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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JP2010532786A5 (enExample
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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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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/107—Emulsions ; Emulsion preconcentrates; Micelles
    • A61K9/1075—Microemulsions or submicron emulsions; Preconcentrates or solids thereof; Micelles, e.g. made of phospholipids or block copolymers
    • 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
    • A61K47/542—Carboxylic acids, e.g. a fatty acid or an amino acid
    • 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
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    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • 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
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    • 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
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
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    • 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
    • A61K47/554—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 a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
    • 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
    • A61K47/69—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6905—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 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
    • A61K47/6907—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 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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    • 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
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    • C12N2320/32—Special delivery means, e.g. tissue-specific

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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

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JP2010532786A true JP2010532786A (ja) 2010-10-14
JP2010532786A5 JP2010532786A5 (enExample) 2011-08-25

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

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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CA2724014C (en) 2008-05-13 2016-09-13 University Of Washington Micellic assemblies
WO2009140421A2 (en) 2008-05-13 2009-11-19 University Of Washington Polymeric carrier
MX2010012238A (es) 2008-05-13 2011-04-11 Univ Washington Copolimeros dibloque y sus complejos con polinucleotidos destinados a introducirlos en celulas.
CA2734917A1 (en) 2008-08-22 2010-02-25 University Of Washington Heterogeneous polymeric micelles for intracellular delivery
EP2364330B1 (en) 2008-11-06 2015-03-25 University Of Washington Multiblock copolymers
EP2352522A4 (en) 2008-11-06 2014-03-05 Univ Washington BISPECIFIC AUXILIARIES FOR INTRA-CELLULAR RELEASE
CA2745926A1 (en) 2008-12-08 2010-07-08 Phaserx, Inc. 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
CN102971002B (zh) * 2010-07-09 2019-04-05 国立大学法人东京大学 核酸递送用组合物及载体组合物、使用其的药物组合物以及核酸递送方法
AU2014296278C1 (en) 2013-07-30 2023-02-02 Genevant Sciences Gmbh Block copolymers and their conjugates or complexes with oligonucleotides
EP3247328B1 (en) 2015-01-21 2026-01-07 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
CA3098623A1 (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
JP2024514408A (ja) 2021-03-08 2024-04-02 エクスティエル エーピー,エルエルシー 炭素材料を熱分解するためのデバイス及び方法
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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JP2006522158A (ja) * 2003-04-03 2006-09-28 アルナイラム ファーマシューティカルズ インコーポレイテッド iRNA複合体

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US20060275903A1 (en) * 2002-02-20 2006-12-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
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

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

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