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

Info

Publication number
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
Authority
JP
Japan
Prior art keywords
irna
hydrophilic
acid
pharmaceutical formulation
agent
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2010516035A
Other languages
English (en)
Japanese (ja)
Other versions
JP2010532786A5 (enExample
Inventor
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.
Original Assignee
ノースイースタン ユニバーシティ
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by ノースイースタン ユニバーシティ filed Critical ノースイースタン ユニバーシティ
Publication of JP2010532786A publication Critical patent/JP2010532786A/ja
Publication of JP2010532786A5 publication Critical patent/JP2010532786A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • 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
    • A61K47/554Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • 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
    • A61P37/00Drugs for immunological or allergic disorders
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00Drugs for disorders of the endocrine system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dispersion Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Nanotechnology (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Botany (AREA)
  • Biochemistry (AREA)
  • Endocrinology (AREA)
  • Oncology (AREA)
  • Virology (AREA)
  • Communicable Diseases (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
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 (enExample) 2011-08-25

Family

ID=40228904

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2010516035A Pending JP2010532786A (ja) 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

Country Status (6)

Country Link
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

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102066444A (zh) 2008-05-13 2011-05-18 华盛顿大学 胶束装配体
WO2009140421A2 (en) 2008-05-13 2009-11-19 University Of Washington Polymeric carrier
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
WO2010021770A1 (en) 2008-08-22 2010-02-25 University Of Washington Heterogeneous polymeric micelles for intracellular delivery
US8822213B2 (en) 2008-11-06 2014-09-02 University Of Washington Bispecific intracellular delivery vehicles
MX2011004242A (es) 2008-11-06 2011-07-20 Univ Washington Copolimeros multibloque.
WO2010077678A2 (en) 2008-12-08 2010-07-08 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
WO2011008857A1 (en) 2009-07-14 2011-01-20 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
AU2014296278C1 (en) 2013-07-30 2023-02-02 Genevant Sciences Gmbh Block copolymers and their conjugates or complexes with oligonucleotides
WO2016118697A1 (en) 2015-01-21 2016-07-28 Phaserx, Inc. Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells
US11684584B2 (en) 2016-12-30 2023-06-27 Genevant Sciences Gmbh Branched peg molecules and related compositions and methods
KR20210018267A (ko) 2018-05-07 2021-02-17 알닐람 파마슈티칼스 인코포레이티드 간외 전달
US20200048305A1 (en) * 2018-08-10 2020-02-13 South Dakota Board Of Regents Glutathione-cholesterol derivatives as brain targeting agents
MX2023010517A (es) 2021-03-08 2023-11-16 Extiel Ap Llc Dispositivo para la pirólisis de materiales carbonosos y método.
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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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.
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)

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2219119C (en) * 1995-05-04 2012-10-23 Nexstar Pharmaceuticals, Inc. Nucleic acid ligand complexes
AU2136501A (en) * 1999-12-10 2001-06-18 Celator Technologies Inc. Lipid carrier compositions with protected surface reactive functions
EP1608735A4 (en) * 2003-04-03 2008-11-05 Alnylam Pharmaceuticals RNAI CONJUGATES

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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.

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
JPN6013038493; 'Progress towards in vivo use of siRNAs.' Mol Ther. 13(4), 2006, 644-670 *
JPN6013038494; 'Therapeutic potential for microRNAs.' Adv Drug Deliv Rev. 59(2-3), 2007, 101-114 *

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

Similar Documents

Publication Publication Date Title
JP2010532786A (ja) 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
US20250205272A1 (en) Modified rna agents with reduced off-target effect
CN101903018B (zh) 用于递送核酸基因的ldl样阳离子纳米微粒、其制备方法以及使用其递送核酸基因的方法
JP2018184453A (ja) 修飾RNAi剤
JP7536007B2 (ja) 修飾二重鎖オリゴヌクレオチド
JP2019503394A (ja) 分岐オリゴヌクレオチド
EP2892505B1 (en) Lipid assemblies comprising anionic lysolipids and use thereof
US20250283071A1 (en) Modified double stranded oligonucleotides
JP5865319B2 (ja) iRNA複合体
US12497613B2 (en) Modified oligonucleotides
Sajid Synthesis, Characterization, and Evaluation of Amphiphilic Cell-Penetrating Peptides as siRNA Delivery Tools for Cancer Therapy

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20100723

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20110704

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20110704

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20130805

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20140109