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公开(公告)号：US20220347099A1
公开(公告)日：2022-11-03
申请号：US17162656
申请日：2021-01-29
发明人： Michael Heartlein , Daniel Anderson , Yizhou Dong , Frank DeRosa
摘要： The present invention provides, among other things, methods of delivering mRNA in vivo, including administering to a subject in need of delivery a composition comprising an mRNA encoding a protein, encapsulated within a liposome such that the administering of the composition results in the expression of the protein encoded by the mRNA in vivo, wherein the liposome comprises a cationic lipid of formula I-c: or a pharmaceutically acceptable salt thereof.
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公开(公告)号：US09629804B2
公开(公告)日：2017-04-25
申请号：US14521161
申请日：2014-10-22
发明人： Michael Heartlein , Daniel Anderson , Yizhou Dong , Frank DeRosa
CPC分类号： A61K9/1272 , A61K38/1709 , A61K38/177 , A61K38/4846 , A61K38/53 , A61K48/00 , A61K48/0033 , A61K48/005
摘要： The present invention provides, among other things, methods of delivering mRNA in vivo, including administering to a subject in need of delivery a composition comprising an mRNA encoding a protein, encapsulated within a liposome such that the administering of the composition results in the expression of the protein encoded by the mRNA in vivo, wherein the liposome comprises a cationic lipid of formula I-c: or a pharmaceutically acceptable salt thereof.
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公开(公告)号：US09315472B2
公开(公告)日：2016-04-19
申请号：US14267530
申请日：2014-05-01
发明人： Yizhou Dong , Akinleye C. Alabi , Hao Yin , Joseph R. Dorkin , Delai Chen , Robert S. Langer , Daniel Griffith Anderson , Ahmed A. Eltoukhy
IPC分类号： C07D251/30 , A61K31/53 , A61K31/713
CPC分类号： C07D251/30 , A61K31/53 , A61K31/713
摘要： The present invention provides novel 1,3,5-triazinane-2,4,6-trione derivatives, such as compounds of any one of Formulae (I) and (II), and salts thereof, and methods of preparing the compounds. Also provided are compositions including a compound of the invention and an agent (e.g., an siRNA, mRNA, or plasmid DNA). The present invention also provides methods and kits using the compositions for delivering an agent to a subject (e.g., to the liver, spleen, or lung of the subject) or cell and for treating and/or preventing a range of diseases, such as genetic diseases, proliferative diseases, hematological diseases, neurological diseases, liver diseases, and lung diseases.
摘要翻译： 本发明提供新的1,3,5-三氮杂环丁烷-2,4,6-三酮衍生物，如式（I）和（II）中任一项的化合物及其盐，以及制备该化合物的方法。 还提供了包含本发明化合物和试剂（例如siRNA，mRNA或质粒DNA）的组合物。 本发明还提供了使用组合物将试剂递送至受试者（例如，受试者的肝脏，脾脏或肺）或细胞的方法和试剂盒，并用于治疗和/或预防一系列疾病，例如遗传 疾病，增殖性疾病，血液疾病，神经系统疾病，肝脏疾病和肺部疾病。
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公开(公告)号：US20240024394A1
公开(公告)日：2024-01-25
申请号：US18334449
申请日：2023-06-14
发明人： Darrell J. Irvine , Karl Dane Wittrup , Ron Weiss , Yingzhong Li , Noor Momin , Yizhou Dong
IPC分类号： A61K35/768 , A61P35/00 , A61K9/00 , C07K14/54 , C12N7/00
CPC分类号： A61K35/768 , A61P35/00 , A61K9/0019 , C07K14/5434 , C12N7/00 , C12N2770/36132
摘要： The present disclosure relates to synthetic oncolytic viruses comprising a lipid nanoparticle comprising one or more types of lipid and a self-amplifying replicon RNA comprising a sequence that encodes an immunomodulatory molecule.
