CY1117858T1 - Ολιγονουκλεοτιδια για την προκληση αλλαγης στην αλληλουχια rna μοριου-στοχου παροντος σε ζωντανο κυτταρο - Google Patents

Ολιγονουκλεοτιδια για την προκληση αλλαγης στην αλληλουχια rna μοριου-στοχου παροντος σε ζωντανο κυτταρο

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Publication number
CY1117858T1
CY1117858T1 CY20161100722T CY161100722T CY1117858T1 CY 1117858 T1 CY1117858 T1 CY 1117858T1 CY 20161100722 T CY20161100722 T CY 20161100722T CY 161100722 T CY161100722 T CY 161100722T CY 1117858 T1 CY1117858 T1 CY 1117858T1
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Cyprus
Prior art keywords
change
target rna
oligonucleotides
induction
molecule
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CY20161100722T
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English (en)
Inventor
Boer Daniel Anton De
Tita Ritsema
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Proqr Therapeutics Ii B.V.
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Publication date
Application filed by Proqr Therapeutics Ii B.V. filed Critical Proqr Therapeutics Ii B.V.
Publication of CY1117858T1 publication Critical patent/CY1117858T1/el

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    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • A—HUMAN NECESSITIES
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/08—Solutions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
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    • 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
    • C12N15/01—Preparation of mutants without inserting foreign genetic material therein; Screening processes therefor
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

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  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Saccharide Compounds (AREA)

Abstract

Η παρούσα εφεύρεση σχετίζεται με το πεδίο της γονιδιακής θεραπείας, ειδικότερα με ολιγονουκλεοτίδια για την πρόκληση αλλαγής στην αλληλουχία RNA μορίου-στόχου παρόντος σε ζωντανό κύτταρο.
CY20161100722T 2012-07-12 2016-07-22 Ολιγονουκλεοτιδια για την προκληση αλλαγης στην αλληλουχια rna μοριου-στοχου παροντος σε ζωντανο κυτταρο CY1117858T1 (el)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201261670681P 2012-07-12 2012-07-12
US201261718801P 2012-10-26 2012-10-26
EP13166465 2013-05-03
EP13172515 2013-06-18
EP13173818 2013-06-26
PCT/NL2013/050534 WO2014011053A1 (en) 2012-07-12 2013-07-12 Oligonucleotides for making a change in the sequence of a target rna molecule present in a living cell
EP13760133.2A EP2852668B1 (en) 2012-07-12 2013-07-12 Oligonucleotides for making a change in the sequence of a target rna molecule present in a living cell

Publications (1)

Publication Number Publication Date
CY1117858T1 true CY1117858T1 (el) 2017-05-17

Family

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Family Applications (1)

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CY20161100722T CY1117858T1 (el) 2012-07-12 2016-07-22 Ολιγονουκλεοτιδια για την προκληση αλλαγης στην αλληλουχια rna μοριου-στοχου παροντος σε ζωντανο κυτταρο

Country Status (24)

Country Link
US (2) US9605255B2 (el)
EP (2) EP3103872B1 (el)
JP (1) JP6373833B2 (el)
KR (1) KR20150037968A (el)
CN (1) CN104640986B (el)
AU (1) AU2013287353B2 (el)
BR (1) BR112015000710A8 (el)
CA (1) CA2878934A1 (el)
CY (1) CY1117858T1 (el)
DK (2) DK3103872T3 (el)
ES (2) ES2584856T3 (el)
HK (1) HK1208491A1 (el)
HR (1) HRP20160854T1 (el)
HU (1) HUE029977T2 (el)
IL (1) IL236654B (el)
MX (1) MX356625B (el)
NZ (1) NZ703824A (el)
PL (1) PL2852668T3 (el)
PT (1) PT2852668T (el)
RS (1) RS54944B1 (el)
RU (1) RU2663110C2 (el)
SI (1) SI2852668T1 (el)
SM (1) SMT201600234B (el)
WO (1) WO2014011053A1 (el)

