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Ammonium tetrathiomolybdate
go.drugbank.com/drugs/DB17641InvestigationalSynonyms: Molybdate(2-), tetrathioxo-, ammonium (1:2), (t-4)-, Molybdate(2-), tetrathioxo-, diammonium, (t-4)-Categories: 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
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.
Vamifeport
go.drugbank.com/drugs/DB17692InvestigationalVamifeport is being investigated for the treatment of beta-thalassemia.
Synonyms: 2-(2-((2-(1h-benzimidazol-2-yl)ethyl)amino)ethyl)-n-((3-fluoropyridin-2-yl)methyl)-1,3-oxazole-4-carboxamide, 4-oxazolecarboxamide, 2-(2-((2-(1h-benzimidazol-2-yl)ethyl)amino)ethyl)-n-((3-fluoro-2-pyridinyl)methyl)-Categories: Heterocyclic Compounds, 1-Ring, Heterocyclic Compounds, 2-RingAmotosalen
go.drugbank.com/drugs/DB17693InvestigationalSynonyms: 7h-furo(3,2-g)(1)benzopyran-7-one, 3-((2-aminoethoxy)methyl)-2,5,9-trimethyl-, 3-((2-aminoethoxy)methyl)-2,5,9-trimethyl-7h-furo(3,2-g)chromen-7-oneCategories: Heterocyclic Compounds, 1-Ring, Heterocyclic Compounds, 2-RingSerpinPC
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.