JP2024526021A - 核酸のための１成分送達系 - Google Patents

核酸のための１成分送達系 Download PDF

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Publication number
JP2024526021A
JP2024526021A JP2023572650A JP2023572650A JP2024526021A JP 2024526021 A JP2024526021 A JP 2024526021A JP 2023572650 A JP2023572650 A JP 2023572650A JP 2023572650 A JP2023572650 A JP 2023572650A JP 2024526021 A JP2024526021 A JP 2024526021A
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JP
Japan
Prior art keywords
occurrence
combination
group
ionic amphiphilic
amphiphilic janus
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Pending
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JP2023572650A
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English (en)
Japanese (ja)
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JP2024526021A5 (https=
Inventor
バージル　パーセク
ウェイスマン ドリュー
ターポン チャン
アトチナ－バッサーマン エレナ
モーリャ ディベンドラ
チー シアオ
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Temple Univ School of Medicine
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Temple Univ School of Medicine
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Publication of JP2024526021A publication Critical patent/JP2024526021A/ja
Publication of JP2024526021A5 publication Critical patent/JP2024526021A5/ja
Pending legal-status Critical Current

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C69/00—Esters of carboxylic acids; Esters of carbonic or haloformic acids
    • C07C69/76—Esters of carboxylic acids having a carboxyl group bound to a carbon atom of a six-membered aromatic ring
    • C07C69/84—Esters of carboxylic acids having a carboxyl group bound to a carbon atom of a six-membered aromatic ring of monocyclic hydroxy carboxylic acids, the hydroxy groups and the carboxyl groups of which are bound to carbon atoms of a six-membered aromatic ring
    • C07C69/92—Esters of carboxylic acids having a carboxyl group bound to a carbon atom of a six-membered aromatic ring of monocyclic hydroxy carboxylic acids, the hydroxy groups and the carboxyl groups of which are bound to carbon atoms of a six-membered aromatic ring with etherified hydroxyl groups
    • 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
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/385—Haptens or antigens, bound to carriers
    • 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
    • A61K47/54—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
    • A61K47/545—Heterocyclic compounds
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C229/00—Compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C229/02—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C229/04—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C229/06—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton
    • C07C229/10—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings
    • C07C229/12—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings to carbon atoms of acyclic carbon skeletons
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C235/00—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms
    • C07C235/42—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to carbon atoms of six-membered aromatic rings and singly-bound oxygen atoms bound to the same carbon skeleton
    • C07C235/44—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to carbon atoms of six-membered aromatic rings and singly-bound oxygen atoms bound to the same carbon skeleton with carbon atoms of carboxamide groups and singly-bound oxygen atoms bound to carbon atoms of the same non-condensed six-membered aromatic ring
    • C07C235/48—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to carbon atoms of six-membered aromatic rings and singly-bound oxygen atoms bound to the same carbon skeleton with carbon atoms of carboxamide groups and singly-bound oxygen atoms bound to carbon atoms of the same non-condensed six-membered aromatic ring having the nitrogen atom of at least one of the carboxamide groups bound to an acyclic carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D295/00—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
    • C07D295/04—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms
    • C07D295/14—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals
    • C07D295/145—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals with the ring nitrogen atoms and the carbon atoms with three bonds to hetero atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
    • C07D295/15—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals with the ring nitrogen atoms and the carbon atoms with three bonds to hetero atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings to an acyclic saturated chain

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Mycology (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2023572650A 2021-05-24 2022-05-24 核酸のための１成分送達系 Pending JP2024526021A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163192236P 2021-05-24 2021-05-24
US63/192,236 2021-05-24
US202163253348P 2021-10-07 2021-10-07
US63/253,348 2021-10-07
US202263316794P 2022-03-04 2022-03-04
US63/316,794 2022-03-04
PCT/US2022/030694 WO2022251191A1 (en) 2021-05-24 2022-05-24 One-component delivery system for nucleic acids

Publications (2)

Publication Number Publication Date
JP2024526021A true JP2024526021A (ja) 2024-07-17
JP2024526021A5 JP2024526021A5 (https=) 2025-06-03

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Family Applications (1)

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JP2023572650A Pending JP2024526021A (ja) 2021-05-24 2022-05-24 核酸のための１成分送達系

Country Status (7)

