JP2023549011A5 - - Google Patents

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Publication number
JP2023549011A5
JP2023549011A5 JP2023517323A JP2023517323A JP2023549011A5 JP 2023549011 A5 JP2023549011 A5 JP 2023549011A5 JP 2023517323 A JP2023517323 A JP 2023517323A JP 2023517323 A JP2023517323 A JP 2023517323A JP 2023549011 A5 JP2023549011 A5 JP 2023549011A5
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JP2023517323A
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JP2023549011A (ja
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Priority claimed from PCT/US2021/050511 external-priority patent/WO2022060871A1/en
Publication of JP2023549011A publication Critical patent/JP2023549011A/ja
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JP2023517323A 2020-09-15 2021-09-15 遺伝子編集のための脂質製剤 Pending JP2023549011A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063078738P 2020-09-15 2020-09-15
US63/078,738 2020-09-15
US202163220340P 2021-07-09 2021-07-09
US63/220,340 2021-07-09
PCT/US2021/050511 WO2022060871A1 (en) 2020-09-15 2021-09-15 Lipid formulations for gene editing

Publications (2)

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JP2023549011A JP2023549011A (ja) 2023-11-22
JP2023549011A5 true JP2023549011A5 (enExample) 2024-09-26

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JP2023517323A Pending JP2023549011A (ja) 2020-09-15 2021-09-15 遺伝子編集のための脂質製剤

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US (3) US12024484B2 (enExample)
EP (1) EP4213882A4 (enExample)
JP (1) JP2023549011A (enExample)
WO (1) WO2022060871A1 (enExample)

