JP6542662B2 - オリゴヌクレオチドアナログのボロン酸結合体 - Google Patents

オリゴヌクレオチドアナログのボロン酸結合体 Download PDF

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JP6542662B2
JP6542662B2 JP2015501712A JP2015501712A JP6542662B2 JP 6542662 B2 JP6542662 B2 JP 6542662B2 JP 2015501712 A JP2015501712 A JP 2015501712A JP 2015501712 A JP2015501712 A JP 2015501712A JP 6542662 B2 JP6542662 B2 JP 6542662B2
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oligonucleotide analogue
alkyl
analogue according
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oligonucleotide
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JP2015512254A (ja
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グンナール ジェイ. ハンソン,
グンナール ジェイ. ハンソン,
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サレプタ セラピューティクス, インコーポレイテッド
サレプタ セラピューティクス, インコーポレイテッド
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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
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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
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/331Universal or degenerate base
    • CCHEMISTRY; METALLURGY
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    • 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)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2015501712A 2012-03-20 2013-03-07 オリゴヌクレオチドアナログのボロン酸結合体 Active JP6542662B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261613385P 2012-03-20 2012-03-20
US61/613,385 2012-03-20
PCT/US2013/029684 WO2013142087A1 (en) 2012-03-20 2013-03-07 Boronic acid conjugates of oligonucleotide analogues

Publications (3)

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JP2015512254A JP2015512254A (ja) 2015-04-27
JP2015512254A5 JP2015512254A5 (enExample) 2016-04-21
JP6542662B2 true JP6542662B2 (ja) 2019-07-10

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JP2015501712A Active JP6542662B2 (ja) 2012-03-20 2013-03-07 オリゴヌクレオチドアナログのボロン酸結合体

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

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KR101829644B1 (ko) 2010-05-28 2018-02-19 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
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KR20140097398A (ko) 2011-11-18 2014-08-06 사렙타 쎄러퓨틱스, 인코퍼레이티드 기능적으로-변형된 올리고뉴클레오티드 및 이의 서브유니트
NZ631132A (en) * 2013-03-14 2017-06-30 Sarepta Therapeutics Inc Exon skipping compositions for treating muscular dystrophy
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WO2016187425A1 (en) * 2015-05-19 2016-11-24 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
MA42695A (fr) 2015-08-28 2018-07-04 Sarepta Therapeutics Inc Oligomères antisens modifiés pour l'inclusion d'exons dans l'atrophie musculaire spinale
WO2017060317A1 (en) * 2015-10-05 2017-04-13 Proqr Therapeutics Ii B.V. Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion

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Publication number Publication date
US20150080340A1 (en) 2015-03-19
JP2015512254A (ja) 2015-04-27
AU2017202883A1 (en) 2017-05-18
KR20140138995A (ko) 2014-12-04
AU2019201989A1 (en) 2019-04-11
EA031110B1 (ru) 2018-11-30
AU2013235691B2 (en) 2017-02-02
IN2014DN08451A (enExample) 2015-05-08
NZ700399A (en) 2016-07-29
ES2706198T3 (es) 2019-03-27
BR112014023347A2 (pt) 2017-07-18
CA2868174A1 (en) 2013-09-26
KR102079284B1 (ko) 2020-02-19
HK1206064A1 (en) 2015-12-31
MX2014011276A (es) 2014-10-06
WO2013142087A1 (en) 2013-09-26
CN104411831B (zh) 2020-08-11
EP2828395A1 (en) 2015-01-28
MX358497B (es) 2018-08-23
AU2013235691A1 (en) 2014-10-16
EP2828395A4 (en) 2015-11-18
EP2828395B1 (en) 2018-10-24
EA201491726A1 (ru) 2015-03-31
US9920085B2 (en) 2018-03-20
CN104411831A (zh) 2015-03-11

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