JP7555123B2 - 有機溶媒不含かつ劣化剤不含のトランスフェクション・コンピテント・ベシクルを含む組成物及びシステム並びにそれらに関連する方法 - Google Patents

有機溶媒不含かつ劣化剤不含のトランスフェクション・コンピテント・ベシクルを含む組成物及びシステム並びにそれらに関連する方法 Download PDF

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JP7555123B2
JP7555123B2 JP2021517982A JP2021517982A JP7555123B2 JP 7555123 B2 JP7555123 B2 JP 7555123B2 JP 2021517982 A JP2021517982 A JP 2021517982A JP 2021517982 A JP2021517982 A JP 2021517982A JP 7555123 B2 JP7555123 B2 JP 7555123B2
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リービット，ブレア
カリス，ピーター
ペトカウ，テリー
ヒル，オースティン
ワグナー，パメラ
クルカーニ，ジェイッシュ
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ザ ユニヴァーシティ オブ ブリティッシュ コロンビア
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JP2021517982A 2018-10-09 2019-10-09 有機溶媒不含かつ劣化剤不含のトランスフェクション・コンピテント・ベシクルを含む組成物及びシステム並びにそれらに関連する方法 Active JP7555123B2 (ja)

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JP2023204608A JP7840578B2 (ja) 2018-10-09 2023-12-04 有機溶媒不含かつ劣化剤不含のトランスフェクション・コンピテント・ベシクルを含む組成物及びシステム並びにそれらに関連する方法
JP2025099115A JP2025143306A (ja) 2018-10-09 2025-06-13 有機溶媒不含かつ劣化剤不含のトランスフェクション・コンピテント・ベシクルを含む組成物及びシステム並びにそれらに関連する方法

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US201862743116P 2018-10-09 2018-10-09
US62/743,116 2018-10-09
PCT/US2019/055472 WO2020077007A1 (en) 2018-10-09 2019-10-09 Compositions and systems comprising transfection-competent vesicles free of organic-solvents and detergents and methods related thereto

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JP2023204608A Active JP7840578B2 (ja) 2018-10-09 2023-12-04 有機溶媒不含かつ劣化剤不含のトランスフェクション・コンピテント・ベシクルを含む組成物及びシステム並びにそれらに関連する方法
JP2025099115A Pending JP2025143306A (ja) 2018-10-09 2025-06-13 有機溶媒不含かつ劣化剤不含のトランスフェクション・コンピテント・ベシクルを含む組成物及びシステム並びにそれらに関連する方法

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JP2025099115A Pending JP2025143306A (ja) 2018-10-09 2025-06-13 有機溶媒不含かつ劣化剤不含のトランスフェクション・コンピテント・ベシクルを含む組成物及びシステム並びにそれらに関連する方法

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MA49421A (fr) 2017-06-15 2020-04-22 Modernatx Inc Formulations d'arn
EP3675817A1 (en) 2017-08-31 2020-07-08 Modernatx, Inc. Methods of making lipid nanoparticles
WO2020061457A1 (en) 2018-09-20 2020-03-26 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
HRP20240586T1 (hr) 2018-10-09 2024-07-19 The University Of British Columbia Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima
CN118697900A (zh) 2019-01-31 2024-09-27 摩登纳特斯有限公司 制备脂质纳米颗粒的方法
JP2023526284A (ja) * 2020-05-15 2023-06-21 トランスレイト バイオ， インコーポレイテッド ｍＲＮＡ送達のための脂質ナノ粒子製剤
EP4192432A1 (en) * 2020-08-06 2023-06-14 ModernaTX, Inc. Methods of preparing lipid nanoparticles
CZ310613B6 (cs) 2020-09-23 2026-01-28 Ústav organické chemie a biochemie AV ČR, v. v. i. Lipidoidy pro transfekci nukleových kyselin a jejich použití
WO2022115645A1 (en) 2020-11-25 2022-06-02 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids, and related methods of use
TW202237068A (zh) * 2020-12-09 2022-10-01 美商建南德克公司 製備脂質奈米顆粒的高通量方法及其用途
CZ310443B6 (cs) 2021-07-19 2025-06-25 Ústav organické chemie a biochemie AV ČR, v. v. i. Cyklohexanové lipidoidy pro transfekci nukleových kyselin a jejich použití
WO2023009421A1 (en) * 2021-07-26 2023-02-02 Modernatx, Inc. Processes for preparing lipid nanoparticle compositions
US12589073B2 (en) * 2021-11-11 2026-03-31 Northwestern University Leveraging lipid-protein interactions to engineer spatial organization in cell-free systems
US20250235559A1 (en) * 2022-02-25 2025-07-24 Incisive Genetics Inc. Gene editing reporter system and guide rna and composition related thereto; composition and method for knocking out dna with more than two grnas; gene editing in the eye; and gene editing using base editors
CA3256897A1 (en) 2022-05-25 2023-11-30 Akagera Medicines, Inc. Lipid nanoparticles for the administration of nucleic acids and their methods of use
US20240000966A1 (en) * 2022-06-30 2024-01-04 Life Technologies Corporation Lipid Compositions For In Vivo Delivery
WO2025184543A1 (en) * 2024-03-01 2025-09-04 Arizona Board Of Regents On Behalf Of The University Of Arizona Compositions and methods for delivery of therapeutic compounds

