CN108135922A - 用于治疗多囊肾病的方法 - Google Patents

用于治疗多囊肾病的方法 Download PDF

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Publication number
CN108135922A
CN108135922A CN201680049290.6A CN201680049290A CN108135922A CN 108135922 A CN108135922 A CN 108135922A CN 201680049290 A CN201680049290 A CN 201680049290A CN 108135922 A CN108135922 A CN 108135922A
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China
Prior art keywords
subject
mir
certain embodiments
modified oligonucleotide
modified
Prior art date
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Pending
Application number
CN201680049290.6A
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English (en)
Chinese (zh)
Inventor
J·R·安德罗萨维奇
B·N·周
V·D·帕特尔
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Regulus Therapeutics Inc
University of Texas System
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Regulus Therapeutics Inc
University of Texas System
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Application filed by Regulus Therapeutics Inc, University of Texas System filed Critical Regulus Therapeutics Inc
Priority to CN202111177217.6A priority Critical patent/CN114404440A/zh
Publication of CN108135922A publication Critical patent/CN108135922A/zh
Pending legal-status Critical Current

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/12Antihypertensives
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • C12N2310/113Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, 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)
CN201680049290.6A 2015-08-26 2016-08-25 用于治疗多囊肾病的方法 Pending CN108135922A (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202111177217.6A CN114404440A (zh) 2015-08-26 2016-08-25 用于治疗多囊肾病的方法

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CN202111177217.6A Division CN114404440A (zh) 2015-08-26 2016-08-25 用于治疗多囊肾病的方法

Publications (1)

Publication Number Publication Date
CN108135922A true CN108135922A (zh) 2018-06-08

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
CN201680049290.6A Pending CN108135922A (zh) 2015-08-26 2016-08-25 用于治疗多囊肾病的方法
CN202111177217.6A Pending CN114404440A (zh) 2015-08-26 2016-08-25 用于治疗多囊肾病的方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
CN202111177217.6A Pending CN114404440A (zh) 2015-08-26 2016-08-25 用于治疗多囊肾病的方法

Country Status (14)

Country Link
US (3) US10633657B2 (https=)
EP (2) EP4268891A3 (https=)
JP (1) JP6929269B2 (https=)
KR (2) KR20250049446A (https=)
CN (2) CN108135922A (https=)
AU (1) AU2016312590B2 (https=)
CA (1) CA2995996A1 (https=)
DK (1) DK3340993T5 (https=)
ES (1) ES2954151T3 (https=)
IL (1) IL257596B (https=)
MA (1) MA44836A (https=)
MX (1) MX2018002354A (https=)
RU (1) RU2742300C2 (https=)
WO (1) WO2017035319A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018106568A1 (en) * 2016-12-05 2018-06-14 Regulus Therapeutics Inc. Methods for treatment of polycystic kidney disease
IL322679A (en) * 2016-12-05 2025-10-01 Regulus Therapeutics Inc Modified oligonucleotides for the treatment of polycystic kidney disease
EP3841220A1 (en) 2018-08-23 2021-06-30 Roche Innovation Center Copenhagen A/S Microrna-134 biomarker
CA3234547A1 (en) 2021-10-08 2023-04-13 Regulus Therapeutics Inc. Methods and compositions for treatment of polycystic kidney disease
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)
IL324441A (en) * 2023-06-16 2026-01-01 Pyc Therapeutics Ltd Compounds and methods for treating kidney diseases
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 (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015123449A2 (en) * 2014-02-12 2015-08-20 Thomas Jefferson University Compositions and methods of using microrna inhibitors

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7683036B2 (en) * 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
NZ549535A (en) 2004-03-17 2010-11-26 Novartis Ag Use of aliskiren for treating renal and other disorders
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
EP3192788A1 (en) 2006-10-03 2017-07-19 Arbutus Biopharma Corporation Lipid containing formulations
US20090105174A1 (en) * 2007-04-20 2009-04-23 Amgen Inc. Nucleic acids hybridizable to micro rna and precursors thereof
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
US10660890B2 (en) * 2013-10-24 2020-05-26 National Institutes Of Health (Nih), U.S. Dept. Of Health And Human Services (Dhhs), U.S. Government Nih Division Of Extramural Inventions And Technology Resources (Deitr) Treatment of polycystic diseases with an HDAC6 inhibitor
WO2018106568A1 (en) 2016-12-05 2018-06-14 Regulus Therapeutics Inc. Methods for treatment of polycystic kidney disease
IL322679A (en) 2016-12-05 2025-10-01 Regulus Therapeutics Inc Modified oligonucleotides for the treatment of polycystic kidney disease

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015123449A2 (en) * 2014-02-12 2015-08-20 Thomas Jefferson University Compositions and methods of using microrna inhibitors

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
LAMA NOUREDDINE等: "MicroRNAs and Polycystic Kidney Disease", 《DRUG DISCOVERY TODAY. DISEASE MODELS》 *

Also Published As

Publication number Publication date
EP3340993B1 (en) 2023-06-14
AU2016312590B2 (en) 2021-12-02
NZ740040A (en) 2024-07-26
US20200231971A1 (en) 2020-07-23
HK1256883A1 (en) 2019-10-04
JP2018528945A (ja) 2018-10-04
RU2018108206A3 (https=) 2020-01-14
RU2018108206A (ru) 2019-09-27
WO2017035319A8 (en) 2021-03-11
US10633657B2 (en) 2020-04-28
ES2954151T3 (es) 2023-11-20
DK3340993T5 (da) 2024-10-07
WO2017035319A1 (en) 2017-03-02
DK3340993T3 (da) 2023-08-21
EP3340993A1 (en) 2018-07-04
CA2995996A1 (en) 2017-03-02
AU2016312590A1 (en) 2018-03-15
RU2742300C2 (ru) 2021-02-04
MX2018002354A (es) 2018-12-10
EP4268891A2 (en) 2023-11-01
EP4268891A3 (en) 2024-01-24
KR20250049446A (ko) 2025-04-11
IL257596B (en) 2022-08-01
US11168325B2 (en) 2021-11-09
KR20180080181A (ko) 2018-07-11
JP6929269B2 (ja) 2021-09-01
CN114404440A (zh) 2022-04-29
US20190153442A1 (en) 2019-05-23
IL257596A (en) 2018-04-30
US20220025372A1 (en) 2022-01-27
MA44836A (fr) 2018-07-04

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Application publication date: 20180608