Country Link
US (1) US20240252677A1 (https=)
EP (1) EP4347550A4 (https=)
JP (1) JP2024526021A (https=)
CN (1) CN119233964A (https=)
AU (1) AU2022280004A1 (https=)
CA (1) CA3220089A1 (https=)
WO (1) WO2022251191A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025038627A2 (en) * 2023-08-14 2025-02-20 The Trustees Of The University Of Pennsylvania Pentaerythritol-based ionizable amphiphilic janus dendrimers, delivery systems comprising thereof, and methods of use thereof
CN119219518B (zh) * 2024-09-26 2025-10-24 上海大学 一种树枝化苯乙炔交联剂及其制备方法和应用
CN119462481B (zh) * 2025-01-17 2025-04-25 北京悦康科创医药科技股份有限公司 Sting激活型佐剂脂质、包含其的组合物及用途

Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2005511533A (ja) * 2001-10-19 2005-04-28 ユニバーシティ オブ ストラスクライド 標的デリバリ用デントリマ
WO2011017500A1 (en) * 2009-08-06 2011-02-10 The Trustees Of The University Of Pennsylvania Amphiphilic janus-dendrimers
WO2014190024A1 (en) * 2013-05-21 2014-11-27 Virgil Percec Modular synthesis of amphiphilic janus glycodendrimers and their self-assembly into glycodendrimersomes
WO2016021683A1 (ja) * 2014-08-07 2016-02-11 武田薬品工業株式会社 カチオン性脂質
JP2016514109A (ja) * 2013-03-08 2016-05-19 ノバルティス アーゲー 活性薬物の送達のための脂質と脂質組成物
WO2016104580A1 (ja) * 2014-12-26 2016-06-30 エーザイ・アール・アンド・ディー・マネジメント株式会社 カチオン性脂質
JP2017532302A (ja) * 2014-09-05 2017-11-02 ノバルティス アーゲー 活性物質の送達用の脂質および脂質組成物
JP2018537403A (ja) * 2015-09-14 2018-12-20 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム リポカチオン性デンドリマーおよびその使用
WO2020146805A1 (en) * 2019-01-11 2020-07-16 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents

Patent Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2005511533A (ja) * 2001-10-19 2005-04-28 ユニバーシティ オブ ストラスクライド 標的デリバリ用デントリマ
WO2011017500A1 (en) * 2009-08-06 2011-02-10 The Trustees Of The University Of Pennsylvania Amphiphilic janus-dendrimers
JP2016514109A (ja) * 2013-03-08 2016-05-19 ノバルティス アーゲー 活性薬物の送達のための脂質と脂質組成物
WO2014190024A1 (en) * 2013-05-21 2014-11-27 Virgil Percec Modular synthesis of amphiphilic janus glycodendrimers and their self-assembly into glycodendrimersomes
WO2016021683A1 (ja) * 2014-08-07 2016-02-11 武田薬品工業株式会社 カチオン性脂質
JP2017532302A (ja) * 2014-09-05 2017-11-02 ノバルティス アーゲー 活性物質の送達用の脂質および脂質組成物
WO2016104580A1 (ja) * 2014-12-26 2016-06-30 エーザイ・アール・アンド・ディー・マネジメント株式会社 カチオン性脂質
JP2018537403A (ja) * 2015-09-14 2018-12-20 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム リポカチオン性デンドリマーおよびその使用
WO2020146805A1 (en) * 2019-01-11 2020-07-16 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents

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Title
ACS MACRO LETTERS, vol. 2, no. 8, JPN6026009449, 2013, pages 720 - 724, ISSN: 0005813266 *
ACS NANO, vol. 14, no. 6, JPN6026009451, 2020, pages 7398 - 7411, ISSN: 0005813268 *
ACS NANO, vol. 8, no. 2, JPN6026009453, 2014, pages 1554 - 1565, ISSN: 0005813270 *
JOURNAL OF THE AMERICAN CHEMICAL SOCIETY, vol. 137, no. 41, JPN6026009460, 2015, pages 13334 - 13344, ISSN: 0005813276 *
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, vol. 111, no. 25, JPN6026009452, 2014, pages 9058 - 9063, ISSN: 0005813269 *
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, vol. 113, no. 9, JPN6026009450, 2016, pages 1134 - 1141, ISSN: 0005813267 *
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PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, vol. 116, no. 31, JPN6026009456, 2019, pages 15378 - 15385, ISSN: 0005813273 *
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, vol. 117, no. 22, JPN6026009458, 2020, pages 11931 - 11939, ISSN: 0005813274 *

Also Published As

Publication number Publication date
EP4347550A1 (en) 2024-04-10
CN119233964A (zh) 2024-12-31
WO2022251191A1 (en) 2022-12-01
US20240252677A1 (en) 2024-08-01
EP4347550A4 (en) 2025-07-30
CA3220089A1 (en) 2022-12-01
AU2022280004A1 (en) 2024-01-04

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