公开(公告)号：US20230257353A1

公开(公告)日：2023-08-17

申请号：US17875982

申请日：2022-07-28

申请人： Massachusetts Institute of Technology

发明人： Lei Miao ,  Linxian Li ,  Daniel Griffith Anderson ,  Yuxuan Huang

IPC分类号： C07D233/40 ,  A61K39/39 ,  C07C237/06 ,  C07C251/08 ,  C07D403/06

CPC分类号： C07D233/40 ,  A61K39/39 ,  C07C237/06 ,  C07C251/08 ,  C07D403/06 ,  A61K2039/53

摘要： Provided herein are lipidoid compounds of Formulae (I) and (II), and pharmaceutically acceptable salts, co-crystals, tautomers, stereoisomers, solvates, hydrates, polymorphs, isotopically labeled derivatives, prodrugs, and compositions thereof. Also provided are methods and kits involving the inventive lipidoid compounds, compositions, or formulations for treating and/or preventing diseases (e.g., genetic disease, proliferative disease, hematological disease, neurological disease, painful condition, psychiatric disorder, metabolic disorder, long-term medical condition, inflammatory disease, autoinflammatory disease, liver disease, lung disease, spleen disease, familial amyloid neuropathy, cardiovascular disease, viral infection, infectious disease, fibrotic condition, or autoimmune disease) in a subject, methods for synthesizing the compounds described herein, and compounds described herein synthesized by the synthetic methods described herein. The compounds are effective carriers for the delivery of an agent such as a polynucleotide (e.g., RNA) to a cell.

公开(公告)号：US11459304B2

公开(公告)日：2022-10-04

申请号：US16556470

申请日：2019-08-30

申请人： Massachusetts Institute of Technology

发明人： Lei Miao ,  Linxian Li ,  Daniel Griffith Anderson ,  Yuxuan Huang

IPC分类号： C07D233/40 ,  C07C251/08 ,  A61K39/39 ,  C07D403/06 ,  C07C237/06 ,  A61K39/00

摘要： Provided herein are lipidoid compounds of Formulae (I) and (II), and pharmaceutically acceptable salts, co-crystals, tautomers, stereoisomers, solvates, hydrates, polymorphs, isotopically labeled derivatives, prodrugs, and compositions thereof. Also provided are methods and kits involving the inventive lipidoid compounds, compositions, or formulations for treating and/or preventing diseases (e.g., genetic disease, proliferative disease, hematological disease, neurological disease, painful condition, psychiatric disorder, metabolic disorder, long-term medical condition, inflammatory disease, autoinflammatory disease, liver disease, lung disease, spleen disease, familial amyloid neuropathy, cardiovascular disease, viral infection, infectious disease, fibrotic condition, or autoimmune disease) in a subject, methods for synthesizing the compounds described herein, and compounds described herein synthesized by the synthetic methods described herein. The compounds are effective carriers for the delivery of an agent such as a polynucleotide (e.g., RNA) to a cell.