CA3045461A1 - Methods for treatment of polycystic kidney disease - Google Patents

Methods for treatment of polycystic kidney disease Download PDF

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Publication number
CA3045461A1
CA3045461A1 CA3045461A CA3045461A CA3045461A1 CA 3045461 A1 CA3045461 A1 CA 3045461A1 CA 3045461 A CA3045461 A CA 3045461A CA 3045461 A CA3045461 A CA 3045461A CA 3045461 A1 CA3045461 A1 CA 3045461A1
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CA
Canada
Prior art keywords
subject
modified oligonucleotide
kidney disease
kidney
polycystic kidney
Prior art date
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Pending
Application number
CA3045461A
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English (en)
French (fr)
Inventor
Charles R. ALLERSON
Vishal D. PATEL
B. Nelson Chau
John R. ANDROSAVICH
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Regulus Therapeutics Inc
University of Texas System
Original Assignee
Regulus Therapeutics Inc
University of Texas System
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Filing date
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Application filed by Regulus Therapeutics Inc, University of Texas System filed Critical Regulus Therapeutics Inc
Publication of CA3045461A1 publication Critical patent/CA3045461A1/en
Pending legal-status Critical Current

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    • 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
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/4353Heterocyclic 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
    • A61K31/436Heterocyclic 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic 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
    • A61K31/496Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic 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
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/517Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with carbocyclic ring systems, e.g. quinazoline, perimidine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/55Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/56Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/58Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids containing heterocyclic rings, e.g. danazol, stanozolol, pancuronium or digitogenin
    • A61K31/585Compounds 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
    • AHUMAN NECESSITIES
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    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22Hormones
    • AHUMAN NECESSITIES
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    • A61K38/22Hormones
    • A61K38/31Somatostatins
    • AHUMAN NECESSITIES
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    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
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    • A61K9/08Solutions
    • AHUMAN NECESSITIES
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    • 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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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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
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    • 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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
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    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/3521Methyl
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    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen

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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Endocrinology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Urology & Nephrology (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CA3045461A 2016-12-05 2017-12-04 Methods for treatment of polycystic kidney disease Pending CA3045461A1 (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 (1)

Publication Number Publication Date
CA3045461A1 true CA3045461A1 (en) 2018-06-14

Family

ID=61074503

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3045461A Pending CA3045461A1 (en) 2016-12-05 2017-12-04 Methods for treatment of polycystic kidney disease

Country Status (15)

Country Link
US (4) US20190345494A1 (enExample)
EP (1) EP3548011B1 (enExample)
JP (1) JP7105775B2 (enExample)
KR (2) KR20240148005A (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)
WO (1) WO2018106568A1 (enExample)
ZA (1) ZA201903608B (enExample)

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
CN120519574A (zh) 2018-08-23 2025-08-22 罗氏创新中心哥本哈根有限公司 微小rna-134生物标志物
CN118103510A (zh) * 2021-10-08 2024-05-28 莱古路斯治疗法股份有限公司 用于治疗多囊性肾病的方法和组合物
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 (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU5417201A (en) * 1996-05-24 2001-09-20 Genzyme Corporation Polycystic kidney disease gene
US7811759B2 (en) * 2004-04-20 2010-10-12 Jian Han Method for detecting ncRNA
GB0523659D0 (en) * 2005-11-21 2005-12-28 Novartis Ag Organic compounds
MX2009003548A (es) 2006-10-03 2009-04-15 Alnylam Pharmaceuticals Inc Formulaciones que contienen lipidos.
CN101821390A (zh) * 2007-06-14 2010-09-01 米尔克斯治疗有限责任公司 用于调节靶rna活性的寡核苷酸
KR101889518B1 (ko) * 2007-10-04 2018-08-17 로슈 이노베이션 센터 코펜하겐 에이/에스 마이크로MIRs
WO2009109665A1 (en) * 2008-03-07 2009-09-11 Santaris Pharma A/S Pharmaceutical compositions for treatment of microrna related diseases
CA2780726A1 (en) * 2009-11-11 2011-05-19 Tariq M. Rana Method for generation and regulation of ips cells and compositions thereof
AU2013331070A1 (en) * 2012-10-19 2015-05-07 Synta Pharmaceuticals Corp. Treating polycystic kidney disease with HSP90 inhibitory compounds
US20160362688A1 (en) * 2014-02-12 2016-12-15 Thomas Jefferson University Compositions and methods of using microrna inhibitors
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

Also Published As

Publication number Publication date
MY202466A (en) 2024-04-30
KR20240148005A (ko) 2024-10-10
JP7105775B2 (ja) 2022-07-25
EP3548011A1 (en) 2019-10-09
ZA201903608B (en) 2021-10-27
KR102780365B1 (ko) 2025-03-18
IL266989B2 (en) 2024-12-01
EP3548011B1 (en) 2023-06-14
US20200392501A1 (en) 2020-12-17
JP2019536803A (ja) 2019-12-19
IL266989A (en) 2019-07-31
CN110225747B (zh) 2025-04-29
EA201991370A1 (ru) 2019-12-30
AU2017372695B2 (en) 2023-07-20
US20220213483A1 (en) 2022-07-07
KR20190093207A (ko) 2019-08-08
IL266989B1 (en) 2024-08-01
US20240141350A1 (en) 2024-05-02
BR112019011597A2 (pt) 2019-10-22
WO2018106568A1 (en) 2018-06-14
AU2017372695A1 (en) 2019-06-13
MA46970A (fr) 2019-10-09
CN110225747A (zh) 2019-09-10
MX2019006337A (es) 2019-09-26
US20190345494A1 (en) 2019-11-14
CL2019001526A1 (es) 2019-10-18

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