JPWO2021188389A5 - - Google Patents

Download PDF

Info

Publication number
JPWO2021188389A5
JPWO2021188389A5 JP2022556074A JP2022556074A JPWO2021188389A5 JP WO2021188389 A5 JPWO2021188389 A5 JP WO2021188389A5 JP 2022556074 A JP2022556074 A JP 2022556074A JP 2022556074 A JP2022556074 A JP 2022556074A JP WO2021188389 A5 JPWO2021188389 A5 JP WO2021188389A5
Authority
JP
Japan
Prior art keywords
lipid
alkyl
compound according
alkynyl
alkenyl
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
JP2022556074A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023518944A5 (https=
JP2023518944A (ja
JP7709981B2 (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/022201 external-priority patent/WO2021188389A2/en
Publication of JP2023518944A publication Critical patent/JP2023518944A/ja
Publication of JP2023518944A5 publication Critical patent/JP2023518944A5/ja
Publication of JPWO2021188389A5 publication Critical patent/JPWO2021188389A5/ja
Application granted granted Critical
Publication of JP7709981B2 publication Critical patent/JP7709981B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2022556074A 2020-03-17 2021-03-12 肝星細胞への治療薬の脂質ナノ粒子送達のためのカチオン性脂質 Active JP7709981B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062990939P 2020-03-17 2020-03-17
US62/990,939 2020-03-17
PCT/US2021/022201 WO2021188389A2 (en) 2020-03-17 2021-03-12 Cationic lipids for lipid nanoparticle delivery of therapeutics to hepatic stellate cells

Publications (4)

Publication Number Publication Date
JP2023518944A JP2023518944A (ja) 2023-05-09
JP2023518944A5 JP2023518944A5 (https=) 2024-03-22
JPWO2021188389A5 true JPWO2021188389A5 (https=) 2024-03-22
JP7709981B2 JP7709981B2 (ja) 2025-07-17

Family

ID=77772142

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022556074A Active JP7709981B2 (ja) 2020-03-17 2021-03-12 肝星細胞への治療薬の脂質ナノ粒子送達のためのカチオン性脂質

Country Status (5)

Country Link
US (1) US20230150926A1 (https=)
EP (1) EP4121018A4 (https=)
JP (1) JP7709981B2 (https=)
KR (1) KR20220154740A (https=)
WO (1) WO2021188389A2 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114105799B (zh) * 2022-01-25 2022-05-10 中山大学附属第七医院(深圳) 一种氨基脂质及其制备方法和应用
EP4562154A2 (en) 2022-07-29 2025-06-04 Massachusetts Institute of Technology COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE SIGNAL REGULATORY PROTEIN ALPHA (SIRPalpha) GENE
CN116999397B (zh) * 2023-07-23 2026-01-16 中国人民解放军空军军医大学 双膜融合靶向纳米递药系统的制备方法及其应用
WO2025189064A1 (en) 2024-03-08 2025-09-12 Genzyme Corporation Lipid nanoparticles
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles
WO2026030375A2 (en) 2024-07-30 2026-02-05 Genzyme Corporation Lipid nanoparticles and methods of manufacture and use thereof