Families Citing this family (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB2613225B (en) 2020-03-04 2024-01-03 Verve Therapeutics Inc Compositions and methods for targeted RNA delivery
MX2022012683A (es) * 2020-04-09 2023-01-11 Verve Therapeutics Inc Edicion de bases de pcsk9 y metodos de uso de la misma para el tratamiento de enfermedades.
JP2023549011A (ja) 2020-09-15 2023-11-22 ヴァーヴ・セラピューティクス,インコーポレーテッド 遺伝子編集のための脂質製剤
EP4281080A4 (en) * 2021-01-20 2025-09-24 Beam Therapeutics Inc NANOMATERIALS COMPRISING A BIODEGRADABLE ELEMENT
KR20240013087A (ko) 2021-05-24 2024-01-30 쑤저우 아보젠 바이오사이언시스 컴퍼니 리미티드 지질 화합물 및 지질 나노입자 조성물
WO2023015223A2 (en) 2021-08-03 2023-02-09 Verve Therapeutics, Inc. Compositions and methods for targeted rna delivery
JP2024536406A (ja) * 2021-10-08 2024-10-04 スージョウ・アボジェン・バイオサイエンシズ・カンパニー・リミテッド 脂質化合物及び脂質ナノ粒子組成物
CN114751835B (zh) * 2022-03-15 2024-09-17 大连民族大学 一类含有多个酯键可电离叔胺脂质及其合成方法和应用
CN116354836A (zh) * 2022-03-25 2023-06-30 深圳市新合生物医疗科技有限公司 阳离子脂质化合物及其制备方法和应用、以及mRNA递送系统
CN114890907B (zh) * 2022-03-31 2023-10-31 荣灿生物医药技术(上海)有限公司 一种阳离子脂质化合物及其制备方法和应用
TW202404568A (zh) * 2022-04-07 2024-02-01 美商Rna免疫公司 用於藥物遞送之化合物及組合物
CN117003808A (zh) * 2022-04-28 2023-11-07 北京科兴中维生物技术有限公司 一种阳离子脂质化合物及其制备方法和用途
CN114874104B (zh) * 2022-04-29 2024-11-29 湖北英纳氏生物科技有限公司 阳离子脂质体SM-102及Lipid5的制备方法
WO2023215790A1 (en) * 2022-05-03 2023-11-09 The Board Of Regents Of The University Of Texas System Co-delivery of inhibitory nucleic acids and genome editors for tumor therapy
WO2023230601A1 (en) 2022-05-27 2023-11-30 Beam Therapeutics Inc. Identification of nanoparticles for preferential tissue or cell targeting
GB2634837A (en) 2022-06-07 2025-04-23 Scribe Therapeutics Inc Compositions and methods for the targeting of PCSK9
CN115286674A (zh) * 2022-06-13 2022-11-04 湖北英纳氏生物科技有限公司 一种阳离子脂质体及其制备方法
CN116969850A (zh) * 2022-06-14 2023-10-31 苏州科锐迈德生物医药科技有限公司 碳酸酯-羧酸酯型脂质化合物及基于其的脂质载体、核酸脂质纳米粒组合物和药物制剂
CN114989182B (zh) * 2022-06-23 2023-06-23 尧唐(上海)生物科技有限公司 脂质化合物、包含其的组合物及应用
WO2024012272A1 (zh) * 2022-07-11 2024-01-18 传信生物医药(苏州)有限公司 脂质化合物及其应用
EP4559896A4 (en) * 2022-07-19 2025-10-22 Univ Hokkaido Nat Univ Corp PH-SENSITIVE CATIONIC LIPID AND LIPID NANOPARTICLE
CN117800859A (zh) * 2022-09-26 2024-04-02 苏州科锐迈德生物医药科技有限公司 具有戊二酸骨架的脂质化合物及基于其的脂质载体、核酸脂质纳米粒组合物和药物制剂
CN115504945B (zh) * 2022-10-09 2025-09-02 南京吉迈生物技术有限公司 可电离的含杂环脂质分子及其在制备脂质纳米粒的应用
CN115417779B (zh) * 2022-11-03 2023-06-16 北京华芢生物技术有限公司 可离子化的阳离子脂c6-a1及由其组成的纳米脂质体颗粒
CN115417780B (zh) * 2022-11-04 2023-06-20 北京华芢生物技术有限公司 可离子化的阳离子脂c5-a2及由其组成的纳米脂质体颗粒
WO2024199282A1 (zh) * 2023-03-28 2024-10-03 苏州艾博生物科技有限公司 脂质化合物和脂质纳米颗粒组合物
CN120957730A (zh) * 2023-03-29 2025-11-14 国立大学法人东北大学 核酸封装脂质纳米粒子的制造方法
JPWO2024248146A1 (enExample) * 2023-05-31 2024-12-05
CN117024323A (zh) * 2023-07-21 2023-11-10 中国科学院基础医学与肿瘤研究所(筹) 一种可降解、可电离的阳离子脂材及其应用
AU2024325364A1 (en) * 2023-08-16 2026-03-26 Beam Therapeutics Inc. Ionizable lipids with branched head groups
CN117986153A (zh) * 2023-09-07 2024-05-07 晟迪生物医药(苏州)有限公司 一种可电离阳离子脂质化合物及其制备方法和应用
WO2025059599A1 (en) * 2023-09-13 2025-03-20 Tessera Therapeutics, Inc. Lipid nanoparticles for delivery of therapeutic payloads to cells
WO2026023622A1 (ja) * 2024-07-22 2026-01-29 富士フイルム株式会社 アミノ脂質又はその塩の製造中間体としての化合物、およびそれを用いたアミノ脂質化合物の製造方法

