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Mubodina
go.drugbank.com/drugs/DB17616ExperimentalIt is a recombinant human minibody directed against complement component C5.
AGT-182
go.drugbank.com/drugs/DB17630InvestigationalAGT-182 is an investigational enzyme replacement therapy engineered by the fusion of the iduronate-2-sulfatase (IDS) enzyme and a human insulin receptor monoclonal antibody.
Nedosiran
go.drugbank.com/drugs/DB17635ApprovedInvestigationalNedosiran is an RNA interference targeting hepatic lactate dehydrogenase, the enzyme responsible for the conversion of glyoxylate to oxalate. Oxalate, particularly calcium oxalate, precipitation is the main cause of kidney stones formati...
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.
AVR-RD-01
go.drugbank.com/drugs/DB17651InvestigationalIt consists of autologous CD34+ cells that transduced with Lentiviral Vector/alpha-galactosidase A (AGA) encoding for the human AGA complementary deoxyribonucleic acid (cDNA) sequence.
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
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.