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2009513151A (ja) 2005-11-02 2009-04-02 プロチバ バイオセラピューティクス インコーポレイティッド 修飾ｓｉＲＮＡ分子およびその使用法
JP2011507534A (ja) 2007-12-27 2011-03-10 プロチバ バイオセラピューティクス インコーポレイティッド 干渉ｒｎａを使用したポロ様キナーゼ発現のサイレンシング方法
JP2014523411A (ja) 2011-06-08 2014-09-11 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
JP2016528890A (ja) 2013-07-09 2016-09-23 プレジデント アンド フェローズ オブ ハーバード カレッジ ＣＲＩＳＰＲ／Ｃａｓ系を用いるゲノム編集の治療用の使用
JP2016539916A (ja) 2013-10-22 2016-12-22 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のＣＮＳ送達及びその使用方法
JP2018509387A (ja) 2015-01-21 2018-04-05 フェーズアールエックス インコーポレイテッド 細胞に治療および診断剤を送達するための方法、組成物、ならびにシステム
JP2018511588A (ja) 2015-03-19 2018-04-26 トランスレイト バイオ， インコーポレイテッド ポンペ病のｍＲＮＡ治療

Family Cites Families (237)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5552155A (en) * 1992-12-04 1996-09-03 The Liposome Company, Inc. Fusogenic lipsomes and methods for making and using same
US5776486A (en) 1993-05-28 1998-07-07 Aphios Corporation Methods and apparatus for making liposomes containing hydrophobic drugs
US5981501A (en) * 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
CA2227989A1 (en) 1995-08-01 1997-02-13 Karen Ophelia Hamilton Liposomal oligonucleotide compositions
US6093816A (en) 1996-06-27 2000-07-25 Isis Pharmaceuticals, Inc. Cationic lipids
US7262173B2 (en) 1997-03-21 2007-08-28 Georgetown University Chemosensitizing with liposomes containing oligonucleotides
AU733310C (en) 1997-05-14 2001-11-29 University Of British Columbia, The 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
US6410328B1 (en) 1998-02-03 2002-06-25 Protiva Biotherapeutics Inc. Sensitizing cells to compounds using lipid-mediated gene and compound delivery
US6693086B1 (en) 1998-06-25 2004-02-17 National Jewish Medical And Research Center Systemic immune activation method using nucleic acid-lipid complexes
US20030022854A1 (en) 1998-06-25 2003-01-30 Dow Steven W. Vaccines using nucleic acid-lipid complexes
WO2000030444A1 (en) 1998-11-25 2000-06-02 Vanderbilt University Cationic liposomes for gene transfer
AU5905400A (en) 1999-07-14 2001-02-05 Board Of Regents, The University Of Texas System Methods and compositions for delivery and retention of active agents to lymph nodes
US7094423B1 (en) 1999-07-15 2006-08-22 Inex Pharmaceuticals Corp. Methods for preparation of lipid-encapsulated therapeutic agents
JP4554883B2 (ja) 2000-10-04 2010-09-29 協和発酵キリン株式会社 脂質膜による微粒子の被覆方法
US7713942B2 (en) 2001-04-04 2010-05-11 Nordic Vaccine Technology A/S Cage-like microparticle complexes comprising sterols and saponins for delivery of polynucleotides
WO2002088370A2 (en) 2001-04-30 2002-11-07 Protiva Biotherapeutics Inc. Autogene nucleic acids encoding a secretable rna polymerase
WO2002087541A1 (en) 2001-04-30 2002-11-07 Protiva Biotherapeutics Inc. Lipid-based formulations for gene transfer
EP1441763A2 (en) 2001-11-07 2004-08-04 Inex Pharmaceuticals Corp. Mucosal adjuvants comprising an oligonucleotide and a cationic lipid
EP1519714B1 (en) 2002-06-28 2010-10-20 Protiva Biotherapeutics Inc. Method and apparatus for producing liposomes
CA2532228C (en) 2003-07-16 2017-02-14 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
EP1664316B1 (en) * 2003-09-15 2012-08-29 Protiva Biotherapeutics Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
US20050266067A1 (en) 2004-03-02 2005-12-01 Shiladitya Sengupta Nanocell drug delivery system
JP4764426B2 (ja) 2004-06-07 2011-09-07 プロチバ バイオセラピューティクス インコーポレイティッド カチオン性脂質および使用方法
CA2569664C (en) 2004-06-07 2013-07-16 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
EP1773857A4 (en) 2004-07-02 2009-05-13 Protiva Biotherapeutics Inc THE IMMUNE SYSTEM STIMULATING SIRNA MOLECULES AND APPLICATIONS THEREOF
US20060051405A1 (en) 2004-07-19 2006-03-09 Protiva Biotherapeutics, Inc. Compositions for the delivery of therapeutic agents and uses thereof
CA2587411A1 (en) 2004-11-17 2006-05-26 Protiva Biotherapeutics, Inc. Sirna silencing of apolipoprotein b
JP2006248978A (ja) 2005-03-10 2006-09-21 Mebiopharm Co Ltd 新規なリポソーム製剤
CA2605068A1 (en) 2005-04-15 2006-10-26 The Board Of Regents Of The University Of Texas System Delivery of sirna by neutral lipid compositions
