JP2024542001A - 脂質アミン - Google Patents

脂質アミン Download PDF

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Publication number
JP2024542001A
JP2024542001A JP2024525459A JP2024525459A JP2024542001A JP 2024542001 A JP2024542001 A JP 2024542001A JP 2024525459 A JP2024525459 A JP 2024525459A JP 2024525459 A JP2024525459 A JP 2024525459A JP 2024542001 A JP2024542001 A JP 2024542001A
Authority
JP
Japan
Prior art keywords
alkyl
salt
lipid
compound according
optionally substituted
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2024525459A
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English (en)
Japanese (ja)
Other versions
JP2024542001A5 (enExample
Inventor
シーパーサウド，モヒンドラ
クロフォード，マシュー
テイト，ダニエル
コーネビス，マーク
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
ModernaTx Inc
Original Assignee
ModernaTx Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by ModernaTx Inc filed Critical ModernaTx Inc
Publication of JP2024542001A publication Critical patent/JP2024542001A/ja
Publication of JP2024542001A5 publication Critical patent/JP2024542001A5/ja
Pending legal-status Critical Current

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07J—STEROIDS
    • C07J41/00—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring
    • C07J41/0033—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005
    • C07J41/0055—Normal 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
    • 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
    • A61K9/48—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
    • 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
    • A61K9/5192—Processes
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07J—STEROIDS
    • C07J41/00—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring
    • C07J41/0033—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005
    • C07J41/0088—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005 containing unsubstituted amino radicals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07J—STEROIDS
    • C07J43/00—Normal steroids having a nitrogen-containing hetero ring spiro-condensed or not condensed with the cyclopenta(a)hydrophenanthrene skeleton
    • C07J43/003—Normal steroids having a nitrogen-containing hetero ring spiro-condensed or not condensed with the cyclopenta(a)hydrophenanthrene skeleton not condensed
    • 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
    • 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
    • A61K9/0043—Nose
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Physics & Mathematics (AREA)
  • Molecular Biology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Steroid Compounds (AREA)
JP2024525459A 2021-10-29 2022-10-28 脂質アミン Pending JP2024542001A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163273421P 2021-10-29 2021-10-29
US63/273,421 2021-10-29
US202263308180P 2022-02-09 2022-02-09
US63/308,180 2022-02-09
US202263395243P 2022-08-04 2022-08-04
US63/395,243 2022-08-04
PCT/US2022/048223 WO2023076598A1 (en) 2021-10-29 2022-10-28 Lipid amines

Publications (2)

Publication Number Publication Date
JP2024542001A true JP2024542001A (ja) 2024-11-13
JP2024542001A5 JP2024542001A5 (enExample) 2025-11-17

Family

ID=84365358

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024525459A Pending JP2024542001A (ja) 2021-10-29 2022-10-28 脂質アミン

Country Status (5)

Country Link
US (1) US20260109732A1 (enExample)
EP (1) EP4423107A1 (enExample)
JP (1) JP2024542001A (enExample)
TW (1) TW202332467A (enExample)
WO (1) WO2023076598A1 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4628497A4 (en) * 2023-01-10 2026-05-13 Jenkem Tech Co Ltd Liaoning CATIONIC STEROID-LIPID COMPOUND AND ITS USES
EP4694871A1 (en) * 2023-04-14 2026-02-18 Beijing Jitai Pharmaceutical Technology Co., Ltd. Lipid nanoparticle compositions and uses thereof
EP4704816A1 (en) * 2023-05-03 2026-03-11 ModernaTX, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
WO2024249920A2 (en) * 2023-05-31 2024-12-05 Northwestern University Lipid-gold salt molecules and formulation in lipid nanoparticle delivery systems
CN118027129A (zh) * 2024-01-04 2024-05-14 江苏申基生物科技有限公司 一种可电离脂质化合物及其应用

