JP2024520756A - ApoE及びApoB修飾脂質ナノ粒子組成物及びその使用 - Google Patents

ApoE及びApoB修飾脂質ナノ粒子組成物及びその使用 Download PDF

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JP2024520756A
JP2024520756A JP2023575397A JP2023575397A JP2024520756A JP 2024520756 A JP2024520756 A JP 2024520756A JP 2023575397 A JP2023575397 A JP 2023575397A JP 2023575397 A JP2023575397 A JP 2023575397A JP 2024520756 A JP2024520756 A JP 2024520756A
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pharmaceutical composition
lipid
peg
polypeptide
lnp
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JP2024520756A5 (enExample
Inventor
ミシェル ルブラン,
グレゴリー ファインスタイン,
ランドール ニュートン トイ,
バート ノルティング,
ナサニエル シルバー,
フィリップ サマヨア,
ジョン エドワード チャタートン,
マシュー マンガニエロ,
ダグラス エー. ローズ,
ライアン エム. デラニー,
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ジェネレーション バイオ カンパニー
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    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
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    • A61K48/0041Medicinal 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 polymeric
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    • A61K47/6921Medicinal 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 particulate, a powder, an adsorbate, a bead or a sphere
    • A61K47/6927Medicinal 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 particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores
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    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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
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    • A61K48/0025Medicinal 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
    • A61K48/0033Medicinal 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
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JP2023575397A 2021-06-07 2022-06-07 ApoE及びApoB修飾脂質ナノ粒子組成物及びその使用 Pending JP2024520756A (ja)

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US202163197881P 2021-06-07 2021-06-07
US63/197,881 2021-06-07
PCT/US2022/032510 WO2022261101A1 (en) 2021-06-07 2022-06-07 Apoe and apob modified lipid nanoparticle compositions and uses thereof

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JP2024520756A true JP2024520756A (ja) 2024-05-24
JP2024520756A5 JP2024520756A5 (enExample) 2025-06-17

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US (1) US20240299580A1 (enExample)
EP (1) EP4351533A4 (enExample)
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KR (1) KR20240019244A (enExample)
CN (1) CN117897143A (enExample)
AU (1) AU2022288618A1 (enExample)
CA (1) CA3221454A1 (enExample)
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Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024119103A1 (en) 2022-12-01 2024-06-06 Generation Bio Co. Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers
EP4626400A1 (en) 2022-12-01 2025-10-08 Generation Bio Co. Novel polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
WO2024119074A1 (en) 2022-12-01 2024-06-06 Generation Bio Co. Stealth lipid nanoparticle compositions for cell targeting
AU2023406303A1 (en) 2022-12-01 2025-05-29 Generation Bio Co. Lipid nanoparticles comprising nucleic acids, ionizable lipids, sterols, lipid anchored polymers and helper lipids, their uses
US20240226133A1 (en) * 2023-01-09 2024-07-11 Claudia Chimisso Dos Santos Microrna-based particle for the treatment of dysregulated immune response
WO2024200820A1 (en) 2023-03-30 2024-10-03 Ose Immunotherapeutics Method of synthesis of targeted lipid nanoparticle and uses thereof
EP4687991A1 (en) 2023-03-30 2026-02-11 Ose Immunotherapeutics Lipid-based nanoparticle targeted at activated immune cells for the expression of immune cell enhancing molecule and use thereof
WO2024226779A1 (en) * 2023-04-26 2024-10-31 Saliogen Therapeutics, Inc. Lipid nanoparticle (lnp) formulations
IT202300015291A1 (it) * 2023-07-20 2025-01-20 Fond Riccardo Terzi E Silvio Albini Sistema di rilascio comprendente nanoparticelle lipidiche o polimeriche comprendenti ormoni tiroidei
WO2025052180A2 (en) 2023-09-07 2025-03-13 Axelyf ehf. Lipids and lipid nanoparticles
WO2025230906A1 (en) * 2024-04-29 2025-11-06 Saliogen Therapeutics, Inc. Lipid nanoparticle (lnp) formulations
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles
CN118453517B (zh) * 2024-07-15 2024-10-08 杭州珞米医疗科技有限公司 一种靶向结合ldlr的组合物及其应用
WO2026052871A1 (en) * 2024-09-09 2026-03-12 Re3B Therapeutics Ug (I.G.) Serum-protein coated fusogenic lipid particles for targeted drug delivery
WO2026064512A1 (en) 2024-09-18 2026-03-26 Generation Bio Co. Polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same

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EP1796712A4 (en) * 2004-09-07 2009-11-11 Kos Life Sciences Inc METHOD AND COMPOSITIONS FOR TREATING HYPERCHOLESTEROLEMIA
AR069328A1 (es) * 2007-11-15 2010-01-13 Alcon Mfg Ltd Liberacion de rna interferente corto o sirna, intermediada por receptor de lipoproteina de baja densidad
WO2019040516A1 (en) * 2017-08-22 2019-02-28 Rubius Therapeutics, Inc. METHODS AND COMPOSITIONS OF LIPID NANOPARTICLES FOR THE PRODUCTION OF MODIFIED ERYTHROID CELLS
EP3934700A4 (en) * 2019-03-06 2022-12-14 Generation Bio Co. NON-ACTIVE LIPID NANOPARTICLES WITH CAPSID-FREE, NON-VIRAL DNA
WO2021046265A1 (en) * 2019-09-06 2021-03-11 Generation Bio Co. Lipid nanoparticle compositions comprising closed-ended dna and cleavable lipids and methods of use thereof
WO2022076547A1 (en) * 2020-10-07 2022-04-14 Precision Biosciences, Inc. Lipid nanoparticle compositions

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US20240299580A1 (en) 2024-09-12
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