CO2024004227A2 - Métodos y composiciones para el tratamiento de la enfermedad renal poliquística - Google Patents

Métodos y composiciones para el tratamiento de la enfermedad renal poliquística

Info

Publication number
CO2024004227A2
CO2024004227A2 CONC2024/0004227A CO2024004227A CO2024004227A2 CO 2024004227 A2 CO2024004227 A2 CO 2024004227A2 CO 2024004227 A CO2024004227 A CO 2024004227A CO 2024004227 A2 CO2024004227 A2 CO 2024004227A2
Authority
CO
Colombia
Prior art keywords
kidney disease
polycystic kidney
treatment
methods
compositions
Prior art date
Application number
CONC2024/0004227A
Other languages
English (en)
Spanish (es)
Inventor
Denis Drygin
Garth A Kinberger
Edmund Chun Yu Lee
Original Assignee
Regulus Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Regulus Therapeutics Inc filed Critical Regulus Therapeutics Inc
Publication of CO2024004227A2 publication Critical patent/CO2024004227A2/es

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    • 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
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-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
    • C12N2310/3222'-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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/333Modified A
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • CCHEMISTRY; METALLURGY
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    • 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
    • C12N2310/33Chemical structure of the base
    • C12N2310/336Modified G

Landscapes

  • 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)
CONC2024/0004227A 2021-10-08 2024-04-05 Métodos y composiciones para el tratamiento de la enfermedad renal poliquística CO2024004227A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163253933P 2021-10-08 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
CO2024004227A2 true CO2024004227A2 (es) 2024-04-18

Family

ID=84246040

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2024/0004227A CO2024004227A2 (es) 2021-10-08 2024-04-05 Métodos y composiciones para el tratamiento de la enfermedad renal poliquística

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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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
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
GB0523659D0 (en) 2005-11-21 2005-12-28 Novartis Ag Organic compounds
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
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
US8691965B2 (en) 2007-06-14 2014-04-08 Mirx Therapeutics Aps Oligonucleotides for modulating target RNA activity
EP2623599B1 (en) 2007-10-04 2019-01-02 Roche Innovation Center Copenhagen A/S Micromirs
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
US20130236453A1 (en) 2012-03-12 2013-09-12 The Ohio State University Methods and Compositions for Modulating Acute Graft-versus-Host Disease using miR-155 Specific Inhibitors
WO2013165320A1 (en) 2012-05-04 2013-11-07 Agency For Science, Technology And Research Treating cancer by increasing expression of socs6
SG11201508925WA (en) * 2013-05-01 2015-11-27 Regulus Therapeutics Inc Microrna compounds and methods for modulating mir-122
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
DK3340993T5 (da) 2015-08-26 2024-10-07 Regulus Therapeutics Inc Forbindelse til anvendelse i fremgangsmåder til behandling af polycystisk nyresygdom
WO2018047148A1 (en) 2016-09-12 2018-03-15 Novartis Ag Compounds for the inhibition of mirna
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
US20200392503A1 (en) 2016-12-05 2020-12-17 Regulus Therapeutics Inc. Methods and compositions for treatment of polycystic kidney disease
US20220380767A1 (en) 2016-12-05 2022-12-01 Regulus Therapeutics Inc. Methods and compositions for treatment of polycystic kidney disease
EP4098746A4 (en) 2020-01-31 2024-01-24 National University Corporation Tokai National Higher Education and Research System SINGLE-STRANDED POLYNUCLEOTIDE
CA3233014A1 (en) 2021-10-08 2023-04-13 Denis Drygin Methods and compositions for avoiding off-target effects
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

Also Published As

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
KR20240073028A (ko) 2024-05-24
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
EP4413132A1 (en) 2024-08-14
TW202320808A (zh) 2023-06-01
US20250188466A1 (en) 2025-06-12

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