KR20150004414A - Kras-관련 질환을 치료하기 위한 유기 조성물 - Google Patents

Kras-관련 질환을 치료하기 위한 유기 조성물 Download PDF

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KR20150004414A
KR20150004414A KR1020147033421A KR20147033421A KR20150004414A KR 20150004414 A KR20150004414 A KR 20150004414A KR 1020147033421 A KR1020147033421 A KR 1020147033421A KR 20147033421 A KR20147033421 A KR 20147033421A KR 20150004414 A KR20150004414 A KR 20150004414A
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rti
strand
kras
modified
seq
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Inventor
브라이언 베텐코트
스튜어트 밀스테인
이반카 토우드자르스카
얼 맥도날드
마이클 쥬니어 슐라바흐
프랑크 피. 슈테그마이어
마르쿠스 바르무트
칼리아니 감파
디터 휘스켄
마크 스텀프
얀 바일러
자이납 자가니
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노파르티스 아게
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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
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • A61P35/02Antineoplastic agents specific for leukemia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • 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
    • C12N15/1135Non-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 against oncogenes or tumor suppressor genes
    • 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
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate

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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)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Oncology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
KR1020147033421A 2012-05-02 2013-04-30 Kras-관련 질환을 치료하기 위한 유기 조성물 Withdrawn KR20150004414A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261641588P 2012-05-02 2012-05-02
US61/641,588 2012-05-02
PCT/US2013/038847 WO2013166004A2 (en) 2012-05-02 2013-04-30 Organic compositions to treat kras-related diseases

Publications (1)

Publication Number Publication Date
KR20150004414A true KR20150004414A (ko) 2015-01-12

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ID=48366565

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KR1020147033421A Withdrawn KR20150004414A (ko) 2012-05-02 2013-04-30 Kras-관련 질환을 치료하기 위한 유기 조성물

Country Status (12)

Country Link
US (3) US20150238515A1 (https=)
EP (3) EP3272868A1 (https=)
JP (1) JP2015517466A (https=)
KR (1) KR20150004414A (https=)
CN (2) CN109481455A (https=)
AU (5) AU2013256471A1 (https=)
BR (1) BR112014027337A2 (https=)
CA (2) CA3173610A1 (https=)
EA (1) EA201492004A1 (https=)
HK (1) HK1206387A1 (https=)
MX (1) MX2014013367A (https=)
WO (1) WO2013166004A2 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20180009999A (ko) * 2016-07-20 2018-01-30 주식회사 녹십자지놈 유전성 발달장애 진단용 조성물 및 이의 용도

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW201620525A (zh) * 2014-03-14 2016-06-16 波士頓生醫公司 使k-ras靜默之非對稱干擾rna組成物及其使用方法
KR102589295B1 (ko) 2014-05-14 2023-10-13 타르그이뮨 테라퓨틱스 아게 개선된 폴리에틸렌이민 폴리에틸렌글리콜 벡터
US11180759B2 (en) * 2016-01-19 2021-11-23 The University Of North Carolina At Chapel Hill Methods and compositions using RNA interference and antisense oligonucleotides for inhibition of KRAS
JP2019513371A (ja) 2016-04-01 2019-05-30 アビディティー バイオサイエンシーズ エルエルシー 核酸ポリペプチド組成物とその使用
WO2017173304A1 (en) * 2016-04-01 2017-10-05 Avidity Biosciences Llc Kras nucleic acids and uses thereof
JP7506601B2 (ja) 2017-12-06 2024-06-26 アビディティー バイオサイエンシーズ,インク. 筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法
AU2020287343A1 (en) * 2019-06-05 2021-12-09 Southern Research Institute Thiarabine- and thiarabine prodrug-based treatments
US12146139B2 (en) * 2019-11-14 2024-11-19 The Board Of Regents Of The University Of Oklahoma Oligonucleotide interference treatments of prostate cancer
US11555190B2 (en) 2020-03-19 2023-01-17 Avidity Biosciences, Inc. Compositions and methods of treating Facioscapulohumeral muscular dystrophy
JP7839554B2 (ja) * 2020-04-07 2026-04-02 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル Krasの阻害のためのrna干渉およびアンチセンスオリゴヌクレオチドを使用する方法および組成物
WO2023034537A1 (en) * 2021-09-02 2023-03-09 Molecular Axiom, Llc Compositions and methods for modulating kras expression
MX2024003266A (es) 2021-09-16 2024-04-03 Avidity Biosciences Inc Composiciones y metodos de tratamiento de la distrofia muscular facioescapulohumeral.
CN120677241A (zh) * 2023-01-18 2025-09-19 美国微哲默理有限责任公司 用于调节kras表达的组合物及其用途
CN120769911A (zh) * 2023-02-22 2025-10-10 艾尔塔米拉治疗公司 用于治疗疾病的抑制kras的组合物和方法
WO2026026715A1 (zh) * 2024-07-31 2026-02-05 前沿生物药业(南京)股份有限公司 一种用于抑制KRAS基因表达的siRNA及其修饰物与应用