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK3219705T3 (da) 2005-12-28 2020-04-14 Vertex Pharma Farmaceutiske sammensætninger af den amorfe form af n-[2,4-bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinolin-3-carboxamid
US8563573B2 (en) 2007-11-02 2013-10-22 Vertex Pharmaceuticals Incorporated Azaindole derivatives as CFTR modulators
US8802868B2 (en) 2010-03-25 2014-08-12 Vertex Pharmaceuticals Incorporated Solid forms of (R)-1(2,2-difluorobenzo[D][1,3]dioxo1-5-yl)-N-(1-(2,3-dihydroxypropyl-6-fluoro-2-(1-hydroxy-2-methylpropan2-yl)-1H-Indol-5-yl)-Cyclopropanecarboxamide
ES2858351T3 (es) 2010-04-22 2021-09-30 Vertex Pharma Compuesto intermedio para proceso de producción de compuestos de cicloalquilcaraboxamido-indol
EP3103872B1 (en) * 2012-07-12 2018-08-29 ProQR Therapeutics II B.V. Oligonucleotides for making a change in the sequence of a target rna molecule present in a living cell
RU2744460C2 (ru) 2014-04-15 2021-03-09 Вертекс Фармасьютикалз Инкорпорейтед Фармацевтические композиции для лечения заболеваний, опосредованных муковисцидозным трансмембранным регулятором проводимости
US10278991B2 (en) 2014-05-14 2019-05-07 Targimmune Therapeutics Ag Polyethyleneimine polyethyleneglycol vectors
JP6746569B2 (ja) 2014-10-07 2020-08-26 バーテックス ファーマシューティカルズ インコーポレイテッドＶｅｒｔｅｘ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｉｎｃｏｒｐｏｒａｔｅｄ 嚢胞性線維症膜貫通コンダクタンス制御因子のモジュレーターの共結晶
GB201418892D0 (en) * 2014-10-23 2014-12-10 Proqr Therapeutics B V DNA editing
US20210395729A1 (en) * 2014-12-12 2021-12-23 Tod M. Woolf Compositions and methods for Editing Nucleic Acids in Cells Utilizing Oligonucleotides
DK3234134T3 (da) 2014-12-17 2020-07-27 Proqr Therapeutics Ii Bv Målrettet rna-redigering
GB201507926D0 (en) * 2015-05-08 2015-06-24 Proqr Therapeutics N V Improved treatments using oligonucleotides
WO2017060317A1 (en) 2015-10-05 2017-04-13 Proqr Therapeutics Ii B.V. Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion
CN108367021A (zh) 2015-10-15 2018-08-03 希望之城 包含硫代磷酸化寡脱氧核苷酸的化合物和组合物及其使用方法
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
NZ751483A (en) * 2016-09-01 2022-07-01 Proqr Therapeutics Ii Bv Chemically modified single-stranded rna-editing oligonucleotides
US11274300B2 (en) 2017-01-19 2022-03-15 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in RNA editing
EP3589751A4 (en) 2017-03-03 2021-11-17 The Regents of The University of California RNA TARGETING OF MUTATIONS VIA SUPPRESSOR RNA AND DEAMINASES
CN107267516B (zh) * 2017-07-28 2020-09-29 佛山科学技术学院 双sgRNA介导的基因精确修饰方法及应用
EP3697904A1 (en) * 2017-10-17 2020-08-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Combination treatment for cystic fibrosis
EP3775210B1 (en) 2018-03-27 2023-08-23 University of Rochester Nucleic acid molecules for pseudouridylation
KR20210129646A (ko) 2019-01-22 2021-10-28 코로 바이오, 인크. Rna-편집 올리고뉴클레오타이드 및 그의 용도
EP4069842A1 (en) 2019-12-02 2022-10-12 Shape Therapeutics Inc. Therapeutic editing
CN113397996A (zh) * 2021-07-23 2021-09-17 河南省人民医院 一种抗菌漱口水及其制备方法
CN114917227B (zh) * 2022-05-19 2023-10-31 中国人民解放军海军军医大学 依伐卡托在制备抗蜱传脑炎病毒、西尼罗病毒、黄热病毒和基孔肯雅热病毒感染药中的应用
EP4432289A1 (en) 2023-03-15 2024-09-18 Shape Therapeutics Inc. Generative sequence screening with conditional gans, diffusion models, and denoising diffusion conditional gans

