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mRNA-3927
go.drugbank.com/drugs/DB17669InvestigationalmRNA-3927 is an investigational mRNA therapy that consists of two mRNAs that encode for the alpha and beta subunits of the mitochondrial enzyme propionyl-CoA carboxylase (PCC) encapsulated within a lipid
HMI-103
go.drugbank.com/drugs/DB17684InvestigationalHMI-102 is an investigational gene therapy designed to encode the phenylalanine-4-hydroxylase (PAH) gene, mutated in people with phenylketonuria.
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]
ATX-F8-117
go.drugbank.com/drugs/DB17712ExperimentalDeveloped by Apitope, it is being investigated for the treatment of hemophilia A.[L46163]
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]
OMS906
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.
Synonyms: Humanized immunoglobulin G4 monoclonal antibody directed against mannan-binding lectin-associated serine protease-3 (MASP-3)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.