CN115667207A - 基于酚酸脂质的阳离子脂质 - Google Patents
基于酚酸脂质的阳离子脂质 Download PDFInfo
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- CN115667207A CN115667207A CN202180039080.XA CN202180039080A CN115667207A CN 115667207 A CN115667207 A CN 115667207A CN 202180039080 A CN202180039080 A CN 202180039080A CN 115667207 A CN115667207 A CN 115667207A
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- optionally substituted
- alkyl
- pharmaceutically acceptable
- acceptable salt
- alkenyl
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- 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
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
- A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
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- 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
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- 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
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- 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
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WO2022204549A1 (fr) | 2021-03-25 | 2022-09-29 | Translate Bio, Inc. | Séquences nucléotidiques optimisées codant pour le domaine extracellulaire de la protéine ace2 humaine ou une portion de celle-ci |
CA3230031A1 (fr) | 2021-09-03 | 2023-03-09 | Patrick Baumhof | Nouvelles nanoparticules lipidiques pour l'administration d'acides nucleiques |
WO2023073228A1 (fr) | 2021-10-29 | 2023-05-04 | CureVac SE | Arn circulaire amélioré pour exprimer des protéines thérapeutiques |
WO2023144330A1 (fr) | 2022-01-28 | 2023-08-03 | CureVac SE | Inhibiteurs de facteurs de transcription codés par un acide nucleique |
WO2023227608A1 (fr) | 2022-05-25 | 2023-11-30 | Glaxosmithkline Biologicals Sa | Vaccin à base d'acide nucléique codant pour un polypeptide antigénique fimh d'escherichia coli |
US20240156949A1 (en) | 2022-10-28 | 2024-05-16 | Glaxosmithkline Biologicals Sa | Nucleic Acid Based Vaccine |
WO2024133515A1 (fr) | 2022-12-20 | 2024-06-27 | Sanofi | Vaccin contre l'arnm de rhinovirus |
WO2024184500A1 (fr) | 2023-03-08 | 2024-09-12 | CureVac SE | Nouvelles formulations de nanoparticules lipidiques pour l'administration d'acides nucléiques |
Citations (6)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO1999052894A1 (fr) * | 1998-04-09 | 1999-10-21 | Meiji Seika Kaisha, Ltd. | Composes heterocycliques a base d'azote anti-agregation plaquettaire et leur utilisation medicale |
CN106659731A (zh) * | 2014-05-30 | 2017-05-10 | 夏尔人类遗传性治疗公司 | 用于递送核酸的可生物降解脂质 |
US20180028664A1 (en) * | 2015-12-10 | 2018-02-01 | Modernatx, Inc. | Compositions and methods for delivery of agents |
WO2019226925A1 (fr) * | 2018-05-24 | 2019-11-28 | Translate Bio, Inc. | Lipides cationiques de thioester |
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 |
WO2020056294A1 (fr) * | 2018-09-14 | 2020-03-19 | Translate Bio, Inc. | Compositions et méthodes de traitement de l'acidémie méthylmalonique |
Family Cites Families (16)
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 |
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 |
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 |
US4668777A (en) | 1981-03-27 | 1987-05-26 | University Patents, Inc. | Phosphoramidite nucleoside compounds |
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 |
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 |
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 |
US20180153822A1 (en) | 2016-11-10 | 2018-06-07 | Translate Bio, Inc. | Process of Preparing mRNA-Loaded Lipid Nanoparticles |
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2021
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- 2021-03-31 CN CN202180039080.XA patent/CN115667207A/zh active Pending
- 2021-03-31 EP EP21720636.6A patent/EP4126813A1/fr active Pending
- 2021-03-31 WO PCT/US2021/025128 patent/WO2021202694A1/fr unknown
Patent Citations (6)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO1999052894A1 (fr) * | 1998-04-09 | 1999-10-21 | Meiji Seika Kaisha, Ltd. | Composes heterocycliques a base d'azote anti-agregation plaquettaire et leur utilisation medicale |
CN106659731A (zh) * | 2014-05-30 | 2017-05-10 | 夏尔人类遗传性治疗公司 | 用于递送核酸的可生物降解脂质 |
US20180028664A1 (en) * | 2015-12-10 | 2018-02-01 | Modernatx, Inc. | Compositions and methods for delivery of agents |
WO2019226925A1 (fr) * | 2018-05-24 | 2019-11-28 | Translate Bio, Inc. | Lipides cationiques de thioester |
WO2020056294A1 (fr) * | 2018-09-14 | 2020-03-19 | Translate Bio, Inc. | Compositions et méthodes de traitement de l'acidémie méthylmalonique |
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 |
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