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Emprumapimod
go.drugbank.com/drugs/DB17603InvestigationalEmprumapimod is a potent, orally active and selective inhibitor of p38α mitogen-activated kinase inhibitor. It was investigated for dilated cardiomyopathy.
Ammonium tetrathiomolybdate
go.drugbank.com/drugs/DB17641InvestigationalCategories: Compounds used in a research, industrial, or household settingTAK-611
go.drugbank.com/drugs/DB17644InvestigationalTAK-611, formerly known as SHP611, is a recombinant human arylsulfatase A. First developed by Takeda, it is being investigated for the treatment of metachromatic leukodystrophy.
SerpinPC
go.drugbank.com/drugs/DB17698InvestigationalSerpinPC is a mutated alpha-1-antitrypsin (α1AT) and serine protease inhibitor being investigated for the treatment of hemophilia.[A258993]
Thalagen
go.drugbank.com/drugs/DB17714ExperimentalThalagen is a branded product developed by San Rocco Therapeutics. … It consists of autologous CD34-positive hematopoietic progenitor cells transduced with TNS9.3.55, a lentiviral vector encoding the normal human beta-globin gene.
MOD-5014
go.drugbank.com/drugs/DB17715InvestigationalMOD-5014 is a long-acting carboxy-terminal peptide (CTP)-modified Factor IIa.[A259088] It is being investigated for the treatment of hemophilia.[L46173]
mRNA-3351
go.drugbank.com/drugs/DB17718ExperimentalmRNA-3351 is a modified mRNA encoding the UGT1A1 gene. Developed by Moderna, it is currently being investigated as the treatment for Crigler-Najjar syndrome
NM3086
go.drugbank.com/drugs/DB17719ExperimentalNM3086 is a recombinant, humanized, aglycosylated, IgG1-kappa anti-properdin monoclonal antibody being developed by Novelmed Therapeutics Inc.[L46193]
Synonyms: NM 3086, NM-3086OMS906
go.drugbank.com/drugs/DB17722InvestigationalOMS906 is a humanized Igg4 monoclonal antibody against mannan-binding lectin-associated serine protease-3 (MASP-3) currently being developed by Omeros.
Rusfertide
go.drugbank.com/drugs/DB17724ApprovedInvestigationalRusfertide is a hepcidin mimetic used to treat erythrocytosis in adults with polycythemia vera. … [L64098] The FDA approved rusfertide on August 28, 2026 for the treatment of erythrocytosis in adults with polycythemia vera, a blood cancer.