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US 12,747,198 B2Granted
Phenolic acid lipid based cationic lipids
AssigneeTranslate Bio, Inc.
InventorsShrirang Karve, Frank DeRosa, Apiwat Wangweerawong, Saswata Karmakar
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Abstract
The present invention provides, in part, phenolic acid lipid compounds of Formula (I), 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.
Claims19
A cationic lipid having a structure according to Formula (I):
- wherein L1 is a bond, (C1-C6)alkyl or (C2-C6)alkenyl;
- wherein X is O or S;
- wherein R1, R2, R3, R4 and R5 are each independently selected from H, OH, optionally substituted (C1-C6)alkyl, optionally substituted (C2-C6)alkenyl, optionally substituted (C2-C6)alkynyl, optionally substituted (C1-C6)alkoxy and —OC(O)R′;
- wherein at least one of R1, R2, R3, R4 or R5 is —OC(O)R′;
- wherein R′ is
- wherein R6 is
- wherein m and p are each independently 0, 1, 2, 3, 4 or 5;
- wherein R7 is selected from H, optionally substituted (C1-C6)alkyl, optionally substituted (C2-C6)alkenyl, optionally substituted (C2-C6)alkynyl, optionally substituted (C1-C6)acyl, —(CH2)kRA or —(CH2)kCH(OR11)RA;
- wherein R8 is selected from H, optionally substituted (C1-C6)alkyl, optionally substituted (C2-C6)alkenyl, optionally substituted (C2-C6)alkynyl, optionally substituted (C1-C6)acyl, —(CH2)nRB or —(CH2)nCH(OR12)RB;
- wherein R9 is selected from H, optionally substituted (C1-C6)alkyl, optionally substituted (C2-C6)alkenyl, optionally substituted (C2-C6)alkynyl, optionally substituted (C1-C6)acyl, —(CH2)qRC or —(CH2)qCH(OR13)RC;
- wherein R10 is selected from H, optionally substituted (C1-C6)alkyl, optionally substituted (C2-C6)alkenyl, optionally substituted (C2-C6)alkynyl, optionally substituted (C1-C6)acyl, —(CH2)rRD or —(CH2)rCH(OR14)RD;
- wherein k, n, q and r are each independently 1,2,3,4 or 5;
- or wherein (i) R7 and R8 or (ii) R9 and R10 together form an optionally substituted 5- or 6-membered heterocycloalkyl or heteroaryl wherein the heterocycloalkyl or heteroaryl comprises 1 to 3 heteroatoms selected from N, O and S;
- wherein R11, R12, R13 and R14 are each independently selected from H, methyl, ethyl or propyl;
- wherein RA, RB, RC and RD are each independently selected from optionally substituted (C6-C20)alkyl, optionally substituted (C6-C20)alkenyl, optionally substituted (C6-C20)alkynyl, optionally substituted (C6-C20)acyl, optionally substituted —OC(O)alkyl, optionally substituted —OC(O)alkenyl, optionally substituted (C1-C6) monoalkylamino, optionally substituted (C1-C6) dialkylamino, optionally substituted (C1-C6)alkoxy, —OH, —NH2;
- wherein at least one of R7, R8, R9, R10 comprises a RA, RB, RC or RD moiety respectively wherein that RA, RB, RC or RD is independently selected from optionally substituted (C6-C20)alkyl, optionally substituted (C6-C20)alkenyl, optionally substituted (C6-C20)alkynyl, optionally substituted (C6-C20)acyl, optionally substituted —OC(O)(C6-C20)alkyl or optionally substituted —OC(O)(C6-C20)alkenyl;
- or a pharmaceutically acceptable salt thereof.
14 more elements
A cationic lipid that is:
- or a pharmaceutically acceptable salt thereof.
A cationic lipid that is:
- or a pharmaceutically acceptable salt thereof.
Dependencies are inferred from the claim text.