JP2011517676A - インビボでｒｎａ干渉を媒介するための組成物および方法 - Google Patents

インビボでｒｎａ干渉を媒介するための組成物および方法 Download PDF

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Publication number
JP2011517676A
JP2011517676A JP2011502065A JP2011502065A JP2011517676A JP 2011517676 A JP2011517676 A JP 2011517676A JP 2011502065 A JP2011502065 A JP 2011502065A JP 2011502065 A JP2011502065 A JP 2011502065A JP 2011517676 A JP2011517676 A JP 2011517676A
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oligonucleotide
dsrna
nucleotides
stranded
tissue
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Pending
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JP2011502065A
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Japanese (ja)
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JP2011517676A5 (enExample
Inventor
アキン・アキンク
朋子 中山
デイビッド・コニーズ
マルクス・シュトッフェル
クリスティアン・ヴォルフルム
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ETH Zurich
Alnylam Pharmaceuticals Inc
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ETH Zurich
Alnylam Pharmaceuticals Inc
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Publication of JP2011517676A publication Critical patent/JP2011517676A/ja
Publication of JP2011517676A5 publication Critical patent/JP2011517676A5/ja
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    • 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
    • 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
    • A61K47/554—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
    • 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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Botany (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2011502065A 2008-03-27 2009-03-26 インビボでｒｎａ干渉を媒介するための組成物および方法 Pending JP2011517676A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
EP08005800.1 2008-03-27
EP08005800A EP2105145A1 (en) 2008-03-27 2008-03-27 Method for muscle-specific delivery lipid-conjugated oligonucleotides
US4038608P 2008-03-28 2008-03-28
US61/040,386 2008-03-28
PCT/US2009/038437 WO2009120887A2 (en) 2008-03-27 2009-03-26 Compositions and methods for mediating rnai in vivo

Publications (2)

Publication Number Publication Date
JP2011517676A true JP2011517676A (ja) 2011-06-16
JP2011517676A5 JP2011517676A5 (enExample) 2012-05-17

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JP2011502065A Pending JP2011517676A (ja) 2008-03-27 2009-03-26 インビボでｒｎａ干渉を媒介するための組成物および方法

Country Status (4)

Country Link
US (1) US8148344B2 (enExample)
EP (2) EP2105145A1 (enExample)
JP (1) JP2011517676A (enExample)
WO (1) WO2009120887A2 (enExample)

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013256452A (ja) * 2012-06-11 2013-12-26 Kawaken Fine Chem Co Ltd メラニン産生抑制剤とその組成物
JP2016540777A (ja) * 2013-12-03 2016-12-28 ノースウェスタン ユニバーシティ リポソーム粒子、前述のものを作製する方法及びその使用
JP2018534275A (ja) * 2015-10-07 2018-11-22 コーメディクス・インコーポレーテッド タウロリジンの皮膚浸透製剤
US10837018B2 (en) 2013-07-25 2020-11-17 Exicure, Inc. Spherical nucleic acid-based constructs as immunostimulatory agents for prophylactic and therapeutic use
JP2022537987A (ja) * 2019-07-02 2022-08-31 アルゴノート アールエヌエー リミテッド アポリポタンパク質ｂアンタゴニスト

