CN104302768A - 治疗β-联蛋白相关疾病的有机组合物 - Google Patents

治疗β-联蛋白相关疾病的有机组合物 Download PDF

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Publication number
CN104302768A
CN104302768A CN201380013297.9A CN201380013297A CN104302768A CN 104302768 A CN104302768 A CN 104302768A CN 201380013297 A CN201380013297 A CN 201380013297A CN 104302768 A CN104302768 A CN 104302768A
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strand
catenin
rnai agent
nucleotides
sequence
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Chinese (zh)
Inventor
D·许斯肯
B·贝滕科特
D·布姆克罗特
S·库奇曼奇
S·米尔斯坦
M·小楚拉巴赫
F·P·斯泰格迈尔
M·瓦尔穆特
J·魏勒
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Arrowhead Pharmaceuticals Inc
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Novartis AG
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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
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/02—Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
    • 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
    • C12N2310/14—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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
CN201380013297.9A 2012-01-09 2013-01-08 治疗β-联蛋白相关疾病的有机组合物 Pending CN104302768A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201261584530P 2012-01-09 2012-01-09
US61/584,530 2012-01-09
US201261598530P 2012-02-14 2012-02-14
US61/598,530 2012-02-14
PCT/IB2013/050159 WO2013105022A2 (en) 2012-01-09 2013-01-08 Organic compositions to treat beta-catenin-related diseases

Publications (1)

Publication Number Publication Date
CN104302768A true CN104302768A (zh) 2015-01-21

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CN201380013297.9A Pending CN104302768A (zh) 2012-01-09 2013-01-08 治疗β-联蛋白相关疾病的有机组合物

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Country Link
US (3) US10023862B2 (enExample)
EP (1) EP2802658A2 (enExample)
JP (1) JP6158833B2 (enExample)
CN (1) CN104302768A (enExample)
AU (1) AU2013208720A1 (enExample)
BR (1) BR112014016870A2 (enExample)
CA (1) CA2860676A1 (enExample)
HK (1) HK1205758A1 (enExample)
WO (1) WO2013105022A2 (enExample)

Cited By (1)

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CN114846142A (zh) * 2019-10-29 2022-08-02 箭头药业股份有限公司 用于抑制β-ENaC表达的RNAi试剂、其组合物和使用方法

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JP6999664B2 (ja) * 2016-10-31 2022-01-18 サムスン ライフ パブリック ウェルフェア ファウンデーション 胆汁酸を含む虚血性再灌流損傷の予防または治療用薬学的組成物
US11813280B2 (en) 2018-01-05 2023-11-14 Dicerna Pharmaceuticals, Inc. Reducing beta-catenin and IDO expression to potentiate immunotherapy
AU2019222767A1 (en) * 2018-02-14 2020-08-27 Deep Genomics Incorporated Oligonucleotide therapy for Wilson disease
CN111500694B (zh) * 2019-01-31 2023-02-24 中国科学院脑科学与智能技术卓越创新中心 Baz2b基因作为靶点在缓解衰老中的应用
EP4259134A4 (en) 2020-12-09 2024-10-30 The Regents Of The University Of Michigan Compositions and methods for treating wnt-driven cancer
US20220273695A1 (en) * 2021-03-01 2022-09-01 Steadman Philippon Research Institute Mcm for gene therapy to activate wnt pathway
AU2022316139A1 (en) * 2021-07-23 2024-01-18 Alnylam Pharmaceuticals, Inc. Beta-catenin (ctnnb1) irna compositions and methods of use thereof
WO2025090541A1 (en) * 2023-10-25 2025-05-01 The Regents Of The University Of California Covalent degraders of oncogenic transcription factors

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AU2013208720A1 (en) 2014-07-24
JP2015503608A (ja) 2015-02-02
CA2860676A1 (en) 2013-07-18
US10023862B2 (en) 2018-07-17
HK1205758A1 (en) 2015-12-24
US20180282728A1 (en) 2018-10-04
BR112014016870A2 (pt) 2017-06-27
US20150291954A1 (en) 2015-10-15
WO2013105022A2 (en) 2013-07-18
WO2013105022A3 (en) 2013-10-31
JP6158833B2 (ja) 2017-07-05
US20250270555A1 (en) 2025-08-28
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