JP2023549011A - 遺伝子編集のための脂質製剤 - Google Patents

遺伝子編集のための脂質製剤 Download PDF

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JP2023549011A
JP2023549011A JP2023517323A JP2023517323A JP2023549011A JP 2023549011 A JP2023549011 A JP 2023549011A JP 2023517323 A JP2023517323 A JP 2023517323A JP 2023517323 A JP2023517323 A JP 2023517323A JP 2023549011 A JP2023549011 A JP 2023549011A
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formula
nanoparticle composition
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JPWO2022060871A5 (https=
JP2023549011A5 (https=
Inventor
ラジーブ，カラントッタチル・ジー
ビスワス，スービク
マリャラ，パドマ
カシエビッチ，リサ・エヌ
ビーチ，アーロン
コレース，トーマス・ブイ
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ヴァーヴ・セラピューティクス，インコーポレーテッド
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/16—Amides, e.g. hydroxamic acids
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • 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
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C219/00—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton
    • C07C219/02—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C219/04—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C219/16—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having at least one of the hydroxy groups esterified by an inorganic acid or a derivative thereof
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C271/00—Derivatives of carbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
    • C07C271/06—Esters of carbamic acids
    • C07C271/08—Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms
    • C07C271/10—Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms with the nitrogen atoms of the carbamate groups bound to hydrogen atoms or to acyclic carbon atoms
    • C07C271/20—Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms with the nitrogen atoms of the carbamate groups bound to hydrogen atoms or to acyclic carbon atoms to carbon atoms of hydrocarbon radicals substituted by nitrogen atoms not being part of nitro or nitroso groups
    • 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
    • C07D295/08—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 singly bound oxygen or sulfur atoms
    • C07D295/084—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 singly bound oxygen or sulfur atoms with the ring nitrogen atoms and the oxygen or sulfur atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
    • C07D295/088—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 singly bound oxygen or sulfur atoms with the ring nitrogen atoms and the oxygen or sulfur 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)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Dispersion Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biomedical Technology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Inorganic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Steroid Compounds (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
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 (3)

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JP2023549011A true JP2023549011A (ja) 2023-11-22
JPWO2022060871A5 JPWO2022060871A5 (https=) 2024-09-26
JP2023549011A5 JP2023549011A5 (https=) 2024-09-26

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

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Families Citing this family (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230011913A (ko) 2020-03-04 2023-01-25 버브 테라퓨틱스, 인크. 표적화된 rna 전달을 위한 조성물 및 방법
GB2632565B (en) * 2020-04-09 2025-06-04 Verve Therapeutics Inc Base editing of PCSK9 and methods of using same for treatment of disease
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
IL303845A (en) * 2021-01-20 2023-08-01 Beam Therapeutics Inc Nanomaterials include a degradable property
CA3211623A1 (en) 2021-05-24 2022-12-01 Bo YING Lipid compounds and lipid nanoparticle compositions
JP2024530647A (ja) 2021-08-03 2024-08-23 ヴァーヴ・セラピューティクス，インコーポレーテッド 標的ｒｎａ送達のための組成物および方法
CA3234127A1 (en) * 2021-10-08 2023-04-13 Suzhou Abogen Biosciences Co., Ltd. Lipid compounds and lipid nanoparticle compositions
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
WO2023196615A1 (en) * 2022-04-07 2023-10-12 RNAimmune, Inc. Compounds and compositions for drug delivery
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
EP4532713A1 (en) 2022-05-27 2025-04-09 Beam Therapeutics Inc. Identification of nanoparticles for preferential tissue or cell targeting
WO2023240076A1 (en) 2022-06-07 2023-12-14 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
EP4393511A4 (en) * 2022-07-11 2025-04-02 Themedium Therapeutics Co., Ltd. LIPID COMPOUND AND ITS USE
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
WO2024203660A1 (ja) * 2023-03-29 2024-10-03 国立大学法人東北大学 核酸内封脂質ナノ粒子の製造方法
JPWO2024248146A1 (https=) * 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
WO2025038855A1 (en) * 2023-08-16 2025-02-20 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

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015095340A1 (en) * 2013-12-19 2015-06-25 Novartis Ag Lipids and lipid compositions for the delivery of active agents
JP2017523965A (ja) * 2014-07-16 2017-08-24 ノバルティス アーゲー 脂質ナノ粒子ホスト中に核酸を封入する方法
JP2019516351A (ja) * 2016-03-30 2019-06-20 インテリア セラピューティクス，インコーポレイテッド Ｃｒｉｓｐｒ／ｃａｓ構成成分のための脂質ナノ粒子製剤
JP2020500539A (ja) * 2016-12-09 2020-01-16 サンガモ セラピューティクス， インコーポレイテッド 標的特異的ヌクレアーゼの送達
WO2020219876A1 (en) * 2019-04-25 2020-10-29 Intellia Therapeutics, Inc. Ionizable amine lipids and lipid nanoparticles

Family Cites Families (56)

