CN110167922B - 用于核酸递送的阳离子脂质及其制备 - Google Patents

用于核酸递送的阳离子脂质及其制备 Download PDF

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CN110167922B
CN110167922B CN201780082052.XA CN201780082052A CN110167922B CN 110167922 B CN110167922 B CN 110167922B CN 201780082052 A CN201780082052 A CN 201780082052A CN 110167922 B CN110167922 B CN 110167922B
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formula
dien
bis
dimethylamino
diyl
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CN110167922A (zh
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丹·培尔
斯里尼瓦·拉米谢蒂
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Ramot at Tel Aviv University Ltd
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    • C07C211/01—Compounds containing amino groups bound to a carbon skeleton having amino groups bound to acyclic carbon atoms
    • C07C211/20—Compounds containing amino groups bound to a carbon skeleton having amino groups bound to acyclic carbon atoms of an acyclic unsaturated carbon skeleton
    • C07C211/22—Compounds containing amino groups bound to a carbon skeleton having amino groups bound to acyclic carbon atoms of an acyclic unsaturated carbon skeleton containing at least two amino groups bound to the carbon skeleton
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • C07C215/04—Compounds containing amino and hydroxy groups bound to the same carbon skeleton having hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being saturated
    • C07C215/06—Compounds containing amino and hydroxy groups bound to the same carbon skeleton having hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being saturated and acyclic
    • C07C215/08—Compounds containing amino and hydroxy groups bound to the same carbon skeleton having hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being saturated and acyclic with only one hydroxy group and one amino group bound to the 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
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    • 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
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    • 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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    • C07C243/10—Hydrazines
    • C07C243/12—Hydrazines having nitrogen atoms of hydrazine groups bound to acyclic carbon atoms
    • C07C243/14—Hydrazines having nitrogen atoms of hydrazine groups bound to acyclic carbon atoms of a saturated carbon skeleton
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    • C07C243/10—Hydrazines
    • C07C243/12—Hydrazines having nitrogen atoms of hydrazine groups bound to acyclic carbon atoms
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    • C07C243/24—Hydrazines having nitrogen atoms of hydrazine groups acylated by carboxylic acids
    • C07C243/26—Hydrazines having nitrogen atoms of hydrazine groups acylated by carboxylic acids with acylating carboxyl groups bound to hydrogen atoms or to acyclic carbon atoms
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    • C07C243/24—Hydrazines having nitrogen atoms of hydrazine groups acylated by carboxylic acids
    • C07C243/26—Hydrazines having nitrogen atoms of hydrazine groups acylated by carboxylic acids with acylating carboxyl groups bound to hydrogen atoms or to acyclic carbon atoms
    • C07C243/28—Hydrazines having nitrogen atoms of hydrazine groups acylated by carboxylic acids with acylating carboxyl groups bound to hydrogen atoms or to acyclic carbon atoms to hydrogen atoms or to carbon atoms of a saturated carbon skeleton
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    • C07C333/02—Monothiocarbamic acids; Derivatives thereof
    • C07C333/10—Monothiocarbamic acids; Derivatives thereof having nitrogen atoms of thiocarbamic groups being part of any of the groups, X being a hetero atom, Y being any atom, e.g., N-acyl-thiocarbamates
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    • C07D233/64—Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings having two double bonds between ring members or between ring members and non-ring members with substituted hydrocarbon radicals attached to ring carbon atoms, e.g. histidine
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    • C07D295/12—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 or doubly bound nitrogen atoms
    • C07D295/125—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 or doubly bound nitrogen atoms with the ring nitrogen atoms and the substituent nitrogen atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
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    • 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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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CN201780082052.XA 2016-11-08 2017-11-07 用于核酸递送的阳离子脂质及其制备 Active CN110167922B (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662418844P 2016-11-08 2016-11-08
US62/418,844 2016-11-08
US201762507829P 2017-05-18 2017-05-18
US62/507,829 2017-05-18
PCT/IL2017/051212 WO2018087753A1 (en) 2016-11-08 2017-11-07 Cationic lipids for nucleic acid delivery and preparation thereof

