BR112015000710A8 - Oligonucleotídeos para fazer uma alteração na sequência de um presente alvo de molécula de rna em uma célula viva - Google Patents

Oligonucleotídeos para fazer uma alteração na sequência de um presente alvo de molécula de rna em uma célula viva

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Publication number
BR112015000710A8
BR112015000710A8 BR112015000710A BR112015000710A BR112015000710A8 BR 112015000710 A8 BR112015000710 A8 BR 112015000710A8 BR 112015000710 A BR112015000710 A BR 112015000710A BR 112015000710 A BR112015000710 A BR 112015000710A BR 112015000710 A8 BR112015000710 A8 BR 112015000710A8
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BR
Brazil
Prior art keywords
oligonucleotides
rna molecule
target rna
living cell
make
Prior art date
Application number
BR112015000710A
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English (en)
Portuguese (pt)
Other versions
BR112015000710A2 (https=
Inventor
Anton De Boer Daniel
Ritsema Tita
Original Assignee
Proqr Therapeutics B V
Proqr Therapeutics Ii Bv
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.)
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Publication date
Application filed by Proqr Therapeutics B V, Proqr Therapeutics Ii Bv filed Critical Proqr Therapeutics B V
Publication of BR112015000710A2 publication Critical patent/BR112015000710A2/pt
Publication of BR112015000710A8 publication Critical patent/BR112015000710A8/pt

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    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
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    • 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
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    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/34Allele or polymorphism specific uses

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
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  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
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  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pulmonology (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)
BR112015000710A 2012-07-12 2013-07-12 Oligonucleotídeos para fazer uma alteração na sequência de um presente alvo de molécula de rna em uma célula viva BR112015000710A8 (pt)

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
BR112015000710A2 BR112015000710A2 (https=) 2017-08-15
BR112015000710A8 true BR112015000710A8 (pt) 2018-04-03

Family

ID=49916369

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112015000710A BR112015000710A8 (pt) 2012-07-12 2013-07-12 Oligonucleotídeos para fazer uma alteração na sequência de um presente alvo de molécula de rna em uma célula viva

Country Status (23)

Country Link
US (2) US9605255B2 (https=)
EP (2) EP2852668B1 (https=)
JP (1) JP6373833B2 (https=)
KR (1) KR20150037968A (https=)
CN (1) CN104640986B (https=)
AU (1) AU2013287353B2 (https=)
BR (1) BR112015000710A8 (https=)
CA (1) CA2878934A1 (https=)
CY (1) CY1117858T1 (https=)
DK (2) DK2852668T3 (https=)
ES (2) ES2584856T3 (https=)
HR (1) HRP20160854T1 (https=)
HU (1) HUE029977T2 (https=)
IL (1) IL236654B (https=)
MX (1) MX356625B (https=)
NZ (1) NZ703824A (https=)
PL (1) PL2852668T3 (https=)
PT (1) PT2852668T (https=)
RS (1) RS54944B1 (https=)
RU (1) RU2663110C2 (https=)
SI (1) SI2852668T1 (https=)
SM (1) SMT201600234B (https=)
WO (1) WO2014011053A1 (https=)

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KR102589295B1 (ko) 2014-05-14 2023-10-13 타르그이뮨 테라퓨틱스 아게 개선된 폴리에틸렌이민 폴리에틸렌글리콜 벡터
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
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US10676737B2 (en) * 2014-12-17 2020-06-09 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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JP6872560B2 (ja) * 2016-05-06 2021-05-19 エム. ウルフ、トッド プログラム可能ヌクレアーゼのあるゲノム編集及びプログラム可能ヌクレアーゼのないゲノム編集のための改善された方法
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US10941402B2 (en) 2016-09-01 2021-03-09 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
GB2574769A (en) 2017-03-03 2019-12-18 Univ California RNA Targeting of mutations via suppressor tRNAs 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
WO2019076919A1 (en) * 2017-10-17 2019-04-25 INSERM (Institut National de la Santé et de la Recherche Médicale) COMBINED TREATMENT OF CYSTIC FIBROSIS
EP3615085A4 (en) * 2018-02-09 2021-01-13 Ohio State Innovation Foundation RNA NANOSTRUCTURES, METHOD OF MANUFACTURING AND USES THEREOF
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GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
KR20210129646A (ko) 2019-01-22 2021-10-28 코로 바이오, 인크. Rna-편집 올리고뉴클레오타이드 및 그의 용도
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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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Also Published As

Publication number Publication date
HUE029977T2 (en) 2017-04-28
IL236654A0 (en) 2015-02-26
US9994856B2 (en) 2018-06-12
MX2015000506A (es) 2015-06-03
RU2015104763A (ru) 2016-08-27
PL2852668T3 (pl) 2016-11-30
KR20150037968A (ko) 2015-04-08
SMT201600234B (it) 2016-08-31
CY1117858T1 (el) 2017-05-17
CN104640986B (zh) 2018-08-28
US9605255B2 (en) 2017-03-28
JP2015523082A (ja) 2015-08-13
DK2852668T3 (en) 2016-05-30
SI2852668T1 (sl) 2016-09-30
CA2878934A1 (en) 2014-01-16
AU2013287353A1 (en) 2015-02-26
EP2852668A1 (en) 2015-04-01
PT2852668T (pt) 2016-07-13
EP3103872B1 (en) 2018-08-29
ES2584856T3 (es) 2016-09-29
NZ703824A (en) 2018-06-29
US20170152518A1 (en) 2017-06-01
HRP20160854T1 (hr) 2016-09-23
IL236654B (en) 2018-02-28
RS54944B1 (sr) 2016-11-30
ES2698522T3 (es) 2019-02-05
AU2013287353B2 (en) 2018-11-08
JP6373833B2 (ja) 2018-08-15
EP3103872A1 (en) 2016-12-14
MX356625B (es) 2018-06-06
DK3103872T3 (en) 2018-10-22
CN104640986A (zh) 2015-05-20
BR112015000710A2 (https=) 2017-08-15
WO2014011053A1 (en) 2014-01-16
RU2663110C2 (ru) 2018-08-01
US20150197744A1 (en) 2015-07-16
EP2852668B1 (en) 2016-04-27
HK1208491A1 (zh) 2016-03-04

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