TW202332467A - 脂質胺 - Google Patents

脂質胺 Download PDF

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Publication number
TW202332467A
TW202332467A TW111141006A TW111141006A TW202332467A TW 202332467 A TW202332467 A TW 202332467A TW 111141006 A TW111141006 A TW 111141006A TW 111141006 A TW111141006 A TW 111141006A TW 202332467 A TW202332467 A TW 202332467A
Authority
TW
Taiwan
Prior art keywords
alkyl
salt
compound
lipid
optionally substituted
Prior art date
Application number
TW111141006A
Other languages
English (en)
Chinese (zh)
Inventor
摩西卓拉 西伯沙德
馬修 克勞福德
丹尼爾 泰特
馬克 況必斯
Original Assignee
美商現代公司
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 美商現代公司 filed Critical 美商現代公司
Publication of TW202332467A publication Critical patent/TW202332467A/zh

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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)
TW111141006A 2021-10-29 2022-10-28 脂質胺 TW202332467A (zh)

Applications Claiming Priority (6)

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

Publications (1)

Publication Number Publication Date
TW202332467A true TW202332467A (zh) 2023-08-16

Family

ID=84365358

Family Applications (1)

Application Number Title Priority Date Filing Date
TW111141006A TW202332467A (zh) 2021-10-29 2022-10-28 脂質胺

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)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Families Citing this family (4)

* 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

Family Cites Families (17)

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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
US6071890A (en) * 1994-12-09 2000-06-06 Genzyme Corporation Organ-specific targeting of cationic amphiphile/DNA complexes for gene therapy
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
EP2217221B1 (en) * 2007-10-17 2018-06-27 Korea Advanced Institute of Science and Technology Ldl-like cationic nanoparticles for deliverying nucleic acid gene, method for preparing thereof and method for deliverying nucleic acid gene using the same
US9999673B2 (en) 2011-01-11 2018-06-19 Alnylam Pharmaceuticals, Inc. PEGylated lipids and their use for drug delivery
KR20140048404A (ko) * 2012-10-11 2014-04-24 포항공과대학교 산학협력단 저밀도 지단백질 유사 나노입자 및 이를 포함하는 간 표적 진단 및 치료용 조성물
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
JP7332478B2 (ja) * 2017-03-15 2023-08-23 モデルナティエックス インコーポレイテッド 脂質ナノ粒子製剤
US11529314B2 (en) * 2017-11-10 2022-12-20 Board Of Regents, The University Of Texas System Lipid-based nanoparticles for encapsulation and sustained release of therapeutic agents
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

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Also Published As

Publication number Publication date
EP4423107A1 (en) 2024-09-04
US20260109732A1 (en) 2026-04-23
JP2024542001A (ja) 2024-11-13
WO2023076598A1 (en) 2023-05-04

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