US20080088046A1 (en) 2006-10-13 2008-04-17 Steffen Panzner Amphoteric liposomes, a method of formulating an amphoteric liposome and a method of loading an amphoteric liposome
US20070087045A1 (en) 2005-10-14 2007-04-19 Industrial Technology Research Institute Lipid carrier and method of preparing the same
WO2007048046A2 (en) 2005-10-20 2007-04-26 Protiva Biotherapeutics, Inc. Sirna silencing of filovirus gene expression
US20070218122A1 (en) 2005-11-18 2007-09-20 Protiva Biotherapeutics, Inc. siRNA silencing of influenza virus gene expression
US8598333B2 (en) 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
US20080171716A1 (en) 2006-08-16 2008-07-17 Protiva Biotherapeutics, Inc. Nucleic acid modulation of toll-like receptor-mediated immune stimulation
JP5484059B2 (ja) 2006-10-06 2014-05-07 エスシーアイエル テクノロジー ゲゼルシャフト ミット ベシュレンクテル ハフツング 脊髄核インプラント
EP2217221B1 (en) 2007-10-17 2018-06-27 Korea Advanced Institute of Science and Technology Ldl-like cationic nanoparticles for deliverying nucleic acid gene, method for preparing thereof and method for deliverying nucleic acid gene using the same
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
US8268796B2 (en) 2008-06-27 2012-09-18 Children's Hospital & Research Center At Oakland Lipophilic nucleic acid delivery vehicle and methods of use thereof
TW201004658A (en) 2008-07-31 2010-02-01 Enzon Pharmaceuticals Inc Nanoparticle compositions for nucleic acids delivery system
CA2740000C (en) 2008-10-09 2017-12-12 Tekmira Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
MX360460B (es) 2008-10-20 2018-11-05 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de transtiretina.
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
TW201021853A (en) 2008-11-17 2010-06-16 Enzon Pharmaceuticals Inc Releasable cationic lipids for nucleic acids delivery systems
US20110305769A1 (en) 2008-11-17 2011-12-15 Enzon Pharmaceuticals, Inc. Branched cationic lipids for nucleic acids delivery system
US10292932B2 (en) 2008-12-26 2019-05-21 Samyang Biopharmaceuticals Corporation Polymeric micelle particle comprising anionic drugs and method of preparing the same
WO2010108934A1 (en) 2009-03-25 2010-09-30 Novartis Ag Pharmaceutical composition containing a drug and sirna
SG10201911942UA (en) 2009-05-05 2020-02-27 Muthiah Manoharan Lipid compositions
KR101766408B1 (ko) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
WO2011000107A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
AP2015008874A0 (en) * 2009-08-14 2015-11-30 Alnylam Pharmaceuticals Inc Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
NZ714887A (en) 2009-09-11 2019-07-26 Ionis Pharmaceuticals Inc Modulation of huntingtin expression
CN107028886A (zh) 2009-11-04 2017-08-11 不列颠哥伦比亚大学 含有核酸的脂质粒子及相关的方法
EP3318248B1 (en) 2009-12-01 2019-04-10 Translate Bio, Inc. Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
EP2525778B1 (en) 2010-01-19 2018-08-01 Polypid Ltd. Sustained-release nucleic acid matrix compositions
WO2011141704A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
KR101198715B1 (ko) 2010-05-14 2012-11-13 한국생명공학연구원 핵산 및 친수성 음이온 화합물의 고효율 포획을 위한 비대칭 리포솜 및 이의 제조방법
US20100239521A1 (en) 2010-06-01 2010-09-23 Mohammad Reza Mozafari Method for the preparation of micro- and nano-sized carrier systems for the encapsulation of bioactive substances
KR20190039347A (ko) 2010-06-03 2019-04-10 알닐람 파마슈티칼스 인코포레이티드 활성제의 전달을 위한 생분해성 지질
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
SI4043040T1 (sl) 2010-08-31 2023-04-28 Glaxosmithkline Biologicals Sa Mali liposomi za dostavo imunogen-kodirajoče RNA
US8466122B2 (en) 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
US9011903B2 (en) 2011-06-08 2015-04-21 Nitto Denko Corporation Cationic lipids for therapeutic agent delivery formulations
ES2795110T3 (es) 2011-06-08 2020-11-20 Translate Bio Inc Lípidos escindibles
EP2729127B1 (en) * 2011-07-04 2018-05-02 Statens Serum Institut Methods for producing liposomes
MX366055B (es) 2011-08-31 2019-06-26 Novartis Ag Liposomas pegilados para admistracion de acido ribonucleico (arn) que codifica para inmunogeno.
