公开(公告)号：US20230357166A1

公开(公告)日：2023-11-09

申请号：US18246263

申请日：2021-09-23

申请人： TRANSLATE BIO, INC.

发明人： Saswata KARMAKAR ,  Ramesh DASARI ,  Ryan LANDIS

IPC分类号： C07D241/04 ,  A61K9/127 ,  A61K48/00

CPC分类号： C07D241/04 ,  A61K9/1272 ,  A61K48/0033

摘要： The present invention provides, in part, piperazine-based lipid compounds of Formula (I), and sub-formulas thereof: or a pharmaceutically acceptable salt thereof. The compounds provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.

公开(公告)号：US20240270707A1

公开(公告)日：2024-08-15

申请号：US18555439

申请日：2022-04-15

申请人： TRANSLATE BIO, INC.

发明人： Shrirang KARVE ,  Frank DEROSA ,  Ryan LANDIS ,  Ramesh DASARI ,  Saswata KARMAKAR

IPC分类号： C07D295/088 ,  A61K9/51 ,  A61K39/00

CPC分类号： C07D295/088 ,  A61K9/5123 ,  A61K2039/53 ,  A61K2039/6018

摘要： The present invention provides, among other things, a novel class of cationic lipid compounds (e.g., cationic lipids having a structure according to Formula (I)) for in vivo delivery of therapeutic agents, such as nucleic acids. It is contemplated that these compounds are capable of highly effective in vivo delivery while maintaining a favorable toxicity profile.