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公开(公告)号：US20230124955A1
公开(公告)日：2023-04-20
申请号：US17893918
申请日：2022-08-23
发明人： Yizhou Dong , Kevin Thomas Love , Robert S. Langer , Daniel Griffith Anderson , Delai Chen , Yi Chen , Arturo Jose Vegas , Akinleye C. Alabi , Yunlong Zhang
IPC分类号： A61K31/7105 , C07D233/64 , C07D403/06 , C07D241/08 , C07C229/12 , C07C229/22 , C07C229/26 , C07D487/06 , C07D207/16 , C07D209/20 , C07C271/22 , C07C229/36 , C07C237/08 , C07C237/12 , C12N15/88 , C12N15/87 , C07C229/24 , C07C279/14 , C07C323/58 , C07D209/24 , C07D265/32 , C07D413/06 , C07D487/04 , C12Q1/02 , G01N33/15 , A61K31/711 , A61K47/22
摘要： Described herein are compounds and compositions characterized, in certain embodiments, by conjugation of various groups, such as lipophilic groups, to an amino or amide group of an amino acid, a linear or cyclic peptide, a linear or cyclic polypeptide, or structural isomer thereof, to provide compounds of the present invention, collectively referred to herein as “APPLs”. Such APPLs are deemed useful for a variety of applications, such as, for example, improved nucleotide delivery. Exemplary APPLs include, but are not limited to, compounds of Formula (I), (II), (III), (IV), (V), and (VI), and salts thereof, as described herein: wherein m, n, p, R′, R1, R2, R3, R4, R5, R8, Z, W, Y, and Z are as defined herein.
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公开(公告)号：US20200281994A1
公开(公告)日：2020-09-10
申请号：US16739407
申请日：2020-01-10
发明人： Darrell J. Irvine , Karl Dane Wittrup , Ron Weiss , Yingzhong Li , Noor Momin , Yizhou Dong
IPC分类号： A61K35/768 , C12N7/00 , C07K14/54 , A61K9/00 , A61P35/00
摘要： The present disclosure relates to synthetic oncolytic viruses comprising a lipid nanoparticle comprising one or more types of lipid and a self-amplifying replicon RNA comprising a sequence that encodes an immunomodulatory molecule.
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公开(公告)号：US20170165290A1
公开(公告)日：2017-06-15
申请号：US15340082
申请日：2016-11-01
发明人： Yizhou Dong , Kevin Thomas Love , Robert S. Langer , Daniel Griffith Anderson , Delai Chen , Yi Chen , Arturo Jose Vegas , Akinleye C. Alabi , Yunlong Zhang
IPC分类号： A61K31/7105 , A61K47/22 , C07D241/08 , A61K31/711
CPC分类号： A61K31/7105 , A61K31/711 , A61K47/22 , A61K2121/00 , C07C229/12 , C07C229/22 , C07C229/24 , C07C229/26 , C07C229/36 , C07C237/08 , C07C237/12 , C07C271/22 , C07C279/14 , C07C323/58 , C07D207/16 , C07D209/20 , C07D209/24 , C07D233/64 , C07D241/08 , C07D265/32 , C07D403/06 , C07D413/06 , C07D487/04 , C07D487/06 , C12N15/87 , C12N15/88 , C12Q1/025 , G01N33/15
摘要： Described herein are compounds and compositions characterized, in certain embodiments, by conjugation of various groups, such as lipophilic groups, to an amino or amide group of an amino acid, a linear or cyclic peptide, a linear or cyclic polypeptide, or structural isomer thereof, to provide compounds of the present invention, collectively referred to herein as “APPLs”. Such APPLs are deemed useful for a variety of applications, such as, for example, improved nucleotide delivery. Exemplary APPLs include, but are not limited to, compounds of Formula (I), (II), (III), (IV), (V), and (VI), and salts thereof, as described herein: wherein m, n, p, R′, R1, R2, R3, R4, R5, R8, Z, W, Y, and Z are as defined herein.
