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US 12,404,232 B2Granted
Compounds and compositions for intracellular delivery of therapeutic agents
AssigneeModernaTX, Inc.
InventorsKerry E. Benenato, Mark Cornebise
Life of this patent
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Abstract
The disclosure features novel lipids and compositions involving the same. Nanoparticle compositions include a novel lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Nanoparticle compositions further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
Claims15
A compound of Formula (I):
- or a salt or isomer thereof, wherein:
- R1 is selected from the group consisting of C5-30 alkyl, C5-20 alkenyl, —R*YR″, —YR″, and —R″M′R′;
- R2 and R3 are independently selected from the group consisting of H, C1-14 alkyl, C2-14 alkenyl, —R*YR″, —YR″, and —R*OR″, or R2 and R3, together with the atom to which they are attached, form a heterocycle or carbocycle;
- R4 is selected from the group consisting of
- each R5 is independently selected from the group consisting of C1-3 alkyl, C2-3 alkenyl, and H;
- each R6 is independently selected from the group consisting of C1-3 alkyl, C2-3 alkenyl, and H;
- M and M′ are independently selected from —C(O)O—, —OC(O)—, —C(O)N(R′)—, —N(R′) C(O)—, —C(O)—, —C(S)—, —C(S)S—, —SC(S)—, —CH(OH)—, —P(O) (OR′)O—, —S(O)2—, —S—S—, an aryl group, and a heteroaryl group;
- R7 is selected from the group consisting of C1-3 alkyl, C2-3 alkenyl, and H;
- each R′ is independently selected from the group consisting of C4-18 alkyl, C4-18 alkenyl, —R*YR″, —YR″, and H;
- each R″ is independently selected from the group consisting of C3-14 alkyl and C3-14 alkenyl;
- each R* is independently selected from the group consisting of C1-12 alkyl and C2-12 alkenyl;
- each Y is independently a C3-6 carbocycle;
- and
- m is selected from 5, 6, 7, 8, 9, 10, 11, 12, and 13.
11 more elements
Dependencies are inferred from the claim text.