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AEM-28
go.drugbank.com/drugs/DB17642InvestigationalAEM-28 is a 28 amino acid mimetic of apolipoprotein E being investigated for lipid disorders, such as homozygous familial hypercholesterolemia. It possesses anti-inflammatory properties.[A258764]
VTX-801
go.drugbank.com/drugs/DB17646InvestigationalVTX-801 is a replication-deficient recombinant adeno-associated viral vector (rAAV) consisting of an AAV liver tropic capsid containing a single-stranded DNA genome carrying a shortened version of the
RP-A501
go.drugbank.com/drugs/DB17647InvestigationalRP-A501 is a gene therapy product consisting of a recombinant adeno-associated virus serotype 9 (rAAV9) capsid containing the human LAMP2B transgene (AAV9:LAMP2B), which encodes lysosome-associated membrane
EGT-301
go.drugbank.com/drugs/DB17649ExperimentalEGT-301 is a gene therapy being investigated for Hunter Syndrome.
Reloxiliase
go.drugbank.com/drugs/DB17653ExperimentalReloxiliase is a first-in-class, oral crystalline formulation of the oxalate-specific, microbial enzyme oxalate decarboxylase. It is being investigated for the treatment of hyperoxaluria.[A258823]
RP-L301
go.drugbank.com/drugs/DB17661InvestigationalRP-L301 is a lentiviral vector containing the human liver and erythroid pyruvate kinase gene.
rAAV9-rsATP7A
go.drugbank.com/drugs/DB17664ExperimentalA recombinant adeno-associated viral vector gene therapy under investigation for the treatment of Menkes disease.
SYNB1353
go.drugbank.com/drugs/DB17666InvestigationalSYNB1353 is a strain of the probiotic bacteria _Escherichia coli_ (_E. coli_) type Nissle and an engineered probiotic drug.
ASP-8731
go.drugbank.com/drugs/DB17696InvestigationalASP-8731 is a novel BACH1 inhibitor that potentially induces fetal hemoglobin (HbF). Developed by Mitobridge, it is being investigated for the treatment of sickle cell disease.[L46093]
BBM-H803
go.drugbank.com/drugs/DB17700InvestigationalBBM-H803 is a recombinant adeno-associated virus (AAV) based gene therapy drug for hemophilia A developed and manufactured by Belief BioMed.