US9126966B2 (en) 2011-08-31 2015-09-08 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use thereof
JP2014530602A (ja) 2011-10-05 2014-11-20 プロティバ バイオセラピューティクス インコーポレイテッド アルデヒドデヒドロゲナーゼをサイレンシングするための組成物および方法
US9579338B2 (en) 2011-11-04 2017-02-28 Nitto Denko Corporation Method of producing lipid nanoparticles for drug delivery
EP3485875A1 (en) 2011-11-04 2019-05-22 Nitto Denko Corporation Single use system for sterelily producing lipid-nucleic acid particles
CA3018046A1 (en) 2011-12-16 2013-06-20 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
EP2817287B1 (en) 2012-02-24 2018-10-03 Arbutus Biopharma Corporation Trialkyl cationic lipids and methods of use thereof
WO2013130824A1 (en) 2012-02-29 2013-09-06 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
BR112014024131A2 (pt) 2012-03-29 2017-07-25 Shire Human Genetic Therapies lipídios catiônicos ionizáveis
WO2013151665A2 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
US20160289674A1 (en) 2012-04-02 2016-10-06 Moderna Therapeutics, Inc. Modified polynucleotides for the production of membrane proteins
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
EP2858679B2 (en) 2012-06-08 2024-06-05 Translate Bio, Inc. Pulmonary delivery of mrna to non-lung target cells
HRP20181855T1 (hr) 2012-06-08 2018-12-28 Nitto Denko Corporation Lipidi za formulacije namijenjene unosu terapijskog sredstva
BR112015022855A2 (pt) 2013-03-14 2017-11-07 Shire Human Genetic Therapies composições e método para produção de um anticorpo in vitro
EP3750903A1 (en) 2013-03-14 2020-12-16 Translate Bio, Inc. Ribonucleic acids with 4'-thio-modified nucleotides and related methods
EP3388834B1 (en) 2013-03-15 2020-04-15 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
CN105143456A (zh) 2013-03-15 2015-12-09 不列颠哥伦比亚大学 用于转染的脂质纳米粒子和相关方法
KR102109188B1 (ko) 2013-04-01 2020-05-11 삼성전자주식회사 양이온성 지질을 포함하는 온도민감성 리포좀 및 그의 용도
JP6620093B2 (ja) 2013-07-23 2019-12-11 アービュートゥス バイオファーマ コーポレイションＡｒｂｕｔｕｓ Ｂｉｏｐｈａｒｍａ Ｃｏｒｐｏｒａｔｉｏｎ メッセンジャーｒｎａを送達するための組成物及び方法
WO2015021465A2 (en) 2013-08-09 2015-02-12 New York University Protein engineered systems for delivery of molecules
ES3032935T3 (en) 2013-10-22 2025-07-29 Translate Bio Inc Lipid formulations for delivery of messenger rna
CN106413811A (zh) 2013-10-22 2017-02-15 夏尔人类遗传性治疗公司 精氨基琥珀酸合成酶缺乏症的mrna疗法
JP6506749B2 (ja) 2013-10-22 2019-04-24 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 フェニルケトン尿症のためのｍＲＮＡ療法
KR102396026B1 (ko) 2014-01-21 2022-05-09 안자리움 바이오사이언시스 아게 하이브리도좀, 이를 포함하는 조성물, 이의 제조 방법 및 이의 용도
ES2745769T3 (es) 2014-03-10 2020-03-03 Editas Medicine Inc Procedimientos y composiciones relacionados con CRISPR/CAS para tratar la amaurosis congénita de Leber 10 (LCA10)
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
PL4023249T3 (pl) * 2014-04-23 2025-03-10 Modernatx, Inc. Szczepionki z kwasem nukleinowym
JP6557722B2 (ja) 2014-05-30 2019-08-07 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 核酸の送達のための生分解性脂質
EP3155116A4 (en) 2014-06-10 2017-12-27 Massachusetts Institute Of Technology Method for gene editing
KR102559979B1 (ko) 2014-06-24 2023-07-25 샤이어 휴먼 지네틱 테라피즈 인크. 핵산의 전달용 입체화학적으로 풍부한 조성물
EP3160448B1 (en) 2014-06-26 2025-08-06 Ramot at Tel-Aviv University Ltd. Liposomal formulations for delivery of nucleic acids
BR112016030852A2 (pt) 2014-07-02 2018-01-16 Shire Human Genetic Therapies encapsulação de rna mensageiro
JP6767976B2 (ja) 2014-12-05 2020-10-14 トランスレイト バイオ， インコーポレイテッド 関節疾患の治療のためのメッセンジャーｒｎａ治療法
EP3247398A4 (en) 2015-01-23 2018-09-26 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
EP4667492A3 (en) 2015-05-26 2026-04-15 Ramot at Tel-Aviv University Ltd. Targeted lipid particles for systemic delivery of nucleic acid molecules to leukocytes
