JP7746280B2 - 新規脂質及びそれらのナノ粒子組成物 - Google Patents

新規脂質及びそれらのナノ粒子組成物

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Publication number
JP7746280B2
JP7746280B2 JP2022558110A JP2022558110A JP7746280B2 JP 7746280 B2 JP7746280 B2 JP 7746280B2 JP 2022558110 A JP2022558110 A JP 2022558110A JP 2022558110 A JP2022558110 A JP 2022558110A JP 7746280 B2 JP7746280 B2 JP 7746280B2
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Prior art keywords
lipid
aspects
cedna
pharmaceutically acceptable
disease
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Japanese (ja)
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JP2023518985A5 (https=
JP2023518985A (ja
JPWO2021195529A5 (https=
Inventor
マシュー ジー. スタントン,
バート ノルティング,
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ジェネレーション バイオ カンパニー
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Publication of JP2023518985A5 publication Critical patent/JP2023518985A5/ja
Publication of JPWO2021195529A5 publication Critical patent/JPWO2021195529A5/ja
Priority to JP2025154013A priority Critical patent/JP2025181861A/ja
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Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/28Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51Nanocapsules; Nanoparticles
    • A61K9/5107Excipients; Inactive ingredients
    • A61K9/5123Organic compounds, e.g. fats, sugars
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D211/00Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
    • C07D211/04Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D211/06Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
    • C07D211/08Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms
    • C07D211/18Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms
    • C07D211/20Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms with hydrocarbon radicals, substituted by singly bound oxygen or sulphur atoms
    • C07D211/22Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms with hydrocarbon radicals, substituted by singly bound oxygen or sulphur atoms by oxygen atoms
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction 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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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
  • Molecular Biology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Plant Pathology (AREA)
  • Optics & Photonics (AREA)
  • Obesity (AREA)
  • Nanotechnology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2022558110A 2020-03-27 2021-03-26 新規脂質及びそれらのナノ粒子組成物 Active JP7746280B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025154013A JP2025181861A (ja) 2020-03-27 2025-09-17 新規脂質及びそれらのナノ粒子組成物

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063000990P 2020-03-27 2020-03-27
US63/000,990 2020-03-27
PCT/US2021/024413 WO2021195529A2 (en) 2020-03-27 2021-03-26 Novel lipids and nanoparticle compositions thereof

Related Child Applications (1)

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Publications (4)

Publication Number Publication Date
JP2023518985A JP2023518985A (ja) 2023-05-09
JP2023518985A5 JP2023518985A5 (https=) 2024-04-04
JPWO2021195529A5 JPWO2021195529A5 (https=) 2024-04-04
JP7746280B2 true JP7746280B2 (ja) 2025-09-30

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JP2025154013A Pending JP2025181861A (ja) 2020-03-27 2025-09-17 新規脂質及びそれらのナノ粒子組成物

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Country Status (11)

Country Link
US (3) US12534435B2 (https=)
EP (1) EP4125821A4 (https=)
JP (2) JP7746280B2 (https=)
KR (1) KR20220160647A (https=)
CN (1) CN115916163A (https=)
AU (1) AU2021241696A1 (https=)
BR (1) BR112022019369A2 (https=)
CA (1) CA3173126A1 (https=)
IL (1) IL296763B2 (https=)
MX (1) MX2022011988A (https=)
WO (1) WO2021195529A2 (https=)

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CN118973614A (zh) 2022-03-28 2024-11-15 日油株式会社 用于向脾脏组织递送核酸的脂质纳米粒子以及使用其向脾脏组织递送核酸的方法
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CN117964577B (zh) * 2024-03-29 2024-06-21 天津全和诚生物技术有限公司 阳离子脂质化合物、其制备方法、包含其的组合物及应用
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WO2019188867A1 (ja) 2018-03-27 2019-10-03 日油株式会社 細胞内動態を改善した新規カチオン性脂質

Also Published As

Publication number Publication date
US20230159459A1 (en) 2023-05-25
WO2021195529A2 (en) 2021-09-30
BR112022019369A2 (pt) 2022-12-13
CA3173126A1 (en) 2021-09-30
JP2023518985A (ja) 2023-05-09
EP4125821A4 (en) 2024-06-26
IL296763B2 (en) 2026-04-01
US20240294474A1 (en) 2024-09-05
IL296763B1 (en) 2025-12-01
US12252470B2 (en) 2025-03-18
CN115916163A (zh) 2023-04-04
EP4125821A2 (en) 2023-02-08
AU2021241696A1 (en) 2022-12-01
KR20220160647A (ko) 2022-12-06
JP2025181861A (ja) 2025-12-11
MX2022011988A (es) 2023-01-04
US12534435B2 (en) 2026-01-27
IL296763A (en) 2022-11-01
WO2021195529A3 (en) 2021-11-18
US20250361208A1 (en) 2025-11-27

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