申请人:Translate Bio, Inc.
公开号:US10961184B2
公开(公告)日:2021-03-30
Disclosed are dendritic anionic lipids which are compounds of Formula (I): wherein R and R1 are non-polar groups, L is a linking moiety, and Dm is a dendritic moiety of m generations, each as defined herein. These dendritic anionic lipids are useful for delivery and expression of m RNA 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.
所公开的树枝状阴离子脂质是式(I)化合物:其中R和R1是非极性基团,L是连接基团,Dm是m代树枝状基团,每代均如本文所定义。这些树枝状阴离子脂质可用于递送和表达 m RNA 和编码蛋白质,例如作为脂质体递送载体的组分,因此可用于治疗各种疾病、失调和病症,例如与一种或多种蛋白质缺乏有关的疾病、失调和病症。