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

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

Info

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
Authority
BR
Brazil
Prior art keywords
adenine
nucleotides
poly
dna
consecutive
Prior art date
Application number
BR112020003026-0A
Other languages
English (en)
Portuguese (pt)
Inventor
Christian Dombrowski
Original Assignee
Intellia Therapeutics, Inc.
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 Intellia Therapeutics, Inc. filed Critical Intellia Therapeutics, Inc.
Publication of BR112020003026A2 publication Critical patent/BR112020003026A2/pt

Links

Classifications

    • 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

Landscapes

  • 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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (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 (https=)
EP (1) EP3668980A1 (https=)
JP (2) JP2020532961A (https=)
KR (2) KR20250004924A (https=)
CN (2) CN111212908B (https=)
AU (2) AU2018318135B2 (https=)
BR (1) BR112020003026A2 (https=)
CA (1) CA3072960A1 (https=)
CO (1) CO2020002989A2 (https=)
MX (1) MX2020001821A (https=)
WO (1) WO2019036513A1 (https=)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
AU2015330699B2 (en) 2014-10-10 2021-12-02 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
JP7229923B2 (ja) 2017-01-06 2023-02-28 エディタス・メディシン、インコーポレイテッド ヌクレアーゼ切断を評価する方法
US11499151B2 (en) 2017-04-28 2022-11-15 Editas Medicine, Inc. Methods and systems for analyzing guide RNA molecules
MX2019014640A (es) 2017-06-09 2020-10-05 Editas Medicine Inc Nucleasas de repeticiones palíndromicas agrupadas y regularmente interespaciadas de proteina asociada 9 (cas9) genomanipuladas.
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
CA3072960A1 (en) * 2017-08-15 2019-02-21 Intellia Therapeutics, Inc. Stabilized nucleic acids encoding messenger ribonucleic acid (mrna)
KR102832035B1 (ko) * 2017-09-29 2025-07-09 인텔리아 테라퓨틱스, 인크. 지질 나노파티클을 이용한 mRNA 전달의 체외 방법
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
US12521451B2 (en) 2019-11-08 2026-01-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy
EP4389901A4 (en) * 2021-08-17 2025-08-06 Sk Bioscience Co Ltd METHOD FOR STABLY MAINTAINING A POLY TAIL (A)
TW202325850A (zh) 2021-11-29 2023-07-01 大陸商上海瑞宏迪醫藥有限公司 Aadc、gdnf多核苷酸及其用於治療帕金森病
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
WO2024179544A1 (zh) * 2023-03-01 2024-09-06 仁景(苏州)生物科技有限公司 编码rna的工程化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
WO2026006390A2 (en) 2024-06-26 2026-01-02 Intellia Therapeutics, Inc. Modified guide rnas for genome editing
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

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
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
CA3072960A1 (en) * 2017-08-15 2019-02-21 Intellia Therapeutics, Inc. Stabilized nucleic acids encoding messenger ribonucleic acid (mrna)

Also Published As

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

Similar Documents

Publication Publication Date Title
US12391722B2 (en) Stabilized nucleic acids encoding messenger ribonucleic acid (mRNA)
JP7618577B2 (ja) 修飾環状ポリリボヌクレオチドを含む組成物及びその使用
KR102950624B1 (ko) 원형 폴리리보뉴클레오티드를 포함하는 조성물 및 이의 용도
KR102728416B1 (ko) A형 혈우병을 위한 유전자 편집용 조성물 및 방법
JP2023529316A (ja) ゲノム編集のための組成物及び方法
EP3935162A1 (en) Circular polyribonucleotides and pharmaceutical compositions thereof
CN118256490A (zh) 一种环状rna、组合物和治疗疾病的方法
CN110023494A (zh) Rna指导的核酸修饰酶及其使用方法
WO2021155171A1 (en) Delivery of compositions comprising circular polyribonucleotides
WO2016138278A2 (en) Compositions for inhibiting checkpoint gene expression and uses thereof
JP2021504343A (ja) 尿素サイクル異常症の治療のためのオルニチントランスカルバミラーゼをコードするポリヌクレオチド
KR20250035055A (ko) Pcsk9의 표적화를 위한 조성물 및 방법
TW201219569A (en) Treatment of Sialidase 4 (NEU4) related diseases by inhibition of natural antisense transcript to NEU4
US20250223588A1 (en) Oligonucleotides with 2'-deoxy-2'-f-2'-c-methyl nucleotides
JP7560616B2 (ja) 膜貫通型セリンプロテアーゼ6(tmprss6)発現を阻害するための組成物および方法
JP2024539089A (ja) 修飾複製可能rnaの機能および関連する組成物を増加させるための変異を決定する方法ならびにそれらの使用
WO2013056670A1 (zh) 小干扰RNA及其应用和抑制plk1基因表达的方法
US20200362382A1 (en) Methods of preparing modified rna
CN117597446A (zh) 非自然发生的5’-非转译区及3’-非转译区及其用途
HK40029231B (zh) 编码信使核糖核酸(mRNA)的稳定核酸
HK40029231A (en) Stabilized nucleic acids encoding messenger ribonucleic acid (mrna)
HK40012328A (en) Rna-guided nucleic acid modifying enzymes and methods of use thereof

Legal Events

Date Code Title Description
B350 Update of information on the portal [chapter 15.35 patent gazette]
B06W Patent application suspended after preliminary examination (for patents with searches from other patent authorities) chapter 6.23 patent gazette]
B11B Dismissal acc. art. 36, par 1 of ipl - no reply within 90 days to fullfil the necessary requirements