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US 2025/0049948 A1Published application
Novel ionizable lipids and lipid nanoparticles and methods of using the same
AssigneeSail Biomedicines
InventorsAlessandra BARTOLOZZI, John PROUDFOOT, Roman ERDMANN, Siddharth PATEL, Alaina HOWE, Dominick SALERNO, Sanmit ADHIKARI, Roman BOGORAD, Arijit ADHIKARI
Life of this patent
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Abstract
Novel ionizable lipids and lipid nanoparticles that can be used in the delivery of therapeutic cargos are disclosed.
Claims26
A compound of formula (I) or (IA):
- a pharmaceutically acceptable salt thereof, or a stereoisomer of any of the foregoing,
- wherein
- Ra is H, C1-C3 branched or unbranched alkyl, C2-C3 branched or unbranched alkenyl, halogen, OH, or SH;
- each R1 and each R2 is independently H, C1-C3 branched or unbranched alkyl, C2-C3 branched or unbranched alkenyl, OH, halogen, SH, or NR10R11 or R1 and R2 are taken together to form a cyclic ring;
- each R10 and R11 is independently H, C1-C3 alkyl, or R10 and R11 are taken together to form a heterocyclic ring;
- m is 1, 2, 3, 4, 5, 6, 7 or 8;
- n is 0, 1, 2, 3 or 4;
- Y is O or S;
- Z is absent, O, S, or NR12, wherein R12 is H, C1-C7 branched or unbranched alkyl, or C2-C7 branched or unbranched alkenyl, provided that when Z is not absent, the adjacent R1 and R2 cannot be OH, NR10R11, or SH;
- each A is each independently C1-C16 branched or unbranched alkyl, or C2-C16 branched or unbranched alkenyl, optionally substituted with heteroatom or optionally substituted with OH, SH, or halogen;
- each B is each independently C1-C16 branched or unbranched alkyl, or C2-C16 branched or unbranched alkenyl, optionally substituted with heteroatom or optionally substituted with OH, SH, or halogen;
- and
- each X is independently a biodegradable moiety.
10 more elements
5-11. (canceled)
A compound of formula (V), (VA), or (VB):
- a pharmaceutically acceptable salt thereof, or a stereoisomer of any of the foregoing,
- wherein
- R1 is H, C1-C3 branched or unbranched alkyl, C2-C3 branched or unbranched alkenyl, OH, halogen, SH, or NR10R11;
- and R2 is OH, halogen, SH, or NR10R11, wherein
- R10 and R11 are each independently H or C1-C3 alkyl or R10 and R11 are taken together to form a heterocyclic ring, or
- R1 and R2 are taken together to form a cyclic ring;
- R20 and R30 are each independently H or C1-C5 alkyl, or R20 and R30 are taken together to form a cyclic ring;
- u is 0, 1, 2, 3, 4, 5, 6, 7, or 8;
- v is 1, 2, 3, or 4;
- y is 1, 2, 3, or 4;
- each A is independently C1-C16 branched or unbranched alkyl, or C2-C16 branched or unbranched alkenyl, optionally substituted with heteroatom or substituted with OH, SH, or halogen;
- each B is each independently C1-C16 branched or unbranched alkyl, or C2-C16 branched or unbranched alkenyl, optionally substituted with heteroatom or substituted with OH, SH, or halogen;
- and
- X is a biodegradable moiety.
11 more elements
16-21. (canceled)
23-26. (canceled)
31-33. (canceled)
37-39. (canceled)
(canceled)
A compound having one of the following formulas:
- Lipid
- No.
- Structure
- 7677 (2140)
- 7676 (2139)
- 7675 (2138)
- 7671 (2142)
- 7670 (2146)
- 7669 (2145)
- 7668 (2133)
- 7667 (2137)
- 7651 (2131)
- 7650 (2130)
- 7649 (2129)
- 7633 (2144)
- 7632 (2143)
- 7631 (2134)
- 7608 (2135)
- 7607 (2136)
- 7596 (2141)
- 7593 (2132)
- 2229
- 2228
- 2227
- 2226
- 2225
- 2216
- 2215
- 2233
- 2234
- 2235
- 2236
- 2237
- 2238
- 2239
- 2241
- 2242
- 2244
- 2245
- 2249
- 2250
- 2276
- 2277
- 2300
- 2301
- 2312
- 2313
- 2314
45 more elements
Dependencies are inferred from the claim text.