Ionizable lipidoid compositions and therapeutic uses thereof
AssigneeFlagship Pioneering Innovations Vii, LLC
InventorsSrinivas Abbina, Chelsea RamEl Martinez, Vandan Paresh Jakharia, Timothy Ray Blake, Ulugbek Barotov
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Abstract
Disclosed are lipidoid compounds having the structure of formula (I): or a salt thereof, wherein the groups are as defined in the application. Also disclosed are nanoparticle compositions comprising a lipidoid of the invention that are capable of delivering a therapeutic agent. The application also discloses pharmaceutical compositions comprising a lipidoid composition of the invention.
Claims32
A lipidoid of formula (I), or a salt thereof;
- wherein:
- each of L1, L2, and L3 represents —(CH2)m;
- m is independently for each occurrence an integer selected from 1-20;
- Y1 is
- and * represents the connection to L1;
- Y2 is
- and * represents the connection to L2;
- Z1, Z2, Z3, and Z4 are each independently O, S, or NH;
- X is optionally substituted saturated or unsaturated cycloalkylene, heterocyclylene, heteroarylene, or arylene;
- R1 and R2 are each independently optionally substituted linear (C4-C20)alkyl, branched (C4-C20)alkyl, linear (C4-C20)alkenyl, branched (C4-C20)alkenyl, linear (C4-C20)alkynyl, or branched (C4-C20)alkynyl, optionally wherein two substituents, together with the intervening atoms, form an aryl or 5-7 membered cycloalkyl ring;
- and
- R3 is hydrogen, hydroxyl, hydroxyalkyl, amido, or an optionally substituted linear C2-C10 alkyl or branched C3-C10 alkyl.
3-6. (canceled)
A lipidoid of formula (II), or a salt thereof;
- wherein:
- L3 is —(CH2)m—;
- m is an integer selected from 1-10;
- X is an optionally substituted saturated or unsaturated cycloalkylene, heterocyclylene, heteroarylene, or arylene;
- R1 and R2 are each independently optionally substituted linear (C4-C20)alkyl, branched (C4-C20)alkyl, linear (C4-C20)alkenyl, branched (C4-C20)alkenyl, linear (C4-C20)alkynyl, or branched (C4-C20)alkynyl, optionally wherein two substituents, together with the intervening atoms, form an aryl or 5-7 membered cycloalkyl ring;
- and
- R3 is hydrogen, hydroxyl, hydroxyalkyl, amido, or an optionally substituted linear C2-C10 alkyl or branched C3-C10 alkyl.
8-9. (canceled)
A lipidoid of formula (V), or a salt thereof;
- wherein:
- L3 represents —(CH2)m—;
- m is an integer selected from 1-10;
- each of Z1, Z2, Z3, and Z4 are independently O, S, or NH;
- X is an optionally substituted saturated or unsaturated cycloalkylene, heterocyclylene, heteroarylene, or arylene;
- R3 is hydrogen, hydroxyl, hydroxyalkyl, amido, or an optionally substituted linear C2-C10 alkyl or branched C3-C10 alkyl.
- R4 and R6 are each independently H, linear C2-C10 alkyl or branched C3-C10 alkyl;
- R5 is H, linear C2-C10 alkyl, or branched C3-C10 alkyl,
- or R5 and R4, taken together with the intervening atoms, form an aryl or a 5- to 7-membered cycloalkyl;
- and
- R7 is H, linear C2-C10 alkyl, or branched C3-C10 alkyl,
- or R7 and R6, taken together with the intervening atoms, form an aryl or a 5- to 7-membered cycloalkyl.
11-16. (canceled)
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23-30. (canceled)
33-34. (canceled)
36-40. (canceled)
45-48. (canceled)
51-63. (canceled)
65-66. (canceled)
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Dependencies are inferred from the claim text.