RU2742300C2 - Способы лечения поликистозной болезни почек - Google Patents

Способы лечения поликистозной болезни почек Download PDF

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RU2742300C2
RU2742300C2 RU2018108206A RU2018108206A RU2742300C2 RU 2742300 C2 RU2742300 C2 RU 2742300C2 RU 2018108206 A RU2018108206 A RU 2018108206A RU 2018108206 A RU2018108206 A RU 2018108206A RU 2742300 C2 RU2742300 C2 RU 2742300C2
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subject
mir
certain embodiments
modified oligonucleotide
kidney
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RU2018108206A
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Russian (ru)
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RU2018108206A (ru
RU2018108206A3 (cg-RX-API-DMAC7.html
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Джон Р. АНДРОСАВИЧ
Б. Нельсон ЧАУ
Вишал Д. ПАТЕЛ
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Регьюлэс Терапьютикс Инк.
Борд Оф Риджентс Оф Дзе Юниверсити Оф Техас Систем
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/12—Antihypertensives
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • 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
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Urology & Nephrology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
RU2018108206A 2015-08-26 2016-08-25 Способы лечения поликистозной болезни почек RU2742300C2 (ru)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562210031P 2015-08-26 2015-08-26
US62/210,031 2015-08-26
PCT/US2016/048603 WO2017035319A1 (en) 2015-08-26 2016-08-25 Methods for treatment of polycystic kidney disease

Publications (3)

Publication Number Publication Date
RU2018108206A RU2018108206A (ru) 2019-09-27
RU2018108206A3 RU2018108206A3 (cg-RX-API-DMAC7.html) 2020-01-14
RU2742300C2 true RU2742300C2 (ru) 2021-02-04

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RU2018108206A RU2742300C2 (ru) 2015-08-26 2016-08-25 Способы лечения поликистозной болезни почек

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US (3) US10633657B2 (cg-RX-API-DMAC7.html)
EP (2) EP3340993B1 (cg-RX-API-DMAC7.html)
JP (1) JP6929269B2 (cg-RX-API-DMAC7.html)
KR (2) KR20250049446A (cg-RX-API-DMAC7.html)
CN (2) CN108135922A (cg-RX-API-DMAC7.html)
AU (1) AU2016312590B2 (cg-RX-API-DMAC7.html)
CA (1) CA2995996A1 (cg-RX-API-DMAC7.html)
DK (1) DK3340993T5 (cg-RX-API-DMAC7.html)
ES (1) ES2954151T3 (cg-RX-API-DMAC7.html)
IL (1) IL257596B (cg-RX-API-DMAC7.html)
MA (1) MA44836A (cg-RX-API-DMAC7.html)
MX (1) MX2018002354A (cg-RX-API-DMAC7.html)
RU (1) RU2742300C2 (cg-RX-API-DMAC7.html)
WO (1) WO2017035319A1 (cg-RX-API-DMAC7.html)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102759999B1 (ko) * 2016-12-05 2025-02-03 레굴루스 테라퓨틱스 인크 다낭성 신장 질환의 치료를 위한 변형된 올리고뉴클레오타이드
EP3548011B1 (en) * 2016-12-05 2023-06-14 Regulus Therapeutics Inc. Methods for treatment of polycystic kidney disease
WO2020038968A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Microrna-134 biomarker
WO2024026354A2 (en) * 2022-07-27 2024-02-01 The General Hospital Corporation Methods and compositions for use in treating autosomal recessive polycystic kidney disease (arpkd)
AU2024304872A1 (en) * 2023-06-16 2025-12-18 PYC Therapeutics Limited Compositions and methods for treatment of kidney disease
WO2025110217A1 (ja) * 2023-11-22 2025-05-30 国立大学法人東海国立大学機構 嚢胞性腎疾患の予防及び／又は治療剤

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008042973A2 (en) * 2006-10-03 2008-04-10 Alnylam Pharmaceuticals, Inc. Lipid containing formulations
WO2008131191A2 (en) * 2007-04-20 2008-10-30 Amgen Inc. Nucleic acids hybridizable to micro rna and precursors thereof

