EA201991360A1 - Модифицированные олигонуклеотиды для лечения поликистозной болезни почек - Google Patents

Модифицированные олигонуклеотиды для лечения поликистозной болезни почек

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Publication number
EA201991360A1
EA201991360A1 EA201991360A EA201991360A EA201991360A1 EA 201991360 A1 EA201991360 A1 EA 201991360A1 EA 201991360 A EA201991360 A EA 201991360A EA 201991360 A EA201991360 A EA 201991360A EA 201991360 A1 EA201991360 A1 EA 201991360A1
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EA
Eurasian Patent Office
Prior art keywords
kidney disease
polycystic kidney
modified oligonucleotides
treatment
methods
Prior art date
Application number
EA201991360A
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English (en)
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.)
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Publication date
Application filed filed Critical
Publication of EA201991360A1 publication Critical patent/EA201991360A1/ru

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    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Urology & Nephrology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)

Abstract

В данном документе представлены способы лечения поликистозной болезни почек, в том числе аутосомно-доминантной поликистозной болезни почек, с использованием модифицированных олигонуклеотидов, нацеленных на miR-17.
EA201991360A 2016-12-05 2017-12-04 Модифицированные олигонуклеотиды для лечения поликистозной болезни почек EA201991360A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662430139P 2016-12-05 2016-12-05
PCT/US2017/064428 WO2018106566A1 (en) 2016-12-05 2017-12-04 Modified oligonucleotides for treatment of polycystic kidney disease

Publications (1)

Publication Number Publication Date
EA201991360A1 true EA201991360A1 (ru) 2019-11-29

Family

ID=60915610

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201991360A EA201991360A1 (ru) 2016-12-05 2017-12-04 Модифицированные олигонуклеотиды для лечения поликистозной болезни почек

Country Status (18)

Country Link
US (3) US20200165606A1 (ru)
EP (1) EP3548503A1 (ru)
JP (2) JP7133553B2 (ru)
KR (1) KR20190085951A (ru)
CN (1) CN110036019A (ru)
AU (1) AU2017370560C1 (ru)
BR (1) BR112019011164A2 (ru)
CA (1) CA3044896A1 (ru)
CL (1) CL2019001522A1 (ru)
CO (1) CO2019006234A2 (ru)
EA (1) EA201991360A1 (ru)
IL (1) IL266871A (ru)
MA (1) MA46999A (ru)
MX (1) MX2019006332A (ru)
PH (1) PH12019501224A1 (ru)
TW (2) TW202300647A (ru)
WO (1) WO2018106566A1 (ru)
ZA (1) ZA201903605B (ru)

Families Citing this family (5)

* 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
WO2018155450A1 (ja) 2017-02-21 2018-08-30 国立大学法人大阪大学 アンチセンスオリゴ核酸
EP3841220A1 (en) 2018-08-23 2021-06-30 Roche Innovation Center Copenhagen A/S Microrna-134 biomarker
CA3117981A1 (en) * 2018-11-13 2020-05-22 Regulus Therapeutics Inc. Microrna compounds and methods for modulating mir-10b activity
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

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2007303205A1 (en) 2006-10-03 2008-04-10 Tekmira Pharmaceuticals Corporation Lipid containing formulations
KR101889518B1 (ko) * 2007-10-04 2018-08-17 로슈 이노베이션 센터 코펜하겐 에이/에스 마이크로ＭＩＲｓ
CA2717792A1 (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
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
MA44836A (fr) * 2015-08-26 2018-07-04 Univ Texas Procédés de traitement du syndrome polykystique des reins

Also Published As

Publication number Publication date
IL266871A (en) 2019-07-31
WO2018106566A1 (en) 2018-06-14
TW202300647A (zh) 2023-01-01
US20230109466A1 (en) 2023-04-06
TWI769197B (zh) 2022-07-01
KR20190085951A (ko) 2019-07-19
JP7133553B2 (ja) 2022-09-08
CN110036019A (zh) 2019-07-19
AU2017370560A1 (en) 2019-06-06
AU2017370560B2 (en) 2021-11-18
MX2019006332A (es) 2019-08-01
TW201821618A (zh) 2018-06-16
AU2017370560C1 (en) 2022-08-11
CO2019006234A2 (es) 2019-08-30
US20200165606A1 (en) 2020-05-28
PH12019501224A1 (en) 2019-09-23
MA46999A (fr) 2019-10-09
ZA201903605B (en) 2023-12-20
US20210095282A1 (en) 2021-04-01
BR112019011164A2 (pt) 2019-10-08
CL2019001522A1 (es) 2019-10-25
JP2019536804A (ja) 2019-12-19
JP2022169726A (ja) 2022-11-09
EP3548503A1 (en) 2019-10-09
CA3044896A1 (en) 2018-06-14

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