Compounds targeting rna-binding proteins or rna-modifying proteins

In a first aspect, the present invention is directed to a compound represented by Formula (I):

• or a pharmaceutically acceptable salt thereof, wherein:
• W is —CH2Y—R3, —S(O)2R3, —SCH2R3, Y—R3, —OC(═O)NR11R12 or —N(C═O)NR11R12;
• V is —C(═O)—, —S(═O)2— or CR13R14;
• X is CR4R5, NR6 or O;
• Y is NR7 or O;
• Z is CR8 or N;
• R1 is halo, —CN, —OR1a, —C═CH, a C3-6 carbocyclyl, or a C1-6 alkyl, wherein the C1-6 alkyl is optionally substituted with one or more R10;
• R1a is H or C1-6 alkyl optionally substituted with one or more R10;
• R10, for each occurrence, is independently selected from halo, —CN and —OR10a;
• R10a, for each occurrence, is independently selected from H and C1-6 alkyl;
• R2 is —CN, —CHRzCN, —C(O)NH2, —CHRzC(O)NH2, 3- to 12-membered carbocyclyl, —(CHRz)-(3- to 12-membered carbocyclyl), 3- to 12-membered heterocyclyl, or —(CHRz)-(3 to 12-membered heterocyclyl), wherein the 3 to 12-membered carbocyclyl and the 3 to 12-membered heterocyclyl represented by R2 or in the group represented by R2 are optionally substituted with one or more R20, and Rz is H or methyl;
• R20, for each occurrence, is independently selected from H, halo, CN, oxo, —C(O)R20a, —C(O)2R20a, —C(O)NR20a, —N(R20a)2, —N(R20a)C(O)R20a, —N(R20a)C(O)2R20a, —N(R20a)C(O)N(R20a)2, —N(R20a)S(O)2R20a, —OR20a, —OC(O)R20a, —OC(O)N(R20a)2, —SR20a, —S(O)2R20a, —S(O)N(R20a)2, —S(O)2N(R20a)2, C1-6 alkyl, C3-8cycloalkyl, and 4- to 7-membered monocyclic heterocyclyl, wherein the C1-6 alkyl, C3-8cycloalkyl, and 4- to 7-membered monocyclic heterocyclyl represented by R20 are each optionally substituted with one or more R25;
• R20a, for each occurrence, is independently selected from H, C1-6 alkyl, C3-8cycloalkyl and 4- to 7-membered monocyclic heterocyclyl, wherein the C1-6 alkyl, C3-8cycloalkyl and 4- to 7-membered monocyclic heterocyclyl represented by R20a are each optionally substituted with one or more R25, or two R20a together with the nitrogen atom from which they are attached form a 4- to 7-membered heterocyclyl optionally substituted with one or more R25;
• R25, for each occurrence, is independently H, C1-6 alkyl, halo, —C═O, —C(O)O(C1-C6 alkyl), 4- to 6-membered heterocyclyl optionally substituted with OH, —N(R25a)2, —OR25a, phenyl optionally substituted with C1-6 alkyl, halo, —N(R25a)2 or —OR25a;
• and
• R25a is H, C1-6alkyl;
• or two R25a together with the nitrogen atom from which they are attached form a 4- to 7-membered heterocyclyl, wherein the C1-6alkyl is optionally substituted with —OCH3;
• R3 is a 3 to 12-membered carbocyclyl or a 3 to 12-membered heterocyclyl, wherein the 3 to 12-membered carbocyclyl and the 3 to 12-membered heterocyclyl represented by R3 are optionally substituted with one or more R30;
• R30, for each occurrence, is independently selected from H, halo, oxo, —CN, —C(O)R30a, —C(O)2R30a, —C(O)NR30a, —N(R30a)2, —N(R30a)C(O)R30a, —N(R30a)C(O)2R30a, —N(R30a)C(O)N(R30a)2, —N(R30a)S(O)2R30a, —OR30a, —OC(O)R30a, —OC(O)N(R30a)2, —SR30a, —S(O)2R30a, —S(O)N(R30a)2, —S(O)2N(R30a)2, C1-6 alkyl, C3-8cycloalkyl, and 4- to 7-membered monocyclic heterocyclyl, wherein the C1-6 alkyl, C3-8cycloalkyl, and 4- to 7-membered monocyclic heterocyclyl represented by R30 are each optionally substituted with one or more R35;
