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US 2024/0391869 A1Published application
Lipocationic dendrimers and uses thereof
AssigneeBoard of Regents, The University of Texas System
InventorsDaniel J. SIEGWART, Kejin ZHOU
Life of this patent
Estimating the term…
Abstract
Modular dendrimers with cationic groups and lipophilic groups are provided herein. In some aspects, the dendrimers provided herein may be formulated in compositions which contain a nucleic acid and one or more helper excipients. In some aspects, these compositions may also be used to treat diseases or disorders with a therapeutic nucleic acid.
Claims24
A composition comprising:
- I. a compound of Formula (I):
- Core-Repeating Unit-Terminating Group (I),
- wherein the core is linked to four to six repeating units and each repeating unit is linked to a nitrogen of the core, wherein the core has the following structure:
- wherein:
- X3 is —NR6— or heterocycloalkanediylC≤8, wherein R6 is hydrogen or alkylC≤8;
- R3 and R4 are each independently amino, alkylaminoC≤12, or dialkylaminoC≤12;
- wherein the alkylamino or dialkylamino are each optionally substituted with amino;
- each repeating unit comprises a degradable diacyl having the formula:
- wherein:
- A1 and A2 are each independently —O—,
- Y3 is —CH2CH2—, and
- R9 is —CH3;
- and
- the terminating group is selected from the group consisting of:
- II. a nucleic acid;
- III. a steroid or a steroid derivative;
- and
- IV. a phospholipid.
15 more elements
A composition comprising:
- I. a compound of Formula (I):
- Core-Repeating Unit-Terminating Group (I),
- wherein the core is linked to four to six repeating units and each repeating unit is linked to a nitrogen of the core, wherein the core has the following structure:
- wherein:
- X3 is —NR6— or heterocycloalkanediylC≤8, wherein R6 is hydrogen or alkylC≤8;
- R3 and R4 are each independently amino, alkylaminoC≤12, or dialkylaminoC≤12;
- wherein the alkylamino or dialkylamino are each optionally substituted with amino;
- each repeating unit comprises a degradable diacyl having the formula:
- wherein:
- A1 and A2 are each independently —O—,
- Y3 is —CH2CH2—, and
- R9 is —CH3;
- and
- the terminating group is selected from the group consisting of:
- II. an mRNA
- III. cholesterol;
- IV. a phospholipid selected from 1,2-distearoyl-sn-glycero-3-phosphocholine (DSPC) and 1,2-dioleoyl-sn-glycero-3-phosphoethanolamine (DOPE);
- and
- V. a PEG lipid, wherein the PEG lipid is a compound of the formula:
- wherein:
- R12 and R13 are each independently alkyl(C≤24), alkenyl(C≤24), or a substituted version of either of these groups;
- Re is hydrogen, alkyl(C≤8), or substituted alkyl(C≤8);
- and
- x is 1-250.
21 more elements
Dependencies are inferred from the claim text.