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8.发明申请AMINO ACID-, PEPTIDE-AND POLYPEPTIDE-LIPIDS, ISOMERS, COMPOSITIONS, AND USES THEREOF 有权氨基酸，肽和多肽脂肪，异构体，组合物及其用途公开(公告)号：US20130158021A1
公开(公告)日：2013-06-20
申请号：US13662002
申请日：2012-10-26
发明人： Yizhou Dong , Kevin Thomas Love , Robert S. Langer , Daniel Griffith Anderson , Delai Chen , Yi Chen , Arturo Jose Vagas , Akinleye Alabi , Yunlong Zhang
IPC分类号： C07C323/58 , C12Q1/02 , C07D241/08 , C12N15/87 , C07D487/04 , C07D413/06 , C07C279/14 , C07C229/12 , C07C229/24 , C07D209/24 , C07D233/64 , C07D265/32 , G01N33/15 , C07D403/06
CPC分类号： A61K31/7105 , A61K31/711 , A61K47/22 , A61K2121/00 , C07C229/12 , C07C229/22 , C07C229/24 , C07C229/26 , C07C229/36 , C07C237/08 , C07C237/12 , C07C271/22 , C07C279/14 , C07C323/58 , C07D207/16 , C07D209/20 , C07D209/24 , C07D233/64 , C07D241/08 , C07D265/32 , C07D403/06 , C07D413/06 , C07D487/04 , C07D487/06 , C12N15/87 , C12N15/88 , C12Q1/025 , G01N33/15
摘要： Described herein are compounds and compositions characterized, in certain embodiments, by conjugation of various groups, such as lipophilic groups, to an amino or amide group of an amino acid, a linear or cyclic peptide, a linear or cyclic polypeptide, or structural isomer thereof, to provide compounds of the present invention, collectively referred to herein as “APPLs”. Such APPLs are deemed useful for a variety of applications, such as, for example, improved nucleotide delivery. Exemplary APPLs include, but are not limited to, compounds of Formula (I), (II), (III), (IV), (V), and (VI), and salts thereof, as described herein: wherein m, n, p, R′, R1, R2, R3, R4, R5, R8, Z, W, Y, and Z are as defined herein.
摘要翻译： 本文描述的是化合物和组合物，在某些实施方案中，通过将各种基团如亲脂基团与氨基酸，线性或环状肽，线性或环状多肽或其结构异构体的氨基或酰胺基团缀合， ，以提供本文共同称为“应用”的本发明化合物。 这样的APPL被认为对于各种应用是有用的，例如改进的核苷酸递送。 示例性应用包括但不限于本文所述的式（I），（II），（III），（IV），（V）和（VI）及其盐的化合物：其中m，n ，p，R'，R 1，R 2，R 3，R 4，R 5，R 8，Z，W，Y和Z如本文所定义。
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公开(公告)号：US20240293319A1
公开(公告)日：2024-09-05
申请号：US18545858
申请日：2023-12-19
发明人： Michael Heartlein , Daniel Anderson , Yizhou Dong , Frank DeRosa
CPC分类号： A61K9/1272 , A61K38/1709 , A61K38/177 , A61K38/4846 , A61K38/53 , A61K48/0033 , A61K48/005 , A61K48/00
摘要： The present invention provides, among other things, methods of delivering mRNA in vivo, including administering to a subject in need of delivery a composition comprising an mRNA encoding a protein, encapsulated within a liposome such that the administering of the composition results in the expression of the protein encoded by the mRNA in vivo, wherein the liposome comprises a cationic lipid of formula I-c:
or a pharmaceutically acceptable salt thereof.-
公开(公告)号：US11890377B2
公开(公告)日：2024-02-06
申请号：US17162656
申请日：2021-01-29
发明人： Michael Heartlein , Daniel Anderson , Yizhou Dong , Frank DeRosa
CPC分类号： A61K9/1272 , A61K38/177 , A61K38/1709 , A61K38/4846 , A61K38/53 , A61K48/005 , A61K48/0033 , A61K48/00
摘要： The present invention provides, among other things, methods of delivering mRNA in vivo, including administering to a subject in need of delivery a composition comprising an mRNA encoding a protein, encapsulated within a liposome such that the administering of the composition results in the expression of the protein encoded by the mRNA in vivo, wherein the liposome comprises a cationic lipid of formula I-c:
or a pharmaceutically acceptable salt thereof.
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