CL2024000990A1 - Métodos y composiciones para el tratamiento de la enfermedad renal poliquística - Google Patents

Métodos y composiciones para el tratamiento de la enfermedad renal poliquística

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Publication number
CL2024000990A1
CL2024000990A1 CL2024000990A CL2024000990A CL2024000990A1 CL 2024000990 A1 CL2024000990 A1 CL 2024000990A1 CL 2024000990 A CL2024000990 A CL 2024000990A CL 2024000990 A CL2024000990 A CL 2024000990A CL 2024000990 A1 CL2024000990 A1 CL 2024000990A1
Authority
CL
Chile
Prior art keywords
kidney disease
polycystic kidney
treatment
methods
compositions
Prior art date
Application number
CL2024000990A
Other languages
English (en)
Spanish (es)
Inventor
Drygin Denis
A Kinberger Garth
Chun Yu Lee Edmund
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 CL2024000990A1 publication Critical patent/CL2024000990A1/es

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    • CCHEMISTRY; METALLURGY
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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
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    • 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
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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

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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)
CL2024000990A 2021-10-08 2024-04-03 Métodos y composiciones para el tratamiento de la enfermedad renal poliquística CL2024000990A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202163253933P 2021-10-08 2021-10-08

Publications (1)

Publication Number Publication Date
CL2024000990A1 true CL2024000990A1 (es) 2024-09-27

Family

ID=84246040

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2024000990A CL2024000990A1 (es) 2021-10-08 2024-04-03 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
WO2023060237A1 (en) 2021-10-08 2023-04-13 Regulus Therapeutics Inc. Methods and compositions for treatment of polycystic kidney disease
KR20260005213A (ko) * 2023-04-12 2026-01-09 레굴루스 테라퓨틱스 인크 다낭성 신장 질환 치료를 위한 변형 올리고뉴클레오타이드
EP4680746A1 (en) * 2023-06-16 2026-01-21 Pyc Therapeutics Limited Compositions and methods for treatment of kidney disease
WO2025193584A1 (en) * 2024-03-11 2025-09-18 Regulus Therapeutics Inc. Compound for use in methods for treatment of polycystic kidney disease

Family Cites Families (33)

* Cited by examiner, † Cited by third party
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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
BRPI0509979A (pt) 2004-04-20 2007-10-16 Genaco Biomedical Products Inc método para detectar ncrna
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
EP2007888A2 (en) 2006-04-03 2008-12-31 Santaris Pharma A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
WO2007126150A1 (ja) 2006-04-27 2007-11-08 National University Corporation Nagoya University 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
CA2927045A1 (en) 2006-10-03 2008-04-10 Muthiah Manoharan Lipid containing formulations
WO2008131191A2 (en) 2007-04-20 2008-10-30 Amgen Inc. Nucleic acids hybridizable to micro rna and precursors thereof
EP2714904B1 (en) 2007-06-14 2017-04-12 Mirx Therapeutics ApS Oligonucleotides for modulation of target rna activity
EP2623599B1 (en) 2007-10-04 2019-01-02 Roche Innovation Center Copenhagen A/S Micromirs
US8211867B2 (en) * 2007-10-29 2012-07-03 Regulus Therapeutics Inc. Targeting microRNAs for the treatment of liver cancer
CA2717792A1 (en) * 2008-03-07 2009-09-11 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
AU2010319555A1 (en) 2009-11-11 2012-06-14 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
SG10201803157XA (en) * 2013-05-01 2018-05-30 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
JP2017511694A (ja) 2014-02-12 2017-04-27 トーマス・ジェファーソン・ユニバーシティ マイクロrna阻害剤を使用するための組成物および方法
JP6929269B2 (ja) 2015-08-26 2021-09-01 レグルス セラピューティクス インコーポレイテッド 多発性嚢胞腎の処置のための方法
WO2018047148A1 (en) 2016-09-12 2018-03-15 Novartis Ag Compounds for the inhibition of mirna
WO2018106566A1 (en) 2016-12-05 2018-06-14 Regulus Therapeutics Inc. Modified oligonucleotides for treatment of polycystic kidney disease
AU2017372695B2 (en) 2016-12-05 2023-07-20 Regulus Therapeutics Inc. Methods for 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
WO2023060237A1 (en) 2021-10-08 2023-04-13 Regulus Therapeutics Inc. Methods and compositions for treatment of polycystic kidney disease
JP2024535445A (ja) 2021-10-08 2024-09-30 レグルス セラピューティクス インコーポレイテッド オフターゲット効果を回避するための方法及び組成物
KR20260005213A (ko) 2023-04-12 2026-01-09 레굴루스 테라퓨틱스 인크 다낭성 신장 질환 치료를 위한 변형 올리고뉴클레오타이드

Also Published As

Publication number Publication date
US20240294913A1 (en) 2024-09-05
JP2024537043A (ja) 2024-10-10
WO2023060237A1 (en) 2023-04-13
TW202320808A (zh) 2023-06-01
IL311734A (en) 2024-05-01
CA3234547A1 (en) 2023-04-13
KR20240073028A (ko) 2024-05-24
AU2022361062A1 (en) 2024-03-21
US20250188466A1 (en) 2025-06-12
CO2024004227A2 (es) 2024-04-18
AR127325A1 (es) 2024-01-10
CN118103510A (zh) 2024-05-28
US12559750B2 (en) 2026-02-24
MX2024003529A (es) 2024-04-01
EP4413132A1 (en) 2024-08-14

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