AU2019351917B2 - Ionizable amine lipids - Google Patents

Ionizable amine lipids Download PDF

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Publication number
AU2019351917B2
AU2019351917B2 AU2019351917A AU2019351917A AU2019351917B2 AU 2019351917 B2 AU2019351917 B2 AU 2019351917B2 AU 2019351917 A AU2019351917 A AU 2019351917A AU 2019351917 A AU2019351917 A AU 2019351917A AU 2019351917 B2 AU2019351917 B2 AU 2019351917B2
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Australia
Prior art keywords
compound
lipid
certain embodiments
rna
formula
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AU2019351917A
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AU2019351917A1 (en
Inventor
Derek LAPLACA
Micah MAETANI
Rubina Giare Parmar
Stephen S. Scully
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Intellia Therapeutics Inc
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Intellia Therapeutics Inc
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    • 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
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
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    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
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    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
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    • 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
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
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    • C07C219/06—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 the hydroxy groups esterified by carboxylic acids having the esterifying carboxyl groups bound to hydrogen atoms or to acyclic carbon atoms of an acyclic saturated carbon skeleton
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    • 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
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    • C07C219/20—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 unsaturated
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    • C07C229/26—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 more than one amino group bound to the carbon skeleton, e.g. lysine
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    • C07C233/34—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups
    • C07C233/35—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom
    • C07C233/36—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom having the carbon atom of the carboxamide group bound to a hydrogen atom or to a carbon atom of an acyclic saturated carbon skeleton
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    • C07C233/46—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by carboxyl groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom
    • C07C233/47—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by carboxyl groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom having the carbon atom of the carboxamide group bound to a hydrogen atom or to a carbon atom of an acyclic saturated carbon skeleton
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AU2019351917A 2018-10-02 2019-10-02 Ionizable amine lipids Ceased AU2019351917B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862740274P 2018-10-02 2018-10-02
US62/740,274 2018-10-02
PCT/US2019/054240 WO2020072605A1 (en) 2018-10-02 2019-10-02 Ionizable amine lipids

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AU2019351917A1 AU2019351917A1 (en) 2021-04-29
AU2019351917B2 true AU2019351917B2 (en) 2025-01-30

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US (1) US20220009878A1 (https=)
EP (1) EP3860972A1 (https=)
JP (2) JP7485659B2 (https=)
KR (1) KR20210093871A (https=)
CN (1) CN113039174B (https=)
AU (1) AU2019351917B2 (https=)
BR (1) BR112021006270A2 (https=)
CA (1) CA3114032A1 (https=)
CO (1) CO2021005774A2 (https=)
EA (1) EA202190916A1 (https=)
IL (2) IL281948B2 (https=)
MX (1) MX2021003455A (https=)
PH (1) PH12021550701A1 (https=)
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TW (1) TW202028170A (https=)
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WO (1) WO2020072605A1 (https=)