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CN110167922A CN110167922A (zh) 2019-08-23
CN110167922B true CN110167922B (zh) 2023-09-19

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US (3) US11851389B2 (https=)
EP (1) EP3538515B1 (https=)
JP (1) JP7075669B2 (https=)
CN (1) CN110167922B (https=)
AU (1) AU2017356699B2 (https=)
DK (1) DK3538515T3 (https=)
ES (1) ES2928358T3 (https=)
MX (1) MX2019005287A (https=)
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WO (1) WO2018087753A1 (https=)

Families Citing this family (68)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
KR102591743B1 (ko) 2018-06-08 2023-10-19 후지필름 가부시키가이샤 화합물 또는 그 염 및 지질 입자
US12419965B2 (en) 2018-10-01 2025-09-23 BioNTech SE RNA particles comprising polysarcosine
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
CA3116576A1 (en) 2018-10-18 2020-04-23 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
MX2021005448A (es) * 2018-11-13 2021-08-11 Oncorus Inc Polinucleotidos encapsulados y metodos de uso.
BR112021010853A2 (pt) * 2018-12-05 2021-08-31 Intellia Therapeutics, Inc. Lipídios de amina modificados
WO2020246581A1 (ja) * 2019-06-07 2020-12-10 富士フイルム株式会社 脂質組成物
EP4059505B1 (en) * 2019-11-15 2024-02-21 FUJIFILM Corporation Lipid composition
CN115335084A (zh) * 2020-03-25 2022-11-11 生物技术公司 包含聚肌氨酸的rna颗粒
EP4132576A1 (en) 2020-04-09 2023-02-15 Suzhou Abogen Biosciences Co., Ltd. Nucleic acid vaccines for coronavirus
WO2021204175A1 (en) 2020-04-09 2021-10-14 Suzhou Abogen Biosciences Co., Ltd. Lipid nanoparticle composition
EP4153560A4 (en) * 2020-05-19 2024-04-24 Ramot at Tel-Aviv University Ltd. LIPID PARTICLES FOR NUCLEIC ACID DELIVERY AND CLINICAL APPLICATIONS THEREOF
ES3054438T3 (en) * 2020-07-16 2026-02-03 Acuitas Therapeutics Inc Cationic lipids for use in lipid nanoparticles
IL301536A (en) * 2020-09-23 2023-05-01 Translate Bio Inc Cationic lipids based on piperazine
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
CN113185421B (zh) * 2020-11-27 2022-01-25 广州市锐博生物科技有限公司 脂质化合物及其组合物
WO2022168085A1 (en) * 2021-02-02 2022-08-11 Ramot At Tel-Aviv University Ltd. Lipids suitable for nucleic acid delivery
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
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
EP4320093A1 (en) * 2021-04-09 2024-02-14 Pfizer Inc. Methods for producing of lipids
US20240207442A1 (en) * 2021-04-22 2024-06-27 The Board Of Regents Of The University Of Texas System All-in-one dendrimer-based lipid nanoparticles enable precise hdr-mediated gene editing in vivo
EP4310071A4 (en) * 2021-05-28 2024-11-20 Beijing Tricisionbio Therapeutics Inc. LIPID COMPOUND AND USE THEREOF IN THE ADMINISTRATION OF NUCLEIC ACID
EP4370134A4 (en) 2021-07-13 2025-08-06 Technion Res & Dev Foundation COMPOSITIONS AND METHODS FOR TREATING A HEALTH CONDITION
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
CN115403761B (zh) * 2021-07-23 2025-04-25 天津键凯科技有限公司 一种多元甘醇修饰的脂质化合物及其制备方法和应用
CN115010681A (zh) * 2021-09-07 2022-09-06 安徽荣灿生物科技有限公司 一类阳离子脂质体分子及其制备和应用
CN117440943B (zh) * 2021-10-15 2025-09-30 厦门赛诺邦格生物科技股份有限公司 含氮的阳离子脂质及其应用
CN113999128B (zh) * 2021-11-25 2024-05-10 苏州科锐迈德生物医药科技有限公司 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
EP4458805A4 (en) * 2021-12-31 2026-01-28 Xiamen Sinopeg Biotech Co Ltd CATIONIC LIPID CONTAINING A FUNCTIONAL GROUP ON A SIDE CHAIN, AND ITS USE
CN114874150A (zh) * 2022-02-22 2022-08-09 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
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