* 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
CA2289702C (en) 1997-05-14 2008-02-19 Inex Pharmaceuticals Corp. 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
US7901708B2 (en) 2002-06-28 2011-03-08 Protiva Biotherapeutics, Inc. Liposomal apparatus and manufacturing methods
DE102004054730A1 (de) 2004-03-28 2006-05-11 Novosom Ag Serumstabile amphotere Liposomen
WO2006007712A1 (en) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
US20070087045A1 (en) 2005-10-14 2007-04-19 Industrial Technology Research Institute Lipid carrier and method of preparing the same
WO2007094683A1 (en) * 2006-02-15 2007-08-23 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.
AU2009238175C1 (en) 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
JP5642673B2 (ja) 2008-07-10 2014-12-17 セリナ・セラピユーテイツクス・インコーポレーテツド 不活性末端基を有するポリオキサゾリン類、保護された開始基から調製されるポリオキサゾリン類及び関連化合物
HUE037082T2 (hu) 2008-11-10 2018-08-28 Arbutus Biopharma Corp Új lipidek és készítmények terápiás hatóanyagok szállítására
ES2613498T3 (es) 2009-07-01 2017-05-24 Protiva Biotherapeutics Inc. Nuevas formulaciones de lípidos para el suministro de agentes terapéuticos a tumores sólidos
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
WO2012000104A1 (en) 2010-06-30 2012-01-05 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
KR102451116B1 (ko) 2011-10-27 2022-10-06 메사추세츠 인스티튜트 오브 테크놀로지 약물 캡슐화 마이크로스피어를 형성할 수 있는, ｎ-말단 상에 관능화된 아미노산 유도체
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
US9687448B2 (en) 2012-12-07 2017-06-27 Alnylam Pharmaceuticals, Inc. Nucleic acid lipid particle formulations
EP3608308B1 (en) 2013-03-08 2021-07-21 Novartis AG Lipids and lipid compositions for the delivery of active agents
EP4019506A1 (en) * 2013-12-19 2022-06-29 Novartis AG Lipids and lipid compositions for the delivery of active agents
PL3086814T3 (pl) 2013-12-23 2020-12-28 Bayer Pharma Aktiengesellschaft Koniugaty środka wiążącego (ADC) z inhibitorami KSP
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
AU2016237148A1 (en) 2015-03-24 2017-11-23 Kyowa Hakko Kirin Co., Ltd. Nucleic acid-containing lipid nanoparticles
US10626393B2 (en) 2015-06-04 2020-04-21 Arbutus Biopharma Corporation 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
EP3319976B1 (en) 2015-07-09 2021-09-01 Insmed Incorporated Compositions and methods for treating lung diseases and lung injury
EP4286012A3 (en) 2015-09-17 2024-05-29 ModernaTX, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
HRP20220652T1 (hr) 2015-12-10 2022-06-24 Modernatx, Inc. Pripravci i postupci unosa terapijskih sredstava
WO2017196969A1 (en) * 2016-05-12 2017-11-16 Z Biotech, Llc Multivalent glycan microarray platform
SG11201808928YA (en) * 2016-06-24 2018-11-29 Eisai R&D Man Co Ltd Cationic lipid
TW201813632A (zh) 2016-09-28 2018-04-16 日商協和醱酵麒麟有限公司 含有核酸之脂質奈米粒子
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
SMT202300097T1 (it) 2017-03-15 2023-05-12 Modernatx Inc Composto e composizioni per il rilascio intracellulare di agenti terapeutici
JP7155239B2 (ja) 2017-04-05 2022-10-18 サイレンス・セラピューティクス・ゲーエムベーハー 産物及び組成物
US12350368B2 (en) 2017-04-14 2025-07-08 The Broad Institute, Inc. Delivery of large payloads
EP3630076A1 (en) * 2017-05-30 2020-04-08 GlaxoSmithKline Biologicals SA Methods for manufacturing a liposome encapsulated rna
EP3662913A4 (en) 2017-08-04 2021-06-30 Kyowa Kirin Co., Ltd. Nucleic-acid-containing lipid nanoparticles
WO2019046809A1 (en) 2017-08-31 2019-03-07 Modernatx, Inc. METHODS OF MANUFACTURING LIPID NANOPARTICLES
PT3688162T (pt) 2017-09-29 2024-04-23 Intellia Therapeutics Inc Formulações
BR112020006256A2 (pt) * 2017-09-29 2020-10-20 Intellia Therapeutics, Inc. método in vitro de administração de mrna usando nanopartículas de lipídio
JP7164547B2 (ja) * 2017-12-27 2022-11-01 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
US12435035B2 (en) 2020-01-09 2025-10-07 Guide Therapeutics, Llc Nanomaterials
KR20230017783A (ko) 2020-04-28 2023-02-06 인텔리아 테라퓨틱스, 인크. 시험관내 세포 전달 방법
IL298363A (en) 2020-05-20 2023-01-01 Flagship Pioneering Innovations Vi Llc Immunogenic compositions and uses thereof
CA3179420A1 (en) 2020-05-20 2021-11-25 Avak Kahvejian Coronavirus antigen compositions and their uses
CA3180101A1 (en) 2020-05-29 2021-12-02 Flagship Pioneering Innovations Vi, Llc Trem compositions and methods relating thereto
CA3182026A1 (en) 2020-05-29 2021-12-02 Flagship Pioneering Innovations Vi, Llc. Trem compositions and methods relating thereto
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

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015095340A1 (en) * 2013-12-19 2015-06-25 Novartis Ag Lipids and lipid compositions for the delivery of active agents
JP2017523965A (ja) * 2014-07-16 2017-08-24 ノバルティス アーゲー 脂質ナノ粒子ホスト中に核酸を封入する方法
JP2019516351A (ja) * 2016-03-30 2019-06-20 インテリア セラピューティクス，インコーポレイテッド Ｃｒｉｓｐｒ／ｃａｓ構成成分のための脂質ナノ粒子製剤
JP2020500539A (ja) * 2016-12-09 2020-01-16 サンガモ セラピューティクス， インコーポレイテッド 標的特異的ヌクレアーゼの送達
WO2020219876A1 (en) * 2019-04-25 2020-10-29 Intellia Therapeutics, Inc. Ionizable amine lipids and lipid nanoparticles

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

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