Family Cites Families (61)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
AU733310C (en) 1997-05-14 2001-11-29 University Of British Columbia, The High efficiency encapsulation of charged therapeutic agents in lipid vesicles
DE10109897A1 (de) 2001-02-21 2002-11-07 Novosom Ag Fakultativ kationische Liposomen und Verwendung dieser
US20050222064A1 (en) 2002-02-20 2005-10-06 Sirna Therapeutics, Inc. Polycationic compositions for cellular delivery of polynucleotides
US7858117B2 (en) 2002-02-21 2010-12-28 Novosom Ag Amphoteric liposomes and their use
EP1519714B1 (en) 2002-06-28 2010-10-20 Protiva Biotherapeutics Inc. Method and apparatus for producing liposomes
DE102004054730A1 (de) 2004-03-28 2006-05-11 Novosom Ag Serumstabile amphotere Liposomen
US20060051405A1 (en) 2004-07-19 2006-03-09 Protiva Biotherapeutics, Inc. Compositions for the delivery of therapeutic agents and uses thereof
US20070087045A1 (en) 2005-10-14 2007-04-19 Industrial Technology Research Institute Lipid carrier and method of preparing the same
EP1989179B1 (en) * 2006-02-15 2011-01-26 Ge Healthcare As Contrast agents
FR2928925B1 (fr) 2008-03-19 2011-01-07 Centre Nat Rech Scient Complexes de bore ou d'aluminium, et leurs utilisations.
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
US8883211B2 (en) 2008-07-10 2014-11-11 Serina Therapeutics, Inc. Polyoxazolines with inert terminating groups, polyoxazolines prepared from protected initiating groups and related compounds
CN111808084A (zh) 2008-11-10 2020-10-23 阿布特斯生物制药公司 用于递送治疗剂的新型脂质和组合物
WO2011000107A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
CN101724210B (zh) 2009-12-22 2012-05-23 浙江科技学院 用于加工pvc透明片材的稳定剂及其制备方法和应用
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
US20120178858A1 (en) 2011-01-10 2012-07-12 Andrew Julian Wnuk Isosorbide-Plasticized Starch And Uses Thereof
US8691750B2 (en) * 2011-05-17 2014-04-08 Axolabs Gmbh Lipids and compositions for intracellular delivery of biologically active compounds
PE20181541A1 (es) 2011-10-27 2018-09-26 Massachusetts Inst Technology Derivados de aminoacidos funcionalizados en la terminal n capaces de formar microesferas encapsuladoras de farmaco
CN102617364B (zh) 2012-03-15 2014-04-23 南京工业大学 羟甲基二胺类化合物及其制备方法与应用
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
WO2014008334A1 (en) 2012-07-06 2014-01-09 Alnylam Pharmaceuticals, Inc. Stable non-aggregating nucleic acid lipid particle formulations
WO2014089239A1 (en) 2012-12-07 2014-06-12 Alnylam Pharmaceuticals, Inc. Improved nucleic acid lipid particle formulations
CN107879960B (zh) 2013-03-08 2021-06-22 诺华股份有限公司 用于传递活性成分的脂质和脂质组合物
EP3083556B1 (en) 2013-12-19 2019-12-25 Novartis AG Lipids and lipid compositions for the delivery of active agents
US10426737B2 (en) * 2013-12-19 2019-10-01 Novartis Ag Lipids and lipid compositions for the delivery of active agents
RU2698697C2 (ru) 2013-12-23 2019-08-29 Байер Фарма Акциенгезельшафт Конъюгаты связующего (ADC) с ингибиторами KSP
CN106794141B (zh) * 2014-07-16 2021-05-28 诺华股份有限公司 将核酸包封在脂质纳米粒主体中的方法
WO2016045732A1 (en) 2014-09-25 2016-03-31 Biontech Rna Pharmaceuticals Gmbh Stable formulations of lipids and liposomes
EP3258952A4 (en) 2015-02-04 2018-12-05 Pacific Biosciences Of California, Inc. Multimeric protected fluorescent reagents
