US 12,269,804 B2
Quinoxalinone compounds, compositions, methods, and kits for increasing genome editing efficiency
Sudipta Mahajan, Framingham, MA (US); Marc Saul Weinberg, Encinitas, CA (US); Diego Sebastian D'Astolfo, San Diego, CA (US); Kevin M. Cottrell, Cambridge, MA (US); Mark A. Morris, Somerville, MA (US); and John Patrick Maxwell, Hingham, MA (US)
Assigned to VERTEX PHARMACEUTICALS INCORPORATED, Boston, MA (US)
Appl. No. 16/962,443
Filed by VERTEX PHARMACEUTICALS INCORPORATED, Boston, MA (US)
PCT Filed Jan. 16, 2019, PCT No. PCT/US2019/013785
§ 371(c)(1), (2) Date Jul. 15, 2020,
PCT Pub. No. WO2019/143677, PCT Pub. Date Jul. 25, 2019.
Claims priority of provisional application 62/618,385, filed on Jan. 17, 2018.
Prior Publication US 2020/0361877 A1, Nov. 19, 2020
Int. Cl. C12N 9/22 (2006.01); C07D 241/44 (2006.01); C07D 403/12 (2006.01); C07D 405/14 (2006.01); C07D 491/048 (2006.01); C07D 491/08 (2006.01); C12N 15/11 (2006.01); C12N 15/90 (2006.01)
CPC C07D 241/44 (2013.01) [C07D 403/12 (2013.01); C07D 405/14 (2013.01); C07D 491/048 (2013.01); C07D 491/08 (2013.01); C12N 9/22 (2013.01); C12N 15/11 (2013.01); C12N 15/907 (2013.01); C12N 2310/20 (2017.05); C12N 2800/80 (2013.01)] 8 Claims
 
1. A compound of formula (III):

OG Complex Work Unit Chemistry
wherein
X is O;
Y is a bond, O, or NR; wherein R is H or C1-C4 alkyl;
R2 is
a) a 5- or 6-membered aryl or heteroaryl ring containing one or two heteroatoms selected from the group consisting of N, O, and S, wherein the aryl and the heteroaryl ring may be substituted by 0, 1, 2, or 3 substituents R3 independently selected from the group consisting of CN, halo, C1-C4-alkyl, C3-C6 cycloalkyl, C1-C4-haloalkyl, C1-C4-alkoxy, C1-C4-haloalkoxy, C(═O) NHR1′, and a 5- or 6-membered heterocycloalkyl or heteroaryl ring wherein each ring contains 1, 2, or 3 heteroatoms selected from N, O, and S; wherein R1′ is C1-C4 alkyl; or wherein two R3 groups connected to adjacent carbon atoms of the aryl or heteroaryl ring may form a fused 5- or 6-membered ring which may contain a heteroatom selected from O, N, and S; or
b) COOR4 wherein R4 is C1-C4-alkyl or benzyl;
wherein each C1-C4-alkyl, C1-C4-haloalkyl, C1-C4-alkoxy, and C1-C4-haloalkoxy may further be substituted with OR5 or NR6R7 wherein each of R5, R6, and R7 is independently H, C1-C4 alkyl, or C3-C6 cycloalkyl; or wherein R6 and R7 and the nitrogen atom to which they are attached form a saturated 5- or 6-membered ring that may contain 0 or 1 further heteroatom selected from N, O, and S and wherein the ring may be further substituted by C1-C4-alkyl; and
wherein W is N and Z is O.