JP7844452B2 - Tesベースのカチオン性脂質 - Google Patents
Tesベースのカチオン性脂質Info
- Publication number
- JP7844452B2 JP7844452B2 JP2023518358A JP2023518358A JP7844452B2 JP 7844452 B2 JP7844452 B2 JP 7844452B2 JP 2023518358 A JP2023518358 A JP 2023518358A JP 2023518358 A JP2023518358 A JP 2023518358A JP 7844452 B2 JP7844452 B2 JP 7844452B2
- Authority
- JP
- Japan
- Prior art keywords
- alkyl
- mol
- lipids
- independently selected
- alkenyl
- 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.)
- Active
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C311/00—Amides of sulfonic acids, i.e. compounds having singly-bound oxygen atoms of sulfo groups replaced by nitrogen atoms, not being part of nitro or nitroso groups
- C07C311/30—Sulfonamides, the carbon skeleton of the acid part being further substituted by singly-bound nitrogen atoms, not being part of nitro or nitroso groups
- C07C311/31—Sulfonamides, the carbon skeleton of the acid part being further substituted by singly-bound nitrogen atoms, not being part of nitro or nitroso groups having the sulfur atoms of the sulfonamide groups bound to acyclic carbon atoms
- C07C311/32—Sulfonamides, the carbon skeleton of the acid part being further substituted by singly-bound nitrogen atoms, not being part of nitro or nitroso groups having the sulfur atoms of the sulfonamide groups bound to acyclic carbon atoms of an acyclic saturated carbon skeleton
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/20—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing sulfur, e.g. dimethyl sulfoxide [DMSO], docusate, sodium lauryl sulfate or aminosulfonic acids
-
- 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
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/007—Pulmonary tract; Aromatherapy
-
- 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
- A61K9/107—Emulsions ; Emulsion preconcentrates; Micelles
- A61K9/1075—Microemulsions or submicron emulsions; Preconcentrates or solids thereof; Micelles, e.g. made of phospholipids or block copolymers
-
- 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
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C309/00—Sulfonic acids; Halides, esters, or anhydrides thereof
- C07C309/63—Esters of sulfonic acids
- C07C309/64—Esters of sulfonic acids having sulfur atoms of esterified sulfo groups bound to acyclic carbon atoms
- C07C309/69—Esters of sulfonic acids having sulfur atoms of esterified sulfo groups bound to acyclic carbon atoms of a carbon skeleton substituted by nitrogen atoms, not being part of nitro or nitroso groups
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C311/00—Amides of sulfonic acids, i.e. compounds having singly-bound oxygen atoms of sulfo groups replaced by nitrogen atoms, not being part of nitro or nitroso groups
- C07C311/30—Sulfonamides, the carbon skeleton of the acid part being further substituted by singly-bound nitrogen atoms, not being part of nitro or nitroso groups
- C07C311/31—Sulfonamides, the carbon skeleton of the acid part being further substituted by singly-bound nitrogen atoms, not being part of nitro or nitroso groups having the sulfur atoms of the sulfonamide groups bound to acyclic carbon atoms
- C07C311/34—Sulfonamides, the carbon skeleton of the acid part being further substituted by singly-bound nitrogen atoms, not being part of nitro or nitroso groups having the sulfur atoms of the sulfonamide groups bound to acyclic carbon atoms of an acyclic unsaturated carbon skeleton
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Life Sciences & Earth Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Dispersion Chemistry (AREA)
- Oncology (AREA)
- Immunology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Communicable Diseases (AREA)
- Pulmonology (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Manufacturing & Machinery (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063082090P | 2020-09-23 | 2020-09-23 | |
| US63/082,090 | 2020-09-23 | ||
| PCT/US2021/051403 WO2022066678A1 (en) | 2020-09-23 | 2021-09-22 | Tes-based cationic lipids |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023542945A JP2023542945A (ja) | 2023-10-12 |
| JP2023542945A5 JP2023542945A5 (https=) | 2024-10-01 |
| JP7844452B2 true JP7844452B2 (ja) | 2026-04-13 |
Family
ID=78179524
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023518358A Active JP7844452B2 (ja) | 2020-09-23 | 2021-09-22 | Tesベースのカチオン性脂質 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20230357140A1 (https=) |
| EP (1) | EP4217340A1 (https=) |
| JP (1) | JP7844452B2 (https=) |
| KR (1) | KR20230092914A (https=) |
| CN (1) | CN116507606B (https=) |
| AU (1) | AU2021347814A1 (https=) |
| BR (1) | BR112023005269A2 (https=) |
| CA (1) | CA3193340A1 (https=) |
| IL (1) | IL301540A (https=) |
| MX (1) | MX2023003345A (https=) |