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005013901A2 (en) * 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
CA2558020A1 (en) 2004-03-17 2005-09-29 Novartis Ag Use of renin inhibitors in therapy
WO2007126150A1 (ja) * 2006-04-27 2007-11-08 National University Corporation Nagoya University 癌の新規治療用組成物
US20120283411A9 (en) * 2006-06-29 2012-11-08 Ironwood Pharmaceuticals, Inc. Methods and compositions for the treatment of gastrointestinal disorders
WO2009143403A1 (en) * 2008-05-23 2009-11-26 Targacept, Inc. Combination therapy for the management hypertension
WO2014082644A1 (en) * 2012-11-30 2014-06-05 WULFF, Peter, Samuel Circular rna for inhibition of microrna
JP2016534069A (ja) * 2013-10-24 2016-11-04 メイヨ・ファウンデーション・フォー・メディカル・エデュケーション・アンド・リサーチ Ｈｄａｃ６阻害剤での多嚢胞性疾患の治療
JP2017511694A (ja) * 2014-02-12 2017-04-27 トーマス・ジェファーソン・ユニバーシティ マイクロｒｎａ阻害剤を使用するための組成物および方法
KR102759999B1 (ko) 2016-12-05 2025-02-03 레굴루스 테라퓨틱스 인크 다낭성 신장 질환의 치료를 위한 변형된 올리고뉴클레오타이드
EP3548011B1 (en) 2016-12-05 2023-06-14 Regulus Therapeutics Inc. Methods for treatment of polycystic kidney disease

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008042973A2 (en) * 2006-10-03 2008-04-10 Alnylam Pharmaceuticals, Inc. Lipid containing formulations
WO2008131191A2 (en) * 2007-04-20 2008-10-30 Amgen Inc. Nucleic acids hybridizable to micro rna and precursors thereof

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
LAMA NOUREDDINE, MicroRNA sand polycystic kidney disease, Drug Discovery Today: Disease Models, Volume 10, Issue 3, Autumn 2013, с. e137-e143 https://doi.org/10.1016/j.ddmod.2013.10.001. *
V. PATEL et al, "miR-17 92 miRNA cluster promotes kidney cyst growth in polycystic kidney disease", PNAS June 25, 2013, 110 (26), с. 10765-10770; https://doi.org/10.1073/pnas.1301693110. *
V. PATEL et al, "miR-17 92 miRNA cluster promotes kidney cyst growth in polycystic kidney disease", PNAS June 25, 2013, 110 (26), с. 10765-10770; https://doi.org/10.1073/pnas.1301693110. LAMA NOUREDDINE, MicroRNA sand polycystic kidney disease, Drug Discovery Today: Disease Models, Volume 10, Issue 3, Autumn 2013, с. e137-e143 https://doi.org/10.1016/j.ddmod.2013.10.001. *

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Publication number Publication date
AU2016312590A1 (en) 2018-03-15
CN114404440A (zh) 2022-04-29
US10633657B2 (en) 2020-04-28
EP3340993A1 (en) 2018-07-04
EP3340993B1 (en) 2023-06-14
ES2954151T3 (es) 2023-11-20
RU2018108206A (ru) 2019-09-27
CN108135922A (zh) 2018-06-08
MA44836A (fr) 2018-07-04
US11168325B2 (en) 2021-11-09
AU2016312590B2 (en) 2021-12-02
WO2017035319A8 (en) 2021-03-11
RU2018108206A3 (cg-RX-API-DMAC7.html) 2020-01-14
IL257596A (en) 2018-04-30
CA2995996A1 (en) 2017-03-02
IL257596B (en) 2022-08-01
EP4268891A3 (en) 2024-01-24
KR20250049446A (ko) 2025-04-11
JP6929269B2 (ja) 2021-09-01
US20220025372A1 (en) 2022-01-27
DK3340993T3 (da) 2023-08-21
DK3340993T5 (da) 2024-10-07
US20200231971A1 (en) 2020-07-23
HK1256883A1 (en) 2019-10-04
WO2017035319A1 (en) 2017-03-02
US20190153442A1 (en) 2019-05-23
EP4268891A2 (en) 2023-11-01
MX2018002354A (es) 2018-12-10
JP2018528945A (ja) 2018-10-04
KR20180080181A (ko) 2018-07-11
NZ740040A (en) 2024-07-26

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