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NZ584793A (en) 2007-10-26 2012-05-25 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
WO2009062169A2 (en) 2007-11-09 2009-05-14 The Board Of Regents Of The University Of Texas System Micro-rnas of the mir-15 family modulate cardiomyocyte survival and cardiac repair
WO2009117418A2 (en) 2008-03-17 2009-09-24 The Board Of Regents Of The University Of Texas System Identification of micro-rnas involved in neuromuscular synapse maintenance and regeneration
EP2105145A1 (en) 2008-03-27 2009-09-30 ETH Zürich Method for muscle-specific delivery lipid-conjugated oligonucleotides
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
US20100233270A1 (en) 2009-01-08 2010-09-16 Northwestern University Delivery of Oligonucleotide-Functionalized Nanoparticles
EP2525780B1 (en) 2010-01-19 2022-09-07 Northwestern University Synthetic nanostructures including nucleic acids and/or other entities
CA2862628C (en) 2012-01-27 2021-08-24 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
US9163235B2 (en) 2012-06-21 2015-10-20 MiRagen Therapeutics, Inc. Inhibitors of the miR-15 family of micro-RNAs
US10568898B2 (en) 2013-08-13 2020-02-25 Northwestern University Lipophilic nanoparticles for drug delivery
SI3071696T1 (sl) 2013-11-22 2019-11-29 Mina Therapeutics Ltd C/EBP alfa kratko delujoči RNA sestavki in postopki uporabe
US10513710B2 (en) 2014-04-18 2019-12-24 University Of Massachusetts Exosomal loading using hydrophobically modified oligonucleotides
AU2015269412B2 (en) 2014-06-04 2020-03-12 Exicure Operating Company Multivalent delivery of immune modulators by liposomal spherical nucleic acids for prophylactic or therapeutic applications
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WO2016081911A2 (en) 2014-11-21 2016-05-26 Northwestern University The sequence-specific cellular uptake of spherical nucleic acid nanoparticle conjugates
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CA3023451A1 (en) 2016-05-06 2017-11-09 Exicure, Inc. Liposomal spherical nucleic acid (sna) constructs presenting antisense oligonucleotides (aso) for specific knockdown of interleukin 17 receptor mrna
WO2018039629A2 (en) 2016-08-25 2018-03-01 Northwestern University Micellar spherical nucleic acids from thermoresponsive, traceless templates
WO2018152327A1 (en) * 2017-02-15 2018-08-23 Northwestern University Enhancing stability and immunomodulatory activity of liposomal spherical nucleic acids
WO2018201090A1 (en) 2017-04-28 2018-11-01 Exicure, Inc. Synthesis of spherical nucleic acids using lipophilic moieties
JP2020526558A (ja) 2017-07-13 2020-08-31 ノースウェスタン ユニバーシティ オリゴヌクレオチド官能化金属有機構造体ナノ粒子を調製するための一般的かつ直接的な方法
WO2019048632A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATORY RNA (PARNA) OF HNF4A AND METHODS OF USE
WO2019048645A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE
US11566246B2 (en) 2018-04-12 2023-01-31 Mina Therapeutics Limited SIRT1-saRNA compositions and methods of use
US12702719B2 (en) 2019-03-06 2026-08-11 Northwestern University Hairpin-like oligonucleotide-conjugated spherical nucleic acid
EP3953473A1 (en) 2019-04-12 2022-02-16 MiNA Therapeutics Limited Sirt1-sarna compositions and methods of use
US12319711B2 (en) 2019-09-20 2025-06-03 Northwestern University Spherical nucleic acids with tailored and active protein coronae
US12378560B2 (en) 2019-10-29 2025-08-05 Northwestern University Sequence multiplicity within spherical nucleic acids
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
EP4304640A1 (en) 2021-03-12 2024-01-17 Northwestern University Antiviral vaccines using spherical nucleic acids
US20240175033A1 (en) 2021-03-26 2024-05-30 Mina Therapeutics Limited TMEM173 saRNA Compositions and Methods of Use
WO2023099884A1 (en) 2021-12-01 2023-06-08 Mina Therapeutics Limited Pax6 sarna compositions and methods of use
GB202117758D0 (en) 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
TW202440929A (zh) 2022-12-14 2024-10-16 加拿大商普羅維登斯治療控股公司 用於感染性疾病的組合物和方法
US20260218178A1 (en) 2022-12-22 2026-07-30 Mina Therapeutics Limited Chemically Modified saRNA Compositions and Methods of Use
WO2026027887A2 (en) 2024-08-02 2026-02-05 Mina Therapeutics Limited Hbg1/2-sarna compositions and methods of use

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Cited By (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013256452A (ja) * 2012-06-11 2013-12-26 Kawaken Fine Chem Co Ltd メラニン産生抑制剤とその組成物
US10837018B2 (en) 2013-07-25 2020-11-17 Exicure, Inc. Spherical nucleic acid-based constructs as immunostimulatory agents for prophylactic and therapeutic use
US10894963B2 (en) 2013-07-25 2021-01-19 Exicure, Inc. Spherical nucleic acid-based constructs as immunostimulatory agents for prophylactic and therapeutic use
JP2016540777A (ja) * 2013-12-03 2016-12-28 ノースウェスタン ユニバーシティ リポソーム粒子、前述のものを作製する方法及びその使用
JP2018534275A (ja) * 2015-10-07 2018-11-22 コーメディクス・インコーポレーテッド タウロリジンの皮膚浸透製剤
JP2022537987A (ja) * 2019-07-02 2022-08-31 アルゴノート アールエヌエー リミテッド アポリポタンパク質ｂアンタゴニスト
JP7463410B2 (ja) 2019-07-02 2024-04-08 アルゴノート アールエヌエー リミテッド アポリポタンパク質ｂアンタゴニスト

Also Published As

Publication number Publication date
EP2105145A1 (en) 2009-09-30
US20100003317A1 (en) 2010-01-07
WO2009120887A3 (en) 2009-12-10
EP2265289A2 (en) 2010-12-29
US8148344B2 (en) 2012-04-03
WO2009120887A2 (en) 2009-10-01

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