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 (enExample
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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)
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  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
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  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (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

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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 (enExample) 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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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 (enExample)
EP (3) EP3017047A4 (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)
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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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EP3766975A1 (en) 2010-10-29 2021-01-20 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using short interfering nucleic acid (sina)
KR102463973B1 (ko) 2013-05-22 2022-11-07 알닐람 파마슈티칼스 인코포레이티드 ＳＥＲＰＩＮＡ1 ｉＲＮＡ 조성물 및 이의 사용 방법
EP3017047A4 (en) 2013-07-03 2017-06-14 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
CN108699557B (zh) * 2015-12-04 2025-08-15 诺华股份有限公司 用于免疫肿瘤学的组合物和方法
WO2017139616A1 (en) * 2016-02-10 2017-08-17 Arrowhead Pharmaceuticals, Inc. Methods of treatment for alpha-1 antitrypsin deficiency
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TW202313978A (zh) 2016-11-23 2023-04-01 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
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CN111971051A (zh) * 2018-04-13 2020-11-20 迪克纳制药公司 用增加tm的核苷酸修饰的双链核酸抑制剂分子
EP3980540A4 (en) 2019-06-06 2023-11-01 Arrowhead Pharmaceuticals, Inc. METHOD FOR TREATING ALPHA-1 ANTITRYPSIN DEFICIENCY (AATD)
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
JP2024533312A (ja) * 2021-09-08 2024-09-12 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー Ｓｅｒｐｉｎａ調節組成物及び方法
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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
JP7846095B2 (ja) 2020-09-15 2026-04-14 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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US11912996B2 (en) 2024-02-27
PT3444350T (pt) 2022-02-04
US11459566B1 (en) 2022-10-04
AU2014284298B2 (en) 2020-12-10
US20190390198A1 (en) 2019-12-26
CA2916499A1 (en) 2015-01-08
US11851658B2 (en) 2023-12-26
US11408002B1 (en) 2022-08-09
US11312960B1 (en) 2022-04-26
JP2024075702A (ja) 2024-06-04
EP3444350A1 (en) 2019-02-20
US20170130221A1 (en) 2017-05-11
US20150011607A1 (en) 2015-01-08
US20220228149A1 (en) 2022-07-21
JP2020188771A (ja) 2020-11-26
ES2905257T3 (es) 2022-04-07
US10370655B2 (en) 2019-08-06
US20220396793A1 (en) 2022-12-15
PL3444350T3 (pl) 2022-03-21
US11312961B1 (en) 2022-04-26
AU2020260479A1 (en) 2020-11-26
EP3017047A4 (en) 2017-06-14
EP4012031A1 (en) 2022-06-15
DK3444350T3 (da) 2022-02-07
WO2015003113A3 (en) 2015-02-26
AU2020260479B2 (en) 2023-06-15
AU2014284298A1 (en) 2016-01-21
HUE058794T2 (hu) 2022-09-28
US20230069235A1 (en) 2023-03-02
US10844381B2 (en) 2020-11-24
US9458457B2 (en) 2016-10-04
US11208658B2 (en) 2021-12-28
US20210180061A1 (en) 2021-06-17
EP3444350B1 (en) 2021-12-01
WO2015003113A2 (en) 2015-01-08
EP3017047A2 (en) 2016-05-11
JP2022078069A (ja) 2022-05-24

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