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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mir
certain embodiments
modified oligonucleotide
kidney
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RU2018108206A
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Russian (ru)
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RU2018108206A3 (enExample
RU2018108206A (ru
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Джон Р. АНДРОСАВИЧ
Б. Нельсон ЧАУ
Вишал Д. ПАТЕЛ
Original Assignee
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)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Urology & Nephrology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (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 (enExample) 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 (enExample)
EP (2) EP4268891A3 (enExample)
JP (1) JP6929269B2 (enExample)
KR (2) KR20250049446A (enExample)
CN (2) CN108135922A (enExample)
AU (1) AU2016312590B2 (enExample)
CA (1) CA2995996A1 (enExample)
DK (1) DK3340993T5 (enExample)
ES (1) ES2954151T3 (enExample)
IL (1) IL257596B (enExample)
MA (1) MA44836A (enExample)
MX (1) MX2018002354A (enExample)
RU (1) RU2742300C2 (enExample)
WO (1) WO2017035319A1 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3045461A1 (en) * 2016-12-05 2018-06-14 Regulus Therapeutics Inc. Methods for treatment of polycystic kidney disease
EP3548503A1 (en) * 2016-12-05 2019-10-09 Regulus Therapeutics Inc. Modified oligonucleotides for treatment of polycystic kidney disease
CN120519574A (zh) 2018-08-23 2025-08-22 罗氏创新中心哥本哈根有限公司 微小rna-134生物标志物
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)
WO2024254639A1 (en) * 2023-06-16 2024-12-19 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)

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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

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EP1648914A4 (en) * 2003-07-31 2009-12-16 Regulus Therapeutics Inc OLIGOMERIC COMPOUNDS AND COMPOSITIONS USEFUL FOR MODULATING SMALL NON-CODING RNA
WO2005089731A2 (en) 2004-03-17 2005-09-29 Novartis Ag Use of renin inhibitors in therapy
JPWO2007126150A1 (ja) * 2006-04-27 2009-09-17 国立大学法人名古屋大学 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
US8569381B2 (en) * 2008-05-23 2013-10-29 Targacept, Inc. Combination therapy for the management of 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
US20160362688A1 (en) * 2014-02-12 2016-12-15 Thomas Jefferson University Compositions and methods of using microrna inhibitors
CA3045461A1 (en) 2016-12-05 2018-06-14 Regulus Therapeutics Inc. Methods for treatment of polycystic kidney disease
EP3548503A1 (en) 2016-12-05 2019-10-09 Regulus Therapeutics Inc. Modified oligonucleotides 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. *

Also Published As

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

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