EP3275448B1 (en) 2015-03-24 2025-10-29 Kyowa Kirin Co., Ltd. Nucleic acid-containing lipid nanoparticles
WO2016197133A1 (en) 2015-06-04 2016-12-08 Protiva Biotherapeutics, Inc. Delivering crispr therapeutics with lipid nanoparticles
JP6768011B2 (ja) 2015-06-23 2020-10-14 バイエル ファーマ アクチエンゲゼルシャフト キネシンスピンドルタンパク質(ksp)阻害剤の抗cd123抗体との抗体薬物複合体
US20180200186A1 (en) 2015-07-09 2018-07-19 Insmed Incorporated Compositions and methods for treating lung diseases and lung injury
JP6948313B6 (ja) 2015-09-17 2022-01-14 モデルナティエックス インコーポレイテッド 治療剤の細胞内送達のための化合物および組成物
PL3386484T3 (pl) 2015-12-10 2022-07-25 Modernatx, Inc. Kompozycje i sposoby dostarczania środków terapeutycznych
LT3436077T (lt) 2016-03-30 2025-06-25 Intellia Therapeutics, Inc. Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams
US11231415B2 (en) * 2016-05-12 2022-01-25 Z Biotech Llc Multivalent glycan microarray platform
WO2017222016A1 (ja) * 2016-06-24 2017-12-28 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
WO2018062413A1 (ja) 2016-09-28 2018-04-05 協和発酵キリン株式会社 核酸含有脂質ナノ粒子
WO2018119514A1 (en) 2016-12-28 2018-07-05 Precision Nanosystems Inc. Compositions for transfecting resistant cell types
US11583504B2 (en) 2016-11-08 2023-02-21 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
AU2017374042C1 (en) * 2016-12-09 2024-07-11 Acuitas Therapeutics, Inc. Delivery of target specific nucleases
EP4186888B1 (en) 2017-03-15 2025-11-26 ModernaTX, Inc. Compound and compositions for intracellular delivery of therapeutic agents
DK3607069T3 (da) 2017-04-05 2022-11-21 Silence Therapeutics Gmbh Produkter og sammensætninger
WO2018191750A2 (en) 2017-04-14 2018-10-18 The Broad Institute Inc. Novel delivery of large payloads
BR112019025104A2 (pt) * 2017-05-30 2020-06-23 Glaxosmithkline Biologicals S.A. Método de fabricar um sistema de entrega não viral, sistema de entrega não viral, e, composição
WO2019027055A1 (ja) 2017-08-04 2019-02-07 協和発酵キリン株式会社 核酸含有脂質ナノ粒子
EP3675817A1 (en) 2017-08-31 2020-07-08 Modernatx, Inc. Methods of making lipid nanoparticles
US20230140670A1 (en) 2017-09-29 2023-05-04 Intellia Therapeutics, Inc. Formulations
KR102832035B1 (ko) * 2017-09-29 2025-07-09 인텔리아 테라퓨틱스, 인크. 지질 나노파티클을 이용한 mRNA 전달의 체외 방법
EP3733647B1 (en) * 2017-12-27 2022-06-15 Eisai R&D Management Co., Ltd. Cationic lipid
CN114127044A (zh) * 2019-04-25 2022-03-01 英特利亚治疗股份有限公司 可电离的胺类脂质和脂质纳米颗粒
IL294318A (en) 2020-01-09 2022-08-01 Guide Therapeutics Llc Nanomaterials
CA3181340A1 (en) 2020-04-28 2021-11-04 Intellia Therapeutics, Inc. Methods of in vitro cell delivery
EP4153223A1 (en) 2020-05-20 2023-03-29 Flagship Pioneering Innovations VI, LLC Immunogenic compositions and uses thereof
IL298362A (en) 2020-05-20 2023-01-01 Flagship Pioneering Innovations Vi Llc Coronavirus antigen compositions and their uses
WO2021243301A2 (en) 2020-05-29 2021-12-02 Flagship Pioneering Innovations Vi, Llc. Trem compositions and methods relating thereto
KR20230029685A (ko) 2020-05-29 2023-03-03 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 Trem 조성물 및 이에 관련된 방법
JP2023549011A (ja) 2020-09-15 2023-11-22 ヴァーヴ・セラピューティクス,インコーポレーテッド 遺伝子編集のための脂質製剤

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