CN112055597A - 用于治疗胆管缺乏相关病况的方法和组合物 - Google Patents

用于治疗胆管缺乏相关病况的方法和组合物 Download PDF

Info

Publication number
CN112055597A
CN112055597A CN201980029432.6A CN201980029432A CN112055597A CN 112055597 A CN112055597 A CN 112055597A CN 201980029432 A CN201980029432 A CN 201980029432A CN 112055597 A CN112055597 A CN 112055597A
Authority
CN
China
Prior art keywords
nucleotides
length
oligonucleotide
oligonucleotides
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
CN201980029432.6A
Other languages
English (en)
Chinese (zh)
Inventor
N·珀塞尔
C·赖
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 CN112055597A publication Critical patent/CN112055597A/zh
Pending legal-status Critical Current

Links

Images

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)
CN201980029432.6A 2018-03-02 2019-02-15 用于治疗胆管缺乏相关病况的方法和组合物 Pending CN112055597A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862637973P 2018-03-02 2018-03-02
US62/637973 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
CN112055597A true CN112055597A (zh) 2020-12-08

Family

ID=67805109

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201980029432.6A Pending CN112055597A (zh) 2018-03-02 2019-02-15 用于治疗胆管缺乏相关病况的方法和组合物

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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130109740A1 (en) * 2010-07-06 2013-05-02 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of beta-catenin by double-stranded rna
US20140079680A1 (en) * 2011-04-01 2014-03-20 Scott & White Healthcare Melatonin-based treatment and diagnosis of bile duct disease

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130109740A1 (en) * 2010-07-06 2013-05-02 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of beta-catenin by double-stranded rna
US20140079680A1 (en) * 2011-04-01 2014-03-20 Scott & White Healthcare Melatonin-based treatment and diagnosis of bile duct disease

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
MICHAEL D. THOMPSON等: "β-Catenin regulation of farnesoid X receptor signaling and bile acid metabolism during murine cholestasis", HEPATOLOGY, vol. 67, no. 3, 26 January 2018 (2018-01-26), pages 7 *

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
CA3092089A1 (en) 2019-09-06
WO2019168686A1 (en) 2019-09-06
US20210010005A1 (en) 2021-01-14
IL277015A (en) 2020-10-29
AU2019228448A1 (en) 2020-09-10

Similar Documents

Publication Publication Date Title
JP7749645B2 (ja) Ａｎｇｐｔｌ３発現を阻害する組成物及び方法
US20220170025A1 (en) Compositions and methods for inhibiting gene expression in the central nervous system
CN114761557B (zh) 具有最小氟含量的小干扰rna的化学修饰
US20250092403A1 (en) Compositions and methods for inhibiting ketohexokinase (khk)
CN111601891A (zh) 用于抑制aldh2表达的组合物和方法
US12084662B2 (en) Compositions and methods for modulating PNPLA3 expression
US11634715B2 (en) Methods and compositions for treating bile duct paucity-associated conditions
CN118355120A (zh) 用于调节apoc3表达的组合物和方法
US20260055414A1 (en) Compositions and methods for inhibiting xdh expression
US20230374522A1 (en) Compositions and methods for modulating scap activity

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
WD01 Invention patent application deemed withdrawn after publication
WD01 Invention patent application deemed withdrawn after publication

Application publication date: 20201208