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH10510813A (ja) * 1994-12-09 1998-10-20 ジェンザイム・コーポレイション 治療分子の細胞内送達のためのカチオン性両親媒性化合物およびプラスミド
JP2011500671A (ja) * 2007-10-17 2011-01-06 韓国科学技術院 核酸遺伝子伝達用低密度リポタンパク質類似（ＬＤＬ−ｌｉｋｅ）陽イオン性ナノ粒子、その製造方法、及びこれを用いた核酸遺伝子の伝達方法
US20150297749A1 (en) * 2012-10-11 2015-10-22 Postech Academy-Industry Foundation Low-density lipoprotein analogue nanoparticles, and composition comprising same for targeted diagnosis and treatment of liver
WO2019094405A1 (en) * 2017-11-10 2019-05-16 Board Of Regents, The University Of Texas System Lipid-based nanoparticles for encapsulation and sustained release of therapeutic agents
JP2020510072A (ja) * 2017-03-15 2020-04-02 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． 脂質ナノ粒子製剤

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5681702A (en) 1994-08-30 1997-10-28 Chiron Corporation Reduction of nonspecific hybridization by using novel base-pairing schemes
DE19631189A1 (de) * 1996-08-02 1998-02-05 Max Delbrueck Centrum Neuartige kationische Amphiphile für den liposomalen Gentransfer
US5942634A (en) * 1997-05-09 1999-08-24 Genzyme Corporation Cationic amphiphiles for cell transfections
US6169078B1 (en) * 1998-05-12 2001-01-02 University Of Florida Materials and methods for the intracellular delivery of substances
WO2000030444A1 (en) * 1998-11-25 2000-06-02 Vanderbilt University Cationic liposomes for gene transfer
FR2797188A1 (fr) * 1999-08-04 2001-02-09 Univ Paris Nord Laboratoire De Composes lipidiques cationiques et leurs utilisation pour le transfert de substances d'interet therapeutique chargees negativement
US9999673B2 (en) 2011-01-11 2018-06-19 Alnylam Pharmaceuticals, Inc. PEGylated lipids and their use for drug delivery
CN105143456A (zh) 2013-03-15 2015-12-09 不列颠哥伦比亚大学 用于转染的脂质纳米粒子和相关方法
JP2017520549A (ja) * 2014-06-26 2017-07-27 ラモット アット テル アビブ ユニバーシティ， リミテッド 核酸の送達のためのリポソーム製剤
EP4710943A3 (en) * 2015-08-28 2026-04-29 Molecular Transfer, Inc. Transfection complexes and methods of using the same
WO2022032154A2 (en) * 2020-08-06 2022-02-10 Modernatx, Inc. Compositions for the delivery of payload molecules to airway epithelium
CA3199784A1 (en) * 2020-11-13 2022-05-19 Modernatx, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH10510813A (ja) * 1994-12-09 1998-10-20 ジェンザイム・コーポレイション 治療分子の細胞内送達のためのカチオン性両親媒性化合物およびプラスミド
JP2011500671A (ja) * 2007-10-17 2011-01-06 韓国科学技術院 核酸遺伝子伝達用低密度リポタンパク質類似（ＬＤＬ−ｌｉｋｅ）陽イオン性ナノ粒子、その製造方法、及びこれを用いた核酸遺伝子の伝達方法
US20150297749A1 (en) * 2012-10-11 2015-10-22 Postech Academy-Industry Foundation Low-density lipoprotein analogue nanoparticles, and composition comprising same for targeted diagnosis and treatment of liver
JP2020510072A (ja) * 2017-03-15 2020-04-02 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． 脂質ナノ粒子製剤
WO2019094405A1 (en) * 2017-11-10 2019-05-16 Board Of Regents, The University Of Texas System Lipid-based nanoparticles for encapsulation and sustained release of therapeutic agents

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
RADCHATAWEDCHAKOON WIDCHAYA ET AL.: "Solid phase synthesis of novel asymmetric hydrophilic head cholesterol-based cationic lipids with po", BIOORGANIC & MEDICINAL CHEMISTRY, vol. 18, JPN6026030462, 2010, pages 330 - 342, ISSN: 0005913196 *

Also Published As

Publication number Publication date
EP4423107A1 (en) 2024-09-04
US20260109732A1 (en) 2026-04-23
TW202332467A (zh) 2023-08-16
WO2023076598A1 (en) 2023-05-04

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