JP7791103B2 - フェノール酸脂質系カチオン性脂質 - Google Patents

フェノール酸脂質系カチオン性脂質

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Publication number
JP7791103B2
JP7791103B2 JP2022559873A JP2022559873A JP7791103B2 JP 7791103 B2 JP7791103 B2 JP 7791103B2 JP 2022559873 A JP2022559873 A JP 2022559873A JP 2022559873 A JP2022559873 A JP 2022559873A JP 7791103 B2 JP7791103 B2 JP 7791103B2
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JP
Japan
Prior art keywords
pharmaceutically acceptable
acceptable salt
alkyl
optionally substituted
cationic lipid
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JP2022559873A
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English (en)
Japanese (ja)
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JP2023520047A5 (https=
JPWO2021202694A5 (https=
JP2023520047A (ja
Inventor
シュリラン・カルブ
フランク・デロサ
アピワット・ワンウィーラウォン
サスワタ・カルマカール
Original Assignee
トランスレイト バイオ， インコーポレイテッド
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Publication of JP2023520047A publication Critical patent/JP2023520047A/ja
Publication of JP2023520047A5 publication Critical patent/JP2023520047A5/ja
Publication of JPWO2021202694A5 publication Critical patent/JPWO2021202694A5/ja
Priority to JP2025244099A priority Critical patent/JP2026048786A/ja
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Publication of JP7791103B2 publication Critical patent/JP7791103B2/ja
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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C229/00—Compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C229/02—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C229/04—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C229/06—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton
    • C07C229/10—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings
    • C07C229/12—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings to carbon atoms of acyclic carbon skeletons
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D213/00—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members
    • C07D213/02—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members
    • C07D213/04—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D213/24—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen or carbon atoms directly attached to the ring nitrogen atom with substituted hydrocarbon radicals attached to ring carbon atoms
    • C07D213/28—Radicals substituted by singly-bound oxygen or sulphur atoms
    • C07D213/30—Oxygen atoms
    • 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
    • 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

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Dispersion Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
JP2022559873A 2020-04-01 2021-03-31 フェノール酸脂質系カチオン性脂質 Active JP7791103B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025244099A JP2026048786A (ja) 2020-04-01 2025-12-10 フェノール酸脂質系カチオン性脂質

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063003698P 2020-04-01 2020-04-01
US63/003,698 2020-04-01
PCT/US2021/025128 WO2021202694A1 (en) 2020-04-01 2021-03-31 Phenolic acid lipid based cationic lipids

Related Child Applications (1)

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JP2025244099A Division JP2026048786A (ja) 2020-04-01 2025-12-10 フェノール酸脂質系カチオン性脂質

Publications (4)

Publication Number Publication Date
JP2023520047A JP2023520047A (ja) 2023-05-15
JP2023520047A5 JP2023520047A5 (https=) 2024-04-11
JPWO2021202694A5 JPWO2021202694A5 (https=) 2024-04-11
JP7791103B2 true JP7791103B2 (ja) 2025-12-23

Family

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JP2022559873A Active JP7791103B2 (ja) 2020-04-01 2021-03-31 フェノール酸脂質系カチオン性脂質
JP2025244099A Pending JP2026048786A (ja) 2020-04-01 2025-12-10 フェノール酸脂質系カチオン性脂質

Family Applications After (1)

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Country Status (4)

Country Link
EP (1) EP4126813A1 (https=)
JP (2) JP7791103B2 (https=)
CN (2) CN121318756A (https=)
WO (1) WO2021202694A1 (https=)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4313115A1 (en) 2021-03-25 2024-02-07 Translate Bio, Inc. Optimized nucleotide sequences encoding the extracellular domain of human ace2 protein or a portion thereof
US20250073351A1 (en) * 2021-04-19 2025-03-06 Translate Bio, Inc. Improved compositions for delivery of mrna
MX2024002726A (es) 2021-09-03 2024-03-20 CureVac SE Nuevas nanoparticulas lipidicas para la administracion de acidos nucleicos.
WO2023073228A1 (en) 2021-10-29 2023-05-04 CureVac SE Improved circular rna for expressing therapeutic proteins
US20250099614A1 (en) 2022-01-28 2025-03-27 CureVac SE Nucleic acid encoded transcription factor inhibitors
WO2023227608A1 (en) 2022-05-25 2023-11-30 Glaxosmithkline Biologicals Sa Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide
WO2024089638A1 (en) 2022-10-28 2024-05-02 Glaxosmithkline Biologicals Sa Nucleic acid based vaccine
WO2024133515A1 (en) 2022-12-20 2024-06-27 Sanofi Rhinovirus mrna vaccine
KR20250153298A (ko) 2023-03-08 2025-10-24 큐어백 에스이 핵산 전달을 위한 신규의 지질 나노입자 제형
WO2024230934A1 (en) 2023-05-11 2024-11-14 CureVac SE Therapeutic nucleic acid for the treatment of ophthalmic diseases
WO2025189064A1 (en) 2024-03-08 2025-09-12 Genzyme Corporation Lipid nanoparticles
WO2026003373A1 (en) 2024-06-28 2026-01-02 Sanofi Phenolic acid lipid based cationic lipids
WO2026008743A1 (en) 2024-07-02 2026-01-08 Sanofi Pasteur Inc. Water-soluble polyanionic polymer as adjuvant for carrier-formulated nucleic acid
WO2026030375A2 (en) 2024-07-30 2026-02-05 Genzyme Corporation Lipid nanoparticles and methods of manufacture and use thereof

