CN104411831B - 寡核苷酸类似物的硼酸缀合物 - Google Patents

寡核苷酸类似物的硼酸缀合物 Download PDF

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Publication number
CN104411831B
CN104411831B CN201380023605.6A CN201380023605A CN104411831B CN 104411831 B CN104411831 B CN 104411831B CN 201380023605 A CN201380023605 A CN 201380023605A CN 104411831 B CN104411831 B CN 104411831B
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oligonucleotide analogue
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hydrocarbyl
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CN104411831A (zh
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贡纳·J·汉森
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Sarepta Therapeutics Inc
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6596—Heterocyclic 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6558—Heterocyclic 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
    • C07F9/65583—Heterocyclic 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
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/331—Universal or degenerate base
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate

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  • Health & Medical Sciences (AREA)
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  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • General Chemical & Material Sciences (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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CN201380023605.6A 2012-03-20 2013-03-07 寡核苷酸类似物的硼酸缀合物 Expired - Fee Related CN104411831B (zh)

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 (2)

Publication Number Publication Date
CN104411831A CN104411831A (zh) 2015-03-11
CN104411831B true CN104411831B (zh) 2020-08-11

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CN201380023605.6A Expired - Fee Related CN104411831B (zh) 2012-03-20 2013-03-07 寡核苷酸类似物的硼酸缀合物

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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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ATE498685T1 (de) 2004-06-28 2011-03-15 Univ Western Australia Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
LT2499249T (lt) 2009-11-12 2018-12-27 The University Of Western Australia Priešprasmės molekulės ir patologijų gydymo būdai
KR20200133284A (ko) 2010-05-28 2020-11-26 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
WO2012031243A2 (en) 2010-09-03 2012-03-08 Avi Biopharma, Inc. dsRNA MOLECULES COMPRISING OLIGONUCLEOTIDE ANALOGS HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS
EA202090338A1 (ru) 2011-11-18 2021-04-30 Сарепта Терапьютикс, Инк. Функционально-модифицированные олигонуклеотиды и их субъединицы
RS58529B1 (sr) * 2013-03-14 2019-04-30 Sarepta Therapeutics Inc Sastavi koji preskaču egzon za tretiranje mišićne distrofije
NZ731587A (en) 2013-03-14 2021-07-30 Sarepta Therapeutics Inc Exon skipping compositions for treating muscular dystrophy
CA2906812A1 (en) 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
EP3297649B1 (en) * 2015-05-19 2023-10-11 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
EP3302489A4 (en) * 2015-06-04 2019-02-06 Sarepta Therapeutics, Inc. METHOD AND COMPOUNDS FOR THE TREATMENT OF DISEASES AND SUFFERING IN CONNECTION WITH LYMPHOCYTES
JP6987041B2 (ja) 2015-08-28 2021-12-22 サレプタ セラピューティクス，インコーポレイテッド 脊髄性筋萎縮症におけるエクソン包含のための修飾アンチセンスオリゴマー
EP3359667A1 (en) * 2015-10-05 2018-08-15 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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AU2009281857A1 (en) * 2008-08-15 2010-02-18 Georgia State University Research Foundation, Inc. Aptamer inhibition of thrombus formation
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GB0501944D0 (en) * 2005-01-31 2005-03-09 Univ Cambridge Tech Compounds for use in chemical detection and/or quantitation
AU2009281857A1 (en) * 2008-08-15 2010-02-18 Georgia State University Research Foundation, Inc. Aptamer inhibition of thrombus formation
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ES2706198T3 (es) 2019-03-27
EP2828395A4 (en) 2015-11-18
KR20140138995A (ko) 2014-12-04
JP2015512254A (ja) 2015-04-27
IN2014DN08451A (https=) 2015-05-08
AU2013235691A1 (en) 2014-10-16
EP2828395A1 (en) 2015-01-28
AU2013235691B2 (en) 2017-02-02
WO2013142087A1 (en) 2013-09-26
US20150080340A1 (en) 2015-03-19
BR112014023347A2 (pt) 2017-07-18
AU2019201989A1 (en) 2019-04-11
CA2868174A1 (en) 2013-09-26
EP2828395B1 (en) 2018-10-24
US9920085B2 (en) 2018-03-20
MX358497B (es) 2018-08-23
NZ700399A (en) 2016-07-29
EA201491726A1 (ru) 2015-03-31
JP6542662B2 (ja) 2019-07-10
CN104411831A (zh) 2015-03-11
AU2017202883A1 (en) 2017-05-18
HK1206064A1 (en) 2015-12-31
MX2014011276A (es) 2014-10-06
KR102079284B1 (ko) 2020-02-19
EA031110B1 (ru) 2018-11-30

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