CA3092089A1 - Methods and compositions for treating bile duct paucity-associated conditions - Google Patents

Methods and compositions for treating bile duct paucity-associated conditions Download PDF

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Publication number
CA3092089A1
CA3092089A1 CA3092089A CA3092089A CA3092089A1 CA 3092089 A1 CA3092089 A1 CA 3092089A1 CA 3092089 A CA3092089 A CA 3092089A CA 3092089 A CA3092089 A CA 3092089A CA 3092089 A1 CA3092089 A1 CA 3092089A1
Authority
CA
Canada
Prior art keywords
nucleotides
oligonucleotide
length
antisense strand
ctnnb1
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3092089A
Other languages
English (en)
French (fr)
Inventor
Natalie PURSELL
Cheng LAI
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.)
Dicerna Pharmaceuticals Inc
Original Assignee
Dicerna Pharmaceuticals Inc
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.)
Filing date
Publication date
Application filed by Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of CA3092089A1 publication Critical patent/CA3092089A1/en
Pending legal-status Critical Current

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Classifications

    • 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]

Landscapes

  • 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)
CA3092089A 2018-03-02 2019-02-15 Methods and compositions for treating bile duct paucity-associated conditions Pending CA3092089A1 (en)

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 (1)

Publication Number Publication Date
CA3092089A1 true CA3092089A1 (en) 2019-09-06

Family

ID=67805109

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3092089A Pending CA3092089A1 (en) 2018-03-02 2019-02-15 Methods and compositions for treating bile duct paucity-associated conditions

Country Status (10)

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=)

Families Citing this family (2)

* 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
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

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

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
JP2021517909A (ja) 2021-07-29
US11634715B2 (en) 2023-04-25
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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