Family Cites Families (120)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL154598B (nl) 1970-11-10 1977-09-15 Organon Nv Werkwijze voor het aantonen en bepalen van laagmoleculire verbindingen en van eiwitten die deze verbindingen specifiek kunnen binden, alsmede testverpakking.
US3901654A (en) 1971-06-21 1975-08-26 Biological Developments Receptor assays of biologically active compounds employing biologically specific receptors
US3817827A (en) 1972-03-30 1974-06-18 Scott Paper Co Soft absorbent fibrous webs containing elastomeric bonding material and formed by creping and embossing
US3935074A (en) 1973-12-17 1976-01-27 Syva Company Antibody steric hindrance immunoassay with two antibodies
US3996345A (en) 1974-08-12 1976-12-07 Syva Company Fluorescence quenching with immunological pairs in immunoassays
US4034074A (en) 1974-09-19 1977-07-05 The Board Of Trustees Of Leland Stanford Junior University Universal reagent 2-site immunoradiometric assay using labelled anti (IgG)
US3993754A (en) 1974-10-09 1976-11-23 The United States Of America As Represented By The United States Energy Research And Development Administration Liposome-encapsulated actinomycin for cancer chemotherapy
US3984533A (en) 1975-11-13 1976-10-05 General Electric Company Electrophoretic method of detecting antigen-antibody reaction
US4086257A (en) 1976-10-12 1978-04-25 Sears Barry D Phosphatidyl quaternary ammonium compounds
US4098876A (en) 1976-10-26 1978-07-04 Corning Glass Works Reverse sandwich immunoassay
CH624011A5 (https=) 1977-08-05 1981-07-15 Battelle Memorial Institute
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4452901A (en) 1980-03-20 1984-06-05 Ciba-Geigy Corporation Electrophoretically transferring electropherograms to nitrocellulose sheets for immuno-assays
US4376110A (en) 1980-08-04 1983-03-08 Hybritech, Incorporated Immunometric assays using monoclonal antibodies
US4522803A (en) 1983-02-04 1985-06-11 The Liposome Company, Inc. Stable plurilamellar vesicles, their preparation and use
US4588578A (en) 1983-08-08 1986-05-13 The Liposome Company, Inc. Lipid vesicles prepared in a monophase
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
US5453566A (en) 1986-03-28 1995-09-26 Calgene, Inc. Antisense regulation of gene expression in plant/cells
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
CA1340323C (en) 1988-09-20 1999-01-19 Arnold E. Hampel Rna catalyst for cleaving specific rna sequences
GB8822492D0 (en) 1988-09-24 1988-10-26 Considine J Apparatus for removing tumours from hollow organs of body
US5725871A (en) 1989-08-18 1998-03-10 Danbiosyst Uk Limited Drug delivery compositions comprising lysophosphoglycerolipid
AU637800B2 (en) 1989-08-31 1993-06-10 City Of Hope Chimeric dna-rna catalytic sequences
US5286634A (en) 1989-09-28 1994-02-15 Stadler Joan K Synergistic method for host cell transformation
US5707644A (en) 1989-11-04 1998-01-13 Danbiosyst Uk Limited Small particle compositions for intranasal drug delivery
US6753423B1 (en) 1990-01-11 2004-06-22 Isis Pharmaceuticals, Inc. Compositions and methods for enhanced biostability and altered biodistribution of oligonucleotides in mammals
US6365730B1 (en) 1990-06-19 2002-04-02 Gene Shears Pty. Limited DNA-Armed ribozymes and minizymes
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
JP3257675B2 (ja) 1990-10-12 2002-02-18 マックス−プランク−ゲゼルシャフト ツール フェルデルング デル ビッセンシャフテン エー.ファウ. 修飾リボザイム
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
US6831166B2 (en) 1992-10-23 2004-12-14 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US5756353A (en) 1991-12-17 1998-05-26 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol-and liposome-based delivery
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
EP0642589A4 (en) 1992-05-11 1997-05-21 Ribozyme Pharm Inc METHOD AND REAGENT TO INHIBIT VIRAL REPLICATION.
ES2105262T3 (es) 1992-05-18 1997-10-16 Minnesota Mining & Mfg Dispositivo de suministro de farmaco a traves de las mucosas.
US5792451A (en) 1994-03-02 1998-08-11 Emisphere Technologies, Inc. Oral drug delivery compositions and methods
WO1994002595A1 (en) 1992-07-17 1994-02-03 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of animal diseases
IL108367A0 (en) 1993-01-27 1994-04-12 Hektoen Inst For Medical Resea Antisense polynzcleotide inhibition of human growth factor-sensitive cancer cells