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US12419965B2 (en) 2018-10-01 2025-09-23 BioNTech SE RNA particles comprising polysarcosine
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
CA3145913A1 (en) 2019-07-29 2021-02-04 James Everett Dahlman 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
IL294318A (en) 2020-01-09 2022-08-01 Guide Therapeutics Llc Nanomaterials
IL297419B2 (en) 2020-04-22 2025-02-01 BioNTech SE Coronavirus vaccine
CA3181340A1 (en) 2020-04-28 2021-11-04 Intellia Therapeutics, Inc. Methods of in vitro cell delivery
EP4228658A4 (en) 2020-10-14 2025-02-19 George Mason Research Foundation, Inc. IONIZABLE LIPIDS AND METHODS FOR THE PRODUCTION AND USE THEREOF
WO2022101486A1 (en) * 2020-11-16 2022-05-19 BioNTech SE Pharmaceutical compositions comprising particles and mrna and methods for preparing and storing the same
US20240041785A1 (en) * 2020-11-16 2024-02-08 BioNTech SE Compositions and methods for stabilization of lipid nanoparticle mrna vaccines
CN117042758A (zh) * 2020-11-16 2023-11-10 生物技术欧洲股份公司 用于稳定化脂质纳米颗粒mRNA疫苗的组合物和方法
TW202237148A (zh) * 2020-11-16 2022-10-01 德商拜恩迪克公司 包含rna之lnp組合物以及製備、儲存及使用彼之方法
WO2022218503A1 (en) * 2021-04-12 2022-10-20 BioNTech SE Lnp compositions comprising rna and methods for preparing, storing and using the same
CN116782889A (zh) * 2020-11-16 2023-09-19 生物技术欧洲股份公司 增强的制剂稳定性和改善的冻干工艺
IL303506A (en) 2020-12-11 2023-08-01 Intellia Therapeutics Inc Polynucleotides, compositions, and methods for genome editing involving deamination
EP4262818A4 (en) * 2020-12-21 2025-03-26 Beam Therapeutics Inc. NANOMATERIALS WITH CARBONATES
JP2024503976A (ja) * 2020-12-21 2024-01-30 ビーム セラピューティクス インク． アセタールを含むナノ材料
EP4262819A4 (en) * 2020-12-21 2024-12-04 Beam Therapeutics Inc. NANOMATERIALS WITH ESTER-BOUND ACETALS
EP4281080A4 (en) * 2021-01-20 2025-09-24 Beam Therapeutics Inc NANOMATERIALS COMPRISING A BIODEGRADABLE ELEMENT
CN116761888A (zh) 2021-01-20 2023-09-15 比姆医疗股份有限公司 纳米材料
CN113993839B (zh) * 2021-02-05 2024-02-09 嘉晨西海(杭州)生物技术有限公司 一种可离子化脂质分子、其制备方法及其在制备脂质纳米颗粒中的应用
CN112961065B (zh) * 2021-02-05 2023-03-14 嘉晨西海(杭州)生物技术有限公司 一种可电离脂质分子及其制备方法及其在制备脂质纳米颗粒的应用
CN117510830A (zh) * 2021-04-08 2024-02-06 厦门赛诺邦格生物科技股份有限公司 一种聚乙二醇化脂质及其修饰的脂质体、含该脂质体的药物组合物及其制剂和应用
CN115197078B (zh) * 2021-04-08 2024-06-04 厦门赛诺邦格生物科技股份有限公司 一种聚乙二醇化脂质及其修饰的脂质体、含该脂质体的药物组合物及其制剂和应用
IL307740A (en) 2021-04-17 2023-12-01 Intellia Therapeutics Inc DNA-dependent protein kinase inhibitors and their compositions and uses
KR20240017793A (ko) 2021-04-17 2024-02-08 인텔리아 테라퓨틱스, 인크. 지질 나노입자 조성물
EP4351651A1 (en) * 2021-05-28 2024-04-17 Renagade Therapeutics Management Inc. Lipid nanoparticles and methods of use thereof
WO2022261292A1 (en) 2021-06-10 2022-12-15 Intellia Therapeutics, Inc. Modified guide rnas comprising an internal linker for gene editing
KR20240038705A (ko) * 2021-06-22 2024-03-25 인텔리아 테라퓨틱스, 인크. 간 유전자의 생체 내 편집 방법
TW202328067A (zh) 2021-09-14 2023-07-16 美商雷納嘉德醫療管理公司 環狀脂質及其使用方法
CN118317944A (zh) 2021-09-14 2024-07-09 雷纳嘉德医疗管理公司 非环状脂质及其使用方法
US20250295755A1 (en) 2021-10-22 2025-09-25 Sail Biomedicines, Inc. Mrna vaccine composition
CA3237303A1 (en) 2021-11-03 2023-05-11 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing
CA3238764A1 (en) 2021-11-23 2023-06-01 Siddharth Patel A bacteria-derived lipid composition and use thereof
US12186387B2 (en) 2021-11-29 2025-01-07 BioNTech SE Coronavirus vaccine
EP4452239A1 (en) 2021-12-20 2024-10-30 Sail Biomedicines, Inc. Compositions comprising mrna and lipid reconstructed plant messenger packs
TW202330457A (zh) * 2022-01-19 2023-08-01 大陸商蘇州艾博生物科技有限公司 脂質化合物和脂質奈米顆粒組合物
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
WO2023222081A1 (zh) 2022-05-19 2023-11-23 仁景(苏州)生物科技有限公司 长链烷基酯胺类脂质化合物及其制备方法和在核酸递送方面的应用