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HRP930935A2 (en) * 1992-06-11 1994-12-31 Astra Ab New dna sequences
CN1123038A (zh) 1993-05-11 1996-05-22 北卡罗来纳大学查珀尔希尔分校 阻止异常剪接的反义寡核苷酸及其使用方法
US6207455B1 (en) 1997-05-01 2001-03-27 Lung-Ji Chang Lentiviral vectors
NZ500740A (en) 1997-05-13 2001-02-23 Univ North Carolina Recombinant lentivirus-based gene transfer vectors comprising 3 vectors from Equine Infectious Anemia Virus (EIAV)
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US6218181B1 (en) 1998-03-18 2001-04-17 The Salk Institute For Biological Studies Retroviral packaging cell line
US6683173B2 (en) 1998-04-03 2004-01-27 Epoch Biosciences, Inc. Tm leveling methods
DE19909769A1 (de) 1999-03-05 2000-09-07 Bundesrepublik Deutschland Let Von SIVagm abgeleitete lentivirale Vektoren, Verfahren zu ihrer Herstellung und ihre Verwendung zur Genübertragung in Säugerzellen
US8314226B2 (en) * 2004-03-29 2012-11-20 The General Hospital Corporation Oligonucleotide complex compositions and methods of use as gene alteration tools
EP2151248A1 (en) 2008-07-30 2010-02-10 Johann Bauer Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses
JP2013518603A (ja) 2010-02-08 2013-05-23 アイシス ファーマシューティカルズ， インコーポレーテッド 反復伸張に関連する疾患または病態の治療に有用な方法および組成物
KR20130051954A (ko) * 2010-04-23 2013-05-21 노파르티스 아게 베타-ＥＮａＣ-관련 질환을 치료하기 위한 유기 조성물
JP2013530154A (ja) 2010-05-28 2013-07-25 サレプタ セラピューティクス， インコーポレイテッド 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ
US20150209448A1 (en) 2012-07-12 2015-07-30 Proqr Therapeutics N.V. Exon replacement with stabilized artificial rnas
EP3103872B1 (en) 2012-07-12 2018-08-29 ProQR Therapeutics II B.V. Oligonucleotides for making a change in the sequence of a target rna molecule present in a living cell

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HRP20160854T1 (hr) 2016-09-23
RS54944B1 (sr) 2016-11-30
HUE029977T2 (en) 2017-04-28
MX356625B (es) 2018-06-06
CN104640986B (zh) 2018-08-28
EP2852668B1 (en) 2016-04-27
KR20150037968A (ko) 2015-04-08
HK1208491A1 (zh) 2016-03-04
US20170152518A1 (en) 2017-06-01
NZ703824A (en) 2018-06-29
AU2013287353B2 (en) 2018-11-08
IL236654A0 (en) 2015-02-26
JP6373833B2 (ja) 2018-08-15
AU2013287353A1 (en) 2015-02-26
US20150197744A1 (en) 2015-07-16
IL236654B (en) 2018-02-28
EP3103872A1 (en) 2016-12-14
MX2015000506A (es) 2015-06-03
RU2663110C2 (ru) 2018-08-01
CN104640986A (zh) 2015-05-20
PL2852668T3 (pl) 2016-11-30
US9605255B2 (en) 2017-03-28
US9994856B2 (en) 2018-06-12
SI2852668T1 (sl) 2016-09-30
BR112015000710A8 (pt) 2018-04-03
EP2852668A1 (en) 2015-04-01
JP2015523082A (ja) 2015-08-13
WO2014011053A1 (en) 2014-01-16
ES2698522T3 (es) 2019-02-05
EP3103872B1 (en) 2018-08-29
PT2852668T (pt) 2016-07-13
ES2584856T3 (es) 2016-09-29
RU2015104763A (ru) 2016-08-27
DK2852668T3 (en) 2016-05-30
CA2878934A1 (en) 2014-01-16
BR112015000710A2 (el) 2017-08-15
SMT201600234B (it) 2016-08-31
DK3103872T3 (en) 2018-10-22

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