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US 2021/0261502 A1Published application
PolyAnionic Delivery of Nucleic Acids
AssigneeTranslate Bio, Inc.
InventorsMichael Heartlein, Frank DeRosa, Shrirang Karve
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Abstract
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.
Claims15
A compound selected from the group consisting of:
-16. (canceled)
Dependencies are inferred from the claim text.