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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    • 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
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • 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
    • C12N15/67—General methods for enhancing the expression
    • 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
    • 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]

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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)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (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)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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)
  • Crystallography & Structural Chemistry (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (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)

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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)

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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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JP2020532961A5 JP2020532961A5 (enExample) 2021-09-30

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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)
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Cited By (2)

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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
JP2025527792A (ja) * 2022-08-26 2025-08-22 シェンチェン、リージェン、バイオテクノロジー、カンパニー、リミテッド 宿主細胞に安定して存在するマルチセグメント化ＰｏｌｙＡポリヌクレオチドおよびその使用

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ES3015369T3 (en) * 2014-10-09 2025-05-05 Seattle Childrens Hospital Dba Seattle Childrens Res Inst Long poly (a) plasmids and methods for introduction of long poly (a) sequences into the plasmid
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
EP3555297A1 (en) 2016-12-19 2019-10-23 Editas Medicine, Inc. Assessing nuclease cleavage
WO2018129368A2 (en) 2017-01-06 2018-07-12 Editas Medicine, Inc. Methods of assessing nuclease cleavage
US11499151B2 (en) 2017-04-28 2022-11-15 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
JP2020532961A (ja) * 2017-08-15 2020-11-19 インテリア セラピューティクス，インコーポレイテッド 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
CA3077391A1 (en) * 2017-09-29 2019-04-04 Intellia Therapeutics, Inc. In vitro method of mrna delivery using lipid nanoparticles
AU2019291918B2 (en) 2018-06-29 2025-06-12 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
US12521451B2 (en) 2019-11-08 2026-01-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy
CA3238867A1 (en) 2021-11-29 2023-06-01 Shanghai Regenelead Therapies Co., Ltd. Aadc/gdnf polynucleotide, and use thereof in treating parkinson's disease
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
US20250388946A1 (en) * 2022-05-31 2025-12-25 Industry-Academic Cooperation Foundation Gyeongsang National University Novel modified polyadenine sequence and use thereof
JP2026507667A (ja) * 2023-03-01 2026-03-04 仁景（▲蘇▼州）生物科技有限公司 Ｒｎａをコードするための工学的に操作されたｄｎａ分子
TW202508610A (zh) 2023-08-23 2025-03-01 大陸商上海瑞宏迪醫藥有限公司 醫藥組成物及其用途
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
TW202617987A (zh) 2024-06-26 2026-05-01 美商英特利亞醫療公司 用於基因體編輯之經修飾之嚮導ｒｎａ
CN121628926A (zh) * 2024-08-29 2026-03-10 仁景(苏州)生物科技有限公司 一种多价流感mRNA疫苗

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Cited By (3)

* Cited by examiner, † Cited by third party
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
JP2025527792A (ja) * 2022-08-26 2025-08-22 シェンチェン、リージェン、バイオテクノロジー、カンパニー、リミテッド 宿主細胞に安定して存在するマルチセグメント化ＰｏｌｙＡポリヌクレオチドおよびその使用

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

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