TW202504886A - 經修飾之胺脂質 - Google Patents

經修飾之胺脂質 Download PDF

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TW202504886A
TW202504886A TW113114385A TW113114385A TW202504886A TW 202504886 A TW202504886 A TW 202504886A TW 113114385 A TW113114385 A TW 113114385A TW 113114385 A TW113114385 A TW 113114385A TW 202504886 A TW202504886 A TW 202504886A
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compound
composition
alkylene
formula
oxy
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TW113114385A
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Chinese (zh)
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史戴分 S 史考利
米卡 馬它尼
藍西 馬兆
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美商英特利亞醫療公司
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Publication of TW202504886A publication Critical patent/TW202504886A/zh

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Families Citing this family (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL314386A (en) * 2018-12-05 2024-09-01 Intellia Therapeutics Inc amine modified lipids
WO2020219876A1 (en) * 2019-04-25 2020-10-29 Intellia Therapeutics, Inc. Ionizable amine lipids and lipid nanoparticles
US12569438B2 (en) 2019-07-29 2026-03-10 Georgia Tech Research Corporation Nanomaterials containing constrained lipids and uses thereof
US12521451B2 (en) 2019-11-08 2026-01-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy
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
JP2023546175A (ja) 2020-10-14 2023-11-01 ジョージ・メイソン・リサーチ・ファウンデーション・インコーポレイテッド 脂質ナノ粒子製造の方法及びそれに由来する組成物
CR20230305A (es) 2020-12-11 2023-11-10 Intellia Therapeutics Inc Polinucleótidos, composiciones y métodos para la edición del genoma que implican desaminación
KR20230131863A (ko) * 2021-01-20 2023-09-14 빔 테라퓨틱스, 인크. 나노물질
IL303845A (en) 2021-01-20 2023-08-01 Beam Therapeutics Inc Nanomaterials include a degradable property
US20240180954A1 (en) 2021-02-04 2024-06-06 Shionogi & Co., Ltd. Cationic lipid
CR20230535A (es) 2021-04-17 2024-02-16 Intellia Therapeutics Inc Inhibidores de proteína cinasa dependiente de adn y composiciones y usos de estos
KR20240032013A (ko) 2021-06-10 2024-03-08 인텔리아 테라퓨틱스, 인크. 유전자 편집을 위한 내부 링커를 포함하는 변형된 가이드 rna
KR20240090727A (ko) 2021-10-22 2024-06-21 세일 바이오메디슨스, 인크. Mrna 백신 조성물
EP4426822A2 (en) 2021-11-03 2024-09-11 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing
CN118488834A (zh) 2021-11-23 2024-08-13 赛欧生物医药股份有限公司 细菌源性脂质组合物和其用途
WO2023122080A1 (en) 2021-12-20 2023-06-29 Senda Biosciences, Inc. Compositions comprising mrna and lipid reconstructed plant messenger packs
CN114213347B (zh) * 2022-02-22 2022-05-20 中国科学院基础医学与肿瘤研究所(筹) 一种双价可电离脂质化合物、组合物及其应用
CN114213346B (zh) * 2022-02-22 2022-05-20 中国科学院基础医学与肿瘤研究所(筹) 一种双价可电离脂质化合物、组合物及其应用
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
CN119384498A (zh) 2022-06-16 2025-01-28 因特利亚治疗公司 用于对细胞进行遗传修饰的方法和组合物
WO2024019936A1 (en) * 2022-07-20 2024-01-25 Beam Therapeutics Inc. Nanomaterials comprising triols
EP4615424A1 (en) 2022-11-10 2025-09-17 Sail Biomedicines, Inc. Rna compositions comprising lipid nanoparticles or lipid reconstructed natural messenger packs
WO2024138189A2 (en) 2022-12-22 2024-06-27 Intellia Therapeutics, Inc. Methods for analyzing nucleic acid cargos of lipid nucleic acid assemblies
WO2024138115A1 (en) 2022-12-23 2024-06-27 Intellia Theraperutics, Inc. Systems and methods for genomic editing
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
EP4698150A1 (en) 2023-04-19 2026-02-25 Sail Biomedicines, Inc. Delivery of polynucleotides from lipid nanoparticles comprising rna and ionizable lipids
WO2024220712A2 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Vaccine compositions
WO2024220752A2 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Rna therapeutic compositions
CN121241046A (zh) * 2023-05-30 2025-12-30 解放生物股份有限公司 脂质化合物、组合物及其用途
US12311033B2 (en) 2023-05-31 2025-05-27 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
EP4725938A1 (en) * 2023-06-07 2026-04-15 InnoVec Biotherapeutics Ionizable lipid compound, lipid carrier comprising same and use thereof
WO2025038855A1 (en) * 2023-08-16 2025-02-20 Beam Therapeutics Inc. Ionizable lipids with branched head groups
WO2025049481A1 (en) 2023-08-28 2025-03-06 Intellia Therapeutics, Inc. Methods of editing an hla-a gene in vitro
AU2024335327A1 (en) 2023-09-01 2026-03-26 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025064401A1 (en) 2023-09-18 2025-03-27 Intellia Therapeutics, Inc. Nuclease resistant single stranded dna product for non-viral delivery to a cell and methods of production thereof
WO2025064396A1 (en) 2023-09-18 2025-03-27 Intellia Therapeutics, Inc. Nuclease resistant double stranded dna product for non-viral delivery to a cell and methods of production thereof
WO2025106930A1 (en) 2023-11-17 2025-05-22 Sail Biomedicines, Inc. Circular polyribonucleotides encoding glucagon-like peptide 2 (glp-2) and uses thereof
WO2025106915A1 (en) 2023-11-17 2025-05-22 Sail Biomedicines, Inc. Circular polyribonucleotides encoding glucagon-like peptide 1 (glp-1) and uses thereof
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025137301A1 (en) 2023-12-20 2025-06-26 Intellia Therapeutics, Inc. Methods for rapid engineering of cells
WO2025159966A1 (en) * 2024-01-23 2025-07-31 Beam Therapeutics Inc. Compositions and methods for altering a nucleobase in a transthyretin polynucleotide
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025193861A2 (en) * 2024-03-12 2025-09-18 Tessera Therapeutics, Inc. Lipid nanoparticles for delivery of therapeutic payloads to cells
WO2025240946A1 (en) 2024-05-17 2025-11-20 Intellia Therapeutics, Inc. Lipid nanoparticles and lipid nanoparticle compositions
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles
CN121620500A (zh) * 2024-06-27 2026-03-06 因诺纬克生物科技香港有限公司 含醚新型可电离脂质及其用途
WO2026035680A1 (en) 2024-08-06 2026-02-12 Kernal Biologics, Inc. Compositions and methods for delivery of agents

