EA031110B1 - Конъюгаты бороновых кислот олигонуклеотидных аналогов - Google Patents

Конъюгаты бороновых кислот олигонуклеотидных аналогов Download PDF

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Publication number
EA031110B1
EA031110B1 EA201491726A EA201491726A EA031110B1 EA 031110 B1 EA031110 B1 EA 031110B1 EA 201491726 A EA201491726 A EA 201491726A EA 201491726 A EA201491726 A EA 201491726A EA 031110 B1 EA031110 B1 EA 031110B1
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EA
Eurasian Patent Office
Prior art keywords
alkyl
oligonucleotide analogue
morpholino
analogue according
oligonucleotide
Prior art date
Application number
EA201491726A
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English (en)
Russian (ru)
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EA201491726A1 (ru
Inventor
Гуннар Дж. Хансон
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Сарепта Терапьютикс, Инк.
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Publication date
Application filed by Сарепта Терапьютикс, Инк. filed Critical Сарепта Терапьютикс, Инк.
Publication of EA201491726A1 publication Critical patent/EA201491726A1/ru
Publication of EA031110B1 publication Critical patent/EA031110B1/ru

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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02Phosphorus compounds
    • C07F9/547Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6596Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having atoms other than oxygen, sulfur, selenium, tellurium, nitrogen or phosphorus as ring hetero atoms
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02Phosphorus compounds
    • C07F9/547Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6558Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system
    • C07F9/65583Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system each of the hetero rings containing nitrogen as ring hetero atom
    • 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
    • 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
    • C12N2310/31Chemical structure of the backbone
    • 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
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
    • 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
    • C12N2310/33Chemical structure of the base
    • C12N2310/331Universal or degenerate base
    • 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
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Veterinary Medicine (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurosurgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
EA201491726A 2012-03-20 2013-03-07 Конъюгаты бороновых кислот олигонуклеотидных аналогов EA031110B1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261613385P 2012-03-20 2012-03-20
PCT/US2013/029684 WO2013142087A1 (en) 2012-03-20 2013-03-07 Boronic acid conjugates of oligonucleotide analogues

Publications (2)

Publication Number Publication Date
EA201491726A1 EA201491726A1 (ru) 2015-03-31
EA031110B1 true EA031110B1 (ru) 2018-11-30

Family

ID=49223176

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201491726A EA031110B1 (ru) 2012-03-20 2013-03-07 Конъюгаты бороновых кислот олигонуклеотидных аналогов

Country Status (14)

Country Link
US (1) US9920085B2 (https=)
EP (1) EP2828395B1 (https=)
JP (1) JP6542662B2 (https=)
KR (1) KR102079284B1 (https=)
CN (1) CN104411831B (https=)
AU (3) AU2013235691B2 (https=)
BR (1) BR112014023347A2 (https=)
CA (1) CA2868174A1 (https=)
EA (1) EA031110B1 (https=)
ES (1) ES2706198T3 (https=)
IN (1) IN2014DN08451A (https=)
MX (1) MX358497B (https=)
NZ (1) NZ700399A (https=)
WO (1) WO2013142087A1 (https=)

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KR20200133284A (ko) 2010-05-28 2020-11-26 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
US10017763B2 (en) 2010-09-03 2018-07-10 Sarepta Therapeutics, Inc. dsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups
HK1201514A1 (en) 2011-11-18 2015-09-04 Sarepta Therapeutics, Inc. Functionally-modified oligonucleotides and subunits thereof
US20140315977A1 (en) * 2013-03-14 2014-10-23 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
CN110218727A (zh) * 2013-03-14 2019-09-10 萨勒普塔医疗公司 用于治疗肌营养不良的外显子跳跃组合物
US20140329762A1 (en) 2013-03-15 2014-11-06 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
AU2016264471B2 (en) * 2015-05-19 2022-01-27 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
US10905709B2 (en) 2015-08-28 2021-02-02 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
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Also Published As

Publication number Publication date
MX2014011276A (es) 2014-10-06
EP2828395A1 (en) 2015-01-28
KR20140138995A (ko) 2014-12-04
US9920085B2 (en) 2018-03-20
ES2706198T3 (es) 2019-03-27
JP2015512254A (ja) 2015-04-27
US20150080340A1 (en) 2015-03-19
CN104411831A (zh) 2015-03-11
KR102079284B1 (ko) 2020-02-19
JP6542662B2 (ja) 2019-07-10
AU2013235691B2 (en) 2017-02-02
MX358497B (es) 2018-08-23
WO2013142087A1 (en) 2013-09-26
EA201491726A1 (ru) 2015-03-31
AU2017202883A1 (en) 2017-05-18
IN2014DN08451A (https=) 2015-05-08
HK1206064A1 (en) 2015-12-31
BR112014023347A2 (pt) 2017-07-18
CA2868174A1 (en) 2013-09-26
EP2828395B1 (en) 2018-10-24
AU2019201989A1 (en) 2019-04-11
CN104411831B (zh) 2020-08-11
EP2828395A4 (en) 2015-11-18
NZ700399A (en) 2016-07-29
AU2013235691A1 (en) 2014-10-16

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