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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Classifications

    • 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 N.A.
    • 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

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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WO2008018795A1 (fr) 2006-08-11 2008-02-14 Prosensa Technologies B.V. Procédés et moyens de traitement de troubles génétiques associés à l'instabilité des répétitions de l'ADN
WO2009054725A2 (fr) 2007-10-26 2009-04-30 Academisch Ziekenhuis Leiden Moyens et procédés pour contrebalancer des troubles musculaires
EP2539357B1 (fr) 2010-02-26 2017-06-14 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
US20120116381A1 (en) 2010-11-05 2012-05-10 Houser Kevin L Surgical instrument with charging station and wireless communication
AU2011343720A1 (en) * 2010-12-15 2013-04-11 Miragen Therapeutics MicroRNA inhibitors comprising locked nucleotides
CN102643807B (zh) * 2011-02-18 2015-06-03 中国科学院上海药物研究所 人miR-484的反义寡聚核苷酸及其应用
EP2699269A1 (fr) * 2011-04-22 2014-02-26 Prosensa Technologies B.V. Nouveaux composés pour traiter, retarder et/ou prévenir un trouble génétique humain, tel que la dystrophie myotonique de type 1 (dm1)
ES2868950T3 (es) * 2011-04-25 2021-10-22 Sanofi Sa Compuestos de microARN y métodos para modular la actividad de miR-21
WO2012149646A1 (fr) * 2011-05-05 2012-11-08 Sunnybrook Research Institute Inhibiteurs d'arnmi et leurs utilisations
BR112013029016A2 (pt) * 2011-05-09 2017-01-17 Cambridge Entpr Ltd métodos de modulação de micrornas no tratamento da hipertensão arterial pulmonar
EP2763703B1 (fr) 2011-10-06 2018-02-14 Miragen Therapeutics, Inc. Régulation de l'homéostasie énergétique du corps entier par la régulation de microarn
ES2907250T3 (es) 2012-01-27 2022-04-22 Biomarin Tech Bv Oligonucleótidos de modulación de ARN con características mejoradas para el tratamiento de la distrofia muscular de Duchenne y de Becker
RS56319B1 (sr) 2012-04-23 2017-12-29 Biomarin Tech Bv Oligonukleotidi koji moduliraju rnk sa poboljšanim karakteristikama za lečenje neuromišićnih poremećaja
UA117098C2 (uk) 2012-04-25 2018-06-25 Рег'Юлес Терап'Ютікс Інк. Сполука, що містить модифікований олігонуклеотид
CN102703456B (zh) * 2012-05-15 2014-04-02 武汉生命之美科技有限公司 DAPK3基因hsa-miR-20a的作用靶位点
CA2876180C (fr) 2012-06-21 2019-11-19 MiRagen Therapeutics, Inc. Inhibiteurs a base d'oligonucleotides comprenant un motif d'acide nucleique bloque
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
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CN103667441B (zh) * 2013-09-10 2016-10-19 山西医科大学第一医院 一种Hsa-miR-145-5p试剂盒及其成熟体模拟物的应用
GB201400598D0 (en) 2014-01-14 2014-03-05 Univ Glasgow Materials and methods for modulation of tendon healing
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
AU2016209386A1 (en) 2015-01-20 2017-08-03 MiRagen Therapeutics, Inc. miR-92 inhibitors and uses thereof
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JP2019512489A (ja) * 2016-03-07 2019-05-16 ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ マイクロｒｎａおよびその使用方法
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Also Published As

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

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