JP7348067B2 - ＳＥＲＰＩＮＡ１ ｉＲＮＡ組成物およびその使用方法 - Google Patents

ＳＥＲＰＩＮＡ１ ｉＲＮＡ組成物およびその使用方法 Download PDF

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JP7348067B2
JP7348067B2 JP2019547592A JP2019547592A JP7348067B2 JP 7348067 B2 JP7348067 B2 JP 7348067B2 JP 2019547592 A JP2019547592 A JP 2019547592A JP 2019547592 A JP2019547592 A JP 2019547592A JP 7348067 B2 JP7348067 B2 JP 7348067B2
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antisense strand
dsrna
strand
nucleotides
double
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JP2020500929A5 (enExample
JP2020500929A (ja
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マーク・ケイ・シュリーゲル
マジャ・ジャナス
バサント・アール・ジャダブ
ドナルド・フォスター
ムティア・マノハラン
カラントッタティル・ジー・ラジーブ
アレクサンダー・ブイ・ケリン
クラウス・カリセ
ジャヤプラカシュ・ケイ・ナーイル
マーティン・エイ・マイアー
シゲオ・マツダ
ムトゥサミー・ジャヤラマン
アルフィカ・セーガル
クリストファー・ブラウン
ケビン・フィッツジェラルド
スチュアート・ミルスタイン
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Alnylam Pharmaceuticals Inc
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2019547592A 2016-11-23 2017-11-21 ＳＥＲＰＩＮＡ１ ｉＲＮＡ組成物およびその使用方法 Active JP7348067B2 (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201662425907P 2016-11-23 2016-11-23
US62/425,907 2016-11-23
US201762548589P 2017-08-22 2017-08-22
US62/548,589 2017-08-22
US201762549099P 2017-08-23 2017-08-23
US62/549,099 2017-08-23
US201762561514P 2017-09-21 2017-09-21
US62/561,514 2017-09-21
PCT/US2017/062701 WO2018098117A1 (en) 2016-11-23 2017-11-21 SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF

Publications (3)

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JP2020500929A JP2020500929A (ja) 2020-01-16
JP2020500929A5 JP2020500929A5 (enExample) 2021-01-07
JP7348067B2 true JP7348067B2 (ja) 2023-09-20

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US (3) US10995336B2 (enExample)
EP (1) EP3545093A1 (enExample)
JP (1) JP7348067B2 (enExample)
KR (1) KR20190082317A (enExample)
CN (1) CN110114463B (enExample)
AU (1) AU2017363604A1 (enExample)
BR (1) BR112019010342A2 (enExample)
CA (1) CA3044562A1 (enExample)
CL (1) CL2019001388A1 (enExample)
CO (1) CO2019005992A2 (enExample)
CR (1) CR20190294A (enExample)
DO (1) DOP2019000132A (enExample)
IL (1) IL266746A (enExample)
MA (1) MA46923A (enExample)
MX (1) MX2019005898A (enExample)
MY (1) MY199978A (enExample)
PH (1) PH12019550076A1 (enExample)
RU (2) RU2761667C2 (enExample)
SG (1) SG10201913877WA (enExample)
TW (2) TW202313978A (enExample)
UY (1) UY37494A (enExample)
WO (1) WO2018098117A1 (enExample)
ZA (1) ZA202103080B (enExample)

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TWI664187B (zh) 2013-05-22 2019-07-01 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
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
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
CA3045045A1 (en) 2017-01-10 2018-07-19 Zhen Li Alpha-1 antitrypsin (aat) rnai agents, compositions including aat rnai agents, and methods of use
MX2020009812A (es) * 2018-03-21 2021-01-08 Regeneron Pharma COMPOSICIONES DE ARNI DE 17ß-HIDROXIESTEROIDE DESHIDROGENASA TIPO 13 (HSD17B13) Y MÉTODOS DE USO DE LAS MISMAS.
US20210388356A1 (en) * 2018-11-09 2021-12-16 Alnylam Pharmaceuticals, Inc. Modified double stranded oligonucleotide
US20220211743A1 (en) * 2019-05-17 2022-07-07 Alnylam Pharmaceuticals, Inc. Oral delivery of oligonucleotides
EP3998355A4 (en) 2019-07-08 2023-10-11 MD Healthcare Inc. Method for diagnosing brain tumor through bacterial metagenomic analysis
WO2021022109A1 (en) * 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. SERPIN FAMILY F MEMBER 2 (SERPINF2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
KR20230011913A (ko) 2020-03-04 2023-01-25 버브 테라퓨틱스, 인크. 표적화된 rna 전달을 위한 조성물 및 방법
JP2023549505A (ja) * 2020-11-13 2023-11-27 ザ ジョージ ワシントン ユニバーシティ， ア コングレッショナリー チャータード ノット－フォー－プロフィット コーポレイション Ｂ－スペクトリン（ｓｐｔｂｎ１）欠損はマウスを高脂肪食起因性肝疾患及び癌への進展から保護する
WO2022162153A1 (en) 2021-01-30 2022-08-04 E-Therapeutics Plc Conjugated oligonucleotide compounds, methods of making and uses thereof
EP4176061A1 (en) * 2021-01-30 2023-05-10 E-Therapeutics plc Nucleic acids containing abasic nucleotides
EP4413130A1 (en) * 2021-10-08 2024-08-14 E-Therapeutics plc Nucleic acids containing abasic nucleosides
IL315546A (en) * 2022-03-16 2024-11-01 Empirico Inc GALNAC compositions to improve siRNA bioavailability
JP2025512835A (ja) 2022-03-28 2025-04-22 エンピリコ インク． 修飾オリゴヌクレオチド
AU2023313030A1 (en) * 2022-07-27 2025-01-09 E-Therapeutics Plc Nucleic acid compounds
CN117070583B (zh) * 2023-10-16 2024-05-14 吉林凯莱英制药有限公司 抑制PCSK9基因表达的siRNA的制备方法
WO2025136994A1 (en) * 2023-12-19 2025-06-26 Sirius Therapeutics, Inc. Modifications of polynucleic acid molecules and uses thereof

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