US5426039A (en) 1993-09-08 1995-06-20 Bio-Rad Laboratories, Inc. Direct molecular cloning of primer extended DNA containing an alkane diol
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5591317A (en) 1994-02-16 1997-01-07 Pitts, Jr.; M. Michael Electrostatic device for water treatment
US5631359A (en) 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
US5639725A (en) 1994-04-26 1997-06-17 Children's Hospital Medical Center Corp. Angiostatin protein
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
ATE219660T1 (de) 1994-09-30 2002-07-15 Inex Pharmaceuticals Corp Mittel zum einbringen polyanionischer materialien in zellen
AU3889595A (en) 1994-10-05 1996-05-02 Amgen, Inc. Method for inhibiting smooth muscle cell proliferation and oligonucleotides for use therein
US5783683A (en) 1995-01-10 1998-07-21 Genta Inc. Antisense oligonucleotides which reduce expression of the FGFRI gene
US5580579A (en) 1995-02-15 1996-12-03 Nano Systems L.L.C. Site-specific adhesion within the GI tract using nanoparticles stabilized by high molecular weight, linear poly (ethylene oxide) polymers
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US5747470A (en) 1995-06-07 1998-05-05 Gen-Probe Incorporated Method for inhibiting cellular proliferation using antisense oligonucleotides to gp130 mRNA
ATE285477T1 (de) 1995-06-07 2005-01-15 Inex Pharmaceutical Corp Herstellung von lipid-nukleinsäure partikeln duch ein hydrophobische lipid-nukleinsäuree komplexe zwischenprodukt und zur verwendung in der gentransfer
US5705385A (en) 1995-06-07 1998-01-06 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US7422902B1 (en) 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US20030216335A1 (en) 2001-11-30 2003-11-20 Jennifer Lockridge Method and reagent for the modulation of female reproductive diseases and conditions
US20040142895A1 (en) 1995-10-26 2004-07-22 Sirna Therapeutics, Inc. Nucleic acid-based modulation of gene expression in the vascular endothelial growth factor pathway
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6713069B1 (en) 1996-04-16 2004-03-30 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Compositions and methods for detecting, preventing, and treating African Hemorrhagic Fever
US20050119470A1 (en) 1996-06-06 2005-06-02 Muthiah Manoharan Conjugated oligomeric compounds and their use in gene modulation
US6111085A (en) 1996-09-13 2000-08-29 Isis Pharmaceuticals, Inc. Carbamate-derivatized nucleosides and oligonucleosides
US6174861B1 (en) 1996-10-22 2001-01-16 The Children's Medical Center Corporation Methods of inhibiting angiogenesis via increasing in vivo concentrations of endostatin protein
US5739119A (en) 1996-11-15 1998-04-14 Galli; Rachel L. Antisense oligonucleotides specific for the muscarinic type 2 acetylcholine receptor MRNA
US6406705B1 (en) 1997-03-10 2002-06-18 University Of Iowa Research Foundation Use of nucleic acids containing unmethylated CpG dinucleotide as an adjuvant
CA2289702C (en) 1997-05-14 2008-02-19 Inex Pharmaceuticals Corp. High efficiency encapsulation of charged therapeutic agents in lipid vesicles
US6110745A (en) 1997-07-24 2000-08-29 Inex Pharmaceuticals Corp. Preparation of lipid-nucleic acid particles using a solvent extraction and direct hydration method
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
WO2000062813A2 (en) 1999-04-20 2000-10-26 The University Of British Columbia Cationic peg-lipids and methods of use
US6852334B1 (en) 1999-04-20 2005-02-08 The University Of British Columbia Cationic peg-lipids and methods of use
CZ20021029A3 (cs) 1999-08-27 2002-08-14 Inex Pharmaceuticals Corp. Kompozice a její pouľití pro stimulaci sekrece cytokinů a indukci imunitní odpovědi
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US7189705B2 (en) 2000-04-20 2007-03-13 The University Of British Columbia Methods of enhancing SPLP-mediated transfection using endosomal membrane destabilizers
ATE398175T1 (de) 2000-12-08 2008-07-15 Coley Pharmaceuticals Gmbh Cpg-artige nukleinsäuren und verfahren zu ihrer verwendung
US7053150B2 (en) 2000-12-18 2006-05-30 Nektar Therapeutics Al, Corporation Segmented polymers and their conjugates
TW593427B (en) 2000-12-18 2004-06-21 Nektar Therapeutics Al Corp Synthesis of high molecular weight non-peptidic polymer derivatives
