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Rexlemestrocel-L
go.drugbank.com/drugs/DB17606InvestigationalRexlemestrocel-L is an investigational human bone marrow-derived allogeneic mesenchymal precursor cell (MPC) product.
Synonyms: Rexlemestrocel-LCDX-6210
go.drugbank.com/drugs/DB17610ExperimentalCDX-6210 is a modified version of a leucine decarboxylase enzyme from _Planctomycetaceae bacterium_.
Ergothioneine
go.drugbank.com/drugs/DB17614InvestigationalSynonyms: L-ergothioneine, L-(+)-ergothioneineCategories: Compounds used in a research, industrial, or household settingZodasiran
go.drugbank.com/drugs/DB17626InvestigationalARO-ANG3 is a subcutaneously administered RNA interference (RNAi)-based investigational medicine targeting angiopoietin-like protein 3 (ANGPTL3).
RGX-111
go.drugbank.com/drugs/DB17632InvestigationalRGX-111 is an adeno-associated virus vector AAV9 designed to deliver the α-L-iduronidase (IDUA) gene.
Synonyms: Adeno-associated virus vector serotype 9 expressing human a-L-iduronidaseRGX-501
go.drugbank.com/drugs/DB17639InvestigationalRGX-501 is an adeno-associated virus vector 8 (AAV8)-mediated gene therapy that delivers a functional copy of the human low-density lipoprotein receptor (LDLR) gene to liver cells.
Phosphocysteamine
go.drugbank.com/drugs/DB17640ExperimentalCategories: Compounds used in a research, industrial, or household settingAEM-28-14
go.drugbank.com/drugs/DB17643ExperimentalAEM-28-14 is an analog of [AEM-28], an apolipoprotein E mimetic peptide, with a 400 percent greater cholesterol-lowering efficacy and a several-fold increase in drug tolerability.
EGT-101
go.drugbank.com/drugs/DB17645ExperimentalDeveloped by Esteve Pharmaceuticals, it is being investigated for Sanfilippo syndrome type A.[L45919]
OTL-201
go.drugbank.com/drugs/DB17652ExperimentalIt consists of a lentiviral vector to insert a functional copy of the human N-Sulfoglucosamine Sulfohydrolase (SGSH) gene into autologous CD34+ hematopoietic stem cells.[L45973]