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017518371A (ja) 2014-05-30 2017-07-06 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド 核酸の送達のための生分解性脂質
WO2018170306A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
JP2019519601A (ja) 2016-05-18 2019-07-11 モダーナティエックス・インコーポレイテッドＭｏｄｅｒｎａＴＸ， Ｉｎｃ． 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5132418A (en) 1980-02-29 1992-07-21 University Patents, Inc. Process for preparing polynucleotides
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US4500707A (en) 1980-02-29 1985-02-19 University Patents, Inc. Nucleosides useful in the preparation of polynucleotides
US4668777A (en) 1981-03-27 1987-05-26 University Patents, Inc. Phosphoramidite nucleoside compounds
US4415732A (en) 1981-03-27 1983-11-15 University Patents, Inc. Phosphoramidite compounds and processes
US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
US4373071A (en) 1981-04-30 1983-02-08 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US4401796A (en) 1981-04-30 1983-08-30 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US5153319A (en) 1986-03-31 1992-10-06 University Patents, Inc. Process for preparing polynucleotides
US5262530A (en) 1988-12-21 1993-11-16 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5047524A (en) 1988-12-21 1991-09-10 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5780014A (en) 1995-04-14 1998-07-14 Inhale Therapeutic Systems Method and apparatus for pulmonary administration of dry powder alpha 1-antitrypsin
US5700642A (en) 1995-05-22 1997-12-23 Sri International Oligonucleotide sizing using immobilized cleavable primers
US5744335A (en) 1995-09-19 1998-04-28 Mirus Corporation Process of transfecting a cell with a polynucleotide mixed with an amphipathic compound and a DNA-binding protein
US6472397B1 (en) * 1998-04-09 2002-10-29 Meiji Seika Kaisha, Ltd. Nitrogen-containing heterocyclic compounds having antiplatelet aggregation effect and medicinal use thereof
HRP20220652T1 (hr) * 2015-12-10 2022-06-24 Modernatx, Inc. Pripravci i postupci unosa terapijskih sredstava
WO2018089801A1 (en) 2016-11-10 2018-05-17 Translate Bio, Inc. Improved process of preparing mrna-loaded lipid nanoparticles
CN112437767B (zh) * 2018-05-24 2023-10-27 川斯勒佰尔公司 硫酯阳离子脂质
EP3849617A1 (en) * 2018-09-14 2021-07-21 Translate Bio, Inc. Composition and methods for treatment of methylmalonic acidemia
CN110526885A (zh) * 2019-08-20 2019-12-03 上海英诺富成生物科技有限公司 麦考酚酸的前体药物及其制备方法

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017518371A (ja) 2014-05-30 2017-07-06 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド 核酸の送達のための生分解性脂質
JP2019519601A (ja) 2016-05-18 2019-07-11 モダーナティエックス・インコーポレイテッドＭｏｄｅｒｎａＴＸ， Ｉｎｃ． 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド
WO2018170306A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
インド国特許発明第２００９ＭＵ１２０９号明細書

Also Published As

Publication number Publication date
EP4126813A1 (en) 2023-02-08
JP2026048786A (ja) 2026-03-17
CN115667207B (zh) 2025-09-16
CN115667207A (zh) 2023-01-31
JP2023520047A (ja) 2023-05-15
WO2021202694A1 (en) 2021-10-07
CN121318756A (zh) 2026-01-13

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