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US 12,698,258 B2Granted
Cystine cationic lipids
AssigneeTranslate Bio, Inc.
InventorsShrirang Karve, Saswata Karmakar, Frank DeRosa
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Abstract
The present invention provides, in part cystine cationic lipid compounds of formula A, 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.
Claims16
A cationic lipid having the following structure:
- or a pharmaceutically acceptable salt thereof,
- wherein
- each R1a is independently hydrogen, R1c, or R1d,
- each R1b is independently R1c or R1d,
- each R1c is independently —(CH2)2C(O)X1R3;
- each R1d is independently —C(O)R4;
- each R2 is independently —[C (R2a)2]cR2b;
- each R2a is independently hydrogen or C1-C6 alkyl;
- R2b is —(OCH2CH2)bOH or —(OCH2CH2)bOCH3;
- each R3 is independently C6-C30 aliphatic;
- each R4 is independently unsubstituted C6-C24 alkyl or C6-C10 alkyl substituted with —OC(O)R5 or —CO2R5, wherein R5 is unsubstituted C6-C16 alkyl;
- each X1 is independently a covalent bond or O;
- each a is independently an integer of 1-10;
- each b is independently an integer of 1-10;
- and
- each c is independently an integer of 1-10.
13 more elements
Dependencies are inferred from the claim text.