Делпацибарт этедесиран

Rec. INN (наименование, зарегистрированное ВОЗ)

2653183-68-5

immunoglobulin G1-kappa, anti-[Homo sapiens TFRC (transferrin receptor, TfR1)], humanized monoclonal antibody; conjugated via a 4-(maleimidomethyl)cyclohexanecarboxamide (MCC) linker with a double-stranded small interfering RNA (siRNA) which causes cleavage of the mRNA that encodes myotonin-protein kinase (MT-PK, myotonic dystrophy 1 protein kinase, DM1 protein kinase, DMPK, DM-kinase, DMK, EC:2.7.11.1);
gamma1 heavy chain humanized (1-445) [VH (Homo sapiens IGHV1-2*06 (87.8%) -(IGHD) -IGHJ4*01 (100%), CDR-IMGT [8.8.9] (26-33.51-58.97-105)) (1-116) -Homo sapiens IGHG1*03, G1m3, nG1m1, G1v14 CH2 A1.3, A1.2, G1v48 CH2 R113 (CH1 R120 (213) (117-214), hinge 1-15 (215-229), CH2 L1.3>A (233), L1.2>A (234), L113>R (327) (230-339), CH3 E12 (355), M14 (357) (340-444), CHS K2>del (445)) (117-445)], (219-214)-disulfide with kappa light chain humanized (1'-214') [V-KAPPA (Homo sapiens IGKV1-NL1*01 (84.2%) -IGKJ4*01 (100%), CDR-IMGT [6.3.9] (27-32.50-52.89-97)) (1'-107') -Homo sapiens IGKC*01 (100%), Km3 A45.1 (153), V101 (191) (108'-214')]; dimer (225-225'': 228-228'')-bisdisulfide, produced in Chinese hamster ovary (CHO) cells, glycoform alfa; substituted on average at one sulfur atom of a reduced cysteine residue 219, 225, 214', 219'', 225'', or 214''' with an all-P-ambo-2'-O-methyl-P-thiouridylyl-(3'→5')-2'-deoxy-2'-fluoro-P-thiouridylyl-(3'→5')-2'-O-methylcytidylyl-(3'→5')-2'-O-methylguanylyl-(3'→5')-2'-O-methyladenylyl-(3'→5')-2'-deoxy-2'-fluoroadenylyl-(3'→5')-2'-O-methylguanylyl-(3'→5')-2'-O-methyladenylyl-(3'→5')-2'-O-methylcytidylyl-(3'→5')-2'-O-methyladenylyl-(3'→5')-2'-O-methylguanylyl-(3'→5')-2'-O-methyluridylyl-(3'→5')-2'-O-methyluridylyl-(3'→5')-2'-deoxy-2'-fluorocytidylyl-(3'→5')-2'-O-methyluridylyl-(3'→5')-2'-deoxy-2'-fluoroadenylyl-(3'→5')-2'-O-methylguanylyl-(3'→5')-2'-O-methylguanylyl-(3'→5')-2'-O-methyl-P-thioguanylyl-(3'→5')-2'-O-methyluridylyl-(3'→5')-2'-O-methyluridine duplex with all-P-ambo-(3RS)-1-({cis- or trans-4-[(6-{[2'-O-methyl-P-thioadenylyl-(5'→3')-2'-O-methyl-P-thioadenylyl-(5'→3')-2'-O-methylguanylyl-(5'→3')-2'-O-methylcytidylyl-(5'→3')-2'-O-methyluridylyl-(5'→3')-2'-O-methyluridylyl-(5'→3')-2'-O-methylcytidylyl-(5'→3')-2'-O-methyluridylyl-(5'→3')-2'-O-methylguanylyl-(5'→3')-2'-O-methyluridylyl-(5'→3')-2'-deoxy-2'-fluorocytidylyl-(5'→3')-2'-deoxy-2'-fluoroadenylyl-(5'→3')-2'-deoxy-2'-fluoroadenylyl-(5'→3')-2'-O-methylguanylyl-(5'→3')-2'-O-methyladenylyl-(5'→3')-2'-O-methyluridylyl-(5'→3')-2'-O-methyl-P-thiocytidylyl-(5'→3')-2'-O-methyl-P-thiocytidylyl-(5'→3')-2'-O-methyl-5'-cytidylyl]oxy}hexyl)carbamoyl]cyclohexyl}methyl)-2,5-dioxopyrrolidin-3-yl (etedesiran) group

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