JP2021535226A5 - - Google Patents

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Publication number
JP2021535226A5
JP2021535226A5 JP2021536679A JP2021536679A JP2021535226A5 JP 2021535226 A5 JP2021535226 A5 JP 2021535226A5 JP 2021536679 A JP2021536679 A JP 2021536679A JP 2021536679 A JP2021536679 A JP 2021536679A JP 2021535226 A5 JP2021535226 A5 JP 2021535226A5
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Japan
Prior art keywords
composition according
substituted
lipids
composition
alkyl
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JP2021536679A
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English (en)
Japanese (ja)
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JPWO2020051220A5 (https=
JP2021535226A (ja
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Priority claimed from PCT/US2019/049552 external-priority patent/WO2020051220A1/en
Publication of JP2021535226A publication Critical patent/JP2021535226A/ja
Publication of JPWO2020051220A5 publication Critical patent/JPWO2020051220A5/ja
Publication of JP2021535226A5 publication Critical patent/JP2021535226A5/ja
Priority to JP2025064066A priority Critical patent/JP2025108518A/ja
Pending legal-status Critical Current

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JP2021536679A 2018-09-04 2019-09-04 核酸を臓器特異的送達するための組成物および方法 Pending JP2021535226A (ja)

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JP2025064066A JP2025108518A (ja) 2018-09-04 2025-04-09 核酸を臓器特異的送達するための組成物および方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862726770P 2018-09-04 2018-09-04
US62/726,770 2018-09-04
PCT/US2019/049552 WO2020051220A1 (en) 2018-09-04 2019-09-04 Compositions and methods for organ specific delivery of nucleic acids

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JP2025064066A Division JP2025108518A (ja) 2018-09-04 2025-04-09 核酸を臓器特異的送達するための組成物および方法

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JP2021535226A JP2021535226A (ja) 2021-12-16
JPWO2020051220A5 JPWO2020051220A5 (https=) 2022-09-14
JP2021535226A5 true JP2021535226A5 (https=) 2022-09-14

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JP2021536679A Pending JP2021535226A (ja) 2018-09-04 2019-09-04 核酸を臓器特異的送達するための組成物および方法
JP2025064066A Pending JP2025108518A (ja) 2018-09-04 2025-04-09 核酸を臓器特異的送達するための組成物および方法

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US (2) US11766408B2 (https=)
EP (1) EP3846857A4 (https=)
JP (2) JP2021535226A (https=)
CN (2) CN120360966A (https=)
AU (2) AU2019335055B2 (https=)
CA (1) CA3111483A1 (https=)
GB (3) GB2617429B (https=)
WO (1) WO2020051220A1 (https=)

