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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Pending
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CN201380013297.9A
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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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Publication of CN104302768A publication Critical patent/CN104302768A/zh
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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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Application Number Title Priority Date Filing Date
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

Country Status (9)

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

Cited By (1)

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Publication number Priority date Publication date Assignee Title
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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EP3134527B1 (en) * 2014-04-22 2018-12-12 Medizinische Hochschule Hannover Lncrnas for therapy and diagnosis of angiogenesis
JP6987084B2 (ja) * 2016-03-16 2021-12-22 ディセルナ ファーマシューティカルズ インコーポレイテッド β−カテニン関連疾患又は障害の治療のための組成物及び方法
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
EP3697423A4 (en) 2018-01-05 2021-08-11 Dicerna Pharmaceuticals, Inc. REDUCED EXPRESSION OF BETA-CATENIN AND IDO TO POTENTIALIZE IMMUNOTHERAPY
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
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
US20240082272A1 (en) 2020-12-09 2024-03-14 The Regents Of The University Of Michigan Compositions and methods for treating wnt-driven cancer
EP4301339A2 (en) * 2021-03-01 2024-01-10 Steadman Philippon Research Institute Compositions comprising catenin mrna and their use for healing bone fractures
CA3226878A1 (en) * 2021-07-23 2023-01-26 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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HK1205758A1 (en) 2015-12-24
US20150291954A1 (en) 2015-10-15
JP2015503608A (ja) 2015-02-02
BR112014016870A2 (pt) 2017-06-27
WO2013105022A2 (en) 2013-07-18
US20180282728A1 (en) 2018-10-04
AU2013208720A1 (en) 2014-07-24
JP6158833B2 (ja) 2017-07-05
WO2013105022A3 (en) 2013-10-31
EP2802658A2 (en) 2014-11-19
CA2860676A1 (en) 2013-07-18
US20250270555A1 (en) 2025-08-28
US10023862B2 (en) 2018-07-17

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