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NTLA-2002
go.drugbank.com/drugs/DB17597InvestigationalNTLA-2002 is a lipid nanoparticle encapsulating single guide RNA (G012267) targeting the human KLKB1 gene and messenger RNA (mRNA000042) encoding Cas9.
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.
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.
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]