公开(公告)号：EP3934673A1

公开(公告)日：2022-01-12

申请号：EP20704372.0

申请日：2020-01-10

申请人： Massachusetts Institute of Technology ,  THE OHIO STATE UNIVERSITY

发明人： IRVINE, Darrell, J. ,  WITTRUP, Karl, Dane ,  WEISS, Ron ,  LI, Yingzhong ,  MOMIN, Noor ,  DONG, Yizhou

IPC分类号： A61K35/768 ,  A61K31/7105 ,  C07K14/54 ,  C12N15/86 ,  C12N15/88

公开(公告)号：EP4074694A1

公开(公告)日：2022-10-19

申请号：EP22158860.1

申请日：2012-10-26

申请人： Massachusetts Institute Of Technology

发明人： DONG, Yizhou ,  LOVE, Kevin Thomas ,  LANGER, Robert S. ,  ANDERSON, Daniel Griffith ,  CHEN, Delai ,  CHEN, Yi ,  VEGAS, Arturo Jose ,  ALABI, Akinleye ,  ZHANG, Yunlong

IPC分类号： C07D207/16 ,  C07D209/20 ,  C07D233/64 ,  C07D241/08 ,  C07D403/06 ,  C07D487/06 ,  C07C227/18 ,  C07C229/12 ,  C07C229/16 ,  C07C229/22 ,  C07C229/26 ,  C07C229/28 ,  A61K48/00 ,  C12N15/87

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', R 1 , R 2 , R 3 , R 4 , R 5 , R 8 , Z, W, Y, and Z are as defined herein.

公开(公告)号：EP4452323A1

公开(公告)日：2024-10-30

申请号：EP22912380.7

申请日：2022-12-20

申请人： Ohio State Innovation Foundation ,  MASSACHUSETTS INSTITUTE OF TECHNOLOGY

发明人： WEISS, Ron ,  DONG, Yizhou ,  DU, Shi ,  LI, Wenqing ,  ZHANG, Yuebao ,  IRVINE, Darrell

IPC分类号： A61K47/28 ,  A61K31/56 ,  A61K31/575 ,  A61K31/7088 ,  A61K47/24 ,  A61P15/10 ,  A61P35/00

公开(公告)号：EP3871696A1

公开(公告)日：2021-09-01

申请号：EP21158237.4

申请日：2014-10-22

申请人： Translate Bio MA, Inc. ,  Massachusetts Institute Of Technology

发明人： HEARTLEIN, Michael ,  ANDERSON, Daniel ,  DONG, Yizhou ,  DEROSA, Frank

IPC分类号： A61K48/00 ,  C12N15/89

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：EP3060257A1

公开(公告)日：2016-08-31

申请号：EP14792713.1

申请日：2014-10-22

申请人： Shire Human Genetic Therapies, Inc. ,  Massachusetts Institute of Technology

发明人： HEARTLEIN, Michael ,  ANDERSON, Daniel ,  DONG, Yizhou ,  DEROSA, Frank

IPC分类号： A61K48/00 ,  C12N15/89

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.

摘要翻译： 本发明尤其提供了在体内递送mRNA的方法，包括向需要递送的受试者施用包含在脂质体内的编码蛋白质的mRNA的组合物，使得组合物的施用导致表达 由mRNA在体内编码的蛋白质，其中所述脂质体包含式Ic阳离子脂质或其药学上可接受的盐。

公开(公告)号：EP2771319A1

公开(公告)日：2014-09-03

申请号：EP12783791.2

申请日：2012-10-26

申请人： Massachusetts Institute Of Technology

发明人： DONG, Yizhou ,  LOVE, Kevin, Thomas ,  LANGER, Robert, S. ,  ANDERSON, Daniel, Griffith ,  CHEN, Delai ,  CHEN, Yi ,  VEGAS, Arturo, Jose ,  ALABI, Akinleye ,  ZHANG, Yunlong

IPC分类号： C07D207/16 ,  C07D209/20 ,  C07D233/64 ,  C07D241/08 ,  C07D403/06 ,  C07D487/06 ,  C07C227/18 ,  C07C229/12 ,  C07C229/16 ,  C07C229/22 ,  C07C229/26 ,  C07C229/28 ,  A61K48/00 ,  C12N15/87

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： worin M，N，P，R”，R1，R2，R3，R4，R5，R8，Z，W，Y，和Z如在定义。