US20030077829A1 (en) 2001-04-30 2003-04-24 Protiva Biotherapeutics Inc.. Lipid-based formulations
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US20050282188A1 (en) 2001-05-18 2005-12-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
US20030130186A1 (en) 2001-07-20 2003-07-10 Chandra Vargeese Conjugates and compositions for cellular delivery
DE10163098B4 (de) 2001-10-12 2005-06-02 Alnylam Europe Ag Verfahren zur Hemmung der Replikation von Viren
AU2003207708A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
WO2003070283A2 (en) 2002-02-22 2003-08-28 Klaus Strebhardt Agent for inhibiting development or progress of proliferative diseases and especially cancer diseases and pharmaceutical composition containing said agent
US7901708B2 (en) 2002-06-28 2011-03-08 Protiva Biotherapeutics, Inc. Liposomal apparatus and manufacturing methods
WO2004011647A1 (en) 2002-07-26 2004-02-05 Chiron Corporation Modified small interfering rna molecules and methods of use
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
US20040167090A1 (en) 2003-02-21 2004-08-26 Monahan Sean D. Covalent modification of RNA for in vitro and in vivo delivery
AU2004227414A1 (en) 2003-04-03 2004-10-21 Alnylam Pharmaceuticals iRNA conjugates
DK1620544T3 (en) 2003-04-17 2019-01-14 Alnylam Pharmaceuticals Inc MODIFIED iRNA AGENTS
ES2559828T3 (es) 2003-07-16 2016-02-16 Protiva Biotherapeutics Inc. ARN de interferencia encapsulado en lípidos
US7514574B2 (en) * 2003-08-07 2009-04-07 Ooi Wong Biodegradable penetration enhancers with multiple hydrophilic moieties
NZ592917A (en) 2003-09-15 2012-12-21 Protiva Biotherapeutics Inc Stable polyethyleneglycol (PEG) dialkyloxypropyl (DAA) lipid conjugates
US7745651B2 (en) 2004-06-07 2010-06-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
US7799565B2 (en) 2004-06-07 2010-09-21 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering RNA
JP2008512500A (ja) 2004-09-10 2008-04-24 ソマジェニックス インコーポレーティッド ウィルス遺伝子発現を効率的に阻害する低分子干渉rnaおよびその使用方法
CA2587411A1 (en) 2004-11-17 2006-05-26 Protiva Biotherapeutics, Inc. Sirna silencing of apolipoprotein b
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
US9005654B2 (en) 2005-07-27 2015-04-14 Protiva Biotherapeutics, Inc. Systems and methods for manufacturing liposomes
WO2007048046A2 (en) 2005-10-20 2007-04-26 Protiva Biotherapeutics, Inc. Sirna silencing of filovirus gene expression
US8101741B2 (en) 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
WO2007056861A1 (en) 2005-11-18 2007-05-24 Protiva Biotherapeutics, Inc. Sirna silencing of influenza virus gene expression
US7871967B2 (en) * 2007-02-12 2011-01-18 Chevron U.S.A. Inc. Diester-based lubricants and methods of making same
HUE037082T2 (hu) * 2008-11-10 2018-08-28 Arbutus Biopharma Corp Új lipidek és készítmények terápiás hatóanyagok szállítására
US20130085091A1 (en) * 2011-09-29 2013-04-04 Chevron U.S.A. Inc. Industrial oil comprising a bio-derived ester
EP2788316B1 (en) * 2011-12-07 2019-04-24 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents
SG11201405157PA (en) * 2012-02-24 2014-10-30 Protiva Biotherapeutics Inc Trialkyl cationic lipids and methods of use thereof
AU2013243948A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
US9603800B2 (en) * 2012-04-12 2017-03-28 Yale University Methods of treating inflammatory and autoimmune diseases and disorders using nanolipogels
US9744103B1 (en) 2013-03-08 2017-08-29 Chad Ricker Infant teething apparatus
WO2015011633A1 (en) 2013-07-23 2015-01-29 Protiva Biotherapeutics, Inc. Compositions and methods for delivering messenger rna
ES2895651T3 (es) * 2013-12-19 2022-02-22 Novartis Ag Lípidos y composiciones lipídicas para la administración de agentes activos
US11219634B2 (en) * 2015-01-21 2022-01-11 Genevant Sciences Gmbh Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells
US10626393B2 (en) 2015-06-04 2020-04-21 Arbutus Biopharma Corporation Delivering CRISPR therapeutics with lipid nanoparticles
US20180245074A1 (en) 2015-06-04 2018-08-30 Protiva Biotherapeutics, Inc. Treating hepatitis b virus infection using crispr
CN109563511A (zh) 2016-06-30 2019-04-02 阿布特斯生物制药公司 用于递送信使rna的组合物和方法
US12077501B2 (en) * 2017-06-14 2024-09-03 Modernatx, Inc. Compounds and compositions for intracellular delivery of agents
EP3764816B1 (en) * 2018-03-16 2026-01-28 Dow Global Technologies Llc Foam control in food