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030083272A1 (en) 1997-09-19 2003-05-01 Lahive & Cockfield, Llp Sense mrna therapy
WO2006007712A1 (en) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
WO2011076807A2 (en) 2009-12-23 2011-06-30 Novartis Ag Lipids, lipid compositions, and methods of using them
SG186085A1 (en) * 2010-06-03 2013-01-30 Alnylam Pharmaceuticals Inc Biodegradable lipids for the delivery of active agents
EP3354644A1 (en) * 2011-06-08 2018-08-01 Translate Bio, Inc. Cleavable lipids
DK2931897T3 (en) * 2012-12-12 2018-02-05 Broad Inst Inc CONSTRUCTION, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTICAL APPLICATIONS
EP3608308B1 (en) * 2013-03-08 2021-07-21 Novartis AG Lipids and lipid compositions for the delivery of active agents
ES2895651T3 (es) 2013-12-19 2022-02-22 Novartis Ag Lípidos y composiciones lipídicas para la administración de agentes activos
EP4019506A1 (en) * 2013-12-19 2022-06-29 Novartis AG Lipids and lipid compositions for the delivery of active agents
RU2571176C1 (ru) * 2014-07-14 2015-12-20 Гиа Маргович Гвичия Светодиодная матрица
EP3169309B1 (en) 2014-07-16 2023-05-10 Novartis AG Method of encapsulating a nucleic acid in a lipid nanoparticle host
US10125092B2 (en) 2014-09-05 2018-11-13 Novartis Ag Lipids and lipid compositions for the delivery of active agents
JP6531983B2 (ja) * 2015-07-31 2019-06-19 パナソニックIpマネジメント株式会社 自動運転装置、自動運転支援方法及び自動運転支援プログラム
EP4286012A3 (en) 2015-09-17 2024-05-29 ModernaTX, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
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
EP3538515B1 (en) * 2016-11-08 2022-07-20 Ramot at Tel-Aviv University Ltd. Cationic lipids for nucleic acid delivery and preparation thereof
IL314386A (en) 2018-12-05 2024-09-01 Intellia Therapeutics Inc amine modified lipids

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