JP2021517909A - 胆管減少症関連状態を治療するための方法及び組成物 - Google Patents

胆管減少症関連状態を治療するための方法及び組成物 Download PDF

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JP2021517909A
JP2021517909A JP2020568662A JP2020568662A JP2021517909A JP 2021517909 A JP2021517909 A JP 2021517909A JP 2020568662 A JP2020568662 A JP 2020568662A JP 2020568662 A JP2020568662 A JP 2020568662A JP 2021517909 A JP2021517909 A JP 2021517909A
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JP2021517909A5 (https=
JPWO2019168686A5 (https=
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ナタリー パーセル
ナタリー パーセル
チェン ライ
チェン ライ
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Dicerna Pharmaceuticals Inc
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Dicerna Pharmaceuticals Inc
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    • 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
    • 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
    • C12N15/1135—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 against oncogenes or tumor suppressor genes
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
JP2020568662A 2018-03-02 2019-02-15 胆管減少症関連状態を治療するための方法及び組成物 Pending JP2021517909A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862637973P 2018-03-02 2018-03-02
US62/637,973 2018-03-02
PCT/US2019/018184 WO2019168686A1 (en) 2018-03-02 2019-02-15 Methods and compositions for treating bile duct paucity-associated conditions

Publications (3)

Publication Number Publication Date
JP2021517909A true JP2021517909A (ja) 2021-07-29
JP2021517909A5 JP2021517909A5 (https=) 2022-02-17
JPWO2019168686A5 JPWO2019168686A5 (https=) 2022-02-17

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ID=67805109

Family Applications (1)

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JP2020568662A Pending JP2021517909A (ja) 2018-03-02 2019-02-15 胆管減少症関連状態を治療するための方法及び組成物

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Country Link
US (1) US11634715B2 (https=)
EP (1) EP3740248A4 (https=)
JP (1) JP2021517909A (https=)
KR (1) KR20200127008A (https=)
CN (1) CN112055597A (https=)
AU (1) AU2019228448A1 (https=)
CA (1) CA3092089A1 (https=)
IL (1) IL277015A (https=)
MX (1) MX2020009074A (https=)
WO (1) WO2019168686A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024262593A1 (ja) * 2023-06-22 2024-12-26 公益財団法人川崎市産業振興財団 核酸の血中安定性を向上させ、標的化効率を向上させる方法

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3092089A1 (en) 2018-03-02 2019-09-06 Dicerna Pharmaceuticals, Inc. Methods and compositions for treating bile duct paucity-associated conditions

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK3037538T3 (da) 2010-07-06 2019-06-24 Dicerna Pharmaceuticals Inc Fremgangsmåder og sammensætninger til specifik inhibering af beta-catenin ved hjælp af dobbeltstrenget rna
US9468670B2 (en) * 2011-04-01 2016-10-18 Texas A&M University System Melatonin-based treatment and diagnosis of bile duct disease
IL316808A (en) * 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
AU2015330670B2 (en) * 2014-10-10 2022-01-06 Novo Nordisk Health Care Ag Therapeutic inhibition of lactate dehydrogenase and agents therefor
CA3092089A1 (en) 2018-03-02 2019-09-06 Dicerna Pharmaceuticals, Inc. Methods and compositions for treating bile duct paucity-associated conditions

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
THOMPSON, M.D., ET AL., HEPATOLOGY (BALTIMORE, MD.), vol. 67, no. 3, JPN6023006568, 26 January 2018 (2018-01-26), pages 955 - 971, ISSN: 0005153970 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024262593A1 (ja) * 2023-06-22 2024-12-26 公益財団法人川崎市産業振興財団 核酸の血中安定性を向上させ、標的化効率を向上させる方法

Also Published As

Publication number Publication date
EP3740248A1 (en) 2020-11-25
KR20200127008A (ko) 2020-11-09
EP3740248A4 (en) 2022-01-26
MX2020009074A (es) 2020-10-08
US11634715B2 (en) 2023-04-25
CA3092089A1 (en) 2019-09-06
WO2019168686A1 (en) 2019-09-06
CN112055597A (zh) 2020-12-08
US20210010005A1 (en) 2021-01-14
IL277015A (en) 2020-10-29
AU2019228448A1 (en) 2020-09-10

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