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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subject
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
The University of Texas System
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Regulus Therapeutics Inc
The University of Texas System
The University of Texas at Austin
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    • 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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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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
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    • 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
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  • Health & Medical Sciences (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Endocrinology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Urology & Nephrology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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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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US (5) US20190345494A1 (enExample)
EP (1) EP3548011B1 (enExample)
JP (1) JP7105775B2 (enExample)
KR (2) KR102780365B1 (enExample)
CN (1) CN110225747B (enExample)
AU (1) AU2017372695B2 (enExample)
CA (1) CA3045461A1 (enExample)
CL (1) CL2019001526A1 (enExample)
EA (1) EA201991370A1 (enExample)
IL (1) IL266989B2 (enExample)
MA (1) MA46970A (enExample)
MX (1) MX2019006337A (enExample)
MY (1) MY202466A (enExample)
NZ (2) NZ794203A (enExample)
WO (1) WO2018106568A1 (enExample)
ZA (1) ZA201903608B (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2742300C2 (ru) 2015-08-26 2021-02-04 Регьюлэс Терапьютикс Инк. Способы лечения поликистозной болезни почек
EP3841220A1 (en) 2018-08-23 2021-06-30 Roche Innovation Center Copenhagen A/S Microrna-134 biomarker
WO2023060237A1 (en) * 2021-10-08 2023-04-13 Regulus Therapeutics Inc. Methods and compositions for treatment of polycystic kidney disease
WO2024254639A1 (en) 2023-06-16 2024-12-19 PYC Therapeutics Limited Compositions and methods for treatment of kidney disease
TW202539703A (zh) * 2024-03-11 2025-10-16 美商雷格勒斯治療公司 治療多囊性腎病之方法

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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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DK2205737T3 (da) * 2007-10-04 2013-05-21 Santaris Pharma As Mikromirer
WO2014063080A1 (en) * 2012-10-19 2014-04-24 Synta Pharmaceuticals Corp. Treating polycystic kidney disease with hsp90 inhibitory compounds
RU2742300C2 (ru) 2015-08-26 2021-02-04 Регьюлэс Терапьютикс Инк. Способы лечения поликистозной болезни почек
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 *
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KR20190093207A (ko) 2019-08-08
NZ753811A (en) 2026-03-27
US20220213483A1 (en) 2022-07-07
EA201991370A1 (ru) 2019-12-30
EP3548011A1 (en) 2019-10-09
MA46970A (fr) 2019-10-09
CL2019001526A1 (es) 2019-10-18
IL266989B2 (en) 2024-12-01
IL266989A (en) 2019-07-31
BR112019011597A2 (pt) 2019-10-22
EP3548011B1 (en) 2023-06-14
MY202466A (en) 2024-04-30
CN110225747A (zh) 2019-09-10
MX2019006337A (es) 2019-09-26
JP2019536803A (ja) 2019-12-19
AU2017372695A1 (en) 2019-06-13
US20190345494A1 (en) 2019-11-14
US20240141350A1 (en) 2024-05-02
IL266989B1 (en) 2024-08-01
JP7105775B2 (ja) 2022-07-25
CN110225747B (zh) 2025-04-29
NZ794203A (en) 2026-03-27
ZA201903608B (en) 2021-10-27
US20200392501A1 (en) 2020-12-17
CA3045461A1 (en) 2018-06-14
WO2018106568A1 (en) 2018-06-14
US20260055406A1 (en) 2026-02-26
KR102780365B1 (ko) 2025-03-18
KR20240148005A (ko) 2024-10-10

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