MA33488B1 - Motifs de modification chimique pour des inhibiteurs et mimétiques de miarn - Google Patents

Motifs de modification chimique pour des inhibiteurs et mimétiques de miarn

Info

Publication number
MA33488B1
MA33488B1 MA34505A MA34505A MA33488B1 MA 33488 B1 MA33488 B1 MA 33488B1 MA 34505 A MA34505 A MA 34505A MA 34505 A MA34505 A MA 34505A MA 33488 B1 MA33488 B1 MA 33488B1
Authority
MA
Morocco
Prior art keywords
mimetics
inhibitors
mirik
chemical modification
modification rules
Prior art date
Application number
MA34505A
Other languages
English (en)
Inventor
Christina Yamada
William S Marshall
Original Assignee
Miragen Therapeutics
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 Miragen Therapeutics filed Critical Miragen Therapeutics
Publication of MA33488B1 publication Critical patent/MA33488B1/fr

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    • 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
    • C12N15/113—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/14—Vasoprotectives; Antihaemorrhoidals; Drugs for varicose therapy; Capillary stabilisers
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • 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
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/332—Abasic residue
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Cardiology (AREA)
  • Public Health (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La présente invention concerne des polynucléotides ayant des motifs de chimie qui confèrent une stabilité, activité et/ou toxicité améliorée(s) par rapport à leur utilisation en tant qu'inhibiteurs de miARN ou mimétiques de miARN. L'invention concerne en outre des compositions pharmaceutiques et formulations comprenant les polynucléotides, et des procédés pour traiter des patients ayant une affection associée à l'expression de miARN ou d'ARNm.
MA34505A 2009-06-08 2010-06-08 Motifs de modification chimique pour des inhibiteurs et mimétiques de miarn MA33488B1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US18503309P 2009-06-08 2009-06-08
PCT/US2010/037821 WO2010144485A1 (fr) 2009-06-08 2010-06-08 Motifs de modification chimique pour des inhibiteurs et mimétiques de miarn

Publications (1)

Publication Number Publication Date
MA33488B1 true MA33488B1 (fr) 2012-08-01

Family

ID=43309198

Family Applications (1)

Application Number Title Priority Date Filing Date
MA34505A MA33488B1 (fr) 2009-06-08 2010-06-08 Motifs de modification chimique pour des inhibiteurs et mimétiques de miarn

Country Status (17)

Country Link
US (2) US20120148664A1 (fr)
EP (1) EP2440566A4 (fr)
JP (1) JP2012529295A (fr)
KR (1) KR20120047892A (fr)
CN (1) CN102803284B (fr)
AU (1) AU2010258875A1 (fr)
BR (1) BRPI1010885A2 (fr)
CA (1) CA2765129A1 (fr)
EA (1) EA022757B1 (fr)
GE (1) GEP20156329B (fr)
MA (1) MA33488B1 (fr)
MX (1) MX2011013176A (fr)
NZ (1) NZ597078A (fr)
SG (1) SG176716A1 (fr)
UA (1) UA105390C2 (fr)
WO (1) WO2010144485A1 (fr)
ZA (1) ZA201109319B (fr)

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NZ584793A (en) 2007-10-26 2012-05-25 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
WO2011106104A2 (fr) 2010-02-26 2011-09-01 Memorial Sloan-Kettering Cancer Center Procédés et compositions destinés à la détection et au traitement du cancer impliquant des miarn et des inhibiteurs des miarn ainsi que des cibles
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EP2652151A2 (fr) 2010-12-15 2013-10-23 Miragen Therapeutics Inhibiteurs de microarn comprenant des nucléotides bloqués
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AU2012246822B2 (en) * 2011-04-22 2017-05-25 Biomarin Technologies B.V. New compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (DM1)
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WO2012149646A1 (fr) * 2011-05-05 2012-11-08 Sunnybrook Research Institute Inhibiteurs d'arnmi et leurs utilisations
WO2012153135A1 (fr) * 2011-05-09 2012-11-15 University Of Glasgow Procédés de modulation de microarn dans le traitement de l'hypertension artérielle pulmonaire
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EP2864482A4 (fr) 2012-06-21 2016-02-10 Miragen Therapeutics Inc Inhibiteurs à base d'oligonucléotides comprenant un motif d'acide nucléique bloqué
US9163235B2 (en) 2012-06-21 2015-10-20 MiRagen Therapeutics, Inc. Inhibitors of the miR-15 family of micro-RNAs
UA116639C2 (uk) 2012-10-09 2018-04-25 Рег'Юлес Терап'Ютікс Інк. Способи лікування синдрому альпорта
CA2902623A1 (fr) 2013-03-15 2014-09-25 MiRagen Therapeutics, Inc. Inhibiteur d'acide nucleique verrouille de mir-145 et utilisations associees
JP2016518842A (ja) 2013-05-01 2016-06-30 レグルス セラピューティクス インコーポレイテッド マイクロＲＮＡ化合物およびｍｉＲ−１２２をモジュレートする方法
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CN104083761A (zh) * 2014-06-25 2014-10-08 北京大学第三医院 microRNA-101抑制剂在制备预防或治疗骨关节炎药物中的应用
MA40463A (fr) 2014-08-07 2017-06-14 Regulus Therapeutics Inc Ciblage de micro-arn pour traiter des troubles métaboliques
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JP6842779B2 (ja) * 2016-10-31 2021-03-17 国立大学法人岐阜大学 二本鎖核酸分子、およびその用途
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Also Published As

Publication number Publication date
EP2440566A1 (fr) 2012-04-18
GEP20156329B (en) 2015-07-27
SG176716A1 (en) 2012-01-30
NZ597078A (en) 2013-11-29
EP2440566A4 (fr) 2013-10-16
BRPI1010885A2 (pt) 2015-09-22
MX2011013176A (es) 2012-04-30
EA201171493A1 (ru) 2012-06-29
CA2765129A1 (fr) 2010-12-16
US20140066491A1 (en) 2014-03-06
UA105390C2 (ru) 2014-05-12
CN102803284B (zh) 2015-11-25
US20120148664A1 (en) 2012-06-14
CN102803284A (zh) 2012-11-28
EA022757B1 (ru) 2016-02-29
WO2010144485A1 (fr) 2010-12-16
JP2012529295A (ja) 2012-11-22
AU2010258875A1 (en) 2012-01-19
ZA201109319B (en) 2013-02-27
KR20120047892A (ko) 2012-05-14

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