Patent ID: 11858884
Assignee: BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A | IPC A  C

Claim 43:
44. A method for delivering a nucleic acid to a cell of a subject, the method comprising administering to said subject said nucleic acid, wherein said nucleic acid is formulated with a nanoparticle comprising a compound having the structure of Formula (I):

Core-Repeating Unit-Terminating Group,

or a pharmaceutically acceptable salt thereof,
wherein:
the core is linked to two to six repeating units, and each repeating unit is linked to a nitrogen of the core, wherein:
the core corresponds to the structure of Formula (II):, wherein, in Formula (II):
X1 is optionally substituted
dialkylamino(C≤12), optionally substituted heterocycloalkyl(C≤12), or optionally substituted heteroaryl(C≤12);
R1 is amino, hydroxy, mercapto, optionally substituted alkylamino(C≤12), or optionally substituted dialkylamino(C≤12); and
a is 1, 2, 3, 4, 5, or 6; or
alternatively, the core corresponds to the structure of Formula (III):, wherein, in Formula (III):
X2 is N(R5)y, wherein:
R5 is hydrogen or optionally substituted alkyl(C≤18); and
y is 0, 1, or 2, provided that the sum of y and z is 3;
R2 is amino, hydroxy, mercapto, optionally substituted alkylamino(C≤12), or optionally substituted dialkylamino(C≤12);
b is 1, 2, 3, 4, 5, or 6; and
z is 1, 2, 3; provided that the sum of z and y is 3; or
alternatively, the core corresponds to the structure of Formula (IV):, wherein, in Formula (IV):
X3 is selected from —NR6—, —O—, optionally substituted alkylaminodiyl(C≤8), optionally substituted alkoxydiyl(C≤8), optionally substituted arenediyl(C≤8), optionally substituted heteroarenediyl(C≤8), and optionally substituted heterocycloalkanediyl(C≤8),
wherein R6 is hydrogen or optionally substituted alkyl(C≤8);
R3 and R4 are each independently amino, hydroxy, mercapto, optionally substituted alkylamino(C≤12), optionally substituted dialkylamino(C≤12), or a group of the formula:, wherein:
e is 1, 2, or 3; and
Rc and Rd are each independently hydrogen, or optionally substituted alkyl(C≤6); and
c and d are each independently 1, 2, 3, 4, 5, or 6;
each repeating unit
is a degradable diacyl group of Formula (VII):, wherein, in Formula (VII):
A1 and A2 are each independently —O—; and
Y3 is optionally substituted alkanediyl(C≤12), optionally substituted alkenediyl(C≤12), or optionally substituted arenediyl(C≤12); and
R9 is optionally substituted alkyl(C≤8); wherein the core contains at least one nitrogen atom and
each terminating group has the structure of Formula (VIII):, wherein, in Formula (VIII):
Y4 is alkanediyl(C≤18) optionally substituted with one or more substituents each independently selected from the group consisting of —OH, —F, —Cl, —Br, —I, —SH, —OCH3, —OCH2CH3, —SCH3, and —OC(O)CH3; and
R10 is hydrogen,
wherein a protein expressed by said nucleic acid is present in a cell of the subject following said administration of said nucleic acid formulated with said nanoparticle.