Patent ID: 8552067

Claim:
A bioactive agent having the formula I(b)[(A2)−(J) n′ )] n −M, wherein A2 is a compound that inhibits activity of the cystic fibrosis transmembrane conductance regulator protein (CFTR); J is a spacer; and M is a macromolecular moiety capable of interacting with a cell that expresses CFTR, or a pharmaceutically acceptable salt or stereoisomer thereof, such that the bioactive agent has the formula I(b): [(A2)−(J) n′ )] n −M as follows: wherein R 1 is phenyl or naphthalenyl; R 2 is phenyl, optionally substituted with any one or more of hydroxyl, C 1-8 alkyl, C 1-8 alkoxy, carboxy, halo, or nitro; R 3 is H or C 1-8 alkyl; R 4 is H; R 5 is H, alkyl, phenyl, carboxy, aryl, heteroaryl, —C(═O)NHNR 9 R 10 , —C(═O)NHN(═R 9 ), —NR 9 R 10 ; —C(═O)NHNHC(═S)NR 9 R 10 , —C(═O)NHNHC(═O)NR 9 R 11 , —C(═O)NHNHC(═O)CR 9 R 10 , —C(═O)R 9 , —CH 2 (CH) z R 9 wherein z is 0-7, or —CH 2 CH 2 NHR 11 ; each of R 9 , and R 10 is the same or different and independently H, C 1-8 alkyl, cycloalkyl, phenyl, or phenylalkyl; R 11 is H, C 1-8 alkyl, cycloalkyl, phenyl, or alkylphenyl; wherein J is a spacer that comprises a first end and a second end, wherein the spacer is attached to the compound of formula A2 at the first end of the spacer through a first linker functional group and wherein the spacer is attached to the macromolecular moiety M at the second end of the spacer through a second linker functional group, and wherein the first end of the spacer is attached to compound A2 at one or more of R 3 , R 4 or R 5 ; n=1 to 500 and n′=0 or 1, and wherein when n′=0, M forms a direct bond with at least one of R 3 , R 4 or R 5 , and wherein M is a lectin.