• R30a, for each occurrence, is independently selected from H, C1-6 alkyl, C3-8cycloalkyl and 4- to 7-membered monocyclic heterocyclyl, wherein the C1-6 alkyl, C3-8cycloalkyl and 4- to 7-membered monocyclic heterocyclyl represented by R30a are each optionally substituted with one or more R35;
• R35, for each occurrence, is independently H, —NH2, C1-6alkyl, halo and —OR35a;
• and
• R35a is H or C1-6 alkyl;
• alternatively, when Y is NR7, R7 and R3 together with the N atom from which R7 is attached can form a 4 to 12-membered heterocyclyl optionally substituted with or more R30;
• R4, R5, R15, R16, R17 and R18 are each independently selected from H, C1-6 alkyl, halo, —NHC(O)CH3, 4- to 6-membered carbocyclyl, or 4- to 6-membered heterocyclyl, wherein the C1-6 alkyl is optionally substituted with one or more substituents independently selected from halo, and wherein the heterocyclyl and carbocyclyl are optionally substituted with halo, —CN, —OR4a, C1-C6 alkyl or ═O (when the carbocyclyl or heterocyclyl are non-aromatic), or wherein R15 and R16 taken together or R17 and R18 taken together are ═NOH or ═NHOCH3;
• R4a is H or C1-6 alkyl, optionally substitute with one or more halo;
• R6 is H or C1-6alkyl optionally substituted with one or more substituents independently selected from halo, —CN and —OR6a;
• R6a is H or C1-6 alkyl optionally substitute with one or more halo;
• R7 is H or C1-6 alkyl optionally substituted with one or more halo;
• R8 is H, halo, —CN, —OR8a or C1-6 alkyl optionally substituted with one or more halo;
• R8a is H or C1-6 alkyl optionally substituted with one or more halo;
• R9, R13 and R14 are each independently selected from H, C1-6alkyl, and C3-6 cycloalkyl, wherein the C1-6 alkyl and the C3-6 cycloalkyl are each optionally substituted with one or more substituents independently selected from halo, —CN, —OR9a and C1-6 alkyl optionally substituted with one or more halo;
• R9a is H or C1-6 alkyl optionally substituted with one or more halo;
• R11 and R12 are each independently H or C1-6 alkyl, or R11 and R12 together with the nitrogen atom from which they are attached form a 4- to 7-membered monocyclic heterocyclyl optionally substituted with one or more substituents independently selected from C1-6 alkyl, halo, —OH, and C1-6 alkoxy;
• or any two of R4, R5, R9, R13, R14, R15, R16, R17 and R18 together form a —C1-5 alkylene-, optionally substituted with one or more substituents independently selected from halo, —ORa, —N(Ra)2 and C1-6 alkyl;
• or R4 and R15 taken together with their intervening carbon atoms form phenyl or a 6 membered heteroaryl;
• and
• Ra, for each occurrence, is independently H or C1-6 alkyl optionally substituted with one or more halo;
• R19a and R19b are each independently H or C1-6 alkyl optionally substituted with one or more halo, or R19a and R19b together with the carbon atom from which they are attached form —C(═O);
• or
• one R20 group and one R30 group taken together form —O—CH2CH2—O— or —O—CH2CH2—NH—;
• and
• n1 is 0, 1 or 2;
• and
• n2 is 0, 1 or 2, provided when X is NR6 or O, n1 and n2 cannot be 0.