Tes-based cationic lipids
AssigneeTranslate Bio, Inc.
InventorsShrirang KARVE, Frank DEROSA
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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.
Claims40
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:
- optionally substituted alkyl, optionally substituted alkenyl, optionally substituted alkynyl and optionally substituted acyl;
- R4 is
- each R5 is independently selected from optionally substituted alkyl, optionally substituted alkenyl and optionally substituted alkynyl;
- each R6 is independently selected from hydrogen, halogen, optionally substituted C1-C6 alkyl, and optionally substituted C2-C6 alkenyl;
- A is —NR9— or —O—;
- D is O or S;
- E and G are each independently selected from —NR10—, —O— and —S—;
- R8, R9, and R10 are each independently selected from hydrogen, optionally substituted C1-C6 alkyl and optionally substituted 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.
The compound according to any one of the preceding claims or a pharmaceutically acceptable salt thereof, wherein R1, R2 and R3 are each independently
The compound according to any one of the preceding claims or a pharmaceutically acceptable salt thereof,
- wherein R1, R2 and R3 are each independently
The compound according to any one of the preceding claims or a pharmaceutically acceptable salt thereof, wherein each R5 is independently selected from optionally substituted alkyl and optionally substituted alkenyl.
The compound according to any one of the preceding claims or a pharmaceutically acceptable salt thereof, wherein each R5 is independently selected from:
The compound of any one of the preceding claims or a pharmaceutically acceptable salt thereof, wherein R4 is
The compound of any one of the preceding claims or a pharmaceutically acceptable salt thereof, wherein R4 is
The compound of any one of the preceding claims or a pharmaceutically acceptable salt thereof, wherein R1, R2 and R3 are each independently:
- and wherein b is 1.
The compound of any one of the preceding claims or a pharmaceutically acceptable salt thereof, wherein R4 is
- and wherein c is 2 or 3.
The compound of any one of the preceding claims or a pharmaceutically acceptable salt thereof, wherein A is NR9, optionally wherein R9 is H.
A compound selected from those listed in Table A or a pharmaceutically acceptable salt thereof.
A composition comprising the cationic lipid of any one of the preceding claims, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.
Dependencies are inferred from the claim text.