| WO (1) | WO2022066678A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4637813A1 (en) | 2022-12-20 | 2025-10-29 | Sanofi Pasteur | Rhinovirus mrna vaccine |
| CN117986153A (zh) * | 2023-09-07 | 2024-05-07 | 晟迪生物医药(苏州)有限公司 | 一种可电离阳离子脂质化合物及其制备方法和应用 |
| WO2026008743A1 (en) | 2024-07-02 | 2026-01-08 | Sanofi Pasteur Inc. | Water-soluble polyanionic polymer as adjuvant for carrier-formulated nucleic acid |
| WO2026052114A1 (zh) * | 2024-09-09 | 2026-03-12 | 北京科兴中维生物技术有限公司 | 脂质纳米颗粒及其应用 |
Citations (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2007510662A (ja) | 2003-11-04 | 2007-04-26 | エリクシアー ファーマシューティカルズ, インコーポレイテッド | 治療化合物およびその使用 |
| WO2015095340A1 (en) | 2013-12-19 | 2015-06-25 | Novartis Ag | Lipids and lipid compositions for the delivery of active agents |
| WO2018033883A2 (en) | 2016-08-18 | 2018-02-22 | University Of Kwazulu Natal | Ph-responsive lipids |
| JP2019519503A (ja) | 2016-05-16 | 2019-07-11 | ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム | カチオン性スルホンアミドアミノ脂質および両親媒性両性イオンアミノ脂質 |
| 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 |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4500707A (en) | 1980-02-29 | 1985-02-19 | University Patents, Inc. | Nucleosides useful in the preparation of polynucleotides |
| US4458066A (en) | 1980-02-29 | 1984-07-03 | University Patents, Inc. | Process for preparing polynucleotides |
| US5132418A (en) | 1980-02-29 | 1992-07-21 | University Patents, Inc. | Process for preparing polynucleotides |
| US4415732A (en) | 1981-03-27 | 1983-11-15 | University Patents, Inc. | Phosphoramidite compounds and processes |
| US4668777A (en) | 1981-03-27 | 1987-05-26 | University Patents, Inc. | Phosphoramidite nucleoside compounds |
| US4973679A (en) | 1981-03-27 | 1990-11-27 | University Patents, Inc. | Process for oligonucleo tide synthesis using phosphormidite intermediates |
| US4401796A (en) | 1981-04-30 | 1983-08-30 | City Of Hope Research Institute | Solid-phase synthesis of polynucleotides |
| US4373071A (en) | 1981-04-30 | 1983-02-08 | 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 |
| US5047524A (en) | 1988-12-21 | 1991-09-10 | Applied Biosystems, Inc. | Automated system for polynucleotide synthesis and purification |
| US5262530A (en) | 1988-12-21 | 1993-11-16 | Applied Biosystems, Inc. | Automated system for polynucleotide synthesis and purification |
| CA2064783C (en) * | 1989-08-29 | 2002-12-17 | Richard C. Franson | Cytoprotective fatty moiety compounds |
| 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 |
| US20100055168A1 (en) * | 2008-04-16 | 2010-03-04 | Abbott Laboratories | Cationic lipids and uses thereof |
| KR101766408B1 (ko) | 2009-06-10 | 2017-08-10 | 알닐람 파마슈티칼스 인코포레이티드 | 향상된 지질 조성물 |
| AU2017357758B2 (en) | 2016-11-10 | 2023-11-16 | Translate Bio, Inc. | Improved process of preparing mRNA-loaded lipid nanoparticles |
| US12479791B2 (en) * | 2018-08-10 | 2025-11-25 | Takeda Pharmaceutical Company Limited | Cationic lipid |
-
2021
- 2021-09-22 WO PCT/US2021/051403 patent/WO2022066678A1/en not_active Ceased
- 2021-09-22 KR KR1020237013612A patent/KR20230092914A/ko active Pending
- 2021-09-22 US US18/246,261 patent/US20230357140A1/en active Pending
- 2021-09-22 CA CA3193340A patent/CA3193340A1/en active Pending
- 2021-09-22 IL IL301540A patent/IL301540A/en unknown
- 2021-09-22 BR BR112023005269A patent/BR112023005269A2/pt unknown
- 2021-09-22 JP JP2023518358A patent/JP7844452B2/ja active Active
- 2021-09-22 MX MX2023003345A patent/MX2023003345A/es unknown
- 2021-09-22 EP EP21791537.0A patent/EP4217340A1/en active Pending
- 2021-09-22 AU AU2021347814A patent/AU2021347814A1/en not_active Abandoned
- 2021-09-22 CN CN202180075717.0A patent/CN116507606B/zh active Active
Patent Citations (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2007510662A (ja) | 2003-11-04 | 2007-04-26 | エリクシアー ファーマシューティカルズ, インコーポレイテッド | 治療化合物およびその使用 |
| WO2015095340A1 (en) | 2013-12-19 | 2015-06-25 | Novartis Ag | Lipids and lipid compositions for the delivery of active agents |
| JP2019519503A (ja) | 2016-05-16 | 2019-07-11 | ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム | カチオン性スルホンアミドアミノ脂質および両親媒性両性イオンアミノ脂質 |
| WO2018033883A2 (en) | 2016-08-18 | 2018-02-22 | University Of Kwazulu Natal | Ph-responsive lipids |
| 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 |
|---|---|
| IL301540A (en) | 2023-05-01 |
| JP2023542945A (ja) | 2023-10-12 |
| CN116507606B (zh) | 2026-03-24 |
| CA3193340A1 (en) | 2022-03-31 |
| AU2021347814A9 (en) | 2024-10-03 |
| AU2021347814A1 (en) | 2023-06-08 |
| EP4217340A1 (en) | 2023-08-02 |
| CN116507606A (zh) | 2023-07-28 |
| BR112023005269A2 (pt) | 2023-04-25 |
| MX2023003345A (es) | 2023-07-24 |
| KR20230092914A (ko) | 2023-06-26 |
| WO2022066678A1 (en) | 2022-03-31 |
| US20230357140A1 (en) | 2023-11-09 |
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