JP2020514383A - 修飾オリゴヌクレオチドおよびその治療上の使用 - Google Patents

修飾オリゴヌクレオチドおよびその治療上の使用 Download PDF

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JP2020514383A
JP2020514383A JP2019551984A JP2019551984A JP2020514383A JP 2020514383 A JP2020514383 A JP 2020514383A JP 2019551984 A JP2019551984 A JP 2019551984A JP 2019551984 A JP2019551984 A JP 2019551984A JP 2020514383 A JP2020514383 A JP 2020514383A
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compound
protein
pharmaceutical composition
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JP2020514383A5 (enExample
Inventor
ロロフ アレクサンダー
ロロフ アレクサンダー
シー ギアネッシ ネイサン
シー ギアネッシ ネイサン
イー コールマン カッサンドラ
イー コールマン カッサンドラ
ピー トンプソン マシュー
ピー トンプソン マシュー
エイ バーティン ポール
エイ バーティン ポール
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Vybyl Holdings Inc
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Vybyl Holdings Inc
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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
    • A61K47/51—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
    • A61K47/68—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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6807—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
    • 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
    • A61K47/42—Proteins; Polypeptides; Degradation products thereof; Derivatives thereof, e.g. albumin, gelatin or zein
    • 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
    • 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
    • A61K47/62—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 a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/643—Albumins, e.g. HSA, BSA, ovalbumin or a Keyhole Limpet Hemocyanin [KHL]
    • 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
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Inorganic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Dermatology (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2019551984A 2017-03-22 2018-03-21 修飾オリゴヌクレオチドおよびその治療上の使用 Pending JP2020514383A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762475185P 2017-03-22 2017-03-22
US62/475,185 2017-03-22
PCT/US2018/023578 WO2018175592A1 (en) 2017-03-22 2018-03-21 Modified oligonucleotides and therapeutic uses thereof

Publications (2)

Publication Number Publication Date
JP2020514383A true JP2020514383A (ja) 2020-05-21
JP2020514383A5 JP2020514383A5 (enExample) 2021-04-30

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JP2019551984A Pending JP2020514383A (ja) 2017-03-22 2018-03-21 修飾オリゴヌクレオチドおよびその治療上の使用

Country Status (9)

Country Link
US (1) US20200046846A1 (enExample)
EP (1) EP3600439A4 (enExample)
JP (1) JP2020514383A (enExample)
KR (1) KR20190123351A (enExample)
CN (1) CN110636865A (enExample)
AU (1) AU2018237139A1 (enExample)
CA (1) CA3057292A1 (enExample)
SG (1) SG11201908771YA (enExample)
WO (1) WO2018175592A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025028656A1 (ja) * 2023-08-02 2025-02-06 日東電工株式会社 オリゴヌクレオチドを製造する方法

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20250270248A1 (en) * 2020-09-16 2025-08-28 Astrazeneca Ab Oligonucleotides conjugated to fatty acids
CN117980003A (zh) * 2021-09-10 2024-05-03 嘉德治疗有限责任公司 核酸的脂肪酸缀合物

Citations (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0821001A1 (en) * 1995-03-31 1998-01-28 Drug Delivery System Institute, Ltd. Amidite derivatives and oligonucleotide derivatives
WO2007112414A2 (en) * 2006-03-27 2007-10-04 Isis Pharmaceuticals, Inc. Conjugated double strand compositions for use in gene modulation
WO2010150004A1 (en) * 2009-06-22 2010-12-29 Sylentis S.A.U. Novel drugs for inhibition of gene expression
WO2012165616A1 (ja) * 2011-06-03 2012-12-06 国立大学法人北海道大学 オリゴヌクレオチド誘導体、オリゴヌクレオチド誘導体を含む治療用医薬組成物及び診断用医薬組成物、並びにｍｉＲＮＡ機能抑制用オリゴヌクレオチド誘導体
WO2016149313A1 (en) * 2015-03-17 2016-09-22 Arrowhead Research Corporation Improved disulfide-containing alkyne linking agents
WO2016149501A2 (en) * 2015-03-18 2016-09-22 The California Institute For Biomedical Research Modified therapeutic agents and compositions thereof
JP2016529230A (ja) * 2013-07-11 2016-09-23 アルニラム ファーマスーティカルズ インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． オリゴヌクレオチド−リガンドコンジュゲートおよびそれらの調製方法
JP2016531156A (ja) * 2013-09-13 2016-10-06 ザ カリフォルニア インスティテュート フォー バイオメディカル リサーチ 修飾された治療剤、及びその組成物
JP2017502024A (ja) * 2013-12-18 2017-01-19 ザ カリフォルニア インスティテュート フォー バイオメディカル リサーチ 修飾された治療剤、ステープル留めされたペプチド脂質複合体、及びそれらの組成物
JP2018527360A (ja) * 2015-09-22 2018-09-20 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 修飾された細胞毒とその治療的使用

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8969543B2 (en) * 2003-04-03 2015-03-03 Bioneer Corporation SiRNA-hydrophilic polymer conjugates for intracellular delivery of siRNA and method thereof
KR101224828B1 (ko) * 2009-05-14 2013-01-22 (주)바이오니아 ｓｉＲＮＡ 접합체 및 그 제조방법
RU2599449C1 (ru) * 2011-12-15 2016-10-10 Байонир Корпорейшн Новые конъюгаты олигонуклеотидов и их применение
WO2017030973A1 (en) * 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery

