US 12,077,483 B2
Modified amine lipids
Stephen S. Scully, Cambridge, MA (US); Micah Maetani, Cambridge, MA (US); and Ramsey Majzoub, Cambridge, MA (US)
Assigned to Intellia Therapeutics, Inc., Cambridge, MA (US)
Appl. No. 17/299,925
Filed by Intellia Therapeutics, Inc., Cambridge, MA (US)
PCT Filed Dec. 5, 2019, PCT No. PCT/US2019/064663
§ 371(c)(1), (2) Date Jun. 4, 2021,
PCT Pub. No. WO2020/118041, PCT Pub. Date Jun. 11, 2020.
Claims priority of provisional application 62/775,783, filed on Dec. 5, 2018.
Prior Publication US 2022/0119341 A1, Apr. 21, 2022
Int. Cl. C07C 219/04 (2006.01); A61P 1/16 (2006.01); C07C 271/20 (2006.01); C07D 205/04 (2006.01); C07D 207/09 (2006.01); C07D 211/46 (2006.01); C07D 211/62 (2006.01); C07D 271/10 (2006.01); C07D 295/08 (2006.01); C12N 15/113 (2010.01); B82Y 5/00 (2011.01); B82Y 30/00 (2011.01); B82Y 40/00 (2011.01)
CPC C07C 219/04 (2013.01) [A61P 1/16 (2018.01); C07C 271/20 (2013.01); C07D 205/04 (2013.01); C07D 207/09 (2013.01); C07D 211/46 (2013.01); C07D 211/62 (2013.01); C07D 271/10 (2013.01); C07D 295/08 (2013.01); C12N 15/113 (2013.01); B82Y 5/00 (2013.01); B82Y 30/00 (2013.01); B82Y 40/00 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01)] 21 Claims
 
1. A compound of Formula IA;

OG Complex Work Unit Chemistry
wherein, independently for each occurrence,
X1 is C1-3 alkylene or

OG Complex Work Unit Chemistry
X2 is selected from O, NH, NMe, and a bond, provided that when X2 is O, R2 taken together with the nitrogen atom and either R1 or a carbon atom of X3 form a 4-membered, 5-membered, or 6-membered ring;
X3 is C2-4 alkylene,
X4 is C1 alkylene or a bond,
X5 is C1 alkylene or a bond,
R1 is C1-3 alkyl,
R2 is C1-3 alkyl, or
R2 taken together with the nitrogen atom and either R1 or a carbon atom of X3 form a 4-membered, 5-membered, or 6-membered ring,
Y1 is selected from a bond, —CH═CH—, —(C═O)O—, and —O(C═O)—,
Y2 is selected from —CH2—CH═CH— and C3-C4 alkylene,
R3 is selected from H, C5-7 cycloalkyl, C8-C10 alkenyl, and C3-18 alkyl, and
R4 is C4-8 alkyl,
or a salt thereof.