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

Info

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
Authority
CY
Cyprus
Prior art keywords
change
target rna
oligonucleotides
induction
molecule
Prior art date
Application number
CY20161100722T
Other languages
Greek (el)
English (en)
Inventor
Boer Daniel Anton De
Tita Ritsema
Original Assignee
Proqr Therapeutics Ii B.V.
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 Proqr Therapeutics Ii B.V. filed Critical Proqr Therapeutics Ii B.V.
Publication of CY1117858T1 publication Critical patent/CY1117858T1/el

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    • 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
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • 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
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • 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
    • 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
    • C12N15/01—Preparation of mutants without inserting foreign genetic material therein; Screening processes therefor
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N2310/11—Antisense
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pulmonology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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

ID=49916369

Family Applications (1)

Application Number Title Priority Date Filing Date
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 (23)

Country Link
US (2) US9605255B2 (enExample)
EP (2) EP2852668B1 (enExample)
JP (1) JP6373833B2 (enExample)
KR (1) KR20150037968A (enExample)
CN (1) CN104640986B (enExample)
AU (1) AU2013287353B2 (enExample)
BR (1) BR112015000710A8 (enExample)
CA (1) CA2878934A1 (enExample)
CY (1) CY1117858T1 (enExample)
DK (2) DK3103872T3 (enExample)
ES (2) ES2584856T3 (enExample)
HR (1) HRP20160854T1 (enExample)
HU (1) HUE029977T2 (enExample)
IL (1) IL236654B (enExample)
MX (1) MX356625B (enExample)
NZ (1) NZ703824A (enExample)
PL (1) PL2852668T3 (enExample)
PT (1) PT2852668T (enExample)
RS (1) RS54944B1 (enExample)
RU (1) RU2663110C2 (enExample)
SI (1) SI2852668T1 (enExample)
SM (1) SMT201600234B (enExample)
WO (1) WO2014011053A1 (enExample)

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RU2569678C2 (ru) 2010-04-22 2015-11-27 Вертекс Фармасьютикалз Инкорпорейтед Способ получения циклоалкилкарбоксамидо-индольных соединений
KR20150037968A (ko) 2012-07-12 2015-04-08 프로큐알 테라퓨틱스 엔.브이. 살아있는 세포 내에 존재하는 목적 rna 분자의 서열을 변화시키기 위한 올리고뉴클레오타이드
PL3925607T5 (pl) 2014-04-15 2026-06-01 Vertex Pharmaceuticals Incorporated Kompozycje farmaceutyczne do leczenia chorób, w których pośredniczy mukowiscydozowy przezbłonowy regulator przewodnictwa
US10278991B2 (en) 2014-05-14 2019-05-07 Targimmune Therapeutics Ag Polyethyleneimine polyethyleneglycol vectors
CA2963945C (en) 2014-10-07 2023-01-10 Vertex Pharmaceuticals Incorporated Co-crystals of modulators of cystic fibrosis transmembrane conductance regulator
GB201418892D0 (en) * 2014-10-23 2014-12-10 Proqr Therapeutics B V DNA editing
CN107532162A (zh) * 2014-12-12 2018-01-02 托德·M·伍尔夫 用于利用寡核苷酸编辑细胞中核酸的组合物和方法
AU2015367378A1 (en) * 2014-12-17 2017-06-15 Proqr Therapeutics Ii B.V. Targeted RNA editing
US20210401869A1 (en) * 2015-02-20 2021-12-30 Rosalind Franklin University Of Medicine And Science Antisense Compounds Targeting Genes Associated with Cystic Fibrosis
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AU2016335032B2 (en) 2015-10-05 2022-04-14 Proqr Therapeutics Ii B.V. Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion
CA3001723A1 (en) 2015-10-15 2017-04-20 City Of Hope Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use thereof
CA3022319A1 (en) * 2016-05-06 2017-11-09 Tod M. Woolf Improved methods for genome editing with and without programmable nucleases
EP3475424A1 (en) 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Single-stranded rna-editing oligonucleotides
WO2018041973A1 (en) * 2016-09-01 2018-03-08 Proqr Therapeutics Ii B.V. 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
CN110612353A (zh) 2017-03-03 2019-12-24 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
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
CN110573184B (zh) * 2018-02-09 2025-05-13 俄亥俄州立创新基金会 Rna纳米结构,其制备方法和用途
ES2959701T3 (es) 2018-03-27 2024-02-27 Univ Rochester Moléculas de ácido nucleico para pseudouridilación
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
CA3127241A1 (en) * 2019-01-22 2020-07-30 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
AU2020245332B2 (en) * 2019-03-28 2026-04-30 Splisense Ltd. Compositions and methods for treating cystic fibrosis
US11827880B2 (en) 2019-12-02 2023-11-28 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
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KR20150037968A (ko) 2012-07-12 2015-04-08 프로큐알 테라퓨틱스 엔.브이. 살아있는 세포 내에 존재하는 목적 rna 분자의 서열을 변화시키기 위한 올리고뉴클레오타이드
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Publication number Publication date
SMT201600234B (it) 2016-08-31
US20150197744A1 (en) 2015-07-16
DK3103872T3 (en) 2018-10-22
AU2013287353A1 (en) 2015-02-26
JP2015523082A (ja) 2015-08-13
NZ703824A (en) 2018-06-29
CA2878934A1 (en) 2014-01-16
HUE029977T2 (en) 2017-04-28
AU2013287353B2 (en) 2018-11-08
ES2698522T3 (es) 2019-02-05
BR112015000710A2 (enExample) 2017-08-15
EP2852668A1 (en) 2015-04-01
WO2014011053A1 (en) 2014-01-16
HRP20160854T1 (hr) 2016-09-23
US9605255B2 (en) 2017-03-28
EP3103872B1 (en) 2018-08-29
IL236654B (en) 2018-02-28
EP2852668B1 (en) 2016-04-27
KR20150037968A (ko) 2015-04-08
MX2015000506A (es) 2015-06-03
BR112015000710A8 (pt) 2018-04-03
EP3103872A1 (en) 2016-12-14
RS54944B1 (sr) 2016-11-30
HK1208491A1 (zh) 2016-03-04
CN104640986A (zh) 2015-05-20
US20170152518A1 (en) 2017-06-01
SI2852668T1 (sl) 2016-09-30
JP6373833B2 (ja) 2018-08-15
DK2852668T3 (en) 2016-05-30
MX356625B (es) 2018-06-06
RU2663110C2 (ru) 2018-08-01
ES2584856T3 (es) 2016-09-29
PL2852668T3 (pl) 2016-11-30
RU2015104763A (ru) 2016-08-27
IL236654A0 (en) 2015-02-26
CN104640986B (zh) 2018-08-28
PT2852668T (pt) 2016-07-13
US9994856B2 (en) 2018-06-12

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