AU2018318135B2 - Stabilized nucleic acids encoding messenger ribonucleic acid (mRNA) - Google Patents

Stabilized nucleic acids encoding messenger ribonucleic acid (mRNA) Download PDF

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Publication number
AU2018318135B2
AU2018318135B2 AU2018318135A AU2018318135A AU2018318135B2 AU 2018318135 B2 AU2018318135 B2 AU 2018318135B2 AU 2018318135 A AU2018318135 A AU 2018318135A AU 2018318135 A AU2018318135 A AU 2018318135A AU 2018318135 B2 AU2018318135 B2 AU 2018318135B2
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poly
nucleotides
adenine
dna
tail
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AU2018318135A1 (en
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Christian Dombrowski
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Intellia Therapeutics Inc
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Intellia Therapeutics Inc
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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
    • 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
    • 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)
  • 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)
AU2018318135A 2017-08-15 2018-08-14 Stabilized nucleic acids encoding messenger ribonucleic acid (mRNA) Active AU2018318135B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2024278094A AU2024278094A1 (en) 2017-08-15 2024-12-04 Stabilized nucleic acids encoding messenger ribonucleic acid (mRNA)

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)

Related Child Applications (1)

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AU2024278094A Division AU2024278094A1 (en) 2017-08-15 2024-12-04 Stabilized nucleic acids encoding messenger ribonucleic acid (mRNA)

Publications (2)

Publication Number Publication Date
AU2018318135A1 AU2018318135A1 (en) 2020-03-05
AU2018318135B2 true AU2018318135B2 (en) 2024-09-12

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AU2018318135A Active AU2018318135B2 (en) 2017-08-15 2018-08-14 Stabilized nucleic acids encoding messenger ribonucleic acid (mRNA)
AU2024278094A Pending AU2024278094A1 (en) 2017-08-15 2024-12-04 Stabilized nucleic acids encoding messenger ribonucleic acid (mRNA)

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Country Status (11)

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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)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
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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
US20240344019A1 (en) * 2021-08-17 2024-10-17 Sk Bioscience Co., Ltd. Method for stably maintaining poly(a) tail
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

Family Cites Families (11)

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Publication number Priority date Publication date Assignee Title
JPH08131163A (ja) * 1994-11-11 1996-05-28 Sankyo Co Ltd ドメイン変換ヘアピン型リボザイム
JP2002360299A (ja) * 2001-04-20 2002-12-17 Seiwa Technics:Kk ｍＲＮＡの不安定性を利用したサイトカインの３’非翻訳領域を組み込んだトランスジェニックマウスのガン、炎症などの病変部位検出方法
WO2012019630A1 (en) * 2010-08-13 2012-02-16 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded protein
WO2013120500A1 (en) * 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded tumour antigen
WO2016005004A1 (en) * 2014-07-11 2016-01-14 Biontech Rna Pharmaceuticals Gmbh Stabilization of poly(a) sequence encoding dna sequences
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
MA41349A (fr) * 2015-01-14 2017-11-21 Univ Temple Éradication de l'herpès simplex de type i et d'autres virus de l'herpès associés guidée par arn
EP3085714B1 (en) * 2015-04-21 2018-03-14 Rohm And Haas Company Co2 abating latex coating composition
WO2017059902A1 (en) * 2015-10-07 2017-04-13 Biontech Rna Pharmaceuticals Gmbh 3' utr sequences for stabilization of rna
KR101786396B1 (ko) * 2016-10-27 2017-11-24 서울대학교산학협력단 전령 rna의 3′말단 서열 분석 방법
AU2018318135B2 (en) * 2017-08-15 2024-09-12 Intellia Therapeutics, Inc. Stabilized nucleic acids encoding messenger ribonucleic acid (mRNA)

Also Published As

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
JP2020532961A (ja) 2020-11-19
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

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