CN110740737A - 用于治疗奥尔波特综合征的方法 - Google Patents

用于治疗奥尔波特综合征的方法 Download PDF

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Publication number
CN110740737A
CN110740737A CN201880037914.1A CN201880037914A CN110740737A CN 110740737 A CN110740737 A CN 110740737A CN 201880037914 A CN201880037914 A CN 201880037914A CN 110740737 A CN110740737 A CN 110740737A
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China
Prior art keywords
subject
modified oligonucleotide
nucleoside
dose
certain embodiments
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Pending
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CN201880037914.1A
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English (en)
Chinese (zh)
Inventor
T·莱特
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Sanofi Aventis France
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Sanofi Aventis France
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Priority to CN202310591916.8A priority Critical patent/CN116585344A/zh
Publication of CN110740737A publication Critical patent/CN110740737A/zh
Pending legal-status Critical Current

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    • 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
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0021—Intradermal administration, e.g. through microneedle arrays or needleless injectors
    • 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
    • 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 nucleic acids [NA]
    • 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
    • C12N2310/322—2'-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
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Dermatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Urology & Nephrology (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Inorganic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN201880037914.1A 2017-05-04 2018-05-04 用于治疗奥尔波特综合征的方法 Pending CN110740737A (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202310591916.8A CN116585344A (zh) 2017-05-04 2018-05-04 用于治疗奥尔波特综合征的方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762501699P 2017-05-04 2017-05-04
US62/501,699 2017-05-04
PCT/US2018/031094 WO2018204788A1 (en) 2017-05-04 2018-05-04 Methods for treatment of alport syndrome

Related Child Applications (1)

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CN110740737A true CN110740737A (zh) 2020-01-31

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CN202310591916.8A Pending CN116585344A (zh) 2017-05-04 2018-05-04 用于治疗奥尔波特综合征的方法

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Country Status (13)

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US (3) US20210100828A1 (https=)
EP (1) EP3618838A1 (https=)
JP (2) JP2020518633A (https=)
KR (1) KR20190141770A (https=)
CN (2) CN110740737A (https=)
AU (1) AU2018261792A1 (https=)
BR (1) BR112019022824A2 (https=)
CA (1) CA3062316A1 (https=)
CL (1) CL2019003153A1 (https=)
MA (1) MA50749A (https=)
MX (1) MX2019013158A (https=)
SG (1) SG11201910099WA (https=)
WO (1) WO2018204788A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN120731923A (zh) * 2025-09-05 2025-10-03 内蒙古医科大学附属医院(内蒙古自治区心血管研究所) 一种Alport综合征小鼠NMD逃逸模型的构建方法及其应用

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022076452A1 (en) * 2020-10-05 2022-04-14 Father Flanagan's Boys' Home Doing Business As Boys Town National Research Hospital Combination therapy for alport renal disease

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104718295A (zh) * 2012-10-09 2015-06-17 莱古路斯治疗法股份有限公司 用于治疗奥尔波特综合征的方法
WO2016180784A1 (en) * 2015-05-08 2016-11-17 Proqr Therapeutics Ii B.V. Improved treatments using oligonucleotides

Family Cites Families (4)

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US7498315B2 (en) * 2004-06-01 2009-03-03 Pronai Therapeutics, Inc. Methods and compositions for the inhibition of gene expression
NZ601660A (en) * 2008-08-25 2014-05-30 Excaliard Pharmaceuticals Inc Antisense oligonucleotides directed against connective tissue growth factor and uses thereof
US20150031750A1 (en) * 2012-03-15 2015-01-29 The Scripps Research Institute Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf
PE20142404A1 (es) * 2012-04-25 2015-02-02 Regulus Therapeutics Inc Compuestos de microarn y metodos de modulacion de la actividad de mir-21

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104718295A (zh) * 2012-10-09 2015-06-17 莱古路斯治疗法股份有限公司 用于治疗奥尔波特综合征的方法
WO2016180784A1 (en) * 2015-05-08 2016-11-17 Proqr Therapeutics Ii B.V. Improved treatments using oligonucleotides

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
IVAN G. GOMEZ ET AL.，: "Anti–microRNA-21 oligonucleotides prevent Alport nephropathy progression by stimulating metabolic pathways", 《J CLIN INVEST》 *
NCT，: "History of Changes for Study: NCT02855268 Study of Weekly RG-012 Injections in Patients With Alport Syndrome (HERA)", 《NCT,HTTPS://CLINICALTRIALS.GOV/CT2/HISTORY/NCT02855268?V_3=VIEW#STUDYPAGETOP》 *
汤森路透: "2015 年全球新药研发报告—第二部分：趋势复杂，亮点频现（Ⅴ）", 《药学进展》 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN120731923A (zh) * 2025-09-05 2025-10-03 内蒙古医科大学附属医院(内蒙古自治区心血管研究所) 一种Alport综合征小鼠NMD逃逸模型的构建方法及其应用
CN120731923B (zh) * 2025-09-05 2025-11-04 内蒙古医科大学附属医院(内蒙古自治区心血管研究所) 一种Alport综合征小鼠NMD逃逸模型的构建方法及其应用

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Publication number Publication date
KR20190141770A (ko) 2019-12-24
US20220133769A1 (en) 2022-05-05
JP2023075111A (ja) 2023-05-30
AU2018261792A1 (en) 2020-01-02
CN116585344A (zh) 2023-08-15
RU2019139261A3 (https=) 2021-09-06
EP3618838A1 (en) 2020-03-11
WO2018204788A1 (en) 2018-11-08
BR112019022824A2 (pt) 2020-06-09
JP2020518633A (ja) 2020-06-25
SG11201910099WA (en) 2019-11-28
US20210100828A1 (en) 2021-04-08
CA3062316A1 (en) 2018-11-08
MA50749A (fr) 2020-03-11
CL2019003153A1 (es) 2020-03-27
US20240115598A1 (en) 2024-04-11
MX2019013158A (es) 2020-02-07
RU2019139261A (ru) 2021-06-04

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Application publication date: 20200131