JP7791182B2 - ピペラジンベースのカチオン性脂質 - Google Patents

ピペラジンベースのカチオン性脂質

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Publication number
JP7791182B2
JP7791182B2 JP2023518356A JP2023518356A JP7791182B2 JP 7791182 B2 JP7791182 B2 JP 7791182B2 JP 2023518356 A JP2023518356 A JP 2023518356A JP 2023518356 A JP2023518356 A JP 2023518356A JP 7791182 B2 JP7791182 B2 JP 7791182B2
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alkyl
pharmaceutically acceptable
formula
independently selected
acceptable salt
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English (en)
Japanese (ja)
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JP2023542944A5 (https=
JP2023542944A (ja
Inventor
サスワタ・カルマカル
ラメシュ・ダサリ
ライアン・ランディス
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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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D241/00—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings
    • C07D241/02—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings not condensed with other rings
    • C07D241/04—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings not condensed with other rings having no double bonds between ring members or between ring members and non-ring members
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal 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
    • A61K48/0025—Medicinal 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
    • A61K48/0033—Medicinal 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
    • 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
    • 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
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Dispersion Chemistry (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Manufacturing & Machinery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Detergent Compositions (AREA)
  • Agricultural Chemicals And Associated Chemicals (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2023518356A 2020-09-23 2021-09-23 ピペラジンベースのカチオン性脂質 Active JP7791182B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063082101P 2020-09-23 2020-09-23
US63/082,101 2020-09-23
PCT/US2021/051763 WO2022066916A1 (en) 2020-09-23 2021-09-23 Piperazine-based cationic lipids

Publications (3)

Publication Number Publication Date
JP2023542944A JP2023542944A (ja) 2023-10-12
JP2023542944A5 JP2023542944A5 (https=) 2024-10-01
JP7791182B2 true JP7791182B2 (ja) 2025-12-23

Family

ID=78414070

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023518356A Active JP7791182B2 (ja) 2020-09-23 2021-09-23 ピペラジンベースのカチオン性脂質

Country Status (12)

Country Link
US (1) US20230357166A1 (https=)
EP (1) EP4216933B1 (https=)
JP (1) JP7791182B2 (https=)
KR (1) KR20230074526A (https=)
CN (1) CN116457027A (https=)
AU (1) AU2021349262A1 (https=)
BR (1) BR112023005207A2 (https=)
CA (1) CA3193347A1 (https=)
ES (1) ES2994955T3 (https=)
IL (1) IL301536A (https=)
MX (1) MX2023003344A (https=)
WO (1) WO2022066916A1 (https=)

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WO2022099003A1 (en) 2020-11-06 2022-05-12 Sanofi Lipid nanoparticles for delivering mrna vaccines
BR112023020027A2 (pt) * 2021-04-15 2023-11-14 Translate Bio Inc Lipídeos catiônicos baseados em tampão "good"
WO2023178167A1 (en) * 2022-03-16 2023-09-21 Translate Bio, Inc. Asymmetric piperazine-based cationic lipids
US20250263385A1 (en) 2022-04-13 2025-08-21 Sanofi "Good" buffer-based cationic lipids
US20240150306A1 (en) * 2022-09-27 2024-05-09 Reinvigoron Theratech, Inc. Compounds with cleavable disulfide moieties
EP4637813A1 (en) 2022-12-20 2025-10-29 Sanofi Pasteur Rhinovirus mrna vaccine
CN116082275B (zh) * 2023-03-13 2023-07-14 北京悦康科创医药科技股份有限公司 一种脾脏中高表达的阳离子脂质化合物、包含其的组合物及用途
EP4734960A1 (en) 2023-06-28 2026-05-06 Sanofi Pasteur Inc. Sterol analogs in lipid nanoparticle formulations
US12599560B2 (en) 2023-11-02 2026-04-14 Longuide Biopharma Corporation Lipids and lipid nanoparticle formulations
CN121219273A (zh) * 2023-11-02 2025-12-26 深圳厚存纳米药业有限公司 脂质和脂质纳米颗粒制剂
US12539279B2 (en) 2023-12-11 2026-02-03 The Governing Council Of The University Of Toronto Supramolecular ionizable lipid molecules with heteroatomic tuning for nucleic acid delivery
WO2025141521A1 (en) 2023-12-29 2025-07-03 Sanofi Lipids having dendritic moieties
WO2025160118A1 (en) * 2024-01-22 2025-07-31 The Trustees Of The University Of Pennsylvania Compositions and methods for hematopoietic stem cell (hsc) targeted delivery of therapeutic agents
WO2025255534A1 (en) * 2024-06-07 2025-12-11 Innovac Therapeutics Hk Limited Diol-based novel ionizable lipids and their uses thereof
WO2026008743A1 (en) 2024-07-02 2026-01-08 Sanofi Pasteur Inc. Water-soluble polyanionic polymer as adjuvant for carrier-formulated nucleic acid
WO2026053147A1 (en) 2024-09-05 2026-03-12 Sanofi Pasteur Inc. Helper lipids and lnp compositions comprising same