WO2023165582A1 (zh) * 2022-03-04 2023-09-07 益杰立科(上海)生物科技有限公司 靶向递送系统和方法
CN114874146B (zh) * 2022-03-25 2023-09-12 圣诺生物医药技术(苏州)有限公司 组氨酸脂质、组氨酸脂质纳米颗粒及其制备方法和应用
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
CN117460711B (zh) * 2022-06-02 2025-07-08 厦门赛诺邦格生物科技股份有限公司 一种含有不饱和键的氨基酸阳离子脂质
CN116444395A (zh) * 2022-06-27 2023-07-18 上海云沂生物医药科技有限公司 一种氨基脂质、其合成方法、颗粒及用途
EP4585586A4 (en) * 2022-09-09 2025-12-03 Xiamen Sinopeg Biotech Co Ltd CARBON CORE CATIONIC LIPID
AU2023413766A1 (en) 2022-12-23 2025-05-29 Biontech Delivery Technologies Gmbh Composition
WO2024160826A1 (en) 2023-01-31 2024-08-08 BioNTech SE Compositions and methods
CN116143707B (zh) * 2023-02-24 2024-04-19 山东大学 一种碱基可电离脂质及其制备方法与应用
CN117384055B (zh) * 2023-04-27 2024-11-19 仁景(苏州)生物科技有限公司 用于递送治疗剂的可电离脂质及其用途
AU2024279278A1 (en) 2023-05-31 2025-12-18 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
CN121729406A (zh) * 2023-06-13 2026-03-24 特拉维夫大学拉莫特有限公司 具有提高的稳定性的可生物降解的氨基甲酸酯脂质
WO2025027019A1 (en) 2023-07-31 2025-02-06 BioNTech SE Method of determining nucleic acid concentration in aqueous solution or dispersion
WO2025064850A1 (en) 2023-09-22 2025-03-27 BioNTech SE Rna constructs with n-terminal degrons to enhance an immune response
WO2025093665A1 (en) 2023-10-31 2025-05-08 BioNTech SE Particles, compositions and methods
WO2025106754A1 (en) 2023-11-15 2025-05-22 BioNTech SE Coronavirus vaccine
FR3155424A1 (fr) 2023-11-15 2025-05-23 BioNTech SE Compositions immunogènes contre le sars-cov-2
WO2025133105A1 (en) 2023-12-21 2025-06-26 Biontech Delivery Technologies Gmbh Compositions and methods
WO2025143892A1 (ko) * 2023-12-29 2025-07-03 주식회사 삼양홀딩스 약물전달용 나노입자의 조성물
WO2025149492A1 (en) 2024-01-08 2025-07-17 BioNTech SE Rna encoding an immune inhibitory il-1 family member
WO2025160118A1 (en) * 2024-01-22 2025-07-31 The Trustees Of The University Of Pennsylvania Compositions and methods for hematopoietic stem cell (hsc) targeted delivery of therapeutic agents
WO2025176732A1 (en) 2024-02-20 2025-08-28 BioNTech SE Compositions and methods
WO2025215636A1 (en) 2024-04-08 2025-10-16 Yeda Research And Development Co. Ltd. Anti-defense system polypeptides and uses thereof
WO2025228979A1 (en) 2024-04-30 2025-11-06 Neovac Ltd. Lipids nanoparticle formulations suitable for lung targeting
WO2025245869A1 (zh) * 2024-05-31 2025-12-04 清华大学 含有精氨酸结构的可电离脂质分子、包含其的脂质纳米颗粒及其用途
WO2025261607A1 (en) 2024-06-21 2025-12-26 BioNTech SE Particles, compositions and methods
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles
WO2026009227A1 (en) 2024-07-04 2026-01-08 Yeda Research And Development Co. Ltd. Compositions for downregulating zeb2 in macrophages and uses thereof
WO2026013059A1 (en) 2024-07-08 2026-01-15 Neovac Ltd. Nanoparticle formulations for treatment of inflammations
WO2026021698A1 (en) 2024-07-23 2026-01-29 BioNTech SE Nucleic acid particle
WO2026047603A1 (en) 2024-08-28 2026-03-05 BioNTech SE Sars-cov-2 immunogenic compositions
WO2026047148A1 (en) 2024-08-29 2026-03-05 BioNTech SE Minimal polypeptide-encoding rnas
WO2026057625A1 (en) 2024-09-11 2026-03-19 BioNTech SE Peptidoglycan hydrolases having a chap domain with engineered active site
WO2026057626A1 (en) 2024-09-11 2026-03-19 BioNTech SE Peptidoglycan hydrolases having multiple enzymatically active domains
WO2026068864A1 (en) 2024-09-30 2026-04-02 BioNTech SE Engineered ch1 and cl domains for the prevention of chain mispairing
WO2026082899A2 (en) 2024-10-16 2026-04-23 BioNTech SE Rna for preventing or treating tuberculosis

