JP2008513513A - ポリヌクレオチドの送達 - Google Patents

ポリヌクレオチドの送達 Download PDF

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Publication number
JP2008513513A
JP2008513513A JP2007532679A JP2007532679A JP2008513513A JP 2008513513 A JP2008513513 A JP 2008513513A JP 2007532679 A JP2007532679 A JP 2007532679A JP 2007532679 A JP2007532679 A JP 2007532679A JP 2008513513 A JP2008513513 A JP 2008513513A
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formulation
decoy
molecule
weight
dsodn
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JP2008513513A5 (https=
Inventor
マヤ ダジー，
ロルフ エールハルト，
ハンス ホフランド，
レスリー マクエボイ，
トニー ムカムール，
ブライアン ビー． シェリバー，
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Anesiva Inc
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Corgentech Inc
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Publication of JP2008513513A publication Critical patent/JP2008513513A/ja
Publication of JP2008513513A5 publication Critical patent/JP2008513513A5/ja
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • 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
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/36—Polysaccharides; Derivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
    • A61K47/38—Cellulose; Derivatives thereof
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0014—Skin, i.e. galenical aspects of topical compositions
    • 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
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/02—Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/06—Antipsoriatics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/14—Drugs for dermatological disorders for baldness or alopecia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/08—Antiallergic agents
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/13—Decoys
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dermatology (AREA)
  • Inorganic Chemistry (AREA)
  • Dispersion Chemistry (AREA)
  • Pulmonology (AREA)
  • Immunology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2007532679A 2004-09-21 2005-09-21 ポリヌクレオチドの送達 Pending JP2008513513A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US61204604P 2004-09-21 2004-09-21
US66349705P 2005-03-18 2005-03-18
PCT/US2005/034110 WO2006034433A2 (en) 2004-09-21 2005-09-21 Delivery of polynucleotides

Publications (2)

Publication Number Publication Date
JP2008513513A true JP2008513513A (ja) 2008-05-01
JP2008513513A5 JP2008513513A5 (https=) 2009-03-05

Family

ID=36090693

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2007532679A Pending JP2008513513A (ja) 2004-09-21 2005-09-21 ポリヌクレオチドの送達

Country Status (7)

Country Link
US (1) US20060069055A1 (https=)
EP (1) EP1799271A4 (https=)
JP (1) JP2008513513A (https=)
AU (1) AU2005286640A1 (https=)
CA (1) CA2583413A1 (https=)
MX (1) MX2007003167A (https=)
WO (1) WO2006034433A2 (https=)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015518705A (ja) * 2012-04-02 2015-07-06 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド
JP2015519881A (ja) * 2012-04-02 2015-07-16 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 核タンパク質の産生のための修飾ポリヌクレオチド
JP2020511552A (ja) * 2017-03-23 2020-04-16 リピド システムス エスペ．ゼット．オ．オ． 単層リポソームにおける親水性化合物の高い効率の封入

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EP1709175A2 (en) * 2003-12-03 2006-10-11 Corgentech, Inc. Hif oligonucleotide decoy molecules
US20070219131A1 (en) * 2004-04-15 2007-09-20 Ben-Sasson Shmuel A Compositions capable of facilitating penetration across a biological barrier
US8241670B2 (en) * 2004-04-15 2012-08-14 Chiasma Inc. Compositions capable of facilitating penetration across a biological barrier
US9486408B2 (en) 2005-12-01 2016-11-08 University Of Massachusetts Lowell Botulinum nanoemulsions
US8530436B2 (en) * 2007-01-29 2013-09-10 Transderm, Inc. Methods and compositions for transdermal delivery of nucleotides
AU2008224883A1 (en) * 2007-03-14 2008-09-18 Sierra Molecular Corporation Compositions, systems, and methods for preservation and/or stabilization of a cell and/or macromolecule
CA2723672C (en) * 2007-05-11 2019-09-03 Adynxx, Inc. Gene expression and pain
JP5294302B2 (ja) * 2008-03-28 2013-09-18 マルホ株式会社 胸腺間質リンパ球増殖因子産生を評価するスクリーニング方法
CN102176900B (zh) 2008-09-17 2017-09-26 克艾思马有限公司 药物组合物和相关的给药方法
KR20140005998A (ko) * 2011-01-24 2014-01-15 안테리오스, 인코퍼레이티드 나노입자 조성물, 이의 제형, 및 그의 용도
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
EP3511022A1 (en) 2012-05-10 2019-07-17 Adynxx, Inc. Formulations for the delivery of active ingredients
JP6705807B2 (ja) 2014-08-15 2020-06-03 エーダイニクス インコーポレイテッド 疼痛を治療するためのオリゴヌクレオチドデコイ
HUE071943T2 (hu) 2015-02-03 2025-10-28 Amryt Endo Inc Akromegália kezelése oktreotid orális alkalmazásával
AU2017360346B2 (en) 2016-11-21 2023-11-23 Eirion Therapeutics, Inc. Transdermal delivery of large agents
US12233073B2 (en) * 2017-06-09 2025-02-25 Regents Of The University Of Minnesota Skin care formulations and skin cancer treatment
CA3128615A1 (en) * 2019-03-01 2020-09-10 Flagship Pioneering Innovations Vi, Llc Topical delivery of polyribonucleic acids
MA55083A (fr) * 2019-03-01 2022-03-16 Flagship Pioneering Innovations Vi Llc Polyribonucléotides et leurs utilisations cosmétiques
US11141457B1 (en) 2020-12-28 2021-10-12 Amryt Endo, Inc. Oral octreotide therapy and contraceptive methods
CN114469897B (zh) * 2022-01-12 2023-02-28 上海市胸科医院 靶向治疗心肌梗死的锂皂石复合递送材料及其制备与应用
US20230406888A1 (en) * 2022-04-27 2023-12-21 Sachi Bioworks Inc. Methods and systems for targeting autoimmune and inflammatory pathways using nanoligomers