Similar Documents

Publication Publication Date Title
CN101291653B (zh) 脂质包封的干扰rna
US20230398082A1 (en) Mrna delivery composition
JP2023071950A (ja) Rna送達のためのイオン化可能なカチオン性脂質
AU2013256008B2 (en) Lipid-based drug carriers for rapid penetration through mucus linings
JP2012509258A (ja) 核酸送達系のための分岐カチオン性脂質
JP7795461B2 (ja) ポリ乳酸塩を含む薬物送達用ナノ粒子組成物製造用キット
JP2024500516A (ja) マンノースを含む脂質ナノ粒子またはその用途
JP7828458B2 (ja) 生分解性エステル結合を含むイオン化可能な脂質およびこれを含む脂質ナノ粒子
JP2004508012A (ja) エンドソーム膜不安定剤を用いたsplp媒介性トランスフェクションの強化方法
JP2025501905A (ja) イオン化脂質、脂質ナノ粒子、及びこれらの使用
JP2025512240A (ja) 硫化物を含むイオン化可能な脂質、及びこれを含む脂質ナノ粒子
KR102911928B1 (ko) 신규 이온화 가능한 지질 및 이를 포함하는 지질나노입자
JPWO2021188389A5 (https=)
CN120835876A (zh) 包括具有支化结构的电离脂质的脂质纳米粒制剂及其用途
KR20240083166A (ko) 폐를 표적장기로 하는 생분해성 지질나노입자 약물 전달 제형 및 이의 활용
WO2024096516A1 (ko) 핵산 약물의 경폐전달을 위한 지질나노입자 제형 및 이의 활용
JPWO2021163339A5 (https=)
US20250057766A1 (en) Lipids for drug delivery, nanoparticles comprising same, and composition for drug delivery comprising nanoparticles
WO2010067617A1 (ja) 脂質膜構造体
KR20240109177A (ko) 가지 친 구조의 이온화 지질을 포함한 지질 나노입자 제형 및 이의 용도
TW201927287A (zh) 藉由具有蛋白質表現於其表面之囊泡遞送藥劑至自噬及凋亡細胞
JP2026505365A (ja) Rnaに対する自然免疫応答を低減するための方法及び修飾
CN119424366A (zh) 靶向次级淋巴器官的脂质纳米颗粒及其制备方法和用途
JP2023510253A (ja) アルキル化ヌクレオシド、ならびにそれらの組成物および核酸送達のための方法
CN121969393A (zh) 稳定性增强型脂质纳米颗粒递送系统