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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JP
Japan
Prior art keywords
lipid
aspects
cedna
pharmaceutically acceptable
disease
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JP2022558110A
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English (en)
Japanese (ja)
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JP2023518985A (ja
JP2023518985A5 (enExample
Inventor
マシュー ジー． スタントン，
バート ノルティング，
Original Assignee
ジェネレーション バイオ カンパニー
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Publication of JP2023518985A publication Critical patent/JP2023518985A/ja
Publication of JP2023518985A5 publication Critical patent/JP2023518985A5/ja
Priority to JP2025154013A priority Critical patent/JP2025181861A/ja
Application granted granted Critical
Publication of JP7746280B2 publication Critical patent/JP7746280B2/ja
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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61K47/28—Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • 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
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules 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
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
    • C07D211/04—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D211/06—Heterocyclic 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
    • C07D211/08—Heterocyclic 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
    • C07D211/18—Heterocyclic 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
    • C07D211/20—Heterocyclic 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
    • C07D211/22—Heterocyclic 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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

Landscapes

  • 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)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (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)
  • General Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Obesity (AREA)
  • Biochemistry (AREA)
  • Optics & Photonics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Nanotechnology (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 (3)

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JP2023518985A JP2023518985A (ja) 2023-05-09
JP2023518985A5 JP2023518985A5 (enExample) 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 (enExample)
EP (1) EP4125821A4 (enExample)
JP (2) JP7746280B2 (enExample)
KR (1) KR20220160647A (enExample)
CN (1) CN115916163A (enExample)
AU (1) AU2021241696A1 (enExample)
BR (1) BR112022019369A2 (enExample)
CA (1) CA3173126A1 (enExample)
IL (1) IL296763B2 (enExample)
MX (1) MX2022011988A (enExample)
WO (1) WO2021195529A2 (enExample)

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JP7746280B2 (ja) 2020-03-27 2025-09-30 ジェネレーション バイオ カンパニー 新規脂質及びそれらのナノ粒子組成物
AU2021307952A1 (en) * 2020-07-17 2023-03-02 Generation Bio Co. Methods for encapsulating polynucleotides into reduced sizes of Lipid nanoparticles and novel formulation thereof
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WO2023190175A1 (ja) 2022-03-28 2023-10-05 日油株式会社 末梢血単核細胞へ核酸を送達するための脂質ナノ粒子およびこれを用いて末梢血単核細胞へ核酸を送達する方法
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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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Also Published As

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

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