CN114404440A - 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
CN114404440A
CN114404440A CN202111177217.6A CN202111177217A CN114404440A CN 114404440 A CN114404440 A CN 114404440A CN 202111177217 A CN202111177217 A CN 202111177217A CN 114404440 A CN114404440 A CN 114404440A
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China
Prior art keywords
mir
certain embodiments
subject
modified oligonucleotide
modified
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Pending
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CN202111177217.6A
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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
The University of Texas System
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Regulus Therapeutics Inc
The University of Texas System
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Publication of CN114404440A publication Critical patent/CN114404440A/zh
Pending legal-status Critical Current

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

Applications Claiming Priority (4)

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
CN201680049290.6A CN108135922A (zh) 2015-08-26 2016-08-25 用于治疗多囊肾病的方法

Related Parent Applications (1)

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

Publications (1)

Publication Number Publication Date
CN114404440A true CN114404440A (zh) 2022-04-29

Family

ID=56855838

Family Applications (2)

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
CN201680049290.6A Pending CN108135922A (zh) 2015-08-26 2016-08-25 用于治疗多囊肾病的方法

Family Applications After (1)

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CN201680049290.6A Pending CN108135922A (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 (cg-RX-API-DMAC7.html)
EP (2) EP4268891A3 (cg-RX-API-DMAC7.html)
JP (1) JP6929269B2 (cg-RX-API-DMAC7.html)
KR (2) KR20250049446A (cg-RX-API-DMAC7.html)
CN (2) CN114404440A (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 (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3548011B1 (en) * 2016-12-05 2023-06-14 Regulus Therapeutics Inc. Methods for treatment of polycystic kidney disease
TWI769197B (zh) * 2016-12-05 2022-07-01 美商雷格勒斯治療公司 用於治療多囊腎病之組成物
EP3841220A1 (en) 2018-08-23 2021-06-30 Roche Innovation Center Copenhagen A/S Microrna-134 biomarker
WO2023060237A1 (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)
WO2024254639A1 (en) * 2023-06-16 2024-12-19 PYC Therapeutics Limited Compositions and methods for treatment of kidney disease
CN122295113A (zh) * 2023-11-22 2026-06-26 国立大学法人东海国立大学机构 囊性肾病的预防和/或治疗剂

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)

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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
RU2426532C2 (ru) 2004-03-17 2011-08-20 Новартис Аг Применение органических соединений
WO2007126150A1 (ja) * 2006-04-27 2007-11-08 National University Corporation Nagoya University 癌の新規治療用組成物
WO2008002971A2 (en) * 2006-06-29 2008-01-03 Microbia, Inc. Methods and compositions for the treatment of gastrointestinal disorders
CA3144493A1 (en) 2006-10-03 2008-04-10 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
US20150299702A1 (en) * 2012-11-30 2015-10-22 Aarhus Universitet Circular rna for inhibition of microrna
EP3060217B1 (en) * 2013-10-24 2022-06-08 Mayo Foundation for Medical Education and Research Treatment of polycystic diseases with an hdac6 inhibitor
EP3548011B1 (en) 2016-12-05 2023-06-14 Regulus Therapeutics Inc. Methods for treatment of polycystic kidney disease
TWI769197B (zh) 2016-12-05 2022-07-01 美商雷格勒斯治療公司 用於治療多囊腎病之組成物

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 (2)

* Cited by examiner, † Cited by third party
Title
HUAN SUN等: "MicroRNA-17 post-transcriptionally regulates polycystic kidney disease-2 gene and promotes cell proliferation" *
LAMA NOUREDDINE等: ""MicroRNAs and Polycystic Kidney Disease"" *

Also Published As

Publication number Publication date
RU2018108206A (ru) 2019-09-27
NZ740040A (en) 2024-07-26
JP2018528945A (ja) 2018-10-04
KR20250049446A (ko) 2025-04-11
IL257596A (en) 2018-04-30
US11168325B2 (en) 2021-11-09
RU2018108206A3 (cg-RX-API-DMAC7.html) 2020-01-14
CA2995996A1 (en) 2017-03-02
IL257596B (en) 2022-08-01
AU2016312590B2 (en) 2021-12-02
US10633657B2 (en) 2020-04-28
ES2954151T3 (es) 2023-11-20
WO2017035319A1 (en) 2017-03-02
KR20180080181A (ko) 2018-07-11
US20190153442A1 (en) 2019-05-23
EP4268891A2 (en) 2023-11-01
HK1256883A1 (en) 2019-10-04
AU2016312590A1 (en) 2018-03-15
DK3340993T5 (da) 2024-10-07
EP4268891A3 (en) 2024-01-24
EP3340993A1 (en) 2018-07-04
DK3340993T3 (da) 2023-08-21
EP3340993B1 (en) 2023-06-14
US20220025372A1 (en) 2022-01-27
MX2018002354A (es) 2018-12-10
WO2017035319A8 (en) 2021-03-11
JP6929269B2 (ja) 2021-09-01
US20200231971A1 (en) 2020-07-23
CN108135922A (zh) 2018-06-08
RU2742300C2 (ru) 2021-02-04
MA44836A (fr) 2018-07-04

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