CN114728887A - 用于治疗剂的细胞内递送的支链尾端脂质化合物和组合物 - Google Patents

用于治疗剂的细胞内递送的支链尾端脂质化合物和组合物 Download PDF

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CN114728887A
CN114728887A CN202080080067.4A CN202080080067A CN114728887A CN 114728887 A CN114728887 A CN 114728887A CN 202080080067 A CN202080080067 A CN 202080080067A CN 114728887 A CN114728887 A CN 114728887A
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alkyl
lipid
peg
compound
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K·E·贝内纳托
M·科内比斯
E·亨尼西
E·S·库马拉辛赫
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ModernaTx Inc
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ModernaTx Inc
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    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C219/00Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton
    • C07C219/02Compounds 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
    • C07C219/04Compounds 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
    • C07C219/06Compounds 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C229/00Compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C229/02Compounds 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
    • C07C229/04Compounds 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
    • C07C229/24Compounds 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 carboxyl group bound to the carbon skeleton, e.g. aspartic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/7088Compounds having three or more nucleosides or nucleotides
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    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
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    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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
    • A61K48/0033Medicinal 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 the non-active part being non-polymeric
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272Non-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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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
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    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C219/00Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton
    • C07C219/02Compounds 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
    • C07C219/18Compounds 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 saturated and containing rings
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    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C229/00Compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C229/02Compounds 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
    • C07C229/04Compounds 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
    • C07C229/06Compounds 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
    • C07C229/10Compounds 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
    • C07C229/12Compounds 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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    • C07C229/00Compounds containing amino and carboxyl groups bound to the same carbon skeleton
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    • C07C229/04Compounds 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
    • C07C229/06Compounds 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
    • C07C229/10Compounds 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
    • C07C229/16Compounds 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 hydrocarbon radicals substituted by amino or carboxyl groups, e.g. ethylenediamine-tetra-acetic acid, iminodiacetic acids
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    • C07C2601/14The ring being saturated

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
CN202080080067.4A 2019-09-19 2020-09-18 用于治疗剂的细胞内递送的支链尾端脂质化合物和组合物 Pending CN114728887A (zh)

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US201962902927P 2019-09-19 2019-09-19
US62/902927 2019-09-19
PCT/US2020/051609 WO2021055833A1 (en) 2019-09-19 2020-09-18 Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents

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CN202510240330.6A Pending CN120423969A (zh) 2019-09-19 2020-09-18 用于治疗剂的细胞内递送的支链尾端脂质化合物和组合物

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AR (1) AR120080A1 (https=)
AU (1) AU2020350759A1 (https=)
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Cited By (13)

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CN115557875A (zh) * 2022-08-19 2023-01-03 上海耐澄生物科技有限公司 一种脂质化合物及脂质纳米颗粒
CN115745820A (zh) * 2023-01-05 2023-03-07 北京悦康科创医药科技股份有限公司 长效低毒的新型阳离子脂质化合物及其组合物
CN115925563A (zh) * 2023-02-28 2023-04-07 清华大学 一种可用于靶向肺部递送核酸的脂质分子及其制备方法与应用
CN116947669A (zh) * 2022-09-30 2023-10-27 荣灿生物医药技术(上海)有限公司 一种转染效率高的可电离脂质化合物及其应用
CN117126071A (zh) * 2022-08-12 2023-11-28 上海蓝鹊生物医药有限公司 含氮链状化合物、其制备方法、包含其的组合物和应用
CN117777089A (zh) * 2022-12-28 2024-03-29 仁景(苏州)生物科技有限公司 脂质化合物和脂质纳米颗粒组合物
WO2024067639A1 (zh) * 2022-09-30 2024-04-04 荣灿生物医药技术(上海)有限公司 一种转染效率高的可电离脂质化合物及其应用
CN117886711A (zh) * 2024-03-13 2024-04-16 北京新合睿恩生物医疗科技有限公司 一种阳离子脂质化合物及其制备方法和应用、及lnp组合物
WO2024145964A1 (zh) * 2023-01-05 2024-07-11 北京悦康科创医药科技股份有限公司 高效低毒且稳定性表达的阳离子脂质化合物及其组合物
WO2024145963A1 (zh) * 2023-01-05 2024-07-11 北京悦康科创医药科技股份有限公司 用于递送核酸的可电离阳离子脂质化合物和组合物及用途
WO2024193525A1 (zh) * 2023-03-17 2024-09-26 尧唐(上海)生物科技有限公司 递送治疗剂的脂质化合物及其制备方法与应用
WO2025146125A1 (zh) * 2024-01-05 2025-07-10 仁景(苏州)生物科技有限公司 用于递送的脂质化合物和脂质纳米颗粒
WO2025153099A1 (zh) * 2024-01-19 2025-07-24 晟迪生物医药(苏州)有限公司 预制载体在制备用于免疫细胞和干细胞的体外基因递送的产品中的应用

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JP2025537047A (ja) * 2023-01-05 2025-11-13 ハンチョウ ティアンロン ファーマシューティカル カンパニー、リミテッド 高効率かつ低毒性で安定的に発現するカチオン性脂質化合物、およびその組成物
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