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
AU2016341309A1 (en) 2015-10-22 2018-06-07 Modernatx, Inc. Cancer vaccines
WO2017070624A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Tropical disease vaccines
AU2016342045A1 (en) 2015-10-22 2018-06-07 Modernatx, Inc. Human cytomegalovirus vaccine
EP3368089B1 (en) 2015-10-26 2025-11-05 Translate Bio Ma, Inc. Nanoparticle formulations for delivery of nucleic acid complexes
PL3386484T3 (pl) 2015-12-10 2022-07-25 Modernatx, Inc. Kompozycje i sposoby dostarczania środków terapeutycznych
PT3391875T (pt) 2015-12-18 2021-12-20 Samyang Holdings Corp Método para preparar micelas poliméricas contendo fármaco aniónico
LT3394030T (lt) 2015-12-22 2022-04-11 Modernatx, Inc. Junginiai ir kompozicijos terapinei medžiagai teikti intraceliuliniu būdu
CN107281103A (zh) 2016-04-11 2017-10-24 百奥迈科生物技术有限公司 具有高含量阳性脂质化合物的脂质体制剂及其应用
KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
US20190298657A1 (en) 2016-05-18 2019-10-03 Modernatx, Inc. Polynucleotides Encoding Acyl-CoA Dehydrogenase, Very Long-Chain for the Treatment of Very Long-Chain Acyl-CoA Dehydrogenase Deficiency
EP3469074B1 (en) 2016-06-13 2020-12-09 Translate Bio, Inc. Messenger rna therapy for the treatment of ornithine transcarbamylase deficiency
WO2017218704A1 (en) 2016-06-14 2017-12-21 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
WO2018064755A1 (en) 2016-10-03 2018-04-12 Precision Nanosystems Inc. Compositions for transfecting resistant cell types
EP3528821A4 (en) 2016-10-21 2020-07-01 ModernaTX, Inc. HUMAN CYTOMEGALOVIRUS VACCINE
CA3040337A1 (en) 2016-10-26 2018-05-03 Curevac Ag Lipid nanoparticle mrna vaccines
US11583504B2 (en) 2016-11-08 2023-02-21 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
AU2017356699B2 (en) 2016-11-08 2021-11-11 Ramot At Tel-Aviv University Ltd. Cationic lipids for nucleic acid delivery and preparation thereof
AU2017357758B2 (en) 2016-11-10 2023-11-16 Translate Bio, Inc. Improved process of preparing mRNA-loaded lipid nanoparticles
CA3043033A1 (en) 2016-11-10 2018-05-17 Translate Bio, Inc. Improved ice-based lipid nanoparticle formulation for delivery of mrna
AU2017374042C1 (en) 2016-12-09 2024-07-11 Acuitas Therapeutics, Inc. Delivery of target specific nucleases
US10383952B2 (en) 2016-12-21 2019-08-20 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
CA3055653A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Lipid nanoparticle formulation
EP4186888B1 (en) 2017-03-15 2025-11-26 ModernaTX, Inc. Compound and compositions for intracellular delivery of therapeutic agents
WO2018213726A1 (en) * 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
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
US20200131498A1 (en) 2017-06-14 2020-04-30 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase
WO2018232120A1 (en) 2017-06-14 2018-12-20 Modernatx, Inc. Compounds and compositions for intracellular delivery of agents
MA49421A (fr) 2017-06-15 2020-04-22 Modernatx Inc Formulations d'arn
EP3641834B1 (en) 2017-06-19 2023-10-04 Translate Bio, Inc. Messenger rna therapy for the treatment of friedreich's ataxia
KR20200036873A (ko) 2017-07-14 2020-04-07 온코루스, 인크. 캡슐화된 폴리뉴클레오티드 및 사용 방법
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
CA3073020A1 (en) 2017-08-16 2019-02-21 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
EP4501322A3 (en) 2017-08-17 2025-04-16 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036028A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICULAR FORMULATIONS
EP3675817A1 (en) 2017-08-31 2020-07-08 Modernatx, Inc. Methods of making lipid nanoparticles
WO2019089828A1 (en) 2017-10-31 2019-05-09 Acuitas Therapeutics, Inc. Lamellar lipid nanoparticles
