JP2020532961A - メッセンジャーリボ核酸（ｍＲＮＡ）をコードする安定化された核酸 - Google Patents

メッセンジャーリボ核酸（ｍＲＮＡ）をコードする安定化された核酸 Download PDF

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JP2020532961A
JP2020532961A JP2020508506A JP2020508506A JP2020532961A JP 2020532961 A JP2020532961 A JP 2020532961A JP 2020508506 A JP2020508506 A JP 2020508506A JP 2020508506 A JP2020508506 A JP 2020508506A JP 2020532961 A JP2020532961 A JP 2020532961A
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adenine
nucleotides
poly
dna
adenine nucleotides
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JP2020532961A5 (enExample
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ドンブロウスキー，クリスチャン
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インテリア セラピューティクス，インコーポレイテッド
インテリア 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 JP2020532961A publication Critical patent/JP2020532961A/ja
Publication of JP2020532961A5 publication Critical patent/JP2020532961A5/ja
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • 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
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/521—Chemokines
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2020508506A 2017-08-15 2018-08-14 メッセンジャーリボ核酸（ｍＲＮＡ）をコードする安定化された核酸 Pending JP2020532961A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023137160A JP2023179428A (ja) 2017-08-15 2023-08-25 メッセンジャーリボ核酸（ｍＲＮＡ）をコードする安定化された核酸

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762545883P 2017-08-15 2017-08-15
US62/545,883 2017-08-15
PCT/US2018/046772 WO2019036513A1 (en) 2017-08-15 2018-08-14 STABILIZED NUCLEIC ACIDS ENCODING MESSENGER RIBONUCLEIC ACID (MRNA)

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JP2023137160A Division JP2023179428A (ja) 2017-08-15 2023-08-25 メッセンジャーリボ核酸（ｍＲＮＡ）をコードする安定化された核酸

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JP2020532961A true JP2020532961A (ja) 2020-11-19
JP2020532961A5 JP2020532961A5 (enExample) 2021-09-30

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JP2020508506A Pending JP2020532961A (ja) 2017-08-15 2018-08-14 メッセンジャーリボ核酸（ｍＲＮＡ）をコードする安定化された核酸
JP2023137160A Pending JP2023179428A (ja) 2017-08-15 2023-08-25 メッセンジャーリボ核酸（ｍＲＮＡ）をコードする安定化された核酸

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US (3) US11673911B2 (enExample)
EP (1) EP3668980A1 (enExample)
JP (2) JP2020532961A (enExample)
KR (2) KR20250004924A (enExample)
CN (2) CN111212908B (enExample)
AU (2) AU2018318135B2 (enExample)
BR (1) BR112020003026A2 (enExample)
CA (1) CA3072960A1 (enExample)
CO (1) CO2020002989A2 (enExample)
MX (1) MX2020001821A (enExample)
WO (1) WO2019036513A1 (enExample)

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JP2024528297A (ja) * 2021-08-17 2024-07-26 エスケー バイオサイエンス カンパニー リミテッド ポリａテールを安定して保持する方法

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EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
US12286727B2 (en) 2016-12-19 2025-04-29 Editas Medicine, Inc. Assessing nuclease cleavage
EP3565907B1 (en) 2017-01-06 2022-05-04 Editas Medicine, Inc. Methods of assessing nuclease cleavage
EP3615672A1 (en) 2017-04-28 2020-03-04 Editas Medicine, Inc. Methods and systems for analyzing guide rna molecules
CN110997908A (zh) 2017-06-09 2020-04-10 爱迪塔斯医药公司 工程化的cas9核酸酶
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
AU2018318135B2 (en) * 2017-08-15 2024-09-12 Intellia Therapeutics, Inc. Stabilized nucleic acids encoding messenger ribonucleic acid (mRNA)
KR20250107976A (ko) * 2017-09-29 2025-07-14 인텔리아 테라퓨틱스, 인크. 지질 나노파티클을 이용한 mRNA 전달의 체외 방법
CA3104856A1 (en) 2018-06-29 2020-01-02 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
WO2023093905A1 (zh) 2021-11-29 2023-06-01 上海瑞宏迪医药有限公司 Aadc、gdnf多核苷酸及其用于治疗帕金森病
WO2023155918A1 (zh) 2022-02-21 2023-08-24 上海瑞宏迪医药有限公司 Vegf结合分子及其医药用途
CN117701602A (zh) * 2022-08-26 2024-03-15 深圳瑞吉生物科技有限公司 在宿主细胞中稳定存在的多段化PolyA多核苷酸及其应用
CN118581123A (zh) * 2023-03-01 2024-09-03 仁景(苏州)生物科技有限公司 编码rna的工程化dna分子
CN119799695A (zh) * 2023-10-10 2025-04-11 深圳瑞吉生物科技有限公司 提高mRNA稳定性以及蛋白表达的多元polyA多核苷酸及其应用
PL446809A1 (pl) * 2023-11-22 2025-05-26 Sieć Badawcza Łukasiewicz - PORT Polski Ośrodek Rozwoju Technologii Cząsteczki RNA ze zmodyfikowanymi sekwencjami końca 3' i ich zastosowanie
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
GB202406051D0 (en) 2024-04-30 2024-06-12 Univ Oxford Innovation Ltd Segmented poly(a) tails

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Publication number Priority date Publication date Assignee Title
JP2024528297A (ja) * 2021-08-17 2024-07-26 エスケー バイオサイエンス カンパニー リミテッド ポリａテールを安定して保持する方法
JP7716573B2 (ja) 2021-08-17 2025-07-31 エスケー バイオサイエンス カンパニー リミテッド ポリａテールを安定して保持する方法

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Publication number Publication date
MX2020001821A (es) 2020-09-18
KR102740888B1 (ko) 2024-12-13
CA3072960A1 (en) 2019-02-21
JP2023179428A (ja) 2023-12-19
US11673911B2 (en) 2023-06-13
US20200172564A1 (en) 2020-06-04
CO2020002989A2 (es) 2020-05-05
US20250346625A1 (en) 2025-11-13
EP3668980A1 (en) 2020-06-24
US20230271997A1 (en) 2023-08-31
CN111212908A (zh) 2020-05-29
US12391722B2 (en) 2025-08-19
WO2019036513A1 (en) 2019-02-21
CN111212908B (zh) 2025-01-24
AU2018318135A1 (en) 2020-03-05
CN119899831A (zh) 2025-04-29
BR112020003026A2 (pt) 2020-08-25
KR20200036025A (ko) 2020-04-06
KR20250004924A (ko) 2025-01-08
AU2024278094A1 (en) 2024-12-19
AU2018318135B2 (en) 2024-09-12

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