Ionizable cationic lipids
AssigneeTranslate Bio, Inc.
InventorsFrank DeRosa, Braydon Charles Guild, Michael Heartlein
Life of this patent
Estimating the term…
Abstract
Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.
Claims19
-89. (canceled)
A method of treating disease in a subject, wherein the method comprises administering an effective amount of a lipid nanoparticle to the subject, wherein said lipid nanoparticle comprises
- mRNA encoding a therapeutic enzyme or protein;
- a cationic lipid having the following structure
- and
- one or more helper lipids, non-cationic lipids, and/or PEG-modified lipid components.
A method of treating disease in a subject, wherein the method comprises administering an effective amount of a lipid nanoparticle to the subject, wherein said lipid nanoparticle comprises
- mRNA encoding a therapeutic enzyme or protein;
- a cationic lipid having the following structure
- and
- one or more helper lipids, non-cationic lipids, and/or PEG-modified lipid components.
Dependencies are inferred from the claim text.