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 (enrdf_load_stackoverflow
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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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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • CCHEMISTRY; METALLURGY
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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
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  • Genetics & Genomics (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (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)

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JP2019500345A true JP2019500345A (ja) 2019-01-10
JP2019500345A5 JP2019500345A5 (enrdf_load_stackoverflow) 2020-01-30

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EP (1) EP3389672A4 (enrdf_load_stackoverflow)
JP (1) JP2019500345A (enrdf_load_stackoverflow)
CA (1) CA3005090A1 (enrdf_load_stackoverflow)
WO (1) WO2017106283A1 (enrdf_load_stackoverflow)

Cited By (2)

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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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AU2015327836B2 (en) 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
AU2016334804B2 (en) 2015-10-09 2022-03-31 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
EP3933041B1 (en) 2015-12-14 2024-01-31 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant retardation
JP7094963B2 (ja) 2017-01-23 2022-07-04 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 Hsd17b13バリアントおよびその使用
CA3059348A1 (en) 2017-04-11 2018-10-18 Regeneron Pharmaceuticals, Inc. Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (hsd17b) family
CA3073515A1 (en) 2017-08-25 2019-02-28 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
AU2018348195B2 (en) 2017-10-11 2025-05-15 Regeneron Pharmaceuticals, Inc. Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation
SMT202500127T1 (it) 2017-10-23 2025-05-12 Stoke Therapeutics Inc Oligomeri antisenso per il trattamento di malattie e condizioni basate sul decadimento dell’rna mediato da un nonsenso
WO2019161105A1 (en) * 2018-02-14 2019-08-22 Deep Genomics Incorporated Oligonucleotide therapy for wilson disease
MX2020008581A (es) * 2018-02-21 2020-09-21 Bristol Myers Squibb Co Oligonucleotidos antisentido de la proteina cinasa del tipo ii delta dependiente del calcio/calmodulina (camk2d) y sus usos.
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.
JP2021523227A (ja) 2018-05-04 2021-09-02 ストーク セラピューティクス,インク. コレステリルエステル蓄積症の処置のための方法及び組成物
TWI869213B (zh) 2018-09-19 2025-01-01 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
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
KR20220070206A (ko) * 2019-09-20 2022-05-30 락티젠 세러퓨틱스 혈소판 감소증 치료용 핵산 분자 및 그의 이용
WO2021074772A1 (en) * 2019-10-14 2021-04-22 Astrazeneca Ab Modulators of pnpla3 expression
EP4069255A4 (en) * 2019-12-04 2024-02-21 Pai, Athma A. IDENTIFICATION OF NON-PRODUCTIVE SPLICE JOINTS
TW202208627A (zh) 2020-05-11 2022-03-01 美商斯托克治療公司 用於病症及疾病之治療的opa1反義寡聚物
WO2021231698A1 (en) * 2020-05-15 2021-11-18 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

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

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