JP2010532786A - Rna薬剤の両親媒性接合を含む混合ミセルおよびその使用 - Google Patents

Rna薬剤の両親媒性接合を含む混合ミセルおよびその使用 Download PDF

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JP2010532786A
JP2010532786A JP2010516035A JP2010516035A JP2010532786A JP 2010532786 A JP2010532786 A JP 2010532786A JP 2010516035 A JP2010516035 A JP 2010516035A JP 2010516035 A JP2010516035 A JP 2010516035A JP 2010532786 A JP2010532786 A JP 2010532786A
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irna
hydrophilic
acid
pharmaceutical formulation
agent
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トーチリン,ヴラディミール
レヴチェンコ,タチアーナ,エス.
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ノースイースタン ユニバーシティ
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/107Emulsions ; Emulsion preconcentrates; Micelles
    • A61K9/1075Microemulsions or submicron emulsions; Preconcentrates or solids thereof; Micelles, e.g. made of phospholipids or block copolymers
    • 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
    • A61K47/542Carboxylic acids, e.g. a fatty acid or an amino acid
    • 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
    • A61K47/543Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • 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
    • A61K47/543Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • A61K47/544Phospholipids
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    • 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
    • A61K47/554Medicinal 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 a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
    • 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
    • A61K47/69Medicinal 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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6905Medicinal 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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion
    • A61K47/6907Medicinal 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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion the form being a microemulsion, nanoemulsion or micelle
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    • A61P31/12Antivirals
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    • A61P35/00Antineoplastic agents
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    • AHUMAN NECESSITIES
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00Drugs for disorders of the endocrine system
    • AHUMAN NECESSITIES
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    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
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    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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JP2010516035A 2007-07-06 2008-07-07 Rna薬剤の両親媒性接合を含む混合ミセルおよびその使用 Pending JP2010532786A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US94843307P 2007-07-06 2007-07-06
PCT/US2008/008326 WO2009009025A1 (en) 2007-07-06 2008-07-07 Mixed micelles including amphipathic conjugates of rna agents, and uses thereof

Publications (2)

Publication Number Publication Date
JP2010532786A true JP2010532786A (ja) 2010-10-14
JP2010532786A5 JP2010532786A5 (https=) 2011-08-25

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US (1) US20110008395A1 (https=)
EP (1) EP2171095A4 (https=)
JP (1) JP2010532786A (https=)
KR (1) KR20100037120A (https=)
CA (1) CA2692748A1 (https=)
WO (1) WO2009009025A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014180256A (ja) * 2013-03-21 2014-09-29 Kinki Univ 遺伝子試料導入用基板及び遺伝子試料導入方法
JP2019526555A (ja) * 2016-08-23 2019-09-19 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 可逆的に修飾されたオリゴヌクレオチドを含む組成物及びその使用

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CN103342789B (zh) 2008-05-13 2016-11-23 华盛顿大学 用于向细胞内递送的二嵌段共聚物和其多核苷酸复合物
WO2009140421A2 (en) 2008-05-13 2009-11-19 University Of Washington Polymeric carrier
KR101764427B1 (ko) 2008-05-13 2017-08-02 유니버시티 오브 워싱톤 미셀성 어셈블리
WO2010021770A1 (en) 2008-08-22 2010-02-25 University Of Washington Heterogeneous polymeric micelles for intracellular delivery
EP2352522A4 (en) 2008-11-06 2014-03-05 Univ Washington BISPECIFIC AUXILIARIES FOR INTRA-CELLULAR RELEASE
EP2364330B1 (en) 2008-11-06 2015-03-25 University Of Washington Multiblock copolymers
US9593169B2 (en) 2008-12-08 2017-03-14 University Of Washington Omega-functionalized polymers, junction-functionalized block copolymers, polymer bioconjugates, and radical chain extension polymerization
GB0910723D0 (en) * 2009-06-22 2009-08-05 Sylentis Sau Novel drugs for inhibition of gene expression
US9238811B2 (en) 2009-07-14 2016-01-19 Northeastern University siRNA phospholipid conjugate
WO2011062965A2 (en) 2009-11-18 2011-05-26 University Of Washington Through Its Center For Commercialization Targeting monomers and polymers having targeting blocks
EP2591792B1 (en) * 2010-07-09 2016-12-21 The University of Tokyo Composition for nucleic acid delivery, carrier composition, pharmaceutical composition using the composition for nucleic acid delivery or the carrier composition, and method for delivering nucleic acid
EP3027221A1 (en) 2013-07-30 2016-06-08 PhaseRx, Inc. Block copolymers and their conjugates or complexes with oligonucleotides
US11219634B2 (en) 2015-01-21 2022-01-11 Genevant Sciences Gmbh Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells
WO2018126084A1 (en) 2016-12-30 2018-07-05 Phaserx, Inc. Branched peg molecules and related compositions and methods
WO2019217459A1 (en) 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
US20200048305A1 (en) * 2018-08-10 2020-02-13 South Dakota Board Of Regents Glutathione-cholesterol derivatives as brain targeting agents
IL305740A (en) 2021-03-08 2023-11-01 Extiel Ap Llc Apparatus and method for pyrolysis of carbon materials
CN113633613B (zh) * 2021-07-20 2023-04-25 河南大学 一种siRNA胶束、制备方法、组合物及其应用

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WO2004094595A2 (en) * 2003-04-17 2004-11-04 Alnylam Pharmaceuticals Inc. MODIFIED iRNA AGENTS
WO2005041859A2 (en) * 2003-04-30 2005-05-12 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery.

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Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014180256A (ja) * 2013-03-21 2014-09-29 Kinki Univ 遺伝子試料導入用基板及び遺伝子試料導入方法
JP2019526555A (ja) * 2016-08-23 2019-09-19 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 可逆的に修飾されたオリゴヌクレオチドを含む組成物及びその使用
US11390642B2 (en) 2016-08-23 2022-07-19 Dicerna Pharmaceuticals, Inc. Compositions comprising reversibly modified oligonucleotides and uses thereof
JP2022106928A (ja) * 2016-08-23 2022-07-20 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 可逆的に修飾されたオリゴヌクレオチドを含む組成物及びその使用
JP7249080B2 (ja) 2016-08-23 2023-03-30 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 可逆的に修飾されたオリゴヌクレオチドを含む組成物及びその使用
US12037358B2 (en) 2016-08-23 2024-07-16 Dicerna Pharmaceuticals, Inc. Compositions comprising reversibly modified oligonucleotides and uses thereof

Also Published As

Publication number Publication date
WO2009009025A1 (en) 2009-01-15
EP2171095A4 (en) 2012-07-11
US20110008395A1 (en) 2011-01-13
KR20100037120A (ko) 2010-04-08
CA2692748A1 (en) 2009-01-15
EP2171095A1 (en) 2010-04-07

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