Families Citing this family (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3349802B1 (en) * 2015-09-14 2021-08-04 The Board of Regents of the University of Texas System Lipocationic dendrimers and uses thereof
EP4316588A3 (en) 2016-05-27 2024-05-15 Transcriptx, Inc. Treatment of primary ciliary dyskinesia with synthetic messenger rna
US12357580B2 (en) 2018-06-19 2025-07-15 The Board Of Regents Of The University Of Texas System Lipid nanoparticle compositions for delivery of mRNA and long nucleic acids
WO2020051220A1 (en) 2018-09-04 2020-03-12 The Board of the Regents of the University of Texas System Compositions and methods for organ specific delivery of nucleic acids
CN112996519B (zh) 2018-09-04 2025-02-28 德克萨斯大学系统董事会 用于核酸的器官特异性递送的组合物和方法
WO2020051541A1 (en) 2018-09-07 2020-03-12 Duke University Nanoparticulate drug delivery systems
CN113166783B (zh) * 2018-10-09 2024-10-11 不列颠哥伦比亚大学 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法
WO2021216577A1 (en) * 2020-04-20 2021-10-28 Board Of Regents, The University Of Texas System Lipid nanoparticle (lnp) delivery systems and uses thereof
JP2023527163A (ja) * 2020-05-19 2023-06-27 ラモット・アット・テル・アビブ・ユニバーシテイ・リミテッド 核酸送達用の脂質粒子およびその臨床応用
US20240115730A1 (en) * 2020-12-18 2024-04-11 Omega Therapeutics, Inc. Tissue-specific nucleic acid delivery by 1,2-dioleoyl-3-trimethylammonium-propane (dotap) lipid nanoparticles
WO2022204043A1 (en) * 2021-03-22 2022-09-29 The Board Of Regents Of The University Of Texas System Compositions and methods for targeted delivery to cells
JP2024511463A (ja) * 2021-03-22 2024-03-13 リコード セラピューティクス，インク． 細胞への標的送達のための組成物および方法
KR20230175204A (ko) * 2021-03-22 2023-12-29 리코드 테라퓨틱스, 인크. 폴리뉴클레오티드 조성물, 관련된 제제, 및 이의 사용 방법
EP4313004A4 (en) * 2021-03-23 2025-01-15 Recode Therapeutics, Inc. Compositions and methods for targeted systemic delivery to cells
JP2024511437A (ja) 2021-03-23 2024-03-13 リコード セラピューティクス，インク． ポリヌクレオチド組成物、関連製剤、およびその使用方法
IL307319A (en) * 2021-03-31 2023-11-01 Rejuvenation Tech Inc Compositions and methods for delivery of rna
AU2022262355A1 (en) * 2021-04-22 2023-10-12 Flagship Labs 114, Inc. Tissue-specific nucleic acid delivery by mixed cationic lipid particles
WO2022226344A1 (en) * 2021-04-22 2022-10-27 The Board Of Regents Of The University Of Texas System All-in-one dendrimer-based lipid nanoparticles enable precise hdr-mediated gene editing in vivo
CN113402404B (zh) * 2021-04-30 2022-03-11 苏州科锐迈德生物医药科技有限公司 脂质化合物及包含其的脂质载体、核酸脂质纳米粒组合物和药物制剂
CN118201605A (zh) 2021-10-25 2024-06-14 杜克大学 基于poegma的脂质纳米粒子
WO2023165583A1 (zh) * 2022-03-04 2023-09-07 益杰立科(上海)生物科技有限公司 靶向眼部细胞的递送系统和方法
CN119654166A (zh) * 2022-04-01 2025-03-18 内诺维什疗法股份有限公司 mRNA递送方法及其组合物
CN114891808B (zh) * 2022-06-21 2023-10-27 珠海丽凡达生物技术有限公司 编码ALDH2多肽的mRNA分子、应用及mRNA药物
EP4551199A1 (en) 2022-07-08 2025-05-14 PolyPlus Transfection Lipid nanoparticles comprising an imidazolium-based cationic lipid
EP4393511A4 (en) * 2022-07-11 2025-04-02 Themedium Therapeutics Co., Ltd. LIPID COMPOUND AND ITS USE
CN116763756A (zh) * 2022-10-11 2023-09-19 中国科学院化学研究所 一种脂质纳米颗粒及其制备方法和应用
KR20250094676A (ko) * 2022-10-25 2025-06-25 나노베이션 테라퓨틱스 인크. 안티-센스 올리고뉴클레오티드 전달을 위한 지질 나노입자 제형
WO2024108740A1 (zh) * 2022-11-21 2024-05-30 成都诺恩基因科技有限公司 适合于肌注给药的核酸-脂质纳米颗粒、制剂及其应用
EP4400491A4 (en) * 2022-11-24 2025-05-07 Themedium Therapeutics Co., Ltd Novel lipids used for nucleic acid delivery and lipid nanoparticle composition
EP4630061A1 (en) * 2022-12-08 2025-10-15 Nanovation Therapeutics Inc. Lipid nanoparticles comprising elevated neutral lipid and a targeting moiety for targeted delivery of nucleic acid
EP4630057A1 (en) * 2022-12-08 2025-10-15 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
CN118236344A (zh) * 2022-12-15 2024-06-25 深圳瑞吉生物科技有限公司 一种核酸脂质纳米载体及其制备方法与应用
KR20250124204A (ko) * 2022-12-23 2025-08-19 턴 바이오테크놀로지스 인코포레이티드 이중 지질 구조
CN117771213A (zh) * 2023-01-10 2024-03-29 北京剂泰医药科技有限公司 脂质纳米颗粒组合物及其用途
CN121057577A (zh) * 2023-01-27 2025-12-02 赛欧生物医药股份有限公司 经修饰的脂质组合物及其用途
WO2024215257A1 (en) * 2023-04-13 2024-10-17 National University Of Singapore Polymersomes and methods of using and making the same
CN116271105B (zh) * 2023-05-18 2023-08-25 上海贝斯昂科生物科技有限公司 一种适于rpe细胞转染的脂质纳米颗粒及其应用
CN119119123A (zh) * 2023-06-08 2024-12-13 西南大学 长春胺类衍生物及其制备方法和应用
TW202525812A (zh) * 2023-10-24 2025-07-01 南韓商綠十字股份有限公司 可離子化脂質及其用途
WO2025117816A1 (en) * 2023-12-01 2025-06-05 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
US12364773B2 (en) * 2023-12-01 2025-07-22 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
US12553042B2 (en) 2023-12-01 2026-02-17 Recode Therapeutics, Inc. Method for quantifying an amount of capped messenger RNA
WO2025137646A1 (en) * 2023-12-22 2025-06-26 Recode Therapeutics, Inc. Gene editing methods and compositions for treating cystic fibrosis
CN118001254B (zh) * 2024-04-09 2024-07-26 北京悦康科创医药科技股份有限公司 靶向抗原呈递细胞的脂质组合物及其应用
WO2025228979A1 (en) 2024-04-30 2025-11-06 Neovac Ltd. Lipids nanoparticle formulations suitable for lung targeting
WO2025240356A1 (en) * 2024-05-14 2025-11-20 Recode Therapeutics, Inc. Lipid nanoparticles for hepatic delivery
WO2026003403A1 (en) 2024-06-28 2026-01-02 Certest Biotec, S.L. Ionizable lipids and lipid nanoparticles containing them for extrahepatic delivery
CN118750468B (zh) * 2024-09-05 2025-02-18 北京悦康科创医药科技股份有限公司 一种核酸-脂质纳米颗粒冻干组合物及其制备方法和应用
WO2026062291A1 (en) 2024-09-23 2026-03-26 Aldexchem Kft. Silicon-containing spleen-targeted lnp delivery systems

