CN106714908A - 原位自组装的毒性rna抑制剂 - Google Patents

原位自组装的毒性rna抑制剂 Download PDF

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Publication number
CN106714908A
CN106714908A CN201580050560.0A CN201580050560A CN106714908A CN 106714908 A CN106714908 A CN 106714908A CN 201580050560 A CN201580050560 A CN 201580050560A CN 106714908 A CN106714908 A CN 106714908A
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CN
China
Prior art keywords
rna
cell
permeable
k4nmes
dmf
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Pending
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CN201580050560.0A
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English (en)
Chinese (zh)
Inventor
马修·D·迪斯尼
苏珊娜·G·勒祖茨泽克
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Scripps Research Institute
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Scripps Research Institute
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Publication of CN106714908A publication Critical patent/CN106714908A/zh
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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)
CN201580050560.0A 2014-07-18 2015-07-17 原位自组装的毒性rna抑制剂 Pending CN106714908A (zh)

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

Publications (1)

Publication Number Publication Date
CN106714908A true CN106714908A (zh) 2017-05-24

Family

ID=55079084

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Application Number Title Priority Date Filing Date
CN201580050560.0A Pending CN106714908A (zh) 2014-07-18 2015-07-17 原位自组装的毒性rna抑制剂

Country Status (9)

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

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2015289524A1 (en) * 2014-07-18 2017-02-02 The Scripps Research Institute Toxic RNA inhibitors self-assembled in situ
WO2018098297A1 (en) * 2016-11-28 2018-05-31 The Scripps Research Institute Precise small molecule recognition of a toxic cug rna repeat expansion
RU2020112854A (ru) 2017-11-30 2021-12-30 Арракис Терапьютикс, Инк. Фотозонды, связывающие нуклеиновые кислоты, и способы их применения
US12571031B2 (en) 2020-09-02 2026-03-10 Promega Corporation Nucleic acid modifying reagents and uses thereof
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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130183702A1 (en) * 2010-09-29 2013-07-18 Kansas State University Research Foundation Protease selective supramolecular assemblies
WO2013138662A1 (en) * 2012-03-16 2013-09-19 4S3 Bioscience, Inc. Antisense conjugates for decreasing expression of dmpk
US20140050778A1 (en) * 2010-12-28 2014-02-20 University Of Rochester Nucleic acid binding compounds, methods of making, and use thereof

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9150612B2 (en) * 2007-02-23 2015-10-06 The Research Foundation Of State University Of New York RNA targeting compounds and methods for making and using same
US9550769B2 (en) * 2012-08-30 2017-01-24 The Scripps Research Institute Small molecules targeting repeat r(CGG) sequences
AU2015289524A1 (en) * 2014-07-18 2017-02-02 The Scripps Research Institute Toxic RNA inhibitors self-assembled in situ

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130183702A1 (en) * 2010-09-29 2013-07-18 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
WO2013138662A1 (en) * 2012-03-16 2013-09-19 4S3 Bioscience, Inc. Antisense conjugates for decreasing expression of dmpk

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
ALEXEI PUSHECHNIKOV等: "Rational Design of Ligands Targeting Triplet Repeating Transcripts That Cause RNA Dominant Disease: Application to Myotonic Muscular Dystrophy Type 1 and Spinocerebellar Ataxia Type 3", 《J. AM. CHEM. SOC.》 *
BARBARA J. CARTER等: "Site-specific cleavage of RNA by Fe(II)bleomycin", 《PROC. NATL. ACAD. SCI. USA》 *
MELISSA M. LEE等: "Rational and Modular Design of Potent Ligands Targeting the RNA That Causes Myotonic Dystrophy 2", 《ACS CHEMICAL BIOLOGY》 *

Also Published As

Publication number Publication date
JP2019068831A (ja) 2019-05-09
AU2015289524A1 (en) 2017-02-02
US10471057B2 (en) 2019-11-12
JP6461306B2 (ja) 2019-01-30
IL250113A0 (en) 2017-03-30
JP2017522323A (ja) 2017-08-10
SG11201700387QA (en) 2017-02-27
US10220031B2 (en) 2019-03-05
EP3169404A1 (en) 2017-05-24
US20170143703A1 (en) 2017-05-25
US20190151310A1 (en) 2019-05-23
WO2016011348A1 (en) 2016-01-21
CA2955428A1 (en) 2016-01-21
EP3169404A4 (en) 2018-02-07

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