JP6487913B2 - 成長ホルモン受容体のモジュレータ - Google Patents

成長ホルモン受容体のモジュレータ Download PDF

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Publication number
JP6487913B2
JP6487913B2 JP2016524320A JP2016524320A JP6487913B2 JP 6487913 B2 JP6487913 B2 JP 6487913B2 JP 2016524320 A JP2016524320 A JP 2016524320A JP 2016524320 A JP2016524320 A JP 2016524320A JP 6487913 B2 JP6487913 B2 JP 6487913B2
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modified
certain embodiments
antisense
compound
nucleic acid
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Japanese (ja)
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JP2016523548A (ja
JP2016523548A5 (enExample
Inventor
バーノット，サンジェイ
フレアー，スーザン・エム
ブイ，フィン−ホア
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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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
    • C12N15/1138—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 against receptors or cell surface proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/02—Drugs for disorders of the endocrine system of the hypothalamic hormones, e.g. TRH, GnRH, CRH, GRH, somatostatin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/02—Drugs for disorders of the endocrine system of the hypothalamic hormones, e.g. TRH, GnRH, CRH, GRH, somatostatin
    • A61P5/04—Drugs for disorders of the endocrine system of the hypothalamic hormones, e.g. TRH, GnRH, CRH, GRH, somatostatin for decreasing, blocking or antagonising the activity of the hypothalamic hormones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/06—Drugs for disorders of the endocrine system of the anterior pituitary hormones, e.g. TSH, ACTH, FSH, LH, PRL, GH
    • A61P5/08—Drugs for disorders of the endocrine system of the anterior pituitary hormones, e.g. TSH, ACTH, FSH, LH, PRL, GH for decreasing, blocking or antagonising the activity of the anterior pituitary hormones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/10—Drugs for disorders of the endocrine system of the posterior pituitary hormones, e.g. oxytocin, ADH
    • A61P5/12—Drugs for disorders of the endocrine system of the posterior pituitary hormones, e.g. oxytocin, ADH for decreasing, blocking or antagonising the activity of the posterior pituitary hormones
    • 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
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • 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
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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
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Endocrinology (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Epidemiology (AREA)
JP2016524320A 2013-07-02 2014-07-01 成長ホルモン受容体のモジュレータ Expired - Fee Related JP6487913B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361842302P 2013-07-02 2013-07-02
US61/842,302 2013-07-02
PCT/US2014/045088 WO2015002971A2 (en) 2013-07-02 2014-07-01 Modulators of growth hormone receptor

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2019030128A Division JP2019088329A (ja) 2013-07-02 2019-02-22 成長ホルモン受容体のモジュレータ

Publications (3)

Publication Number Publication Date
JP2016523548A JP2016523548A (ja) 2016-08-12
JP2016523548A5 JP2016523548A5 (enExample) 2017-08-10
JP6487913B2 true JP6487913B2 (ja) 2019-03-20

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Family Applications (2)

Application Number Title Priority Date Filing Date
JP2016524320A Expired - Fee Related JP6487913B2 (ja) 2013-07-02 2014-07-01 成長ホルモン受容体のモジュレータ
JP2019030128A Pending JP2019088329A (ja) 2013-07-02 2019-02-22 成長ホルモン受容体のモジュレータ

Family Applications After (1)

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JP2019030128A Pending JP2019088329A (ja) 2013-07-02 2019-02-22 成長ホルモン受容体のモジュレータ

Country Status (13)

Country Link
US (4) US20170073689A1 (enExample)
EP (2) EP3730614A3 (enExample)
JP (2) JP6487913B2 (enExample)
KR (1) KR102236784B1 (enExample)
CN (1) CN105452461B (enExample)
AU (2) AU2014284398B2 (enExample)
BR (1) BR112015033069A2 (enExample)
CA (1) CA2917229A1 (enExample)
ES (1) ES2787600T3 (enExample)
IL (2) IL243093B (enExample)
MX (1) MX375446B (enExample)
RU (1) RU2700244C2 (enExample)
WO (1) WO2015002971A2 (enExample)

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PL3119888T3 (pl) 2014-03-19 2021-12-20 Ionis Pharmaceuticals, Inc. Kompozycje do modulacji ekspresji ataksyny 2
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
MX373283B (es) * 2014-05-01 2020-05-20 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion del receptor de la hormona del crecimiento.
AU2016381174A1 (en) 2015-12-31 2018-05-31 Ionis Pharmaceuticals, Inc. Methods for reducing Ataxin-2 expression
CN110177544A (zh) 2016-11-29 2019-08-27 普尔泰克健康有限公司 用于递送治疗剂的外泌体
JP7379387B2 (ja) 2018-06-05 2023-11-14 エフ． ホフマン－ラ ロシュ アーゲー Ａｔｘｎ２発現を制御するためのオリゴヌクレオチド
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WO2015002971A2 (en) 2015-01-08
RU2016102833A (ru) 2017-08-07
EP3017044B1 (en) 2020-02-26
AU2014284398B2 (en) 2019-10-31
CN105452461A (zh) 2016-03-30
US20230147811A1 (en) 2023-05-11
EP3730614A3 (en) 2020-12-30
EP3730614A2 (en) 2020-10-28
HK1223652A1 (en) 2017-08-04
IL272828A (en) 2020-04-30
CA2917229A1 (en) 2015-01-08
EP3017044A2 (en) 2016-05-11
EP3017044A4 (en) 2017-06-14
AU2020200570A1 (en) 2020-02-20
KR102236784B1 (ko) 2021-04-05
RU2700244C2 (ru) 2019-09-13
MX2015017863A (es) 2016-11-30
US20190225970A1 (en) 2019-07-25
CN105452461B (zh) 2021-04-13
RU2016102833A3 (enExample) 2018-03-28
ES2787600T3 (es) 2020-10-16
IL243093B (en) 2020-03-31
AU2014284398A1 (en) 2016-01-07
JP2016523548A (ja) 2016-08-12
JP2019088329A (ja) 2019-06-13
US20170073689A1 (en) 2017-03-16
BR112015033069A2 (pt) 2017-11-07
WO2015002971A3 (en) 2015-03-05
KR20160037895A (ko) 2016-04-06

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