JP7592493B2 - 治療用タンパク質の標的細胞に特異的な産生のための、および、標的細胞に関連する疾患、疾病、または障害の処置のための、融合性脂質ナノ粒子、および、融合性脂質ナノ粒子の製造方法と使用方法 - Google Patents

治療用タンパク質の標的細胞に特異的な産生のための、および、標的細胞に関連する疾患、疾病、または障害の処置のための、融合性脂質ナノ粒子、および、融合性脂質ナノ粒子の製造方法と使用方法 Download PDF

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JP7592493B2
JP7592493B2 JP2020558508A JP2020558508A JP7592493B2 JP 7592493 B2 JP7592493 B2 JP 7592493B2 JP 2020558508 A JP2020558508 A JP 2020558508A JP 2020558508 A JP2020558508 A JP 2020558508A JP 7592493 B2 JP7592493 B2 JP 7592493B2
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ショルツ,マシュー,レイン
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オイシン バイオテクノロジーズ,インク.
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JP2020558508A 2018-04-18 2019-04-18 治療用タンパク質の標的細胞に特異的な産生のための、および、標的細胞に関連する疾患、疾病、または障害の処置のための、融合性脂質ナノ粒子、および、融合性脂質ナノ粒子の製造方法と使用方法 Active JP7592493B2 (ja)

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JP2024060375A JP2024105237A (ja) 2018-04-18 2024-04-03 治療用タンパク質の標的細胞に特異的な産生のための、および、標的細胞に関連する疾患、疾病、または障害の処置のための、融合性脂質ナノ粒子、および、融合性脂質ナノ粒子の製造方法と使用方法

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US201862659676P 2018-04-18 2018-04-18
US62/659,676 2018-04-18
US201962821084P 2019-03-20 2019-03-20
US62/821,084 2019-03-20
PCT/US2019/028207 WO2019204666A1 (en) 2018-04-18 2019-04-18 Fusogenic lipid nanoparticles and methods for the manufacture and use thereof for the target cell-specific production of a therapeutic protein and for the treatment of a disease, condition, or disorder associated with a target cell

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JP2024060375A Division JP2024105237A (ja) 2018-04-18 2024-04-03 治療用タンパク質の標的細胞に特異的な産生のための、および、標的細胞に関連する疾患、疾病、または障害の処置のための、融合性脂質ナノ粒子、および、融合性脂質ナノ粒子の製造方法と使用方法

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JP2021526505A JP2021526505A (ja) 2021-10-07
JP2021526505A5 JP2021526505A5 (https=) 2022-05-30
JPWO2019204666A5 JPWO2019204666A5 (https=) 2022-05-30
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JP2024060375A Pending JP2024105237A (ja) 2018-04-18 2024-04-03 治療用タンパク質の標的細胞に特異的な産生のための、および、標的細胞に関連する疾患、疾病、または障害の処置のための、融合性脂質ナノ粒子、および、融合性脂質ナノ粒子の製造方法と使用方法

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AU2018206485B2 (en) 2017-01-09 2024-12-05 Oisin Biotechnologies Fusogenic lipid nanoparticles and methods for manufacturing and use for therapeutic protein production and for treatment
WO2019204666A1 (en) 2018-04-18 2019-10-24 Oisin Biotechnologies, Inc. Fusogenic lipid nanoparticles and methods for the manufacture and use thereof for the target cell-specific production of a therapeutic protein and for the treatment of a disease, condition, or disorder associated with a target cell
SG11202105079QA (en) * 2018-11-14 2021-06-29 Flagship Pioneering Innovations V Inc Fusosome compositions for cns delivery
US12496274B2 (en) 2018-11-14 2025-12-16 Flagship Pioneering Innovations V, Inc. Compositions and methods for compartment-specific cargo delivery
CA3094859A1 (en) * 2020-10-01 2022-04-01 Entos Pharmaceuticals Inc. Proteolipid vesicles formulated with fusion associated small transmembrane proteins
CN114540416A (zh) * 2022-02-18 2022-05-27 上海交通大学 一种表达载体、脂质纳米粒、抗肿瘤药物及其制备方法和用途
WO2024036148A1 (en) * 2022-08-08 2024-02-15 A2 Biotherapeutics, Inc. Compositions and methods for treating blood cancers
CN116712406B (zh) * 2023-04-23 2026-03-31 深圳复生命生物医药有限公司 一种病毒样纳米粒子核酸药物递送系统、制备方法及应用

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002517996A (ja) 1998-06-16 2002-06-25 トーマス ジェファーソン ユニヴァーシティー 短型のApaf−1およびその使用方法
US20060002895A1 (en) 1998-03-11 2006-01-05 Board Of Regents, The University Of Texas System Induction of apoptic or cytotoxic gene expression by adenoviral mediated gene codelivery
US20140314831A1 (en) 2010-09-29 2014-10-23 Innovascreen Inc. Recombinant polypeptides for membrane fusion and uses thereof
JP2015525209A (ja) 2012-05-23 2015-09-03 ジ・オハイオ・ステート・ユニバーシティ 脂質ナノ粒子組成物と、それを作製する方法、及びそれを使用する方法
US20160010110A1 (en) 2013-03-24 2016-01-14 Oisin Biotechnologies Systems and methods for the targeted production of a therapeutic protein within a target cell
JP2017014278A (ja) 2011-06-08 2017-01-19 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド mRNA送達のための脂質ナノ粒子組成物および方法

