JP6461306B2 - インサイチュで自己組織化した、毒性のあるｒｎａ阻害剤 - Google Patents

インサイチュで自己組織化した、毒性のあるｒｎａ阻害剤 Download PDF

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JP6461306B2
JP6461306B2 JP2017502695A JP2017502695A JP6461306B2 JP 6461306 B2 JP6461306 B2 JP 6461306B2 JP 2017502695 A JP2017502695 A JP 2017502695A JP 2017502695 A JP2017502695 A JP 2017502695A JP 6461306 B2 JP6461306 B2 JP 6461306B2
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rna
dmf
k4nmes
mmol
cell
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JP2017522323A (ja
JP2017522323A5 (enExample
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ディズニー，マシュー・ディー
ラズチェック，スザンヌ・ジー
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ザ スクリプス リサーチ インスティテュート
ザ スクリプス リサーチ インスティテュート
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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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/496—Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
    • 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
    • A61K31/702—Oligosaccharides, i.e. having three to five saccharide radicals attached to each other by glycosidic linkages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/07—Tetrapeptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/54—Medicinal 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
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/54—Medicinal 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
    • A61K47/55—Medicinal 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 the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/54—Medicinal 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
    • A61K47/555—Medicinal 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 pre-targeting systems involving an organic compound, other than a peptide, protein or antibody, for targeting specific cells
    • A61K47/557—Medicinal 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 pre-targeting systems involving an organic compound, other than a peptide, protein or antibody, for targeting specific cells the modifying agent being biotin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • Pain & Pain Management (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
JP2017502695A 2014-07-18 2015-07-17 インサイチュで自己組織化した、毒性のあるｒｎａ阻害剤 Active JP6461306B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462026266P 2014-07-18 2014-07-18
US62/026,266 2014-07-18
PCT/US2015/040902 WO2016011348A1 (en) 2014-07-18 2015-07-17 Toxic rna inhibitors self-assembled in situ

Related Child Applications (1)

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JP2018241478A Division JP2019068831A (ja) 2014-07-18 2018-12-25 インサイチュで自己組織化した、毒性のあるｒｎａ阻害剤

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JP2017522323A JP2017522323A (ja) 2017-08-10
JP2017522323A5 JP2017522323A5 (enExample) 2018-08-30
JP6461306B2 true JP6461306B2 (ja) 2019-01-30

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JP2018241478A Pending JP2019068831A (ja) 2014-07-18 2018-12-25 インサイチュで自己組織化した、毒性のあるｒｎａ阻害剤

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Country Status (9)

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US (2) US10220031B2 (enExample)
EP (1) EP3169404A4 (enExample)
JP (2) JP6461306B2 (enExample)
CN (1) CN106714908A (enExample)
AU (1) AU2015289524A1 (enExample)
CA (1) CA2955428A1 (enExample)
IL (1) IL250113A0 (enExample)
SG (1) SG11201700387QA (enExample)
WO (1) WO2016011348A1 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6461306B2 (ja) * 2014-07-18 2019-01-30 ザ スクリプス リサーチ インスティテュート インサイチュで自己組織化した、毒性のあるｒｎａ阻害剤
WO2018098297A1 (en) * 2016-11-28 2018-05-31 The Scripps Research Institute Precise small molecule recognition of a toxic cug rna repeat expansion
MX2020003695A (es) 2017-11-30 2020-08-03 Arrakis Therapeutics Inc Fotosondas de union a acido nucleico y usos de las mismas.
KR20230079375A (ko) * 2020-09-02 2023-06-07 프로메가 코포레이션 핵산 변형 시약 및 이의 용도
JP2025507917A (ja) * 2022-03-01 2025-03-21 プロメガ コーポレイション 核酸架橋試薬及びその使用

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2678967C (en) * 2007-02-23 2013-10-01 The Research Foundation Of State University Of New York Rna targeting compounds and methods for making and using same
WO2012050896A2 (en) * 2010-09-29 2012-04-19 Kansas State University Research Foundation Protease selective supramolecular assemblies
US20140050778A1 (en) * 2010-12-28 2014-02-20 University Of Rochester Nucleic acid binding compounds, methods of making, and use thereof
US9610362B2 (en) * 2012-03-16 2017-04-04 Valerion Therapeutics, Llc Antisense conjugates for decreasing expression of DMPK
US9550769B2 (en) * 2012-08-30 2017-01-24 The Scripps Research Institute Small molecules targeting repeat r(CGG) sequences
JP6461306B2 (ja) * 2014-07-18 2019-01-30 ザ スクリプス リサーチ インスティテュート インサイチュで自己組織化した、毒性のあるｒｎａ阻害剤

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Publication number Publication date
JP2019068831A (ja) 2019-05-09
JP2017522323A (ja) 2017-08-10
EP3169404A1 (en) 2017-05-24
US10471057B2 (en) 2019-11-12
IL250113A0 (en) 2017-03-30
US20190151310A1 (en) 2019-05-23
CN106714908A (zh) 2017-05-24
US10220031B2 (en) 2019-03-05
WO2016011348A1 (en) 2016-01-21
CA2955428A1 (en) 2016-01-21
SG11201700387QA (en) 2017-02-27
AU2015289524A1 (en) 2017-02-02
US20170143703A1 (en) 2017-05-25
EP3169404A4 (en) 2018-02-07

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