AU2017438994A1 (en) 2017-11-09 2020-05-28 Immunovaccine Technologies Inc. Pharmaceutical compositions, methods for preparation comprising sizing of lipid vesicle particles, and uses thereof
US12036282B2 (en) 2017-12-06 2024-07-16 Newsouth Innovations Pty Limited Liposomal system for drug delivery
US11951210B2 (en) 2017-12-12 2024-04-09 The Johns Hopkins Universty Methods for making giant vesicles and their use
US11167043B2 (en) 2017-12-20 2021-11-09 Translate Bio, Inc. Composition and methods for treatment of ornithine transcarbamylase deficiency
US20210024928A1 (en) 2018-02-16 2021-01-28 Mina Therapeutics Limited C/ebp alpha sarna compositions and methods of use
US20210002640A1 (en) 2018-02-28 2021-01-07 Exicure, Inc. Liposomal spherical nucleic acid (sna) constructs for survival of motor neuron (sma) inhibitors
WO2019191341A1 (en) 2018-03-27 2019-10-03 Factor Bioscience Inc. Nucleic acid-based therapeutics
WO2019191780A1 (en) 2018-03-30 2019-10-03 Arcturus Therapeutics, Inc. Lipid particles for nucleic acid delivery
AU2019254591B2 (en) 2018-04-17 2025-12-11 CureVac SE Novel RSV RNA molecules and compositions for vaccination
US11679159B2 (en) 2018-04-29 2023-06-20 Precision NanoSystems ULC Compositions for transfecting resistant cell types
CN112055695A (zh) 2018-05-01 2020-12-08 弗莱德哈钦森癌症研究中心 用于基因表达的纳米粒子和其用途
US20210189397A1 (en) 2018-05-10 2021-06-24 Northwestern University Self-manageable abnormal scar treatment with spherical nucleic acid (sna) technology
WO2019226650A1 (en) 2018-05-23 2019-11-28 Modernatx, Inc. Delivery of dna
JP7488193B2 (ja) 2018-05-24 2024-05-21 トランスレイト バイオ， インコーポレイテッド チオエステルカチオン性脂質
EP3823634A4 (en) 2018-07-21 2022-08-03 University of Tennessee Research Foundation NEUTRAL LIPOSOMES CONTAINING BIOLOGICAL ACTIVE INGREDIENTS
JP7422977B2 (ja) 2018-07-23 2024-01-29 トランスレイト バイオ， インコーポレイテッド メッセンジャーｒｎａ用乾燥粉末製剤
WO2020028133A1 (en) 2018-07-30 2020-02-06 Trucode Gene Repair, Inc. Lipid nanoparticle formulations comprising nucleic acid mimics
EP3846857A4 (en) * 2018-09-04 2022-10-12 The Board Of Regents Of The University Of Texas System COMPOSITIONS AND METHODS FOR ORGAN-SPECIFIC DELIVERY OF NUCLEIC ACIDS
WO2020051243A1 (en) 2018-09-04 2020-03-12 Ohio State Innovation Foundation Lipid nanoparticles and methods of using thereof
EP3849617A1 (en) 2018-09-14 2021-07-21 Translate Bio, Inc. Composition and methods for treatment of methylmalonic acidemia
WO2020061367A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
WO2020061457A1 (en) 2018-09-20 2020-03-26 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
HRP20240586T1 (hr) 2018-10-09 2024-07-19 The University Of British Columbia Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima
EP3876914A2 (en) 2018-11-09 2021-09-15 Translate Bio, Inc. Messenger rna therapy for treatment of ocular diseases
AU2019376006A1 (en) 2018-11-09 2021-06-03 Arbutus Biopharma Corporation Cationic lipids containing silicon
EP3876999A4 (en) 2018-11-09 2022-08-31 Arbutus Biopharma Corporation NEGATIVELY CHARGED PEG-LIPID CONJUGATES
CA3118559A1 (en) 2018-11-09 2020-05-14 Arbutus Biopharma Corporation Lipid nanoparticle formulations
AU2019374871B2 (en) 2018-11-09 2025-09-11 Translate Bio, Inc. PEG lipidoid compounds
CA3119449A1 (en) 2018-11-12 2020-05-22 Translate Bio, Inc. Methods for inducing immune tolerance
JP7667077B2 (ja) 2018-11-16 2025-04-22 日東電工株式会社 Ｒｎａ干渉送達製剤及び悪性腫瘍のための方法
WO2020106946A1 (en) 2018-11-21 2020-05-28 Translate Bio, Inc. TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF NEBULIZED mRNA ENCODING CFTR
JP2022516318A (ja) 2019-01-04 2022-02-25 オンコラス， インコーポレイテッド カプセル化ポリヌクレオチド及び使用方法
US12201724B2 (en) 2019-01-24 2025-01-21 CannaClear Inc. Lecithin vesicles for oral delivery