Family Cites Families (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US7053150B2 (en) 2000-12-18 2006-05-30 Nektar Therapeutics Al, Corporation Segmented polymers and their conjugates
US7314956B2 (en) 2001-08-08 2008-01-01 Vaxim, Inc. Multifunctional carrier for the delivery of a pharmacological agent or genetic material into a cell
US8871709B2 (en) 2003-02-05 2014-10-28 Queensland University of Technolgy Synthetic chimeric proteins comprising epidermal growth factor and vitronectin
US7985424B2 (en) 2004-04-20 2011-07-26 Dendritic Nanotechnologies Inc. Dendritic polymers with enhanced amplification and interior functionality
SI1771206T1 (en) 2004-05-05 2018-06-29 Silence Therapeutics Gmbh Lipids, lipid complexes and use thereof
US7799565B2 (en) 2004-06-07 2010-09-21 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering RNA
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
US9006487B2 (en) 2005-06-15 2015-04-14 Massachusetts Institute Of Technology Amine-containing lipids and uses thereof
WO2007073489A2 (en) 2005-12-22 2007-06-28 Trustees Of Boston University Molecules for gene delivery and gene therapy, and methods of use thereof
CA2658768C (en) 2006-07-21 2016-05-17 Massachusetts Institute Of Technology End-modified poly(beta-amino esters) and uses thereof
US20110165223A1 (en) * 2008-01-02 2011-07-07 The Johns Hopkins University Antitumor Immunization by Liposomal Delivery of Vaccine to the Spleen
AU2009238175C1 (en) 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
ES2646630T3 (es) 2008-11-07 2017-12-14 Massachusetts Institute Of Technology Lipidoides aminoalcohólicos y usos de los mismos
US8163861B2 (en) 2009-01-15 2012-04-24 Living Proof, Inc. Beta-amino ester compounds and uses thereof
AP2012006053A0 (en) 2009-06-02 2012-02-29 Brian Charles Keller Pure peg-lipid conjugates.
CN101591428B (zh) 2009-06-29 2011-05-25 浙江大学 一种聚酯树枝状大分子的制备方法
HUE026646T2 (en) * 2010-07-06 2016-07-28 Glaxosmithkline Biologicals Sa Preferred liposomes containing lipids of PKA value for delivery of RNA
US9326939B2 (en) * 2010-07-31 2016-05-03 The Scripps Research Institute Liposome targeting compounds and related uses
WO2012090223A1 (en) 2010-12-29 2012-07-05 Indian Institute Of Science Poly(ether imine) dendrimers and uses thereof
CN107043334B (zh) 2011-06-08 2020-06-16 日东电工株式会社 用于定向药物输送和增强siRNA活性的化合物
PL3586861T3 (pl) 2011-06-08 2022-05-23 Translate Bio, Inc. Kompozycje nanocząstek lipidowych i sposoby dostarczania mrna
JP6294229B2 (ja) * 2011-10-18 2018-03-14 ダイセルナ ファーマシューティカルズ， インコーポレイテッドＤｉｃｅｒｎａ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． アミン陽イオン性脂質およびその使用
ES2762873T3 (es) * 2012-03-29 2020-05-26 Translate Bio Inc Lípidos catiónicos ionizables
US9750819B2 (en) * 2012-05-23 2017-09-05 Ohio State Innovation Foundation Lipid nanoparticle compositions and methods of making and methods of using the same
EP2885318A4 (en) 2012-08-14 2016-03-30 Angiochem Inc PEPTIDE DENDRIMER CONJUGATES AND ITS USES
JP6241193B2 (ja) 2012-10-23 2017-12-06 コニカミノルタ株式会社 透明電極、電子デバイス及び有機エレクトロルミネッセンス素子
US20140187501A1 (en) * 2012-12-28 2014-07-03 Blend Therapeutics, Inc. Targeted Conjugates Encapsulated in Particles and Formulations Thereof
