DK3340993T5 - Forbindelse til anvendelse i fremgangsmåder til behandling af polycystisk nyresygdom - Google Patents

Forbindelse til anvendelse i fremgangsmåder til behandling af polycystisk nyresygdom Download PDF

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Publication number
DK3340993T5
DK3340993T5 DK16760627.6T DK16760627T DK3340993T5 DK 3340993 T5 DK3340993 T5 DK 3340993T5 DK 16760627 T DK16760627 T DK 16760627T DK 3340993 T5 DK3340993 T5 DK 3340993T5
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Denmark
Prior art keywords
compound
methods
kidney disease
polycystic kidney
treating polycystic
Prior art date
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DK16760627.6T
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Danish (da)
English (en)
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DK3340993T3 (da
Inventor
John R Androsavich
B Nelson Chau
Vishal Patel
Original Assignee
Regulus Therapeutics Inc
Univ Texas
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Publication of DK3340993T3 publication Critical patent/DK3340993T3/da
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Publication of DK3340993T5 publication Critical patent/DK3340993T5/da

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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)
DK16760627.6T 2015-08-26 2016-08-25 Forbindelse til anvendelse i fremgangsmåder til behandling af polycystisk nyresygdom DK3340993T5 (da)

Applications Claiming Priority (2)

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

Publications (2)

Publication Number Publication Date
DK3340993T3 DK3340993T3 (da) 2023-08-21
DK3340993T5 true DK3340993T5 (da) 2024-10-07

Family

ID=56855838

Family Applications (1)

Application Number Title Priority Date Filing Date
DK16760627.6T DK3340993T5 (da) 2015-08-26 2016-08-25 Forbindelse til anvendelse i fremgangsmåder til behandling af polycystisk nyresygdom

Country Status (14)

Country Link
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

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
MX2009003548A (es) 2006-10-03 2009-04-15 Alnylam Pharmaceuticals Inc Formulaciones que contienen lipidos.
US20090105174A1 (en) * 2007-04-20 2009-04-23 Amgen Inc. Nucleic acids hybridizable to micro rna and precursors thereof
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

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
RU2742300C2 (ru) 2021-02-04
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
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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