CN113272282B - 具有插入的酯、硫酯、二硫化物和酸酐部分的2,5-二氧代哌嗪脂质 - Google Patents

具有插入的酯、硫酯、二硫化物和酸酐部分的2,5-二氧代哌嗪脂质 Download PDF

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CN113272282B
CN113272282B CN201980088233.2A CN201980088233A CN113272282B CN 113272282 B CN113272282 B CN 113272282B CN 201980088233 A CN201980088233 A CN 201980088233A CN 113272282 B CN113272282 B CN 113272282B
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cationic lipid
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formula
compound
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CN113272282A (zh
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Y·张
F·德罗莎
S·卡夫
M·哈特莱因
S·卡马卡
R·兰迪斯
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Translate Bio Inc
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    • C07D241/02—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings not condensed with other rings
    • C07D241/06—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings not condensed with other rings having one or two double bonds between ring members or between ring members and non-ring members
    • C07D241/08—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings not condensed with other rings having one or two double bonds between ring members or between ring members and non-ring members with oxygen atoms directly attached to ring carbon atoms
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    • C07D241/04—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings not condensed with other rings having no double bonds between ring members or between ring members and non-ring members
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    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • A—HUMAN NECESSITIES
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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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    • 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
    • A61K47/24—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61K48/0041—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 the non-active part being polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • 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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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
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    • C12Y201/00—Transferases transferring one-carbon groups (2.1)
    • C12Y201/03—Carboxy- and carbamoyltransferases (2.1.3)
    • C12Y201/03003—Ornithine carbamoyltransferase (2.1.3.3)

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  • Medicinal Preparation (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Nitrogen And Oxygen As The Only Ring Hetero Atoms (AREA)
CN201980088233.2A 2018-11-09 2019-11-07 具有插入的酯、硫酯、二硫化物和酸酐部分的2,5-二氧代哌嗪脂质 Active CN113272282B (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862758179P 2018-11-09 2018-11-09
US62/758,179 2018-11-09
US201962871510P 2019-07-08 2019-07-08
US62/871,510 2019-07-08
PCT/US2019/060344 WO2020097384A1 (en) 2018-11-09 2019-11-07 2,5-dioxopiperazine lipids with intercalated ester, thioester, disulfide and anhydride moieities

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CN113272282A CN113272282A (zh) 2021-08-17
CN113272282B true CN113272282B (zh) 2024-11-08

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US (1) US20230071228A1 (https=)
EP (1) EP3877368A1 (https=)
JP (2) JP7483294B2 (https=)
KR (1) KR102860605B1 (https=)
CN (1) CN113272282B (https=)
AU (1) AU2019376660B2 (https=)
BR (1) BR112021008974A2 (https=)
CA (1) CA3117882A1 (https=)
IL (1) IL282723B1 (https=)
MX (1) MX2021005482A (https=)
WO (1) WO2020097384A1 (https=)

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MX2024002726A (es) 2021-09-03 2024-03-20 CureVac SE Nuevas nanoparticulas lipidicas para la administracion de acidos nucleicos.
WO2023073228A1 (en) 2021-10-29 2023-05-04 CureVac SE Improved circular rna for expressing therapeutic proteins
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US20230071228A1 (en) 2023-03-09
JP2024099679A (ja) 2024-07-25
IL282723A (en) 2021-06-30
CA3117882A1 (en) 2020-05-14
AU2019376660A8 (en) 2021-06-10
JP2022512945A (ja) 2022-02-07
AU2019376660B2 (en) 2025-06-26
KR102860605B1 (ko) 2025-09-15
JP7483294B2 (ja) 2024-05-15
WO2020097384A1 (en) 2020-05-14
EP3877368A1 (en) 2021-09-15
KR20210102248A (ko) 2021-08-19
BR112021008974A2 (pt) 2021-08-03
CN113272282A (zh) 2021-08-17
MX2021005482A (es) 2021-09-08

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