AU2017372695B2 - Methods for treatment of polycystic kidney disease - Google Patents

Methods for treatment of polycystic kidney disease Download PDF

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Publication number
AU2017372695B2
AU2017372695B2 AU2017372695A AU2017372695A AU2017372695B2 AU 2017372695 B2 AU2017372695 B2 AU 2017372695B2 AU 2017372695 A AU2017372695 A AU 2017372695A AU 2017372695 A AU2017372695 A AU 2017372695A AU 2017372695 B2 AU2017372695 B2 AU 2017372695B2
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Australia
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kidney
mir
certain embodiments
modified oligonucleotide
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AU2017372695A
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AU2017372695A1 (en
Inventor
Charles R. ALLERSON
John R. ANDROSAVICH
B. Nelson Chau
Vishal D. PATEL
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Regulus Therapeutics Inc
University of Texas System
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Regulus Therapeutics Inc
University of Texas System
University of Texas at Austin
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    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/436—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/496—Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/517—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with carbocyclic ring systems, e.g. quinazoline, perimidine
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/55—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
    • 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
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/58—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids containing heterocyclic rings, e.g. danazol, stanozolol, pancuronium or digitogenin
    • A61K31/585—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids containing heterocyclic rings, e.g. danazol, stanozolol, pancuronium or digitogenin containing lactone rings, e.g. oxandrolone, bufalin
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22—Hormones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22—Hormones
    • A61K38/31—Somatostatins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/08—Solutions
    • 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
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2310/30—Chemical structure
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    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
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  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
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  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Endocrinology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Urology & Nephrology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
AU2017372695A 2016-12-05 2017-12-04 Methods for treatment of polycystic kidney disease Active AU2017372695B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662430164P 2016-12-05 2016-12-05
US62/430,164 2016-12-05
PCT/US2017/064432 WO2018106568A1 (en) 2016-12-05 2017-12-04 Methods for treatment of polycystic kidney disease

Publications (2)

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AU2017372695A1 AU2017372695A1 (en) 2019-06-13
AU2017372695B2 true AU2017372695B2 (en) 2023-07-20

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AU2017372695A Active AU2017372695B2 (en) 2016-12-05 2017-12-04 Methods for treatment of polycystic kidney disease

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US (4) US20190345494A1 (cg-RX-API-DMAC7.html)
EP (1) EP3548011B1 (cg-RX-API-DMAC7.html)
JP (1) JP7105775B2 (cg-RX-API-DMAC7.html)
KR (2) KR20240148005A (cg-RX-API-DMAC7.html)
CN (1) CN110225747B (cg-RX-API-DMAC7.html)
AU (1) AU2017372695B2 (cg-RX-API-DMAC7.html)
CA (1) CA3045461A1 (cg-RX-API-DMAC7.html)
CL (1) CL2019001526A1 (cg-RX-API-DMAC7.html)
EA (1) EA201991370A1 (cg-RX-API-DMAC7.html)
IL (1) IL266989B2 (cg-RX-API-DMAC7.html)
MA (1) MA46970A (cg-RX-API-DMAC7.html)
MX (1) MX2019006337A (cg-RX-API-DMAC7.html)
MY (1) MY202466A (cg-RX-API-DMAC7.html)
WO (1) WO2018106568A1 (cg-RX-API-DMAC7.html)
ZA (1) ZA201903608B (cg-RX-API-DMAC7.html)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA44836A (fr) 2015-08-26 2018-07-04 Univ Texas Procédés de traitement du syndrome polykystique des reins
WO2020038968A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Microrna-134 biomarker
KR20240073028A (ko) * 2021-10-08 2024-05-24 레굴루스 테라퓨틱스 인크 다낭성 신장 질환의 치료를 위한 방법 및 조성물
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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WO2005103298A2 (en) * 2004-04-20 2005-11-03 Genaco Biomedical Products, Inc. Method for detecting ncrna
WO2008151639A2 (en) * 2007-06-14 2008-12-18 Querdenker Aps Oligonucleotides for modulation of target rna activity
WO2009109665A1 (en) * 2008-03-07 2009-09-11 Santaris Pharma A/S Pharmaceutical compositions for treatment of microrna related diseases
WO2011060100A1 (en) * 2009-11-11 2011-05-19 Sanford-Burnham Medical Research Institute Method for generation and regulation of ips cells and compositions thereof
WO2015123449A2 (en) * 2014-02-12 2015-08-20 Thomas Jefferson University Compositions and methods of using microrna inhibitors

