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Loxoribine
go.drugbank.com/drugs/DB17589ExperimentalSynonyms: 7-Allyl-7,8-dihydro-8-oxoguanosineCategories: Heterocyclic Compounds, 2-RingEtuvetidigene Autotemcel
go.drugbank.com/drugs/DB17593ApprovedInvestigational[L54693] WAS is a rare X-linked disorder caused by a mutation in the gene encoding the WAS protein, leading to a compromised immune system. … [L54698] First approved by the FDA on December 9, 2025, etuvetidigene autotemcel is used to treat Wiskott–Aldrich syndrome (WAS) in selected pediatric and adult patients.
NP-6A4
go.drugbank.com/drugs/DB17599ExperimentalNP-6A4 is a peptide designed to specifically bind and activate angiotensin II receptor type 2 (AT2R). It has an Orphan Drug designation from the FDA for pediatric cardiomyopathy.[A258523]
Hemoximer
go.drugbank.com/drugs/DB17600InvestigationalHemoximer is a modified version of hemoglobin that undergoes pyridoxylation, for the purpose of lowering the oxygen affinity, and coupling with polyoxyethylene (POE), to increase its molecular weight.
Ixmyelocel-T
go.drugbank.com/drugs/DB17601InvestigationalIxmyelocel-T is an expanded, autologous multicellular therapy cultured from bone-marrow mononuclear cells. Developed by Vericel Corporation, it is being investigated for cardiomyopathy.
Synonyms: Ixmyelocel-TCY-1503
go.drugbank.com/drugs/DB17605InvestigationalCY-1503 is a pentasaccharide ethyl glycoside consisting of one alpha-D-sialylosyl residue as a sodium salt, two beta-D-galactopyranosyl residues, one 2-acetamido-beta-Dglucopyranosyl unit, and one alpha-L-fucopyranosyl
CDX-1135
go.drugbank.com/drugs/DB17607InvestigationalCDX-1135, formerly TP-10, is a soluble, recombinant human Complement Receptor Type 1 (sCR1) developed by Avant Immunotherapeutics (now Celldex).
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 … It is being investigated in Wilson Disease.[L45924]
RP-A501
go.drugbank.com/drugs/DB17647Investigationalprotein 2 (LAMP-2). … RP-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
Synonyms: Adeno-associated virus serotype 9 vector containing the human lysosome-associated membrane glycoprotein 2 isoform B transgene (AAV9.LAMP2B)