Gene delivery agents
AssigneeTIBA Biotech LLC
InventorsPoulami Talukder, Maria Varghese, Heliang Song, Nathan Ivanowsky
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Abstract
Nanoparticle compositions for delivery of nucleic acids to subjects including aminoalkyl branched lipid-like molecules as carriers, and therapeutic or immunogenic nucleic acid agents enclosed within the nanoparticle containing are described. Also provided are methods for treating or preventing diseases or conditions in a subject by administering the nanoparticle compositions that provide immune responses and synergistic therapeutic or preventive effects.
Claims24
A compound of Formula I:
- in which
- YA is H or C1-C4 alkyl;
- ZA is C1-C30 amino, C1-C30 amido, C1-C30 carbonyl, C1-C30 carboxylate, or triazolyl;
- ZB is
- each of LC or LD is O;
- ZC is ZC2-XC-XC-ZC3, in which XC is S, ZC2 is C4-C10 alkylene, and ZC3 is C6-C12 alkyl or alkenyl;
- ZD is C4-C10 alkylene substituted with C6-C12 disulfanyl, in which disulfanyl contains C6-C12 alkyl or alkenyl;
- LK1 is
- or C3-C8 alkylene;
- LK2 is a bond or C1-C8 alkylene;
- each of LK3 and LK4 is —(CH2)2—;
- and
- wherein the compound has 40 to 100 carbon atoms.
A compound selected from the group consisting of Compounds 3, 5, 6, 8, 9, 13, 15, 18, 20-22, and 25-44 as shown below:
Dependencies are inferred from the claim text.