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104603102A (zh) * 2012-07-06 2015-05-06 协和发酵麒麟株式会社 阳离子性脂质
CN105152939A (zh) * 2008-11-10 2015-12-16 阿尔尼拉姆医药品有限公司 用于递送治疗剂的脂质和组合物
WO2015199952A1 (en) * 2014-06-25 2015-12-30 Acuitas Therapeutics Inc. Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL58413C (https=) * 1941-12-04
US3703554A (en) * 1969-07-22 1972-11-21 Scm Corp Terpenylamino ethers and thioethers
GB1367089A (en) * 1970-07-23 1974-09-18 Marumo H Surface modifier for synthetic high polymers
US4034040A (en) * 1971-05-24 1977-07-05 Pfizer Inc. Xylene-diamines as antiviral agents
JP3544429B2 (ja) * 1996-04-25 2004-07-21 三菱製紙株式会社 可逆性感熱記録材料
US20100047897A1 (en) 2005-04-21 2010-02-25 Santosh K Verma Process for producing modified reconstituted sendai viral envelope specific for drug and/or gene delivery to liver cells
AU2010315124B2 (en) 2009-11-04 2016-12-01 Marina Biotech, Inc. Activity generating delivery molecules
WO2013016058A1 (en) 2011-07-22 2013-01-31 Merck Sharp & Dohme Corp. Novel bis-nitrogen containing cationic lipids for oligonucleotide delivery
WO2016021673A1 (ja) 2014-08-06 2016-02-11 協和発酵キリン株式会社 ｓｍａｌｌ ＲＮＡ含有医薬組成物の薬効を予測する方法
AU2016253972B2 (en) 2015-04-27 2020-01-02 Acuitas Therapeutics Inc. Nucleoside-modified RNA for inducing an adaptive immune response
HRP20230209T1 (hr) 2015-10-28 2023-04-14 Acuitas Therapeutics Inc. Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105152939A (zh) * 2008-11-10 2015-12-16 阿尔尼拉姆医药品有限公司 用于递送治疗剂的脂质和组合物
CN104603102A (zh) * 2012-07-06 2015-05-06 协和发酵麒麟株式会社 阳离子性脂质
WO2015199952A1 (en) * 2014-06-25 2015-12-30 Acuitas Therapeutics Inc. Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids

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