HK1232163A1 - 用於抑制α- 抗胰蛋白酶基因表達的組合物及方法 - Google Patents

用於抑制α- 抗胰蛋白酶基因表達的組合物及方法

Info

Publication number
HK1232163A1
HK1232163A1 HK17106007.9A HK17106007A HK1232163A1 HK 1232163 A1 HK1232163 A1 HK 1232163A1 HK 17106007 A HK17106007 A HK 17106007A HK 1232163 A1 HK1232163 A1 HK 1232163A1
Authority
HK
Hong Kong
Prior art keywords
antitrypsin
alpha
compositions
methods
gene expression
Prior art date
Application number
HK17106007.9A
Other languages
English (en)
Inventor
‧伍德爾
‧路易斯
‧維基菲爾德
‧埃爾米達
‧坎納
Original Assignee
Arrowhead 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 Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of HK1232163A1 publication Critical patent/HK1232163A1/zh

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Classifications

    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • 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 N.A.
    • 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
    • C12N2310/321—2'-O-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
    • C12N2310/322—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
    • 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
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Communicable Diseases (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
HK17106007.9A 2014-06-17 2017-06-16 用於抑制α- 抗胰蛋白酶基因表達的組合物及方法 HK1232163A1 (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462013288P 2014-06-17 2014-06-17
PCT/US2015/035976 WO2015195628A2 (en) 2014-06-17 2015-06-16 Compositions and methods for inhibiting gene expression of alpha-1 antitrypsin

Publications (1)

Publication Number Publication Date
HK1232163A1 true HK1232163A1 (zh) 2018-01-05

Family

ID=54835647

Family Applications (1)

Application Number Title Priority Date Filing Date
HK17106007.9A HK1232163A1 (zh) 2014-06-17 2017-06-16 用於抑制α- 抗胰蛋白酶基因表達的組合物及方法

Country Status (13)

Country Link
US (4) US10006025B2 (zh)
EP (1) EP3157541A4 (zh)
CN (2) CN113444725A (zh)
AR (1) AR100840A1 (zh)
AU (2) AU2015277376B2 (zh)
BR (1) BR112016029781A2 (zh)
CA (2) CA3219720A1 (zh)
EA (1) EA201692318A1 (zh)
HK (1) HK1232163A1 (zh)
IL (1) IL249597A0 (zh)
MX (3) MX2016015870A (zh)
TW (1) TW201620526A (zh)
WO (1) WO2015195628A2 (zh)

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KR20170063949A (ko) 2014-10-06 2017-06-08 엑시큐어, 인크. 항-ｔｎｆ 화합물
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KR20230048446A (ko) 2016-03-07 2023-04-11 애로우헤드 파마슈티컬스 인코포레이티드 치료용 화합물을 위한 표적화 리간드
JP7186094B2 (ja) 2016-05-06 2022-12-08 イグジキュア オペレーティング カンパニー インターロイキン１７受容体ｍＲＮＡの特異的ノックダウンのためのアンチセンスオリゴヌクレオチド（ＡＳＯ）を提示するリポソーム系球状核酸（ＳＮＡ）構築物
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Also Published As

Publication number Publication date
WO2015195628A3 (en) 2016-02-18
WO2015195628A2 (en) 2015-12-23
MX2020010524A (es) 2020-11-06
US10006025B2 (en) 2018-06-26
AU2015277376A1 (en) 2016-12-08
AU2021204399A1 (en) 2021-07-22
US20190284558A1 (en) 2019-09-19
US20150361427A1 (en) 2015-12-17
CA2951700A1 (en) 2015-12-23
IL249597A0 (en) 2017-02-28
BR112016029781A2 (pt) 2017-10-31
CN113444725A (zh) 2021-09-28
CA3219720A1 (en) 2015-12-23
EA201692318A1 (ru) 2017-06-30
TW201620526A (zh) 2016-06-16
MX2016015870A (es) 2017-04-27
AR100840A1 (es) 2016-11-02
CN106535917B (zh) 2021-07-06
US11384355B2 (en) 2022-07-12
EP3157541A4 (en) 2018-04-04
CA2951700C (en) 2024-01-02
AU2015277376B2 (en) 2021-04-01
EP3157541A2 (en) 2017-04-26
US20230002768A1 (en) 2023-01-05
US20180258430A1 (en) 2018-09-13
MX2023003672A (es) 2023-04-18
CN106535917A (zh) 2017-03-22

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