JP2024502948A - ナノ材料のためのイオン化可能な脂質 - Google Patents

ナノ材料のためのイオン化可能な脂質 Download PDF

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Publication number
JP2024502948A
JP2024502948A JP2023538794A JP2023538794A JP2024502948A JP 2024502948 A JP2024502948 A JP 2024502948A JP 2023538794 A JP2023538794 A JP 2023538794A JP 2023538794 A JP2023538794 A JP 2023538794A JP 2024502948 A JP2024502948 A JP 2024502948A
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optionally substituted
saturated
branched
compound
hydrocarbon chain
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JP2024502948A5 (https=
JPWO2022159421A5 (https=
Inventor
ミナ ファウジ シハタ、
グレゴリー ローレンス ハミルトン、
ニーラジ ナレンドラ パトワルダン、
コリー デイン サゴ、
ミロニ バルワントクマール チュハブラ、
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ビーム セラピューティクス インク.
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Publication of JP2024502948A publication Critical patent/JP2024502948A/ja
Publication of JP2024502948A5 publication Critical patent/JP2024502948A5/ja
Publication of JPWO2022159421A5 publication Critical patent/JPWO2022159421A5/ja
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    • CCHEMISTRY; METALLURGY
    • 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
    • CCHEMISTRY; METALLURGY
    • 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
    • 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
    • A61K47/14Esters of carboxylic acids, e.g. fatty acid monoglycerides, medium-chain triglycerides, parabens or PEG fatty acid esters
    • 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
    • A61K47/24Organic 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
    • 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
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/107Emulsions ; Emulsion preconcentrates; Micelles
    • 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07JSTEROIDS
    • C07J41/00Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring
    • C07J41/0033Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005
    • C07J41/0055Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005 the 17-beta position being substituted by an uninterrupted chain of at least three carbon atoms which may or may not be branched, e.g. cholane or cholestane derivatives, optionally cyclised, e.g. 17-beta-phenyl or 17-beta-furyl derivatives
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07JSTEROIDS
    • C07J41/00Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring
    • C07J41/0033Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005
    • C07J41/0088Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005 containing unsubstituted amino radicals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C2601/00Systems containing only non-condensed rings
    • C07C2601/12Systems containing only non-condensed rings with a six-membered ring
    • C07C2601/14The ring being saturated
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C2603/00Systems containing at least three condensed rings
    • C07C2603/02Ortho- or ortho- and peri-condensed systems
    • C07C2603/40Ortho- or ortho- and peri-condensed systems containing four condensed rings
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C2603/00Systems containing at least three condensed rings
    • C07C2603/56Ring systems containing bridged rings
    • C07C2603/58Ring systems containing bridged rings containing three rings
    • C07C2603/70Ring systems containing bridged rings containing three rings containing only six-membered rings
    • C07C2603/74Adamantanes

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Physics & Mathematics (AREA)
  • Biomedical Technology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Dispersion Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Steroid Compounds (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
JP2023538794A 2021-01-20 2022-01-19 ナノ材料のためのイオン化可能な脂質 Pending JP2024502948A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163139730P 2021-01-20 2021-01-20
US63/139,730 2021-01-20
PCT/US2022/012867 WO2022159421A1 (en) 2021-01-20 2022-01-19 Ionizable lipids for nanomaterials

Publications (3)

Publication Number Publication Date
JP2024502948A true JP2024502948A (ja) 2024-01-24
JP2024502948A5 JP2024502948A5 (https=) 2025-01-24
JPWO2022159421A5 JPWO2022159421A5 (https=) 2025-01-24

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JP2023538794A Pending JP2024502948A (ja) 2021-01-20 2022-01-19 ナノ材料のためのイオン化可能な脂質

Country Status (7)

Country Link
US (1) US20230348359A1 (https=)
EP (1) EP4281114A4 (https=)
JP (1) JP2024502948A (https=)
CN (1) CN116782951A (https=)
AU (1) AU2022210299A1 (https=)
CA (1) CA3203784A1 (https=)
WO (1) WO2022159421A1 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12569438B2 (en) 2019-07-29 2026-03-10 Georgia Tech Research Corporation Nanomaterials containing constrained lipids and uses thereof
US12435035B2 (en) 2020-01-09 2025-10-07 Guide Therapeutics, Llc Nanomaterials
IL303845A (en) 2021-01-20 2023-08-01 Beam Therapeutics Inc Nanomaterials include a degradable property
KR20230131863A (ko) 2021-01-20 2023-09-14 빔 테라퓨틱스, 인크. 나노물질
US12311033B2 (en) 2023-05-31 2025-05-27 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2025035202A1 (en) * 2023-08-04 2025-02-20 Nanovation Therapeutics Inc. Ionizable lipids comprising macrocyclic rings for the delivery of therapeutic agents
WO2025038855A1 (en) * 2023-08-16 2025-02-20 Beam Therapeutics Inc. Ionizable lipids with branched head groups
WO2025137243A1 (en) * 2023-12-19 2025-06-26 Amplitude Therapeutics, Inc. Methods for measuring self-amplifying rna and trans amplifying rna activity in vivo
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014115473A (ja) * 2012-12-10 2014-06-26 Konica Minolta Inc 光学フィルムのロール体とその製造方法、包装体、偏光板および液晶表示装置
JP2014534220A (ja) * 2011-10-27 2014-12-18 マサチューセッツ インスティテュート オブ テクノロジー 薬物内包微小球の形成が可能なn−末端で官能基化されたアミノ酸誘導体
WO2015095340A1 (en) * 2013-12-19 2015-06-25 Novartis Ag Lipids and lipid compositions for the delivery of active agents
JP2017500865A (ja) * 2013-12-19 2017-01-12 ノバルティス アーゲー レプチンmRNAの組成物および製剤

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014534220A (ja) * 2011-10-27 2014-12-18 マサチューセッツ インスティテュート オブ テクノロジー 薬物内包微小球の形成が可能なn−末端で官能基化されたアミノ酸誘導体
JP2014115473A (ja) * 2012-12-10 2014-06-26 Konica Minolta Inc 光学フィルムのロール体とその製造方法、包装体、偏光板および液晶表示装置
WO2015095340A1 (en) * 2013-12-19 2015-06-25 Novartis Ag Lipids and lipid compositions for the delivery of active agents
JP2017500865A (ja) * 2013-12-19 2017-01-12 ノバルティス アーゲー レプチンmRNAの組成物および製剤

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
MELISSA P. LOKUGAMAGE ET AL.: ""Constrained Nanoparticles Deliver siRNA and sgRNA to T Cells In Vivo without Targeting Ligands"", ADVANCED MATERIALS, vol. 31, no. 41, JPN6025043884, 29 August 2019 (2019-08-29), ISSN: 0005717934 *

Also Published As

Publication number Publication date
CN116782951A (zh) 2023-09-19
US20230348359A1 (en) 2023-11-02
WO2022159421A1 (en) 2022-07-28
EP4281114A4 (en) 2025-08-06
EP4281114A1 (en) 2023-11-29
AU2022210299A1 (en) 2023-06-29
CA3203784A1 (en) 2022-07-28

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