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HUE058794T2 - Módszerek és készítmények az alfa-1 antitripszin specifikus gátlására kettõs szálú RNS által - Google Patents

Módszerek és készítmények az alfa-1 antitripszin specifikus gátlására kettõs szálú RNS által

Info

Publication number
HUE058794T2
HUE058794T2 HUE18196106A HUE18196106A HUE058794T2 HU E058794 T2 HUE058794 T2 HU E058794T2 HU E18196106 A HUE18196106 A HU E18196106A HU E18196106 A HUE18196106 A HU E18196106A HU E058794 T2 HUE058794 T2 HU E058794T2
Authority
HU
Hungary
Prior art keywords
antitrypsin
alpha
compositions
double
methods
Prior art date
Application number
HUE18196106A
Other languages
English (en)
Inventor
Bob D Brown
Henryk T Dudek
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 HUE058794T2 publication Critical patent/HUE058794T2/hu

Links

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
    • A61P35/00—Antineoplastic agents
    • 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 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
    • C12N2310/32—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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--

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)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Electric Double-Layer Capacitors Or The Like (AREA)
  • Medicinal Preparation (AREA)
HUE18196106A 2013-07-03 2014-07-03 Módszerek és készítmények az alfa-1 antitripszin specifikus gátlására kettõs szálú RNS által HUE058794T2 (hu)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361842551P 2013-07-03 2013-07-03
US201361891548P 2013-10-16 2013-10-16

Publications (1)

Publication Number Publication Date
HUE058794T2 true HUE058794T2 (hu) 2022-09-28

Family

ID=52133228

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE18196106A HUE058794T2 (hu) 2013-07-03 2014-07-03 Módszerek és készítmények az alfa-1 antitripszin specifikus gátlására kettõs szálú RNS által

Country Status (11)

Country Link
US (11) US9458457B2 (hu)
EP (3) EP4012031A1 (hu)
JP (4) JP2016528887A (hu)
AU (2) AU2014284298B2 (hu)
CA (1) CA2916499A1 (hu)
DK (1) DK3444350T3 (hu)
ES (1) ES2905257T3 (hu)
HU (1) HUE058794T2 (hu)
PL (1) PL3444350T3 (hu)
PT (1) PT3444350T (hu)
WO (1) WO2015003113A2 (hu)

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PT3444350T (pt) 2013-07-03 2022-02-04 Dicerna Pharmaceuticals Inc Métodos e composições para a inibição específica de alfa-1 antitripsina através de arn de cadeia dupla
TW201620526A (zh) 2014-06-17 2016-06-16 愛羅海德研究公司 用於抑制α－１抗胰蛋白酶基因表現之組合物及方法
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CA3033788A1 (en) 2016-08-17 2018-02-22 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
TWI788312B (zh) 2016-11-23 2023-01-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法
US20180193270A1 (en) 2016-11-29 2018-07-12 PureTech Health LLC Exosomes for delivery of therapeutic agents
JP7383478B2 (ja) * 2016-12-22 2023-11-20 インテリア セラピューティクス，インコーポレイテッド アルファ１アンチトリプシン欠乏症を治療するための組成物および方法
CN110268060B (zh) 2017-01-10 2024-07-26 箭头药业股份有限公司 α-1抗胰蛋白酶(AAT)RNAi物质、包含AAT RNAi物质的组合物和使用方法
SE1750774A1 (en) * 2017-06-19 2018-12-11 Hepgene Medical Ab Novel nucleic acid molecules and their use in therapy
KR20200144100A (ko) * 2018-04-13 2020-12-28 다이서나 파마수이티컬, 인크. Tm-증가 뉴클레오타이드로 변형된 이중-가닥 핵산 억제제 분자
AU2021345026A1 (en) * 2020-09-15 2023-05-11 Arrowhead Pharmaceuticals, Inc. Methods for the reduction of z-aat protein levels
TW202308660A (zh) * 2021-08-25 2023-03-01 美商戴瑟納製藥股份有限公司 用於抑制α－１抗胰蛋白酶表現之組合物及方法
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Also Published As

Publication number Publication date
EP3444350B1 (en) 2021-12-01
JP2016528887A (ja) 2016-09-23
CA2916499A1 (en) 2015-01-08
US11208658B2 (en) 2021-12-28
AU2020260479A1 (en) 2020-11-26
WO2015003113A3 (en) 2015-02-26
EP3017047A2 (en) 2016-05-11
JP2022078069A (ja) 2022-05-24
US20150011607A1 (en) 2015-01-08
US11312960B1 (en) 2022-04-26
PL3444350T3 (pl) 2022-03-21
AU2020260479B2 (en) 2023-06-15
PT3444350T (pt) 2022-02-04
US20220228149A1 (en) 2022-07-21
US11912996B2 (en) 2024-02-27
US11851658B2 (en) 2023-12-26
ES2905257T3 (es) 2022-04-07
US10844381B2 (en) 2020-11-24
US20170130221A1 (en) 2017-05-11
JP2020188771A (ja) 2020-11-26
AU2014284298B2 (en) 2020-12-10
EP4012031A1 (en) 2022-06-15
US11459566B1 (en) 2022-10-04
US20220396793A1 (en) 2022-12-15
US20210180061A1 (en) 2021-06-17
EP3017047A4 (en) 2017-06-14
US10370655B2 (en) 2019-08-06
US11408002B1 (en) 2022-08-09
US11312961B1 (en) 2022-04-26
WO2015003113A2 (en) 2015-01-08
EP3444350A1 (en) 2019-02-20
DK3444350T3 (da) 2022-02-07
US20190390198A1 (en) 2019-12-26
US9458457B2 (en) 2016-10-04
US20230069235A1 (en) 2023-03-02
JP2024075702A (ja) 2024-06-04
AU2014284298A1 (en) 2016-01-21

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