US12502354B2 (en) 2019-01-24 2025-12-23 Northwestern University High density lipoprotein mimetic nanoparticles using lipid conjugated core scaffolds
CN118697900A (zh) 2019-01-31 2024-09-27 摩登纳特斯有限公司 制备脂质纳米颗粒的方法
AU2020213609B2 (en) 2019-01-31 2025-08-07 Newsouth Innovations Pty Limited Liposomal nanoparticle
SG11202110036RA (en) 2019-03-19 2021-10-28 Arcturus Therapeutics Inc Method of making lipid-encapsulated rna nanoparticles
JPWO2020189773A1 (https=) 2019-03-20 2020-09-24
KR102732019B1 (ko) 2019-04-15 2024-11-20 글로벌 라이프 사이언시즈 솔루션즈 캐나다 유엘씨 T 세포 유전자 발현의 비바이러스 변형
AU2020268798A1 (en) 2019-05-03 2021-11-04 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules with shortened sense strands
MA55896A (fr) 2019-05-07 2022-03-16 Modernatx Inc Polynucléotides servant à perturber l'activité de cellule immunitaire et procédés pour les utiliser
CA3140423A1 (en) 2019-05-14 2020-11-19 Translate Bio, Inc. Improved process of preparing mrna-loaded lipid nanoparticles
EP3982932A4 (en) 2019-06-14 2023-10-25 Particella, Inc. COMPOSITIONS AND METHODS FOR BIOLOGICAL RELEASE VEHICLES
EP3986452A1 (en) 2019-06-18 2022-04-27 CureVac AG Rotavirus mrna vaccine
ES2973521T3 (es) 2019-06-20 2024-06-20 Prec Nanosystems Ulc Lípidos ionizables para la administración de ácidos nucleicos
KR102928289B1 (ko) 2019-06-29 2026-02-20 글로벌 라이프 사이언시즈 솔루션즈 캐나다 유엘씨 핵산 전달을 위한 이온화 가능한 지질
CA3146675A1 (en) 2019-07-23 2021-01-28 Translate Bio, Inc. Stable compositions of mrna-loaded lipid nanoparticles and processes of making
CA3145913A1 (en) 2019-07-29 2021-02-04 James Everett Dahlman Nanomaterials containing constrained lipids and uses thereof
AU2020321021A1 (en) 2019-07-30 2022-03-10 Verndari, Inc. Virus-like particle vaccines
US20220280639A1 (en) 2019-07-31 2022-09-08 Modernatx, Inc. Compositions and methods for delivery of rna interference agents to immune cells
EP4009955A1 (en) 2019-08-07 2022-06-15 ModernaTX, Inc. Compositions and methods for enhanced delivery of agents
CN114901253A (zh) 2019-08-14 2022-08-12 爱康泰生治疗公司 用于递送核酸的改进的脂质纳米颗粒
CN114502204A (zh) 2019-08-14 2022-05-13 库尔维科公司 具有降低的免疫刺激性质的rna组合和组合物
EP4021407A1 (en) 2019-08-30 2022-07-06 GlaxoSmithKline Biologicals S.A. Jet mixing lipid nanoparticle manufacturing process
EP4031191A4 (en) 2019-09-18 2023-10-25 The Regents of the University of California PROTEIN PRODUCING NANOLIPOSOMES AND USES THEREOF
AU2020350759A1 (en) 2019-09-19 2022-03-31 Modernatx, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
KR20240130161A (ko) 2019-10-22 2024-08-29 제네반트 사이언시즈 게엠베하 오르니틴 트랜스카르바밀라제 (otc) 구축물 및 그의 사용 방법
JP2023502576A (ja) 2019-11-22 2023-01-25 ジェネレーション バイオ カンパニー イオン化可能な脂質およびそれらのナノ粒子組成物
WO2021108731A1 (en) 2019-11-25 2021-06-03 The Methodist Hospital Aposome compositions and methods of use
EP3838266A1 (en) 2019-12-20 2021-06-23 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Microfluidic production of biofunctionalized giant unilamellar vesicles for targeted intracellular cargo delivery
IL294441A (en) 2019-12-31 2022-09-01 Fred Hutchinson Cancer Center Nanoparticle systems to stimulate and maintain immune system responsiveness at treatment sites
CA3169669A1 (en) 2020-01-31 2021-08-05 Modernatx, Inc. Methods of preparing lipid nanoparticles
EP3858332A1 (en) 2020-01-31 2021-08-04 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Bottom-up assembly of synthetic extracellular vesicles
KR20220144416A (ko) 2020-02-04 2022-10-26 큐어백 아게 코로나바이러스 백신
CN113186203B (zh) 2020-02-13 2022-12-30 斯微(上海)生物科技股份有限公司 治疗或者预防冠状病毒病的疫苗试剂
CN115427021A (zh) 2020-02-25 2022-12-02 翻译生物公司 制备负载mrna的脂质纳米颗粒的改进方法
CN113999184B (zh) 2020-02-28 2024-11-12 深圳深信生物科技有限公司 一种胺基脂质化合物、其制备方法和应用
JP7746280B2 (ja) 2020-03-27 2025-09-30 ジェネレーション バイオ カンパニー 新規脂質及びそれらのナノ粒子組成物
CN113456591B (zh) 2020-03-31 2022-06-14 武汉合生科技有限公司 糖基聚醚类化合物脂质体及其制备方法和药物
