JP2020518633A - アルポート症候群を治療するための方法 - Google Patents

アルポート症候群を治療するための方法 Download PDF

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JP2020518633A
JP2020518633A JP2019560355A JP2019560355A JP2020518633A JP 2020518633 A JP2020518633 A JP 2020518633A JP 2019560355 A JP2019560355 A JP 2019560355A JP 2019560355 A JP2019560355 A JP 2019560355A JP 2020518633 A JP2020518633 A JP 2020518633A
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nucleoside
modified oligonucleotide
dose
certain embodiments
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JP2020518633A5 (enExample
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ティモシー・ライト
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Sanofi SA
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Sanofi SA
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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
    • A61K31/712—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, needleless injectors
    • 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
    • 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)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Dermatology (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Urology & Nephrology (AREA)
  • Inorganic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
JP2019560355A 2017-05-04 2018-05-04 アルポート症候群を治療するための方法 Pending JP2020518633A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023023048A JP2023075111A (ja) 2017-05-04 2023-02-17 アルポート症候群を治療するための方法

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

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JP2020518633A true JP2020518633A (ja) 2020-06-25
JP2020518633A5 JP2020518633A5 (enExample) 2021-06-10

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JP2023023048A Pending JP2023075111A (ja) 2017-05-04 2023-02-17 アルポート症候群を治療するための方法

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US (3) US20210100828A1 (enExample)
EP (1) EP3618838A1 (enExample)
JP (2) JP2020518633A (enExample)
KR (1) KR20190141770A (enExample)
CN (2) CN116585344A (enExample)
AU (1) AU2018261792A1 (enExample)
BR (1) BR112019022824A2 (enExample)
CA (1) CA3062316A1 (enExample)
CL (1) CL2019003153A1 (enExample)
MA (1) MA50749A (enExample)
MX (1) MX2019013158A (enExample)
SG (1) SG11201910099WA (enExample)
WO (1) WO2018204788A1 (enExample)

Families Citing this family (2)

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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
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

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015511494A (ja) * 2012-03-15 2015-04-20 キュアナ，インク． 脳由来神経栄養因子（ｂｄｎｆ）に対する天然アンチセンス転写物の阻害によるｂｄｎｆ関連の疾患の処置
JP2015133967A (ja) * 2004-06-01 2015-07-27 プロネイ・セラピューティクス・インコーポレイテッドＰｒｏｎａｉ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｉｎｃ． 遺伝子発現の阻害に関する方法および組成物
JP2015536301A (ja) * 2012-10-09 2015-12-21 レグルス セラピューティクス インコーポレイテッド アルポート症候群の処置方法
JP2016041067A (ja) * 2008-08-25 2016-03-31 エクスカリアード・ファーマシューティカルズ，インコーポレイテッド 結合組織成長因子を対象とするアンチセンスオリゴヌクレオチドおよびその使用

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112014026639A8 (pt) * 2012-04-25 2018-01-16 Regulus Therapeutics Inc compostos , métodos de inibição da atividade de mir-21 , método para diminuir a expressão de colágeno , método para tratar , prevenir ou atrasar o início de uma doença , método de tratamento de um distúrbio fibroproliferativo e usos de um composto.
GB201507926D0 (en) * 2015-05-08 2015-06-24 Proqr Therapeutics N V Improved treatments using oligonucleotides

Patent Citations (4)

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Publication number Priority date Publication date Assignee Title
JP2015133967A (ja) * 2004-06-01 2015-07-27 プロネイ・セラピューティクス・インコーポレイテッドＰｒｏｎａｉ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｉｎｃ． 遺伝子発現の阻害に関する方法および組成物
JP2016041067A (ja) * 2008-08-25 2016-03-31 エクスカリアード・ファーマシューティカルズ，インコーポレイテッド 結合組織成長因子を対象とするアンチセンスオリゴヌクレオチドおよびその使用
JP2015511494A (ja) * 2012-03-15 2015-04-20 キュアナ，インク． 脳由来神経栄養因子（ｂｄｎｆ）に対する天然アンチセンス転写物の阻害によるｂｄｎｆ関連の疾患の処置
JP2015536301A (ja) * 2012-10-09 2015-12-21 レグルス セラピューティクス インコーポレイテッド アルポート症候群の処置方法

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CLINICALTRIALS.GOV ARCHIVE, vol. NCT02855268, JPN6022003831, 1 August 2016 (2016-08-01), ISSN: 0004898067 *
EUROPEAN MEDICINES AGENCY, vol. EMA/COMP/127039, JPN6022003829, 2015, ISSN: 0004898068 *

Also Published As

Publication number Publication date
US20240115598A1 (en) 2024-04-11
CN110740737A (zh) 2020-01-31
WO2018204788A1 (en) 2018-11-08
EP3618838A1 (en) 2020-03-11
CN116585344A (zh) 2023-08-15
SG11201910099WA (en) 2019-11-28
RU2019139261A3 (enExample) 2021-09-06
KR20190141770A (ko) 2019-12-24
CL2019003153A1 (es) 2020-03-27
CA3062316A1 (en) 2018-11-08
AU2018261792A1 (en) 2020-01-02
RU2019139261A (ru) 2021-06-04
BR112019022824A2 (pt) 2020-06-09
US20210100828A1 (en) 2021-04-08
MX2019013158A (es) 2020-02-07
MA50749A (fr) 2020-03-11
JP2023075111A (ja) 2023-05-30
US20220133769A1 (en) 2022-05-05

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