US20140186332A1 (en) 2012-12-28 2014-07-03 NX Pharmagen Biomarkers of preterm birth
US9820942B2 (en) * 2013-03-06 2017-11-21 Biomics Biotechnologies Co., Ltd. Lipidosome preparation, preparation method and application thereof
US10130649B2 (en) 2013-03-15 2018-11-20 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
ES2707966T3 (es) 2013-10-22 2019-04-08 Translate Bio Inc Terapia de ARNm para la deficiencia en síntesis de argininosuccinato
AU2014361781B2 (en) * 2013-12-12 2021-04-01 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing
US20170143848A1 (en) 2014-03-24 2017-05-25 Shire Human Genetic Therapies, Inc. Mrna therapy for the treatment of ocular diseases
US9833416B2 (en) * 2014-04-04 2017-12-05 Ohio State Innovation Foundation Oligonucleotide lipid nanoparticle compositions, methods of making and methods of using the same
SG10201912038TA (en) * 2014-04-23 2020-02-27 Modernatx Inc Nucleic acid vaccines
WO2015191693A2 (en) * 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
CN103999853B (zh) 2014-06-11 2015-07-22 中国农业大学 荧光树枝状纳米大分子在制备药物载体中的应用
EP3169309B1 (en) * 2014-07-16 2023-05-10 Novartis AG Method of encapsulating a nucleic acid in a lipid nanoparticle host
US10583201B2 (en) * 2014-10-10 2020-03-10 Massachusetts Eye And Ear Infirmary Efficient delivery of therapeutic molecules in vitro and in vivo
WO2016090262A1 (en) 2014-12-05 2016-06-09 Shire Human Genetic Therapies, Inc. Messenger rna therapy for treatment of articular disease
AU2015360794B2 (en) 2014-12-08 2021-07-08 The Board Of Regents Of The University Of Texas System Lipocationic polymers and uses thereof
US11219634B2 (en) * 2015-01-21 2022-01-11 Genevant Sciences Gmbh Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells
WO2016118725A1 (en) * 2015-01-23 2016-07-28 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
EP3349802B1 (en) * 2015-09-14 2021-08-04 The Board of Regents of the University of Texas System Lipocationic dendrimers and uses thereof
EP3353309A4 (en) * 2015-09-25 2019-04-10 Tarveda Therapeutics, Inc. COMPOSITIONS AND METHOD FOR GENERIC EDITING
TWI773666B (zh) * 2016-03-30 2022-08-11 美商英特利亞醫療公司 Crispr/cas 組分之脂質奈米粒子調配物
US20190167811A1 (en) * 2016-04-13 2019-06-06 Modernatx, Inc. Lipid compositions and their uses for intratumoral polynucleotide delivery
CN109414451B (zh) 2016-05-16 2022-08-12 德克萨斯大学系统董事会 用于作为纳米粒递送tRNA的组合物及其使用方法
KR102469450B1 (ko) * 2016-05-18 2022-11-22 모더나티엑스, 인크. 인터류킨-12 (il12)를 코딩하는 폴리뉴클레오티드 및 그의 용도
WO2018029586A1 (en) * 2016-08-07 2018-02-15 Novartis Ag Mrna-mediated immunization methods
BR112019008481A2 (pt) * 2016-10-26 2020-03-03 Curevac Ag Vacinas de mrna de nanopartículas lipídicas
EP3315125A1 (en) * 2016-10-31 2018-05-02 Silence Therapeutics (London) Ltd Lipid nanoparticle formulation
CA3103528A1 (en) * 2018-06-19 2019-12-26 The Board Of Regents Of The University Of Texas System Lipid nanoparticle compositions for delivery of mrna and long nucleic acids
WO2020051220A1 (en) 2018-09-04 2020-03-12 The Board of the Regents of the University of Texas System Compositions and methods for organ specific delivery of nucleic acids
CN112996519B (zh) * 2018-09-04 2025-02-28 德克萨斯大学系统董事会 用于核酸的器官特异性递送的组合物和方法

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