KR20240073028A - 다낭성 신장 질환의 치료를 위한 방법 및 조성물 - Google Patents

다낭성 신장 질환의 치료를 위한 방법 및 조성물 Download PDF

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Publication number
KR20240073028A
KR20240073028A KR1020247010026A KR20247010026A KR20240073028A KR 20240073028 A KR20240073028 A KR 20240073028A KR 1020247010026 A KR1020247010026 A KR 1020247010026A KR 20247010026 A KR20247010026 A KR 20247010026A KR 20240073028 A KR20240073028 A KR 20240073028A
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South Korea
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subject
nucleobase
modified oligonucleotide
compound
certain embodiments
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Korean (ko)
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데니스 드라이진
가스 에이. 킨베르거
에드먼드 천 유 리
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레굴루스 테라퓨틱스 인크
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Publication of KR20240073028A publication Critical patent/KR20240073028A/ko
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    • 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
    • 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
    • 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
    • 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
    • C12N2310/321—2'-O-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
    • C12N2310/322—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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/333—Modified A
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/336—Modified G

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Urology & Nephrology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020247010026A 2021-10-08 2022-10-07 다낭성 신장 질환의 치료를 위한 방법 및 조성물 Pending KR20240073028A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163253933P 2021-10-08 2021-10-08
US63/253,933 2021-10-08
PCT/US2022/077766 WO2023060237A1 (en) 2021-10-08 2022-10-07 Methods and compositions for treatment of polycystic kidney disease

Publications (1)

Publication Number Publication Date
KR20240073028A true KR20240073028A (ko) 2024-05-24

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ID=84246040

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KR1020247010026A Pending KR20240073028A (ko) 2021-10-08 2022-10-07 다낭성 신장 질환의 치료를 위한 방법 및 조성물

Country Status (14)

Country Link
US (2) US20240294913A1 (https=)
EP (1) EP4413132A1 (https=)
JP (1) JP2024537043A (https=)
KR (1) KR20240073028A (https=)
CN (1) CN118103510A (https=)
AR (1) AR127325A1 (https=)
AU (1) AU2022361062A1 (https=)
CA (1) CA3234547A1 (https=)
CL (1) CL2024000990A1 (https=)
CO (1) CO2024004227A2 (https=)
IL (1) IL311734A (https=)
MX (1) MX2024003529A (https=)
TW (1) TW202320808A (https=)
WO (1) WO2023060237A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3234547A1 (en) 2021-10-08 2023-04-13 Regulus Therapeutics Inc. Methods and compositions for treatment of polycystic kidney disease
WO2024215846A1 (en) * 2023-04-12 2024-10-17 Regulus Therapeutics Inc. Modified oligonucleotide for treating polycystic kidney disease
IL324441A (en) * 2023-06-16 2026-01-01 Pyc Therapeutics Ltd Compounds and methods for treating kidney diseases
WO2025193584A1 (en) * 2024-03-11 2025-09-18 Regulus Therapeutics Inc. Compound for use in methods for treatment of polycystic kidney disease

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NZ549535A (en) 2004-03-17 2010-11-26 Novartis Ag Use of aliskiren for treating renal and other disorders
CN101056991A (zh) 2004-04-20 2007-10-17 基因耐克生物医学制品有限公司 检测ncRNA的方法
EP1791567B1 (en) 2004-08-10 2015-07-29 Alnylam Pharmaceuticals Inc. Chemically modified oligonucleotides
US20070213292A1 (en) * 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
US20070092882A1 (en) * 2005-10-21 2007-04-26 Hui Wang Analysis of microRNA
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CA2648132C (en) 2006-04-03 2019-05-28 Santaris Pharma A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
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
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EP2217248B1 (en) * 2007-10-29 2016-11-30 Regulus Therapeutics Inc. Targeting micrornas for the treatment of liver cancer
EP2268811A1 (en) * 2008-03-07 2011-01-05 Santaris Pharma A/S Pharmaceutical compositions for treatment of microrna related diseases
WO2009143403A1 (en) 2008-05-23 2009-11-26 Targacept, Inc. Combination therapy for the management hypertension
EP2499239A1 (en) 2009-11-11 2012-09-19 Sanford-Burnham Medical Research Institute METHOD FOR GENERATION AND REGULATION OF iPS CELLS AND COMPOSITIONS THEREOF
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IL322679A (en) * 2016-12-05 2025-10-01 Regulus Therapeutics Inc Modified oligonucleotides for the treatment of polycystic kidney disease
US20200392503A1 (en) 2016-12-05 2020-12-17 Regulus Therapeutics Inc. Methods and compositions for treatment of polycystic kidney disease
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CA3234547A1 (en) 2021-10-08 2023-04-13 Regulus Therapeutics Inc. Methods and compositions for treatment of polycystic kidney disease
WO2024215846A1 (en) 2023-04-12 2024-10-17 Regulus Therapeutics Inc. Modified oligonucleotide for treating polycystic kidney disease

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Publication number Publication date
US20240294913A1 (en) 2024-09-05
JP2024537043A (ja) 2024-10-10
CA3234547A1 (en) 2023-04-13
WO2023060237A1 (en) 2023-04-13
CN118103510A (zh) 2024-05-28
AR127325A1 (es) 2024-01-10
US12559750B2 (en) 2026-02-24
AU2022361062A1 (en) 2024-03-21
IL311734A (en) 2024-05-01
MX2024003529A (es) 2024-04-01
CL2024000990A1 (es) 2024-09-27
CO2024004227A2 (es) 2024-04-18
EP4413132A1 (en) 2024-08-14
TW202320808A (zh) 2023-06-01
US20250188466A1 (en) 2025-06-12

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Patent event date: 20240326

Patent event code: PA01051R01D

Comment text: International Patent Application

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