JP2019500345A - 肝臓病の処置のための組成物および方法 - Google Patents

肝臓病の処置のための組成物および方法 Download PDF

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JP2019500345A
JP2019500345A JP2018529229A JP2018529229A JP2019500345A JP 2019500345 A JP2019500345 A JP 2019500345A JP 2018529229 A JP2018529229 A JP 2018529229A JP 2018529229 A JP2018529229 A JP 2018529229A JP 2019500345 A JP2019500345 A JP 2019500345A
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seq
protein
nucleobases
intron
target protein
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JP2019500345A5 (https=
Inventor
アズナレズ，イザベル
エム． ナッシュ，ヒュー
エム． ナッシュ，ヒュー
ダブリュ． ホール，サミュエル
ダブリュ． ホール，サミュエル
ジン，エンシュアン
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コールド スプリング ハーバー ラボラトリー
コールド スプリング ハーバー ラボラトリー
ストーク セラピューティクス，インク．
ストーク セラピューティクス，インク．
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Publication of JP2019500345A publication Critical patent/JP2019500345A/ja
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    • 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
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • 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
    • 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
    • C12N2310/30—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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
JP2018529229A 2015-12-14 2016-12-14 肝臓病の処置のための組成物および方法 Pending JP2019500345A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562267238P 2015-12-14 2015-12-14
US62/267,238 2015-12-14
US201662319015P 2016-04-06 2016-04-06
US62/319,015 2016-04-06
PCT/US2016/066564 WO2017106283A1 (en) 2015-12-14 2016-12-14 Compositions and methods for treatment of liver diseases

Publications (2)

Publication Number Publication Date
JP2019500345A true JP2019500345A (ja) 2019-01-10
JP2019500345A5 JP2019500345A5 (https=) 2020-01-30

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EP (1) EP3389672A4 (https=)
JP (1) JP2019500345A (https=)
CA (1) CA3005090A1 (https=)
WO (1) WO2017106283A1 (https=)

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JP2022544702A (ja) * 2019-08-19 2022-10-20 ストーク セラピューティクス，インク． スプライシングおよびタンパク質発現を調節するための組成物および方法
JP2023534557A (ja) * 2020-07-23 2023-08-09 エフ． ホフマン－ラ ロシュ アーゲー Ｒｎａ結合タンパク質部位を標的とするオリゴヌクレオチド

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KR20190139869A (ko) 2017-04-11 2019-12-18 리제너론 파마슈티칼스 인코포레이티드 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정
SG11202001590RA (en) 2017-08-25 2020-03-30 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
EP3461837A1 (en) * 2017-09-28 2019-04-03 Secarna Pharmaceuticals GmbH & Co. KG Inhibitor inhibiting the expression of pprx1
US10961583B2 (en) 2017-10-11 2021-03-30 Regeneron Phramaceuticals, Inc. Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation
GB2610100B (en) 2017-10-23 2023-08-16 Stoke Therapeutics Inc Antisense oligomers for treatment of non-sense mediated RNA decay based conditions and diseases
CN112041439A (zh) * 2018-02-14 2020-12-04 深度基因组学公司 用于威尔森病的寡核苷酸疗法
KR20200140805A (ko) * 2018-02-21 2020-12-16 브리스톨-마이어스 스큅 컴퍼니 Camk2d 안티센스 올리고뉴클레오티드 및 그의 용도
JP7507093B2 (ja) 2018-03-21 2024-06-27 リジェネロン・ファーマシューティカルズ・インコーポレイテッド １７β－水酸化ステロイド脱水素酵素１３型（ＨＳＤ１７Ｂ１３）ｉＲＮＡ組成物およびその使用方法
US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
TWI856973B (zh) * 2018-09-19 2024-10-01 美商Ionis製藥公司 Pnpla3表現之調節劑
US10913951B2 (en) * 2018-10-31 2021-02-09 University of Pittsburgh—of the Commonwealth System of Higher Education Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure
KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
CN111849968A (zh) * 2019-04-30 2020-10-30 中美瑞康核酸技术(南通)研究院有限公司 寡核苷酸分子及其在急性间歇性卟啉症治疗中的应用
US20230212565A1 (en) * 2019-09-20 2023-07-06 Ractigen Therapeutics Nucleic acid molecule for treating thrombocytopenia and application thereof
EP4045062A1 (en) * 2019-10-14 2022-08-24 Astrazeneca AB Modulators of pnpla3 expression
WO2021113773A2 (en) 2019-12-04 2021-06-10 Pai Athma A Identifying non-productive splice sites
TWI817107B (zh) * 2020-04-15 2023-10-01 中央研究院 寡核苷酸共軛物及其製備與應用
BR112022022889A2 (pt) 2020-05-11 2023-04-04 Stoke Therapeutics Inc Oligômeros antissentido de opa1 para tratamento de condições e doenças
EP4150078A1 (en) * 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
WO2022109030A1 (en) * 2020-11-17 2022-05-27 Skyhawk Therapeutics, Inc. Rna-targeting splicing modifiers for treatment of pnpla3-associated conditions and diseases
WO2025226980A2 (en) * 2024-04-25 2025-10-30 Insitro, Inc. Compositions and methods for treating metabolic dysfunction-associated steatotic liver disease

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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022544702A (ja) * 2019-08-19 2022-10-20 ストーク セラピューティクス，インク． スプライシングおよびタンパク質発現を調節するための組成物および方法
JP2023534557A (ja) * 2020-07-23 2023-08-09 エフ． ホフマン－ラ ロシュ アーゲー Ｒｎａ結合タンパク質部位を標的とするオリゴヌクレオチド

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WO2017106283A1 (en) 2017-06-22
CA3005090A1 (en) 2017-06-22
EP3389672A1 (en) 2018-10-24
EP3389672A4 (en) 2019-08-14

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