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
WO2024002985A1 (en) 2022-06-26 2024-01-04 BioNTech SE Coronavirus vaccine
CA3260437A1 (en) 2022-07-20 2024-01-25 Beam Therapeutics Inc. NANOMATERIALS INCLUDING TRIOLS
US20260035338A1 (en) * 2022-10-13 2026-02-05 Shenzhen Shenxin Biotechnology Co., Ltd. Amino lipid compound and lipid nanoparticle for delivering bioactive ingredient
WO2024102434A1 (en) 2022-11-10 2024-05-16 Senda Biosciences, Inc. Rna compositions comprising lipid nanoparticles or lipid reconstructed natural messenger packs
EP4626600A1 (en) * 2022-11-30 2025-10-08 Agency for Science, Technology and Research (A*STAR) Methods of synthesising ionisable lipids
EP4638783A2 (en) 2022-12-22 2025-10-29 Intellia Therapeutics, Inc. Methods for analyzing nucleic acid cargos of lipid nucleic acid assemblies
CN120418427A (zh) 2022-12-23 2025-08-01 因特利亚治疗公司 用于基因组编辑的系统和方法
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
WO2024220752A2 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Rna therapeutic compositions
WO2024220712A2 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Vaccine compositions
WO2024220625A1 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Delivery of polynucleotides from lipid nanoparticles comprising rna and ionizable lipids
CN121039098A (zh) * 2023-04-28 2025-11-28 日东电工株式会社 脂质、包含其的药物组合物、和递送活性药物成分的方法
AU2024312860A1 (en) * 2023-06-21 2026-01-22 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025000542A1 (en) * 2023-06-30 2025-01-02 Shanghai Vitalgen Biopharma Co., Ltd. Ionizable cationic lipid compounds for delivery of biologically active agents
WO2025049481A1 (en) 2023-08-28 2025-03-06 Intellia Therapeutics, Inc. Methods of editing an hla-a gene in vitro
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
WO2025106915A1 (en) 2023-11-17 2025-05-22 Sail Biomedicines, Inc. Circular polyribonucleotides encoding glucagon-like peptide 1 (glp-1) and uses thereof
WO2025106930A1 (en) 2023-11-17 2025-05-22 Sail Biomedicines, Inc. Circular polyribonucleotides encoding glucagon-like peptide 2 (glp-2) 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
CN118255678B (zh) * 2024-03-28 2024-12-24 荣灿生物医药技术(上海)有限公司 一种可电离脂质化合物及其应用
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
WO2026035680A1 (en) 2024-08-06 2026-02-12 Kernal Biologics, Inc. Compositions and methods for delivery of agents

Citations (3)

* 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
WO2017075531A1 (en) * 2015-10-28 2017-05-04 Acuitas Therapeutics, Inc. Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
WO2018170306A1 (en) * 2017-03-15 2018-09-20 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents

Family Cites Families (8)

* 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
US20060051405A1 (en) 2004-07-19 2006-03-09 Protiva Biotherapeutics, Inc. Compositions for the delivery of therapeutic agents and uses thereof
US20100055169A1 (en) * 2008-04-16 2010-03-04 Abbott Laboratories Cationic lipids and uses thereof
CA3009891C (en) 2009-12-23 2020-09-15 Novartis Ag Lipids, lipid compositions, and methods of using them
WO2013086354A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
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
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
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

Patent Citations (3)

* 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
WO2017075531A1 (en) * 2015-10-28 2017-05-04 Acuitas Therapeutics, Inc. Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
WO2018170306A1 (en) * 2017-03-15 2018-09-20 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents

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