IL236654B - Oligonucleotides to change the sequence of an RNA target molecule that is found in a living cell - Google Patents

Oligonucleotides to change the sequence of an RNA target molecule that is found in a living cell

Info

Publication number
IL236654B
IL236654B IL236654A IL23665415A IL236654B IL 236654 B IL236654 B IL 236654B IL 236654 A IL236654 A IL 236654A IL 23665415 A IL23665415 A IL 23665415A IL 236654 B IL236654 B IL 236654B
Authority
IL
Israel
Prior art keywords
oligonucleotides
sequence
making
change
rna molecule
Prior art date
Application number
IL236654A
Other languages
English (en)
Hebrew (he)
Other versions
IL236654A0 (en
Original Assignee
Proqr Therapeutics Ii Bv
Proqr Therapeutics N 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 Bv, Proqr Therapeutics N V filed Critical Proqr Therapeutics Ii Bv
Publication of IL236654A0 publication Critical patent/IL236654A0/en
Publication of IL236654B publication Critical patent/IL236654B/en

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    • 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
    • C12N15/1138Non-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
    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/08Solutions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • 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
    • C12N15/01Preparation of mutants without inserting foreign genetic material therein; Screening processes therefor
    • 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
    • C12N2310/11Antisense
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/31Combination therapy
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/34Allele or polymorphism specific uses

Landscapes

  • 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)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (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)
IL236654A 2012-07-12 2015-01-11 Oligonucleotides to change the sequence of an RNA target molecule that is found in a living cell IL236654B (en)

Applications Claiming Priority (6)

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

Publications (2)

Publication Number Publication Date
IL236654A0 IL236654A0 (en) 2015-02-26
IL236654B true IL236654B (en) 2018-02-28

Family

ID=49916369

Family Applications (1)

Application Number Title Priority Date Filing Date
IL236654A IL236654B (en) 2012-07-12 2015-01-11 Oligonucleotides to change the sequence of an RNA target molecule that is found in a living cell

Country Status (23)

Country Link
US (2) US9605255B2 (enExample)
EP (2) EP3103872B1 (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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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
RU2569678C2 (ru) 2010-04-22 2015-11-27 Вертекс Фармасьютикалз Инкорпорейтед Способ получения циклоалкилкарбоксамидо-индольных соединений
WO2014011053A1 (en) 2012-07-12 2014-01-16 Proqr Therapeutics B.V. Oligonucleotides for making a change in the sequence of a target rna molecule present in a living cell
PT3131582T (pt) 2014-04-15 2018-10-08 Vertex Pharma Composições farmacêuticas para o tratamento de doenças mediadas pelo regulador de condutância transmembranar da fibrose quística
RU2771104C2 (ru) 2014-05-14 2022-04-26 Таргиммьюн Терапьютикс Аг Улучшенные полиэтилениминовые полиэтиленгликолевые векторы
CN107250113B (zh) 2014-10-07 2019-03-29 弗特克斯药品有限公司 囊性纤维化跨膜传导调节蛋白的调节剂的共晶
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
DK3234134T3 (da) * 2014-12-17 2020-07-27 Proqr Therapeutics Ii Bv Målrettet rna-redigering
US20210401869A1 (en) * 2015-02-20 2021-12-30 Rosalind Franklin University Of Medicine And Science Antisense Compounds Targeting Genes Associated with Cystic Fibrosis
GB201507926D0 (en) * 2015-05-08 2015-06-24 Proqr Therapeutics N V Improved treatments using oligonucleotides
KR20180053753A (ko) 2015-10-05 2018-05-23 프로큐알 테라퓨틱스 Ⅱ 비.브이. 삼핵산 반복 확장과 관련된 유전질환의 예방 또는 치료용 단일 가닥 안티센스 올리고뉴클레오타이드의 용도
EP3985116A1 (en) 2015-10-15 2022-04-20 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
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
ES2837076T3 (es) 2016-09-01 2021-06-29 Proqr Therapeutics Ii Bv Oligonucleótidos para la edición de ARN de cadena sencilla modificados químicamente
US11274300B2 (en) 2017-01-19 2022-03-15 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in RNA editing
JP2020508685A (ja) 2017-03-03 2020-03-26 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア サプレッサーtRNA及びデアミナーゼによる変異のRNAターゲティング
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
AU2019243946B2 (en) 2018-03-27 2025-03-06 Proqr Therapeutics Ii B.V. Nucleic acid molecules for pseudouridylation
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
WO2020154343A1 (en) * 2019-01-22 2020-07-30 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
CA3159944A1 (en) 2019-12-02 2021-06-10 David HUSS 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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EP2872632A1 (en) 2012-07-12 2015-05-20 ProQR Therapeutics B.V. Exon replacement with stabilized artificial rnas

Also Published As

Publication number Publication date
BR112015000710A2 (enExample) 2017-08-15
EP3103872A1 (en) 2016-12-14
RS54944B1 (sr) 2016-11-30
JP2015523082A (ja) 2015-08-13
DK3103872T3 (en) 2018-10-22
MX356625B (es) 2018-06-06
CY1117858T1 (el) 2017-05-17
HRP20160854T1 (hr) 2016-09-23
AU2013287353A1 (en) 2015-02-26
RU2663110C2 (ru) 2018-08-01
DK2852668T3 (en) 2016-05-30
CN104640986B (zh) 2018-08-28
KR20150037968A (ko) 2015-04-08
EP2852668A1 (en) 2015-04-01
US20170152518A1 (en) 2017-06-01
IL236654A0 (en) 2015-02-26
HK1208491A1 (zh) 2016-03-04
CA2878934A1 (en) 2014-01-16
MX2015000506A (es) 2015-06-03
PT2852668T (pt) 2016-07-13
ES2584856T3 (es) 2016-09-29
SI2852668T1 (sl) 2016-09-30
CN104640986A (zh) 2015-05-20
US9605255B2 (en) 2017-03-28
EP3103872B1 (en) 2018-08-29
JP6373833B2 (ja) 2018-08-15
WO2014011053A1 (en) 2014-01-16
AU2013287353B2 (en) 2018-11-08
HUE029977T2 (en) 2017-04-28
ES2698522T3 (es) 2019-02-05
US9994856B2 (en) 2018-06-12
NZ703824A (en) 2018-06-29
PL2852668T3 (pl) 2016-11-30
EP2852668B1 (en) 2016-04-27
SMT201600234B (it) 2016-08-31
US20150197744A1 (en) 2015-07-16
BR112015000710A8 (pt) 2018-04-03
RU2015104763A (ru) 2016-08-27

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