Lipid containing formulations
Compositions and methods useful in administering nucleic acid based therapies, for example association complexes such as liposomes and lipoplexes are described.
1. A lipid nanoparticle comprising a compound of formula (XV) and a therapeutic agent
wherein;
L 1 and L 2 are both bonds;
each of R 1 and R 2 is independently alkyl, alkenyl or alkynyl; each of which is optionally substituted with one or more substituents;
X is —C(O)NH—, —C(S)NH—, —C(O)C 1-3 alkylC(O)NH—; or —C(O)C 1-3 alkylC(O)O—;
m is an integer from 0-11; and n is an integer from 1-500.
2. The lipid nanoparticle of claim 1 , wherein both R 1 and R 2 are alkyl.
3. The lipid nanoparticle of claim 1 , wherein both R 1 and R 2 are alkenyl.
4. The lipid nanoparticle of claim 1 , wherein the compound of formula (XV) is a compound of formula (XV′):
5. The lipid nanoparticle of claim 1 , wherein, X is —C(O)C 1-3 alkylC(O)O—.
6. The lipid nanoparticle of claim 1 , wherein m is an integer from 1-10.
7. The lipid nanoparticle of claim 6 , wherein in is an integer from 2-4.
8. The lipid nanoparticle of claim 7 , wherein in is 2.
9. The lipid nanoparticle of claim 1 , wherein n is an integer from 1-500.
10. The lipid nanoparticle of claim 9 , wherein n is an integer from 40-400.
11. The lipid nanoparticle of claim 10 , wherein n is an integer from 40-50.
12. The lipid nanoparticle of claim 1 , wherein the compound of formula (XV) has a formula (XVI) below:
wherein the repeating PEG moiety has an average molecular weight of 2000 with n value between 42 and 47.
13. The lipid nanoparticle of claim 1 , wherein the therapeutic agent siRNA.
14. The lipid nanoparticle particle of claim 1 , wherein the therapeutic agent mRNA.
15. The lipid nanoparticle of claim 1 , wherein X is —C(O)NH—.
16. The lipid nanoparticle of claim 1 , wherein X is —C(S)NH—.
17. The lipid nanoparticle of claim 1 , wherein X is —C(O)C 1-3 alky C(O)NH—.
18. The lipid nanoparticle of claim 1 , wherein each of R 1 and R 2 is independently unsubstituted alkyl, alkenyl or alkynyl.
19. The lipid nanoparticle of claim 18 , wherein each of R 1 and R 2 is independently unsubstituted alkyl.
20. The lipid nanoparticle of claim 18 , wherein each of R 1 and R 2 is independently unsubstituted alkenyl.