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE69132032T2 (de) 1990-11-23 2000-12-21 Peptech Ltd., North Ryde Verzögerung, verhinderung und/oder rückgängigmachen von zellalterung
US5869337A (en) 1993-02-12 1999-02-09 President And Fellows Of Harvard College Regulated transcription of targeted genes and other biological events
CA2289702C (en) 1997-05-14 2008-02-19 Inex Pharmaceuticals Corp. High efficiency encapsulation of charged therapeutic agents in lipid vesicles
EP1018560A4 (en) 1997-08-20 2002-10-02 Dnavec Research Inc CANCER TREATMENT VECTORS
AU1137699A (en) 1997-11-07 1999-05-31 Dalhousie University Novel reovirus-derived proteins, nucleic acids encoding same, and uses therefor
CA2352486A1 (en) 1998-11-25 2000-06-02 Genetica, Inc. Methods and reagents for increasing proliferative capacity and preventing replicative senescence
CA2325088A1 (en) * 2000-12-01 2002-06-01 Fusogenix Inc. Novel membrane fusion proteins derived from poikilothermic reovirus
WO2002101076A2 (en) 2001-06-13 2002-12-19 Eastern Virginia Medical School Methods for targeted expression of therapeutic nucleic acid
CN100361710C (zh) 2004-06-07 2008-01-16 成都康弘生物科技有限公司 肿瘤细胞专一表达免疫调节因子gm-csf的溶瘤性腺病毒重组体的构建及其应用
JPWO2006043354A1 (ja) 2004-10-20 2008-05-22 独立行政法人放射線医学総合研究所 組込型の低線量放射線誘導性ベクター
EP2167688A4 (en) 2007-06-12 2010-07-28 Univ Case Western Reserve TARGETED CELL DEATH
MX2010012473A (es) 2008-05-23 2011-02-23 Siwa Corp Metodos, composiciones y aparato para facilitar la regeneracion.
HUE042177T2 (hu) * 2009-12-01 2019-06-28 Translate Bio Inc Szteroidszármazék mRNS szállítására humán genetikai betegségekben
WO2011146862A1 (en) 2010-05-21 2011-11-24 Bellicum Pharmaceuticals, Inc. Methods for inducing selective apoptosis
WO2012019053A2 (en) * 2010-08-04 2012-02-09 The Ohio State University Methods for impairing the p53/hdm2 auto-regulatory loop in multiple myeloma development using mir-192, mir-194 and mir-215
US20140189897A1 (en) 2011-06-21 2014-07-03 Mayo Foundation For Medical Education And Research Transgenic animals capable of being induced to delete senescent cells
JP2014532071A (ja) * 2011-10-14 2014-12-04 エスティーシー. ユーエヌエムStc.Unm カーゴの経皮送達を含む標的送達用の多孔性ナノ粒子に担持された脂質二重層(プロトセル)及びその方法
US20150064137A1 (en) 2012-04-17 2015-03-05 Kythera Biopharmaceuticals, Inc. Use of engineered viruses to specifically kill senescent cells
US9687448B2 (en) * 2012-12-07 2017-06-27 Alnylam Pharmaceuticals, Inc. Nucleic acid lipid particle formulations
WO2014153163A1 (en) 2013-03-14 2014-09-25 Dicerna Pharmaceuticals, Inc. Process for formulating an anionic agent
US10130649B2 (en) * 2013-03-15 2018-11-20 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
CA2966234A1 (en) 2014-12-15 2016-06-23 Bellicum Pharmaceuticals, Inc. Methods for controlled elimination of therapeutic cells
GB201503133D0 (en) 2015-02-24 2015-04-08 Ucl Business Plc And Syncona Partners Llp Chimeric protein
WO2016185481A2 (en) 2015-05-20 2016-11-24 Yeda Research And Development Co. Ltd. Method of targeting senescent cells
AU2018206485B2 (en) * 2017-01-09 2024-12-05 Oisin Biotechnologies Fusogenic lipid nanoparticles and methods for manufacturing and use for therapeutic protein production and for treatment
WO2019204666A1 (en) 2018-04-18 2019-10-24 Oisin Biotechnologies, Inc. Fusogenic lipid nanoparticles and methods for the manufacture and use thereof for the target cell-specific production of a therapeutic protein and for the treatment of a disease, condition, or disorder associated with a target cell
WO2020163408A2 (en) 2019-02-04 2020-08-13 Oisin Biotechnologies, Inc. Fusogenic lipid nanoparticles for the target cell-specific production of rapamycin inducible therapeutic proteins

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060002895A1 (en) 1998-03-11 2006-01-05 Board Of Regents, The University Of Texas System Induction of apoptic or cytotoxic gene expression by adenoviral mediated gene codelivery
JP2002517996A (ja) 1998-06-16 2002-06-25 トーマス ジェファーソン ユニヴァーシティー 短型のApaf−1およびその使用方法
US20140314831A1 (en) 2010-09-29 2014-10-23 Innovascreen Inc. Recombinant polypeptides for membrane fusion and uses thereof
JP2017014278A (ja) 2011-06-08 2017-01-19 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド mRNA送達のための脂質ナノ粒子組成物および方法
JP2015525209A (ja) 2012-05-23 2015-09-03 ジ・オハイオ・ステート・ユニバーシティ 脂質ナノ粒子組成物と、それを作製する方法、及びそれを使用する方法
US20160010110A1 (en) 2013-03-24 2016-01-14 Oisin Biotechnologies Systems and methods for the targeted production of a therapeutic protein within a target cell

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