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014028487A1 (en) 2012-08-13 2014-02-20 Massachusetts Institute Of Technology Amine-containing lipidoids and uses thereof
JP2017520563A (ja) 2014-06-24 2017-07-27 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立体化学的に濃縮した組成物
WO2020097384A1 (en) 2018-11-09 2020-05-14 Translate Bio, Inc. 2,5-dioxopiperazine lipids with intercalated ester, thioester, disulfide and anhydride moieities
WO2021226468A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Improved compositions for cftr mrna therapy
WO2021226463A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia

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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
US5132418A (en) 1980-02-29 1992-07-21 University Patents, Inc. Process for preparing 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
US7101995B2 (en) * 2001-08-27 2006-09-05 Mirus Bio Corporation Compositions and processes using siRNA, amphipathic compounds and polycations
KR101766408B1 (ko) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
ES3032935T3 (en) * 2013-10-22 2025-07-29 Translate Bio Inc Lipid formulations for delivery of messenger rna
WO2016205691A1 (en) * 2015-06-19 2016-12-22 Massachusetts Institute Of Technology Alkenyl substituted 2,5-piperazinediones and their use in compositions for delivering an agent to a subject or cell
AU2017356699B2 (en) * 2016-11-08 2021-11-11 Ramot At Tel-Aviv University Ltd. Cationic lipids for nucleic acid delivery and preparation thereof
AU2017357758B2 (en) 2016-11-10 2023-11-16 Translate Bio, Inc. Improved process of preparing mRNA-loaded lipid nanoparticles
JP7422977B2 (ja) * 2018-07-23 2024-01-29 トランスレイト バイオ， インコーポレイテッド メッセンジャーｒｎａ用乾燥粉末製剤

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014028487A1 (en) 2012-08-13 2014-02-20 Massachusetts Institute Of Technology Amine-containing lipidoids and uses thereof
JP2017520563A (ja) 2014-06-24 2017-07-27 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立体化学的に濃縮した組成物
WO2020097384A1 (en) 2018-11-09 2020-05-14 Translate Bio, Inc. 2,5-dioxopiperazine lipids with intercalated ester, thioester, disulfide and anhydride moieities
WO2021226468A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Improved compositions for cftr mrna therapy
WO2021226463A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia

Also Published As

Publication number Publication date
AU2021349262A1 (en) 2023-06-08
US20230357166A1 (en) 2023-11-09
KR20230074526A (ko) 2023-05-30
CN116457027A (zh) 2023-07-18
AU2021349262A9 (en) 2025-03-13
ES2994955T3 (en) 2025-02-04
MX2023003344A (es) 2023-06-05
EP4216933B1 (en) 2024-09-18
WO2022066916A1 (en) 2022-03-31
IL301536A (en) 2023-05-01
JP2023542944A (ja) 2023-10-12
EP4216933A1 (en) 2023-08-02
CA3193347A1 (en) 2022-03-31
EP4216933C0 (en) 2024-09-18
BR112023005207A2 (pt) 2023-04-25

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