JP2016528887A - 二本鎖ＲＮＡによるα−１アンチトリプシンの特異的阻害のための方法及び組成物 - Google Patents

二本鎖ＲＮＡによるα−１アンチトリプシンの特異的阻害のための方法及び組成物 Download PDF

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JP2016528887A
JP2016528887A JP2016524365A JP2016524365A JP2016528887A JP 2016528887 A JP2016528887 A JP 2016528887A JP 2016524365 A JP2016524365 A JP 2016524365A JP 2016524365 A JP2016524365 A JP 2016524365A JP 2016528887 A JP2016528887 A JP 2016528887A
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strand
rna
antitrypsin
dsna
nucleic acid
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JP2016528887A5 (https=
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ボブ， ディー ブラウン，
ボブ， ディー ブラウン，
ヘンリク， ティー ドゥデク，
ヘンリク， ティー ドゥデク，
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Dicerna Pharmaceuticals Inc
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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)
  • 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)
JP2016524365A 2013-07-03 2014-07-03 二本鎖ＲＮＡによるα−１アンチトリプシンの特異的阻害のための方法及び組成物 Pending JP2016528887A (ja)

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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JP2020094030A Division JP2020188771A (ja) 2013-07-03 2020-05-29 二本鎖ＲＮＡによるα−１アンチトリプシンの特異的阻害のための方法及び組成物

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JP2016528887A true JP2016528887A (ja) 2016-09-23
JP2016528887A5 JP2016528887A5 (https=) 2017-08-10

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JP2016524365A Pending JP2016528887A (ja) 2013-07-03 2014-07-03 二本鎖ＲＮＡによるα−１アンチトリプシンの特異的阻害のための方法及び組成物
JP2020094030A Withdrawn JP2020188771A (ja) 2013-07-03 2020-05-29 二本鎖ＲＮＡによるα−１アンチトリプシンの特異的阻害のための方法及び組成物
JP2022019996A Withdrawn JP2022078069A (ja) 2013-07-03 2022-02-10 二本鎖ＲＮＡによるα－１アンチトリプシンの特異的阻害のための方法及び組成物
JP2024045063A Withdrawn JP2024075702A (ja) 2013-07-03 2024-03-21 二本鎖ＲＮＡによるα－１アンチトリプシンの特異的阻害のための方法及び組成物

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JP2020094030A Withdrawn JP2020188771A (ja) 2013-07-03 2020-05-29 二本鎖ＲＮＡによるα−１アンチトリプシンの特異的阻害のための方法及び組成物
JP2022019996A Withdrawn JP2022078069A (ja) 2013-07-03 2022-02-10 二本鎖ＲＮＡによるα－１アンチトリプシンの特異的阻害のための方法及び組成物
JP2024045063A Withdrawn JP2024075702A (ja) 2013-07-03 2024-03-21 二本鎖ＲＮＡによるα－１アンチトリプシンの特異的阻害のための方法及び組成物

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

Cited By (3)

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Publication number Priority date Publication date Assignee Title
JP2023075164A (ja) * 2016-12-22 2023-05-30 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
JP2023541170A (ja) * 2020-09-15 2023-09-28 アローヘッド ファーマシューティカルズ インコーポレイテッド Ｚ－ａａｔタンパク質のレベルを低下させる方法
JP2025013768A (ja) * 2018-05-11 2025-01-28 ビーム セラピューティクス インク． プログラム可能塩基エディターシステムを用いて病原性変異を抑制する方法

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WO2012058210A1 (en) 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA)
EA038792B1 (ru) 2013-05-22 2021-10-20 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ НА ОСНОВЕ RNAi Serpina1 И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
US9458457B2 (en) 2013-07-03 2016-10-04 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA
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
WO2017093969A1 (en) * 2015-12-04 2017-06-08 Novartis Ag Compositions and methods for immunooncology
WO2017139616A1 (en) * 2016-02-10 2017-08-17 Arrowhead Pharmaceuticals, Inc. Methods of treatment for alpha-1 antitrypsin deficiency
KR20240056729A (ko) * 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
IL308824A (en) 2016-08-17 2024-01-01 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
CA3043768A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
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
JP2021520355A (ja) * 2018-04-13 2021-08-19 ディセルナ ファーマシューティカルズ インコーポレイテッド Ｔｍ上昇ヌクレオチドで修飾された二本鎖核酸阻害剤分子
MX2021015003A (es) 2019-06-06 2022-01-24 Arrowhead Pharmaceuticals Inc Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (aatd).
EP4392556A1 (en) * 2021-08-25 2024-07-03 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting ?lpha-1 antitrypsin expression
MX2024002988A (es) * 2021-09-08 2024-06-11 Flagship Pioneering Innovations Vi Llc Composiciones y metodos moduladores de la serpina.
WO2024229229A2 (en) * 2023-05-02 2024-11-07 Sirnaomics, Inc. Products and compositions

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Cited By (5)

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Publication number Priority date Publication date Assignee Title
JP2023075164A (ja) * 2016-12-22 2023-05-30 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
US12460205B2 (en) 2016-12-22 2025-11-04 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
JP2025013768A (ja) * 2018-05-11 2025-01-28 ビーム セラピューティクス インク． プログラム可能塩基エディターシステムを用いて病原性変異を抑制する方法
JP2023541170A (ja) * 2020-09-15 2023-09-28 アローヘッド ファーマシューティカルズ インコーポレイテッド Ｚ－ａａｔタンパク質のレベルを低下させる方法
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US11312961B1 (en) 2022-04-26
AU2014284298B2 (en) 2020-12-10
US10370655B2 (en) 2019-08-06
DK3444350T3 (da) 2022-02-07
JP2024075702A (ja) 2024-06-04
JP2020188771A (ja) 2020-11-26
EP4012031A1 (en) 2022-06-15
AU2020260479B2 (en) 2023-06-15
US11208658B2 (en) 2021-12-28
PL3444350T3 (pl) 2022-03-21
US11851658B2 (en) 2023-12-26
JP2022078069A (ja) 2022-05-24
AU2014284298A1 (en) 2016-01-21
US20220228149A1 (en) 2022-07-21
US9458457B2 (en) 2016-10-04
HUE058794T2 (hu) 2022-09-28
WO2015003113A3 (en) 2015-02-26
US11312960B1 (en) 2022-04-26
US20220396793A1 (en) 2022-12-15
EP3017047A2 (en) 2016-05-11
US20190390198A1 (en) 2019-12-26
ES2905257T3 (es) 2022-04-07
US11912996B2 (en) 2024-02-27
US11459566B1 (en) 2022-10-04
US20210180061A1 (en) 2021-06-17
CA2916499A1 (en) 2015-01-08
WO2015003113A2 (en) 2015-01-08
EP3017047A4 (en) 2017-06-14
US20170130221A1 (en) 2017-05-11
US20150011607A1 (en) 2015-01-08
EP3444350B1 (en) 2021-12-01
US11408002B1 (en) 2022-08-09
EP3444350A1 (en) 2019-02-20
US20230069235A1 (en) 2023-03-02
US10844381B2 (en) 2020-11-24
PT3444350T (pt) 2022-02-04
AU2020260479A1 (en) 2020-11-26

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