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JP2002193813A (ja) * 2000-12-27 2002-07-10 Anges Mg Inc デコイを含む薬学的組成物およびその使用方法
WO2002066070A1 (fr) * 2001-02-20 2002-08-29 Anges Mg, Inc. Compositions pharmaceutiques contenant un leurre et procede d'utilisation associe
WO2003063911A1 (en) * 2002-02-01 2003-08-07 Anges Mg, Inc. Decoy-containing pharmaceutical compositions and method of using the same
JP2003524586A (ja) * 1998-05-21 2003-08-19 アイシス・ファーマシューティカルス・インコーポレーテッド オリゴヌクレオチドの非−非経口投与のための組成物と方法
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WO2003085110A2 (en) * 2002-04-05 2003-10-16 Santaris Pharma A/S Oligomeric compounds for the modulation hif-1alpha expression
CA2493902A1 (en) * 2002-05-29 2003-12-04 Anges Mg, Inc. Decoy composition for treating and preventing inflammatory disease
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Patent Citations (6)

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Publication number Priority date Publication date Assignee Title
WO1996035430A1 (en) * 1995-05-12 1996-11-14 Fujisawa Pharmaceutical Co., Ltd. REMEDY AND PREVENTIVE FOR DISEASES CAUSED BY NF-λB
JP2003524586A (ja) * 1998-05-21 2003-08-19 アイシス・ファーマシューティカルス・インコーポレーテッド オリゴヌクレオチドの非−非経口投与のための組成物と方法
US20030206958A1 (en) * 2000-12-22 2003-11-06 Cattaneo Maurizio V. Chitosan biopolymer for the topical delivery of active agents
JP2002193813A (ja) * 2000-12-27 2002-07-10 Anges Mg Inc デコイを含む薬学的組成物およびその使用方法
WO2002066070A1 (fr) * 2001-02-20 2002-08-29 Anges Mg, Inc. Compositions pharmaceutiques contenant un leurre et procede d'utilisation associe
WO2003063911A1 (en) * 2002-02-01 2003-08-07 Anges Mg, Inc. Decoy-containing pharmaceutical compositions and method of using the same

Cited By (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015518705A (ja) * 2012-04-02 2015-07-06 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド
JP2015519881A (ja) * 2012-04-02 2015-07-16 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 核タンパク質の産生のための修飾ポリヌクレオチド
JP2017121244A (ja) * 2012-04-02 2017-07-13 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド
JP2017121241A (ja) * 2012-04-02 2017-07-13 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． ヒト疾患に関連するタンパク質の産生のための修飾ポリヌクレオチド
JP2017121243A (ja) * 2012-04-02 2017-07-13 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． タンパク質の産生のための修飾ポリヌクレオチド
JP2017123847A (ja) * 2012-04-02 2017-07-20 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． 核タンパク質の産生のための修飾ポリヌクレオチド
JP2020511552A (ja) * 2017-03-23 2020-04-16 リピド システムス エスペ．ゼット．オ．オ． 単層リポソームにおける親水性化合物の高い効率の封入
JP7404225B2 (ja) 2017-03-23 2023-12-25 リピド システムス エスペ．ゼット．オ．オ． 単層リポソームにおける親水性化合物の高い効率の封入

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