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US 12,655,095 B2Granted
TES-based cationic lipids
AssigneeTranslate Bio, Inc.
InventorsShrirang Karve, Frank Derosa
Life of this patent
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Abstract
The present invention provides, in part, TES-based lipid compounds of Formula (Ia), 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.
Claims22
A compound having a structure according to Formula (Ia):
- or a pharmaceutically acceptable salt thereof, wherein
- R1, R2 and R3 are each independently selected from:
- C1-C20 alkyl, C2-C20 alkenyl, C2-C20 alkynyl, and acyl;
- R4 is
- each R5 is independently selected from C1-C20 alkyl, C2-C20 alkenyl, and C2-C20 alkynyl;
- each R6 is independently selected from hydrogen, halogen, C1-C6 alkyl, and C2-C6 alkenyl;
- A is —NR9— or —O—;
- D is O;
- E and G are each independently selected from —NR10— and —O—;
- R8, R9, and R10 are each independently selected from hydrogen, C1-C6 alkyl, and C2-C6 alkenyl;
- each b is independently selected from 0, 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10;
- and
- each c is independently selected from 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10;
- wherein acyl refers to RZ—(C═O)— and RZ is C1-C20 alkyl, C2-C20 alkenyl, or C2-C20 alkynyl.
11 more elements
A compound selected from the group consisting of:
- or a pharmaceutically acceptable salt thereof.
Dependencies are inferred from the claim text.