JP2025511899A - イオン化可能なカチオン性脂質および脂質ナノ粒子 - Google Patents

イオン化可能なカチオン性脂質および脂質ナノ粒子 Download PDF

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JP2025511899A
JP2025511899A JP2024559484A JP2024559484A JP2025511899A JP 2025511899 A JP2025511899 A JP 2025511899A JP 2024559484 A JP2024559484 A JP 2024559484A JP 2024559484 A JP2024559484 A JP 2024559484A JP 2025511899 A JP2025511899 A JP 2025511899A
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alkyl
cycloalkyl
lipid
aryl
lnp
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JP2025511899A5 (enExample
Inventor
プリヤ プラカシュ カルマリ，
スティーブン タニス，
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キャプスタン セラピューティクス， インコーポレイテッド
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    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K9/48—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
    • C07C217/00—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton
    • C07C217/02—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C217/04—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C217/06—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one etherified hydroxy group and one amino group bound to the carbon skeleton, which is not further substituted
    • C07C217/08—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one etherified hydroxy group and one amino group bound to the carbon skeleton, which is not further substituted the oxygen atom of the etherified hydroxy group being further bound to an acyclic carbon atom
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C219/00—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton
    • C07C219/02—Compounds 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
    • C07C219/04—Compounds 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
    • C07C219/06—Compounds 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C237/00—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups
    • C07C237/02—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton
    • C07C237/04—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C237/08—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated having the nitrogen atom of at least one of the carboxamide groups bound to an acyclic carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C271/00—Derivatives of carbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
    • C07C271/06—Esters of carbamic acids
    • C07C271/08—Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms
    • C07C271/10—Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms with the nitrogen atoms of the carbamate groups bound to hydrogen atoms or to acyclic carbon atoms
    • C07C271/16—Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms with the nitrogen atoms of the carbamate groups bound to hydrogen atoms or to acyclic carbon atoms to carbon atoms of hydrocarbon radicals substituted by singly-bound oxygen atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C275/00—Derivatives of urea, i.e. compounds containing any of the groups, the nitrogen atoms not being part of nitro or nitroso groups
    • C07C275/04—Derivatives of urea, i.e. compounds containing any of the groups, the nitrogen atoms not being part of nitro or nitroso groups having nitrogen atoms of urea groups bound to acyclic carbon atoms
    • C07C275/06—Derivatives of urea, i.e. compounds containing any of the groups, the nitrogen atoms not being part of nitro or nitroso groups having nitrogen atoms of urea groups bound to acyclic carbon atoms of an acyclic and saturated carbon skeleton
    • C07C275/14—Derivatives of urea, i.e. compounds containing any of the groups, the nitrogen atoms not being part of nitro or nitroso groups having nitrogen atoms of urea groups bound to acyclic carbon atoms of an acyclic and saturated carbon skeleton being further substituted by nitrogen atoms not being part of nitro or nitroso groups
    • 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
    • C07D211/36—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 hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D211/40—Oxygen atoms
    • C07D211/44—Oxygen atoms attached in position 4
    • C07D211/46—Oxygen atoms attached in position 4 having a hydrogen atom as the second substituent in position 4
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D295/00—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
    • C07D295/04—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms
    • C07D295/08—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly bound oxygen or sulfur atoms
    • C07D295/084—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly bound oxygen or sulfur atoms with the ring nitrogen atoms and the oxygen or sulfur atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
    • C07D295/088—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly bound oxygen or sulfur atoms with the ring nitrogen atoms and the oxygen or sulfur atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings to an acyclic saturated chain

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Optics & Photonics (AREA)
  • Biomedical Technology (AREA)
  • Nanotechnology (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2024559484A 2022-04-05 2023-04-05 イオン化可能なカチオン性脂質および脂質ナノ粒子 Pending JP2025511899A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202263362501P 2022-04-05 2022-04-05
US63/362,501 2022-04-05
US202263366462P 2022-06-15 2022-06-15
US63/366,462 2022-06-15
US202363489381P 2023-03-09 2023-03-09
US63/489,381 2023-03-09
PCT/US2023/017647 WO2023196444A1 (en) 2022-04-05 2023-04-05 Ionizable cationic lipids and lipid nanoparticles

Publications (2)

Publication Number Publication Date
JP2025511899A true JP2025511899A (ja) 2025-04-16
JP2025511899A5 JP2025511899A5 (enExample) 2026-04-15

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JP2024559484A Pending JP2025511899A (ja) 2022-04-05 2023-04-05 イオン化可能なカチオン性脂質および脂質ナノ粒子

