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US 12,240,824 B2Granted
Vitamin cationic lipids
AssigneeTranslate Bio, Inc.
InventorsYi Zhang, Shrirang Karve, Saswata Karmakar, Frank DeRosa, Michael Heartlein
Life of this patent
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Abstract
Disclosed are cationic lipids comprising a vitamin substructure and an ionizable nitrogen-containing group. Cationic lipids 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.
Claims17
A liposome encapsulating an mRNA encoding a peptide or polypeptide, wherein the liposome comprises
- one or more cationic lipids, and
- optionally one or more non-cationic lipids, optionally one or more cholesterol-based lipids and optionally one or more PEG-modified lipids;
- and
- wherein the liposome comprises at least one cationic lipid having the structure according to Formula D-A:
- wherein
- represents a single or double bond;
- X1 is NH2, guanidine, amidine, a mono- or dialkylamine, 5- to 6-membered heterocycloalkyl, 5- to 6-membered nitrogen-containing heteroaryl, or
- R3a and R3b are each independently C1-C30-alkyl, C2-C30-alkenyl, or C2-C30-alkynyl;
- n is independently an integer having a value between 1 and 6;
- X2 is O or S;
- Z is O or a covalent bond;
- R1 is C1-C30-alkylene or C2-C30-alkenylene;
- and
- R2 is H or C1-C4-alkyl.
11 more elements
Dependencies are inferred from the claim text.