Family Cites Families (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5030453A (en) 1983-03-24 1991-07-09 The Liposome Company, Inc. Stable plurilamellar vesicles
US6977244B2 (en) 1996-10-04 2005-12-20 Board Of Regents, The University Of Texas Systems Inhibition of Bcl-2 protein expression by liposomal antisense oligodeoxynucleotides
US5891467A (en) 1997-01-31 1999-04-06 Depotech Corporation Method for utilizing neutral lipids to modify in vivo release from multivesicular liposomes
EP2314700A1 (en) 1999-01-28 2011-04-27 Medical College of Georgia Research Institute, Inc Composition and method for in vivo and in vitro attenuation of gene expression using double stranded RNA
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
EP1272630A2 (en) 2000-03-16 2003-01-08 Genetica, Inc. Methods and compositions for rna interference
US6680068B2 (en) 2000-07-06 2004-01-20 The General Hospital Corporation Drug delivery formulations and targeting
WO2002100435A1 (en) 2001-06-11 2002-12-19 Centre Hospitalier Universitaire De Montreal Compositions and methods for enhancing nucleic acid transfer into cells
CA2456977C (en) 2001-08-16 2011-06-07 The Trustees Of The University Of Pennsylvania Synthesis and use of reagents for improved dna lipofection and/or slow release prodrug and drug therapies
US7901708B2 (en) 2002-06-28 2011-03-08 Protiva Biotherapeutics, Inc. Liposomal apparatus and manufacturing methods
MXPA05003287A (es) 2002-09-28 2005-07-05 Massachusetts Inst Technology Influenza terapeutica.
EP2305812A3 (en) * 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
ES2343318T3 (es) 2002-11-26 2010-07-28 University Of Massachusetts Administracion de arnsis.
US20040208921A1 (en) 2003-01-14 2004-10-21 Ho Rodney J. Y. Lipid-drug formulations and methods for targeted delivery of lipid-drug complexes to lymphoid tissues
ES2559828T3 (es) 2003-07-16 2016-02-16 Protiva Biotherapeutics Inc. ARN de interferencia encapsulado en lípidos
CN1845993B (zh) 2003-08-28 2010-06-23 诺瓦提斯公司 具有平端和3'修饰的干扰rna双链体
WO2005045035A2 (en) * 2003-10-23 2005-05-19 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF NOGO AND NOGO RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
EP1714970A1 (en) * 2005-04-22 2006-10-25 Universität des Saarlandes Use of inhibitors of RNAse A-family enzymes for stabilizing oligonucleotides having RNA interfering activity
US8101741B2 (en) 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
WO2007107162A2 (en) 2006-03-23 2007-09-27 Santaris Pharma A/S Small internally segmented interfering rna
GB0608838D0 (en) 2006-05-04 2006-06-14 Novartis Ag Organic compounds
WO2008036841A2 (en) * 2006-09-22 2008-03-27 Dharmacon, Inc. Tripartite oligonucleotide complexes and methods for gene silencing by rna interference
EP2121925A2 (en) * 2007-03-02 2009-11-25 MDRNA, Inc. Nucleic acid compounds for inhibiting ras gene expression and uses thereof
BRPI0811170B8 (pt) 2007-05-22 2021-05-25 Arcturus Therapeutics Inc oligonucleotídeos de rna e complexos de rna substituídos por hidroximetila
CA2704737A1 (en) * 2007-09-18 2009-09-03 Intradigm Corporation Compositions comprising k-ras sirna and methods of use
WO2009062199A1 (en) * 2007-11-09 2009-05-14 Fox Chase Cancer Center EGFR/NEDD9/TGF-β LNTERACTOME AND METHODS OF USE THEREOF FOR THE IDENTIFICATION OF AGENTS HAVING EFFICACY IN THE TREATMENT OF HYPERPROLIFERATIVE DISORDERS
WO2009082817A1 (en) 2007-12-27 2009-07-09 Protiva Biotherapeutics, Inc. Silencing of polo-like kinase expression using interfering rna
US8188060B2 (en) 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation
US20110218334A1 (en) * 2008-07-11 2011-09-08 Alnylam Pharmaceuticals, Inc. PHOSPHOROTHIOATE OLIGONUCLEOTIDES AND NON-NUCLEOSIDIC PHOSPHOROTHIOATES AS DELIVERY AGENTS FOR iRNA AGENTS
CN104651362A (zh) * 2009-04-03 2015-05-27 戴瑟纳制药公司 利用不对称双链rna特异性抑制kras的方法和组合物
WO2010115202A2 (en) * 2009-04-03 2010-10-07 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of kras by blunt ended double-stranded rna
WO2011076807A2 (en) 2009-12-23 2011-06-30 Novartis Ag Lipids, lipid compositions, and methods of using them
US20130116302A1 (en) * 2010-03-12 2013-05-09 Max-Planck-Gesellschaft Zur Foerderung Der Wissenschaften E.V. Pharmaceutical composition for the treatment of chlamydial infection

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20180009999A (ko) * 2016-07-20 2018-01-30 주식회사 녹십자지놈 유전성 발달장애 진단용 조성물 및 이의 용도

Also Published As

Publication number Publication date
CN104583406A (zh) 2015-04-29
AU2020200247A1 (en) 2020-02-06
CA3173610A1 (en) 2013-11-07
US20180153919A1 (en) 2018-06-07
BR112014027337A2 (pt) 2017-07-18
EA201492004A1 (ru) 2015-08-31
EP3736333A1 (en) 2020-11-11
US20150238515A1 (en) 2015-08-27
EP2844757A2 (en) 2015-03-11
MX2014013367A (es) 2014-12-08
JP2015517466A (ja) 2015-06-22
CN109481455A (zh) 2019-03-19
AU2022202991A1 (en) 2022-05-26
US20260069625A1 (en) 2026-03-12
CA2872304A1 (en) 2013-11-07
WO2013166004A3 (en) 2014-01-03
WO2013166004A2 (en) 2013-11-07
AU2013256471A1 (en) 2014-11-13
EP3272868A1 (en) 2018-01-24
HK1206387A1 (en) 2016-01-08
AU2016204832A1 (en) 2016-07-28
AU2017261481A1 (en) 2017-11-30
CA2872304C (en) 2022-12-06

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Patent event date: 20141127

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
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WITN Application deemed withdrawn, e.g. because no request for examination was filed or no examination fee was paid