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US (2) US20230320995A1 (enExample)
EP (1) EP4504694A4 (enExample)
JP (1) JP2025511899A (enExample)
KR (1) KR20250009428A (enExample)
CN (1) CN119013250A (enExample)
AU (1) AU2023249179A1 (enExample)
CA (1) CA3254658A1 (enExample)
IL (1) IL315577A (enExample)
MX (1) MX2024012161A (enExample)
WO (1) WO2023196444A1 (enExample)

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WO2024192291A1 (en) 2023-03-15 2024-09-19 Renagade Therapeutics Management Inc. Delivery of gene editing systems and methods of use thereof
EP4680595A2 (en) 2023-03-15 2026-01-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
EP4719352A1 (en) * 2023-05-31 2026-04-08 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2025006799A1 (en) 2023-06-27 2025-01-02 Capstan Therapeutics, Inc. Extracorporeal and ex vivo engineering of select cell populations from peripheral blood
US20250127728A1 (en) * 2023-10-05 2025-04-24 Capstan Therapeutics, Inc. Constrained Ionizable Cationic Lipids and Lipid Nanoparticles
WO2025076113A1 (en) 2023-10-05 2025-04-10 Capstan Therapeutics, Inc. Ionizable cationic lipids with conserved spacing and lipid nanoparticles
US20250144234A1 (en) 2023-11-02 2025-05-08 Capstan Therapeutics Inc. RNA for In vivo Transfection with Increased Expression
CN122228239A (zh) * 2023-11-15 2026-06-16 上海尧唐生物科技股份有限公司 递送治疗剂的脂质化合物及其制备方法与应用
CN117263882B (zh) * 2023-11-21 2024-02-23 深圳瑞吉生物科技有限公司 一种阳离子脂质化合物、包含其的组合物及应用
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
IL328626A (en) 2023-12-15 2026-07-01 Capstan Therapeutics Inc Humanized anti-CD8 antibodies and their uses
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
WO2025166238A1 (en) 2024-01-31 2025-08-07 Orna Therapeutics, Inc. Fast-shedding polyethylene glycol lipids
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
US20250302763A1 (en) 2024-02-22 2025-10-02 Capstan Therapeutics, Inc. Immune engineering amplification
WO2025180454A1 (zh) * 2024-02-28 2025-09-04 成都凌泰氪生物技术有限公司 一种亲脂性化合物、其制备方法及应用
US20250381150A1 (en) 2024-03-08 2025-12-18 Genzyme Corporation Lipid nanoparticles
WO2025217452A1 (en) * 2024-04-11 2025-10-16 Capstan Therapeutics, Inc. Constrained ionizable cationic lipids and lipid nanoparticles
WO2025217454A2 (en) * 2024-04-11 2025-10-16 Capstan Therapeutics, Inc. Ionizable cationic lipids and lipid nanoparticles
WO2025232898A1 (en) * 2024-05-10 2025-11-13 Yoltech Therapeutics Co., Ltd Lipid compounds and lipid nanoparticle compositions
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles
WO2026030375A2 (en) 2024-07-30 2026-02-05 Genzyme Corporation Lipid nanoparticles and methods of manufacture and use thereof
WO2026122685A1 (en) 2024-12-03 2026-06-11 Orna Therapeutics, Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2026142951A1 (en) 2024-12-23 2026-07-02 Genzyme Corporation Pharmaceutical system
WO2026165530A1 (en) 2025-02-03 2026-08-06 Renagade Therapeutics Management Inc. Hydrophilic polymer lipids for use in lipid nanoparticles

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CA2223088A1 (en) * 1995-06-07 1996-12-19 Bob Dale Brown Novel carbamate-based cationic lipids
CN110003066B (zh) * 2013-11-18 2021-09-03 阿克丘勒斯治疗公司 用于rna递送的可电离的阳离子脂质
US10426737B2 (en) * 2013-12-19 2019-10-01 Novartis Ag Lipids and lipid compositions for the delivery of active agents
CN106794141B (zh) * 2014-07-16 2021-05-28 诺华股份有限公司 将核酸包封在脂质纳米粒主体中的方法
WO2020219876A1 (en) * 2019-04-25 2020-10-29 Intellia Therapeutics, Inc. Ionizable amine lipids and lipid nanoparticles
EP4008741A4 (en) * 2019-08-02 2023-06-28 Daikin Industries, Ltd. Fluorine-containing isocyanul compound

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CA3254658A1 (en) 2023-10-12
US20240299311A1 (en) 2024-09-12
KR20250009428A (ko) 2025-01-17
US20230320995A1 (en) 2023-10-12
AU2023249179A1 (en) 2024-09-26
WO2023196444A9 (en) 2025-08-21
IL315577A (en) 2024-11-01
EP4504694A4 (en) 2026-07-15
WO2023196444A1 (en) 2023-10-12
EP4504694A1 (en) 2025-02-12
CN119013250A (zh) 2024-11-22
MX2024012161A (es) 2024-11-08

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