JP2024542099A - 核酸を含む脂質調合物、及び嚢胞性線維症の治療方法 - Google Patents

核酸を含む脂質調合物、及び嚢胞性線維症の治療方法 Download PDF

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JP2024542099A
JP2024542099A JP2024526594A JP2024526594A JP2024542099A JP 2024542099 A JP2024542099 A JP 2024542099A JP 2024526594 A JP2024526594 A JP 2024526594A JP 2024526594 A JP2024526594 A JP 2024526594A JP 2024542099 A JP2024542099 A JP 2024542099A
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lipid
composition
mrna
mol
sequence
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JP2024542099A5 (https=
JPWO2023081776A5 (https=
Inventor
ジー ペレス－ガルシア，カルロス
清 立川
大輝 松田
チブクラ，パドマナブ
プラカシュ カルマリ，プリヤ
バオ，ヤンジエ
ボイド リー ベガ，ジェレル
ムクタバーラム，ラジェシュ
サギ，アミット
ペイ，イーホア
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Arcturus Therapeutics Inc
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Arcturus Therapeutics Inc
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Publication of JP2024542099A publication Critical patent/JP2024542099A/ja
Publication of JP2024542099A5 publication Critical patent/JP2024542099A5/ja
Publication of JPWO2023081776A5 publication Critical patent/JPWO2023081776A5/ja
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    • 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
    • A61K47/24—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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A61K9/0078—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a nebulizer such as a jet nebulizer, ultrasonic nebulizer, e.g. in the form of aqueous drug solutions or dispersions
    • 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
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C333/00—Derivatives of thiocarbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
    • C07C333/02—Monothiocarbamic acids; Derivatives thereof
    • C07C333/04—Monothiocarbamic acids; Derivatives thereof having nitrogen atoms of thiocarbamic groups bound to hydrogen atoms or to acyclic carbon atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D519/00—Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00
    • 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

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Dispersion Chemistry (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Optics & Photonics (AREA)
  • Nanotechnology (AREA)
  • Otolaryngology (AREA)
  • Pulmonology (AREA)
  • Biomedical Technology (AREA)
  • Dermatology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2024526594A 2021-11-03 2022-11-03 核酸を含む脂質調合物、及び嚢胞性線維症の治療方法 Pending JP2024542099A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163275402P 2021-11-03 2021-11-03
US63/275,402 2021-11-03
PCT/US2022/079244 WO2023081776A1 (en) 2021-11-03 2022-11-03 Lipid formulations containing nucleic acids and methods of treatment for cystic fibrosis

Publications (3)

Publication Number Publication Date
JP2024542099A true JP2024542099A (ja) 2024-11-13
JP2024542099A5 JP2024542099A5 (https=) 2025-11-12
JPWO2023081776A5 JPWO2023081776A5 (https=) 2025-11-12

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ID=86242208

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JP2024526594A Pending JP2024542099A (ja) 2021-11-03 2022-11-03 核酸を含む脂質調合物、及び嚢胞性線維症の治療方法

Country Status (6)

Country Link
US (1) US20230159449A1 (https=)
EP (1) EP4426266A4 (https=)
JP (1) JP2024542099A (https=)
AU (1) AU2022381750A1 (https=)
CA (1) CA3236675A1 (https=)
WO (1) WO2023081776A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12070509B2 (en) * 2020-05-01 2024-08-27 Arcturus Therapeutics, Inc. Nucleic acids and methods of treatment for cystic fibrosis
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
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
CN117653606B (zh) * 2023-11-14 2024-05-10 广东康百医药科技有限公司 一种间充质干细胞外泌体冻干保护剂及其应用
WO2025111297A1 (en) * 2023-11-21 2025-05-30 Modernatx, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
WO2025166323A2 (en) 2024-02-02 2025-08-07 Editas Medicine, Inc. Crispr-related methods and compositions targeting lipoprotein (a) expression
WO2025213138A1 (en) 2024-04-05 2025-10-09 Editas Medicine, Inc. Crispr/rna-guided nuclease related methods and compositions for treating primary open angle glaucoma
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2019013752A (es) * 2017-05-16 2020-07-20 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr optimizado en codones.
ES3039647T3 (en) * 2017-05-31 2025-10-23 Arcturus Therapeutics Inc Synthesis and structure of high potency rna therapeutics
US12083224B2 (en) * 2018-03-30 2024-09-10 Arcturus Therapeutics, Inc. Lipid particles for nucleic acid delivery
KR20210091120A (ko) * 2018-08-29 2021-07-21 트랜슬레이트 바이오 인코포레이티드 Mrna-로딩된 지질 나노입자를 제조하는 개선된 공정
WO2021183563A1 (en) * 2020-03-09 2021-09-16 Arcturus Therapeutics, Inc. Coronavirus vaccine compositions and methods

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Publication number Publication date
EP4426266A4 (en) 2025-11-05
WO2023081776A1 (en) 2023-05-11
AU2022381750A1 (en) 2024-05-16
US20230159449A1 (en) 2023-05-25
CA3236675A1 (en) 2023-05-11
EP4426266A1 (en) 2024-09-11

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