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mRNA-3705
go.drugbank.com/drugs/DB17668InvestigationalmRNA-3705 is an investigational mRNA encoding methylmalonic CoA mutase (MUT) enzyme. Developed by Moderna TX Inc., it is being investigated for the treatment of methylmalonic academia.[A258843]
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 … Developed by Moderna Inc., it is being investigated for the treatment of propionic acidemia.[L45998]
ARCT-810
go.drugbank.com/drugs/DB17683InvestigationalARCT-810 is an mRNA replacement therapy that delivers ornithine transcarbamylase mRNA to hepatocytes.
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/DB17712ExperimentalATX-F8-117 is an investigational peptide derived from Factor VIII. Developed 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 is being investigated for the treatment of beta-thalassemia.[L46168]
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]