BR112020003026A2 - ácidos nucleicos estabilizados codificando ácido ribonucleico mensageiro (mrna) - Google Patents

ácidos nucleicos estabilizados codificando ácido ribonucleico mensageiro (mrna) Download PDF

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Publication number
BR112020003026A2
BR112020003026A2 BR112020003026-0A BR112020003026A BR112020003026A2 BR 112020003026 A2 BR112020003026 A2 BR 112020003026A2 BR 112020003026 A BR112020003026 A BR 112020003026A BR 112020003026 A2 BR112020003026 A2 BR 112020003026A2
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Brazil
Prior art keywords
adenine
nucleotides
poly
dna
consecutive
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BR112020003026-0A
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English (en)
Portuguese (pt)
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Christian Dombrowski
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Intellia Therapeutics, Inc.
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Publication of BR112020003026A2 publication Critical patent/BR112020003026A2/pt

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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02Compounds 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475Growth factors; Growth regulators
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52Cytokines; Lymphokines; Interferons
    • C07K14/521Chemokines
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector 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)
  • Crystallography & Structural Chemistry (AREA)
  • Saccharide Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
BR112020003026-0A 2017-08-15 2018-08-14 ácidos nucleicos estabilizados codificando ácido ribonucleico mensageiro (mrna) BR112020003026A2 (pt)

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)

Publications (1)

Publication Number Publication Date
BR112020003026A2 true BR112020003026A2 (pt) 2020-08-25

Family

ID=63638316

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112020003026-0A BR112020003026A2 (pt) 2017-08-15 2018-08-14 ácidos nucleicos estabilizados codificando ácido ribonucleico mensageiro (mrna)

Country Status (11)

Country Link
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
Publication number Priority date Publication date Assignee Title
US20180250424A1 (en) 2014-10-10 2018-09-06 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
WO2018119010A1 (en) 2016-12-19 2018-06-28 Editas Medicine, Inc. Assessing nuclease cleavage
US12110545B2 (en) 2017-01-06 2024-10-08 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
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES
MX2020001821A (es) * 2017-08-15 2020-09-18 Intellia Therapeutics Inc Ácidos nucleicos estabilizados que codifican el ácido ribonucleico mensajero (arnm).
KR102832035B1 (ko) * 2017-09-29 2025-07-09 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
WO2023022490A1 (ko) * 2021-08-17 2023-02-23 에스케이바이오사이언스 주식회사 폴리 a 테일을 안정적으로 유지하는 방법
US20250051750A1 (en) 2021-11-29 2025-02-13 Shanghai Regenelead Therapies Co., Ltd. Aadc/gdnf polynucleotide, and use thereof in treating parkinson's disease
AU2023220237A1 (en) 2022-02-21 2024-08-15 Shanghai Regenelead Therapies Co., Ltd Vegf-binding molecule and pharmaceutical use thereof
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
KR20250161542A (ko) * 2023-03-01 2025-11-17 리누아진 바이오테크놀로지 씨오., 엘티디. Rna를 코딩하기 위한 조작된 dna 분자
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)

* Cited by examiner, † Cited by third party
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
US9943612B2 (en) * 2014-10-09 2018-04-17 Seattle Children's Hospital 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
MX2020001821A (es) * 2017-08-15 2020-09-18 Intellia Therapeutics Inc Ácidos nucleicos estabilizados que codifican el ácido ribonucleico mensajero (arnm).

Also Published As

Publication number Publication date
CO2020002989A2 (es) 2020-05-05
WO2019036513A1 (en) 2019-02-21
KR20250004924A (ko) 2025-01-08
US20250346625A1 (en) 2025-11-13
US20200172564A1 (en) 2020-06-04
CN111212908B (zh) 2025-01-24
AU2024278094A1 (en) 2024-12-19
EP3668980A1 (en) 2020-06-24
CA3072960A1 (en) 2019-02-21
JP2020532961A (ja) 2020-11-19
JP2023179428A (ja) 2023-12-19
AU2018318135B2 (en) 2024-09-12
CN119899831A (zh) 2025-04-29
CN111212908A (zh) 2020-05-29
KR20200036025A (ko) 2020-04-06
US12391722B2 (en) 2025-08-19
AU2018318135A1 (en) 2020-03-05
US11673911B2 (en) 2023-06-13
MX2020001821A (es) 2020-09-18
KR102740888B1 (ko) 2024-12-13
US20230271997A1 (en) 2023-08-31

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B06W Patent application suspended after preliminary examination (for patents with searches from other patent authorities) chapter 6.23 patent gazette]