KR20250049446A - 다낭포성 신장 질환의 치료 방법 - Google Patents

다낭포성 신장 질환의 치료 방법 Download PDF

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Publication number
KR20250049446A
KR20250049446A KR1020257010343A KR20257010343A KR20250049446A KR 20250049446 A KR20250049446 A KR 20250049446A KR 1020257010343 A KR1020257010343 A KR 1020257010343A KR 20257010343 A KR20257010343 A KR 20257010343A KR 20250049446 A KR20250049446 A KR 20250049446A
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South Korea
Prior art keywords
certain embodiments
mir
subject
modified oligonucleotide
modified
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KR1020257010343A
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Korean (ko)
Inventor
존 알. 안드로사비치
비. 넬슨 차우
비샬 디. 파텔
Original Assignee
레굴루스 테라퓨틱스 인크
보드 오브 리전츠, 더 유니버시티 오브 텍사스 시스템
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Publication of KR20250049446A publication Critical patent/KR20250049446A/ko
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
    • 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
    • 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
    • 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)
KR1020257010343A 2015-08-26 2016-08-25 다낭포성 신장 질환의 치료 방법 Pending KR20250049446A (ko)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562210031P 2015-08-26 2015-08-26
US62/210,031 2015-08-26
KR1020187007997A KR20180080181A (ko) 2015-08-26 2016-08-25 다낭포성 신장 질환의 치료 방법
PCT/US2016/048603 WO2017035319A1 (en) 2015-08-26 2016-08-25 Methods for treatment of polycystic kidney disease

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
KR1020187007997A Division KR20180080181A (ko) 2015-08-26 2016-08-25 다낭포성 신장 질환의 치료 방법

Publications (1)

Publication Number Publication Date
KR20250049446A true KR20250049446A (ko) 2025-04-11

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
KR1020257010343A Pending KR20250049446A (ko) 2015-08-26 2016-08-25 다낭포성 신장 질환의 치료 방법
KR1020187007997A Ceased KR20180080181A (ko) 2015-08-26 2016-08-25 다낭포성 신장 질환의 치료 방법

Family Applications After (1)

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KR1020187007997A Ceased KR20180080181A (ko) 2015-08-26 2016-08-25 다낭포성 신장 질환의 치료 방법

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

Family Cites Families (12)

* 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
EP3105327A4 (en) * 2014-02-12 2017-10-18 Thomas Jefferson University Compositions and methods of using microrna inhibitors
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

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
CN108135922A (zh) 2018-06-08
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
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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Patent event date: 20250328

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Filing date: 20180321

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Patent event date: 20250425

Comment text: Request for Examination of Application