US11559577B2 (en) 2020-03-31 2023-01-24 Engimata, Inc. Immunogenic composition forming a vaccine, and a method for its manufacture
WO2021207273A1 (en) 2020-04-07 2021-10-14 The Regents Of The University Of California Manufacturing of synthetic exosomes for cns and non-cns delivery of therapeutics
WO2021207630A1 (en) 2020-04-10 2021-10-14 Northwestern University Oxidized tumor cell lysates encapsulated in liposomal spherical nucleic acids as potent cancer immunotherapeutics
IL297419B2 (en) 2020-04-22 2025-02-01 BioNTech SE Coronavirus vaccine
CA3176844A1 (en) 2020-05-01 2021-11-04 Carlos G. PEREZ-GARCIA Nucleic acids and methods of treatment for cystic fibrosis
EP4146680A1 (en) 2020-05-07 2023-03-15 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
WO2021226468A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Improved compositions for cftr mrna therapy
JP2023526284A (ja) 2020-05-15 2023-06-21 トランスレイト バイオ， インコーポレイテッド ｍＲＮＡ送達のための脂質ナノ粒子製剤
IL298241A (en) 2020-05-18 2023-01-01 Generation Bio Co New lipids and their nanoparticle preparations
CN115968278A (zh) 2020-05-18 2023-04-14 麦氏生物科技有限公司 脂质-聚合物组合物及使用方法
WO2021236855A1 (en) 2020-05-19 2021-11-25 Orna Therapeutics, Inc. Circular rna compositions and methods
US20220049251A1 (en) 2020-05-20 2022-02-17 Nitto Denko Corporation dsRNA Directed to Coronavirus Proteins
CA3170740A1 (en) 2020-05-29 2021-12-02 Curevac Ag Nucleic acid based combination vaccines
CA3180557A1 (en) 2020-05-29 2021-12-02 Lorena Lerner Encapsulated rna replicons and methods of use
EP4175664A2 (en) 2020-07-06 2023-05-10 Janssen Biotech, Inc. Prostate neoantigens and their uses
US20230338288A1 (en) 2020-07-14 2023-10-26 The Research Foundation For The State University Of New York Asymmetric charged vesicles and methods of preparing and use thereof
AU2021307952A1 (en) 2020-07-17 2023-03-02 Generation Bio Co. Methods for encapsulating polynucleotides into reduced sizes of Lipid nanoparticles and novel formulation thereof
GB202011367D0 (en) 2020-07-22 2020-09-02 Micropore Tech Limited Method of preparing liposomes
WO2022020534A1 (en) 2020-07-23 2022-01-27 The Regents Of The University Of California Compositions comprising mir-690 and methods therefor
MA71659A (fr) 2020-08-06 2025-05-30 Modernatx, Inc. Compositions pour l'administration de molécules de charge utile à l'épithélium des voies respiratoires
EP4192432A1 (en) 2020-08-06 2023-06-14 ModernaTX, Inc. Methods of preparing lipid nanoparticles
US20230338495A1 (en) 2020-08-19 2023-10-26 Vitruviae LLC Vaccine Compositions and Antibodies For Lyme Disease
EP4203987A4 (en) 2020-08-25 2024-10-16 The Regents of The University of California COMPOSITIONS AND METHODS FOR THE TREATMENT OF OCULAR NEUROINFLAMMATION
EP4157344A2 (en) 2020-08-31 2023-04-05 CureVac SE Multivalent nucleic acid based coronavirus vaccines
CA3189903A1 (en) 2020-09-11 2022-03-17 Wilfred Jefferies Vaccine for viral pathogens

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2009513151A (ja) 2005-11-02 2009-04-02 プロチバ バイオセラピューティクス インコーポレイティッド 修飾ｓｉＲＮＡ分子およびその使用法
JP2011507534A (ja) 2007-12-27 2011-03-10 プロチバ バイオセラピューティクス インコーポレイティッド 干渉ｒｎａを使用したポロ様キナーゼ発現のサイレンシング方法
JP2014523411A (ja) 2011-06-08 2014-09-11 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
JP2016528890A (ja) 2013-07-09 2016-09-23 プレジデント アンド フェローズ オブ ハーバード カレッジ ＣＲＩＳＰＲ／Ｃａｓ系を用いるゲノム編集の治療用の使用
JP2016539916A (ja) 2013-10-22 2016-12-22 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のＣＮＳ送達及びその使用方法
JP2018509387A (ja) 2015-01-21 2018-04-05 フェーズアールエックス インコーポレイテッド 細胞に治療および診断剤を送達するための方法、組成物、ならびにシステム
JP2018511588A (ja) 2015-03-19 2018-04-26 トランスレイト バイオ， インコーポレイテッド ポンペ病のｍＲＮＡ治療

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