AU2014284298B2 - Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA - Google Patents

Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA Download PDF

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Publication number
AU2014284298B2
AU2014284298B2 AU2014284298A AU2014284298A AU2014284298B2 AU 2014284298 B2 AU2014284298 B2 AU 2014284298B2 AU 2014284298 A AU2014284298 A AU 2014284298A AU 2014284298 A AU2014284298 A AU 2014284298A AU 2014284298 B2 AU2014284298 B2 AU 2014284298B2
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strand
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rna
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AU2014284298A1 (en
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Bob D. Brown
Henryk T. Dudek
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Novo Nordisk AS
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Novo Nordisk AS
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    • 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 nucleic acids [NA]
    • 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. ==--==--==--

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  • 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)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (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)
AU2014284298A 2013-07-03 2014-07-03 Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA Ceased AU2014284298B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2020260479A AU2020260479B2 (en) 2013-07-03 2020-10-29 Methods and Compositions for the Specific Inhibition of Alpha-1 Antitrypsin by Double-Stranded RNA

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201361842551P 2013-07-03 2013-07-03
US61/842,551 2013-07-03
US201361891548P 2013-10-16 2013-10-16
US61/891,548 2013-10-16
PCT/US2014/045365 WO2015003113A2 (en) 2013-07-03 2014-07-03 Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded rna

Related Child Applications (1)

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AU2020260479A Division AU2020260479B2 (en) 2013-07-03 2020-10-29 Methods and Compositions for the Specific Inhibition of Alpha-1 Antitrypsin by Double-Stranded RNA

Publications (2)

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AU2014284298A1 AU2014284298A1 (en) 2016-01-21
AU2014284298B2 true AU2014284298B2 (en) 2020-12-10

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AU2020260479A Ceased AU2020260479B2 (en) 2013-07-03 2020-10-29 Methods and Compositions for the Specific Inhibition of Alpha-1 Antitrypsin by Double-Stranded RNA

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US (11) US9458457B2 (enExample)
EP (3) EP3444350B1 (enExample)
JP (4) JP2016528887A (enExample)
AU (2) AU2014284298B2 (enExample)
CA (1) CA2916499A1 (enExample)
DK (1) DK3444350T3 (enExample)
ES (1) ES2905257T3 (enExample)
HU (1) HUE058794T2 (enExample)
PL (1) PL3444350T3 (enExample)
PT (1) PT3444350T (enExample)
WO (1) WO2015003113A2 (enExample)

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US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
EP2632472B1 (en) 2010-10-29 2017-12-13 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using short interfering nucleic acids (sina)
MX379208B (es) 2013-05-22 2025-03-10 Alnylam Pharmaceuticals Inc Composiciones de arni de serpina1 y sus metodos de uso.
ES2905257T3 (es) 2013-07-03 2022-04-07 Dicerna Pharmaceuticals Inc Métodos y composiciones para la inhibición específica de alfa-1 antitripsina mediante ARN bicatenario
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
WO2017139616A1 (en) * 2016-02-10 2017-08-17 Arrowhead Pharmaceuticals, Inc. Methods of treatment for alpha-1 antitrypsin deficiency
CA3024448C (en) * 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
US10576167B2 (en) 2016-08-17 2020-03-03 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
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic agents
KR102551664B1 (ko) 2016-12-22 2023-07-05 인텔리아 테라퓨틱스, 인크. 알파-1 항트립신 결핍을 치료하기 위한 조성물 및 방법
TW202436621A (zh) 2017-01-10 2024-09-16 美商愛羅海德製藥公司 α-1抗胰蛋白酶 (AAT) RNAi 藥劑、包含AAT RNAi 藥劑之組合物及使用方法
SE540888C2 (en) * 2017-06-19 2018-12-11 Hepgene Medical Ab Novel nucleic acid molecules and their use in therapy
EP3773609A4 (en) * 2018-04-13 2022-05-18 Dicerna Pharmaceuticals, Inc. DOUBLE-STRANDED NUCLEIC ACID INHIBITOR MOLECULES MODIFIED WITH Tm-INCREASING NUCLEOTIDES
EP3790964A4 (en) * 2018-05-11 2022-06-08 Beam Therapeutics, Inc. METHODS FOR REMOVAL OF PATHOGENIC MUTATIONS USING PROGRAMMABLE BASE EDITOR SYSTEMS
CA3192372A1 (en) * 2020-09-15 2022-03-24 Dawn CHRISTIANSON Methods for the reduction of z-aat protein levels
JP2024532019A (ja) 2021-08-25 2024-09-05 ノボ ノルディスク エー／エス アルファ－１アンチトリプシン発現を阻害するための組成物及び方法
WO2024229229A2 (en) * 2023-05-02 2024-11-07 Sirnaomics, Inc. Products and compositions

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Also Published As

Publication number Publication date
ES2905257T3 (es) 2022-04-07
US11208658B2 (en) 2021-12-28
EP3444350B1 (en) 2021-12-01
US11851658B2 (en) 2023-12-26
JP2020188771A (ja) 2020-11-26
US11408002B1 (en) 2022-08-09
AU2020260479A1 (en) 2020-11-26
US10370655B2 (en) 2019-08-06
EP4012031A1 (en) 2022-06-15
US11312961B1 (en) 2022-04-26
PT3444350T (pt) 2022-02-04
US20170130221A1 (en) 2017-05-11
US11912996B2 (en) 2024-02-27
JP2022078069A (ja) 2022-05-24
US10844381B2 (en) 2020-11-24
AU2020260479B2 (en) 2023-06-15
US20210180061A1 (en) 2021-06-17
US20220228149A1 (en) 2022-07-21
WO2015003113A3 (en) 2015-02-26
US20190390198A1 (en) 2019-12-26
US20230069235A1 (en) 2023-03-02
JP2016528887A (ja) 2016-09-23
PL3444350T3 (pl) 2022-03-21
US20150011607A1 (en) 2015-01-08
US20220396793A1 (en) 2022-12-15
US11459566B1 (en) 2022-10-04
DK3444350T3 (da) 2022-02-07
JP2024075702A (ja) 2024-06-04
WO2015003113A2 (en) 2015-01-08
HUE058794T2 (hu) 2022-09-28
EP3017047A4 (en) 2017-06-14
EP3444350A1 (en) 2019-02-20
US11312960B1 (en) 2022-04-26
EP3017047A2 (en) 2016-05-11
CA2916499A1 (en) 2015-01-08
AU2014284298A1 (en) 2016-01-21
US9458457B2 (en) 2016-10-04

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Free format text: FORMER OWNER(S): DICERNA PHARMACEUTICALS, INC.

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