RU2700244C2 - Модуляторы рецептора гормона роста - Google Patents

Модуляторы рецептора гормона роста Download PDF

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RU2700244C2
RU2700244C2 RU2016102833A RU2016102833A RU2700244C2 RU 2700244 C2 RU2700244 C2 RU 2700244C2 RU 2016102833 A RU2016102833 A RU 2016102833A RU 2016102833 A RU2016102833 A RU 2016102833A RU 2700244 C2 RU2700244 C2 RU 2700244C2
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antisense
modified
ghr
nucleic acid
compound
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RU2016102833A
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Russian (ru)
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RU2016102833A (ru
RU2016102833A3 (OSRAM
Inventor
Санджай Бханот
Сьюзан М. Фрайер
Хунх-Хоа БУИ
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Ионис Фармасьютикалз, Инк.
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    • C—CHEMISTRY; METALLURGY
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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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
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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
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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 ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end

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  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
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  • Chemical & Material Sciences (AREA)
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  • Molecular Biology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • 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)
RU2016102833A 2013-07-02 2014-07-01 Модуляторы рецептора гормона роста RU2700244C2 (ru)

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

Publications (3)

Publication Number Publication Date
RU2016102833A RU2016102833A (ru) 2017-08-07
RU2016102833A3 RU2016102833A3 (OSRAM) 2018-03-28
RU2700244C2 true RU2700244C2 (ru) 2019-09-13

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US (4) US20170073689A1 (OSRAM)
EP (2) EP3017044B1 (OSRAM)
JP (2) JP6487913B2 (OSRAM)
KR (1) KR102236784B1 (OSRAM)
CN (1) CN105452461B (OSRAM)
AU (2) AU2014284398B2 (OSRAM)
BR (1) BR112015033069A2 (OSRAM)
CA (1) CA2917229A1 (OSRAM)
ES (1) ES2787600T3 (OSRAM)
IL (2) IL243093B (OSRAM)
MX (1) MX375446B (OSRAM)
RU (1) RU2700244C2 (OSRAM)
WO (1) WO2015002971A2 (OSRAM)

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WO2015143245A1 (en) 2014-03-19 2015-09-24 Isis Pharmaceuticals, Inc. Methods for modulating ataxin 2 expression
BR122020024446B8 (pt) 2014-05-01 2022-06-28 Ionis Pharmaceuticals Inc Compostos para modulação da expressão do receptor do hormônio de crescimento
EP3397288A4 (en) 2015-12-31 2019-09-11 Ionis Pharmaceuticals, Inc. PROCESS FOR REDUCING ATAXIN-2 EXPRESSION
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic agents
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
KR102585973B1 (ko) * 2018-07-03 2023-10-10 에프. 호프만-라 로슈 아게 타우 발현을 조절하기 위한 올리고뉴클레오티드
TWI843738B (zh) 2018-07-25 2024-06-01 美商Ionis製藥公司 用於減少atxn2表現之化合物及方法
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
US11286485B2 (en) 2019-04-04 2022-03-29 Hoffmann-La Roche Inc. Oligonucleotides for modulating ATXN2 expression
JP7668032B2 (ja) * 2019-10-24 2025-04-24 ジェネヴァント サイエンシズ ゲーエムベーハー 先端巨大症を処置するためのコンジュゲート及び方法
TW202442242A (zh) * 2023-04-21 2024-11-01 德商瑟卡爾納製藥有限兩合公司 Nrp1-特異性反義寡核苷酸及其等在預防及/或治療疾病的用途

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