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Devafidugene civaparvovec
go.drugbank.com/drugs/DB16790InvestigationalDeveloped by Sangamo Therapeutics, Inc., devafidugene civaparvovec is being investigated for the treatment of mucopolysaccharidosis Type I (MPS I).[L46716] … It uses proprietary zinc finger nucleases (ZFN) that insert a functional copy of the alpha-L-iduronidase (IDUA) enzyme.
Delandistrogene moxeparvovec
go.drugbank.com/drugs/DB16802ApprovedInvestigationalMicrodystrophin is a truncated, functional form of dystrophin. … [L47026] DMD is an X-linked genetic disorder characterized by mutations in the dystrophin gene, leading to a deficiency in functional dystrophin protein.
Isaralgagene civaparvovec
go.drugbank.com/drugs/DB16808InvestigationalIsaralgagene civaparvovec, formerly known as ST-920, is liver-tropic rAAV 2/6 vector carrying the cDNA for human alpha-galactosidase A (α-Gal A) enzyme. … It is developed by Sangamo Therapeutics and investigated in Fabry Disease.
Cinchonidine
go.drugbank.com/drugs/DB16831ExperimentalSynonyms: Quinine bisulfate impurity B, Quinine sulfate impurity BCategories: Cytochrome P-450 CYP2D6 Inhibitors, Cytochrome P-450 Enzyme InhibitorsBentamapimod
go.drugbank.com/drugs/DB16851InvestigationalCategories: Heterocyclic Compounds, 1-Ring, Heterocyclic Compounds, 2-RingRemibrutinib
go.drugbank.com/drugs/DB16852ApprovedInvestigationalRemibrutinib is a selective Bruton's tyrosine kinase inhibitor. The FDA approved remibrutinib on September 30, 2025 as the only oral, targeted treatment for chronic spontaneous urticaria. … [L54101] Bruton's tyrosine kinase plays a role in B cell receptor and Fc receptor signalling, releasing histamine and pro-inflammatory mediators.
Categories: MATE 1 Inhibitors, P-glycoprotein inhibitorsTyrosol
go.drugbank.com/drugs/DB16855ExperimentalSynonyms: p-Tyrosol, 4-HydroxyphenylethanolCategories: Cytochrome P-450 Substrates, Cytochrome P-450 CYP2A6 SubstratesIndigo
go.drugbank.com/drugs/DB16862InvestigationalSynonyms: D&C Blue No. 6Categories: Heterocyclic Compounds, 2-Ring, Compounds used in a research, industrial, or household setting