌 선형 dna

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
DK3500682T3 (da) * 2016-08-16 2020-08-24 Touchlight Ip Ltd Fremstilling af lukket, lineært dna
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
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

Similar Documents

Publication Publication Date Title
KR20220133999A (ko) 변형된 뉴클레오티드들을 갖는 닫힌 선형 dna
KR20220134001A (ko) 닫힌 선형 dna의 제조방법
US10940171B2 (en) Microbial production of pure single stranded nucleic acids
CN104059942B (zh) 新城疫病毒耐热活疫苗载体系统及其应用
US5928914A (en) Methods and compositions for transforming cells
KR20210105382A (ko) 단백질을 코딩하는 rna
KR20230054840A (ko) rAAV 비리온의 유도 생산을 위한 안정화된 세포주
CN115968300A (zh) 用于体内转导的载体和方法
CN112501269B (zh) 一种快速鉴定高亲和力tcr抗原交叉反应活性的方法
JP3304932B2 (ja) 共有結合で閉鎖された環状ｄｎａを精製するための新規な方法
CN101688231B (zh) 由茶碱调控其靶特异性核糖核酸的取代活性的变构反式剪接i型核酶
JP2023011736A (ja) 核酸封入ａａｖ中空粒子
US20240360472A1 (en) Polynucleotides and uses thereof
US6534314B1 (en) Methods and compositions for transforming cells
KR20230169221A (ko) 비-바이러스 상동성 매개 말단 접합
KR20220018410A (ko) 세포질에서 자가전사가 가능하고 유전체 편집을 제공하는 rna/dna 시스템
KR20150021839A (ko) 암특이적 유전자를 표적하는 트랜스-스플라이싱 라이보자임의 조절 유도체를 포함하는 재조합 아데노바이러스 및 이의 용도
RU2686102C1 (ru) Рекомбинантный вектор для создания плазмидных генетических конструкций, обладающих повышенной длительностью экспрессии целевых генов
RU2846879C1 (ru) МОДИФИЦИРОВАННАЯ БАКУЛОВИРУСНАЯ СИСТЕМА ДЛЯ УЛУЧШЕННОГО ПОЛУЧЕНИЯ ДНК С ЗАМКНУТЫМИ КОНЦАМИ (ceDNA)
CN100410382C (zh) 一种可诱生细胞免疫应答的共表达载体和真核表达载体
CN111850018B (zh) 多MHC基因型和抗原MiniGene组合文库及其构建方法和应用
CN101311270A (zh) 一种可诱生细胞免疫应答的真核表达载体及其疫苗
KR20240023100A (ko) 유전자 발현을 조절하기 위한 조성물 및 방법
US20130184332A1 (en) Vectors Containing the Max Gene
KR20220067468A (ko) 세포질에서 자가 전사가 가능하고 발현억제 rna를 제공하는 rna/dna 시스템

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

R18-X000 Changes to party contact information recorded

St.27 status event code: A-3-3-R10-R18-oth-X000

D21 Rejection of application intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D21-EXM-PE0902 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

T11 Administrative time limit extension requested

Free format text: ST27 STATUS EVENT CODE: U-3-3-T10-T11-OTH-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000