Patent Citations (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0821001A1 (en) * 1995-03-31 1998-01-28 Drug Delivery System Institute, Ltd. Amidite derivatives and oligonucleotide derivatives
WO2007112414A2 (en) * 2006-03-27 2007-10-04 Isis Pharmaceuticals, Inc. Conjugated double strand compositions for use in gene modulation
WO2010150004A1 (en) * 2009-06-22 2010-12-29 Sylentis S.A.U. Novel drugs for inhibition of gene expression
WO2012165616A1 (ja) * 2011-06-03 2012-12-06 国立大学法人北海道大学 オリゴヌクレオチド誘導体、オリゴヌクレオチド誘導体を含む治療用医薬組成物及び診断用医薬組成物、並びにｍｉＲＮＡ機能抑制用オリゴヌクレオチド誘導体
JP2016529230A (ja) * 2013-07-11 2016-09-23 アルニラム ファーマスーティカルズ インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． オリゴヌクレオチド−リガンドコンジュゲートおよびそれらの調製方法
JP2016531156A (ja) * 2013-09-13 2016-10-06 ザ カリフォルニア インスティテュート フォー バイオメディカル リサーチ 修飾された治療剤、及びその組成物
JP2017502024A (ja) * 2013-12-18 2017-01-19 ザ カリフォルニア インスティテュート フォー バイオメディカル リサーチ 修飾された治療剤、ステープル留めされたペプチド脂質複合体、及びそれらの組成物
WO2016149313A1 (en) * 2015-03-17 2016-09-22 Arrowhead Research Corporation Improved disulfide-containing alkyne linking agents
WO2016149501A2 (en) * 2015-03-18 2016-09-22 The California Institute For Biomedical Research Modified therapeutic agents and compositions thereof
JP2018527360A (ja) * 2015-09-22 2018-09-20 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 修飾された細胞毒とその治療的使用

Non-Patent Citations (8)

* Cited by examiner, † Cited by third party
Title
DOVYDENKO, ILYA, BIOMATERIALS, vol. 76, JPN6022014460, 2016, pages 408 - 417, ISSN: 0004753570 *
MACKELLAR, CALUM: "SYNTHESIS AND PHYSICAL PROPERTIES OF ANTI-HIV ANTISENSE OLIGONUCLEOTIDES BEARING TERMINAL LIPOPHILIC", NUCLEIC ACIDS RESEARCH, vol. 20(13), JPN5021009884, 1992, pages 3411 - 3417, ISSN: 0004753566 *
MING, XIN, BIOMATERIALS, vol. 34, JPN6022014462, 2013, pages 7939 - 7949, ISSN: 0004753571 *
NICOLI, ELENA, PLOS ONE, vol. 10(4), e0122581, JPN6022014464, 9 April 2015 (2015-04-09), pages 16 - 1616, ISSN: 0004753572 *
NIKAN, MEHRAN: "DOCOSAHEXAENOIC ACID CONJUGATION ENHANCES DISTRIBUTION AND SAFETY OF SIRNA UPON LOCAL ADMINISTRATION", MOLECULAR THERAPY - NUCLEIC ACIDS, vol. 5, JPN5021009888, 2016, pages 344 - 1, ISSN: 0004753569 *
NIKAN, MEHRAN: "SYNTHESIS AND EVALUATION OF PARENCHYMAL RETENTION AND EFFICACY OF A METABOLICALLY STABLE O-PHOSPHOCH", BIOCONJUGATE CHEMISTRY, vol. 28(6), JPN5021009887, 10 May 2017 (2017-05-10), US, pages 1758 - 1766, ISSN: 0004753573 *
RAOUANE, MOUNA ET AL: "LIPID CONJUGATED OLIGONUCLEOTIDES: A USEFUL STRATEGY FOR DELIVERY", BIOCONJUGATE CHEMISTRY, vol. 23(6), JPN5021009885, 20 June 2012 (2012-06-20), pages 1091 - 1104, ISSN: 0004753567 *
ZHANG, JINGYU: "DESIGN, SYNTHESIS AND ANTI-INFLUENZA VIRUS ACTIVITIES OF TERMINAL MODIFIED ANTISENSE OLIGONUCLEOTIDE", TETRAHEDRON LETTERS, vol. 55(1), JPN5021009886, 2014, NL, pages 94 - 97, ISSN: 0004753568 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025028656A1 (ja) * 2023-08-02 2025-02-06 日東電工株式会社 オリゴヌクレオチドを製造する方法

Also Published As

Publication number Publication date
AU2018237139A1 (en) 2019-10-17
EP3600439A1 (en) 2020-02-05
KR20190123351A (ko) 2019-10-31
CN110636865A (zh) 2019-12-31
SG11201908771YA (en) 2019-10-30
WO2018175592A1 (en) 2018-09-27
US20200046846A1 (en) 2020-02-13
EP3600439A4 (en) 2021-01-13
CA3057292A1 (en) 2018-09-27

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