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AU5417201A (en) * 1996-05-24 2001-09-20 Genzyme Corporation Polycystic kidney disease gene
GB0523659D0 (en) * 2005-11-21 2005-12-28 Novartis Ag Organic compounds
CA2927045A1 (en) 2006-10-03 2008-04-10 Muthiah Manoharan Lipid containing formulations
MX2010003299A (es) * 2007-10-04 2010-04-14 Santaris Pharma As Oligonucleotidos muy cortos (micromirs).
CA2888462A1 (en) * 2012-10-19 2014-04-14 Synta Pharmaceuticals Corp. Treating polycystic kidney disease with hsp90 inhibitory compounds
MA44836A (fr) * 2015-08-26 2018-07-04 Univ Texas Procédés de traitement du syndrome polykystique des reins
US11157828B2 (en) * 2016-12-08 2021-10-26 Microsoft Technology Licensing, Llc Tomography and generative data modeling via quantum boltzmann training

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WO2005103298A2 (en) * 2004-04-20 2005-11-03 Genaco Biomedical Products, Inc. Method for detecting ncrna
WO2008151639A2 (en) * 2007-06-14 2008-12-18 Querdenker Aps Oligonucleotides for modulation of target rna activity
WO2009109665A1 (en) * 2008-03-07 2009-09-11 Santaris Pharma A/S Pharmaceutical compositions for treatment of microrna related diseases
WO2011060100A1 (en) * 2009-11-11 2011-05-19 Sanford-Burnham Medical Research Institute Method for generation and regulation of ips cells and compositions thereof
WO2015123449A2 (en) * 2014-02-12 2015-08-20 Thomas Jefferson University Compositions and methods of using microrna inhibitors

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ANDRIUS SERVA, BETTINA KNAPP, YUEH-TSO TSAI, CHRISTOPH CLAAS, TAUTVYDAS LISAUSKAS, PETR MATULA, NATHALIE HARDER, LARS KADERALI, KA: "miR-17-5p Regulates Endocytic Trafficking through Targeting TBC1D2/Armus", PLOS ONE, PUBLIC LIBRARY OF SCIENCE, vol. 7, no. 12, 1 January 2012 (2012-01-01), pages e52555 - e52555, XP055111293, ISSN: 19326203, DOI: 10.1371/journal.pone.0052555 *
HUAN SUN ET AL: "MicroRNA-17 post-transcriptionally regulates polycystic kidney disease-2 gene and promotes cell proliferation", MOLECULAR BIOLOGY REPORTS, vol. 37, no. 6, (2009), pages 2951 - 2958 *
V. PATEL ET AL: "miR-17?92 miRNA cluster promotes kidney cyst growth in polycystic kidney disease", PROCEEDINGS NATIONAL ACADEMY OF SCIENCES PNAS, vol. 110, no. 26, 12 June 2013 (2013-06-12), US, pages 10765 - 10770, *

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MY202466A (en) 2024-04-30
JP7105775B2 (ja) 2022-07-25
US20200392501A1 (en) 2020-12-17
JP2019536803A (ja) 2019-12-19
KR20240148005A (ko) 2024-10-10
EA201991370A1 (ru) 2019-12-30
BR112019011597A2 (pt) 2019-10-22
CN110225747A (zh) 2019-09-10
CL2019001526A1 (es) 2019-10-18
ZA201903608B (en) 2021-10-27
AU2017372695A1 (en) 2019-06-13
KR20190093207A (ko) 2019-08-08
EP3548011B1 (en) 2023-06-14
MA46970A (fr) 2019-10-09
EP3548011A1 (en) 2019-10-09
US20190345494A1 (en) 2019-11-14
MX2019006337A (es) 2019-09-26
IL266989A (en) 2019-07-31
US20240141350A1 (en) 2024-05-02
CN110225747B (zh) 2025-04-29
IL266989B2 (en) 2024-12-01
US20220213483A1 (en) 2022-07-07
WO2018106568A1 (en) 2018-06-14
CA3045461A1 (en) 2018-06-14
KR102780365B1 (ko) 2025-03-18
IL266989B1 (en) 2024-08-01

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