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OTL-102
go.drugbank.com/drugs/DB18642ExperimentalIt is being investigated for the treatment of X-linked chronic granulomatous disease.
OTL-203
go.drugbank.com/drugs/DB18643InvestigationalOTL-203 is an autologous CD34+ enriched cell therapy that contains hematopoietic stem and progenitor cells transduced ex-vivo using a lentiviral vector encoding alpha-L-iduronidase gene.
Synonyms: Autologous CD34+ enriched cell population that contains hematopoietic stem and progenitor cells transduced ex-vivo using a lentiviral vector encoding alpha-L-iduronidase geneViltolarsen
go.drugbank.com/drugs/DB15005ApprovedInvestigationalDuchenne muscular dystrophy (DMD) is an X-linked recessive allelic disorder characterized by a lack of functional dystrophin protein, which leads to progressive ambulatory, pulmonary, and cardiac function
Fucose
go.drugbank.com/drugs/DB15236InvestigationalFucose is under investigation in clinical trial NCT03354533 (Study of ORL-1F (L-fucose) in Patients With Leukocyte Adhesion Deficiency Type II).
Synonyms: L-FUCOSEUmbilical Cord Blood Hematopoietic Stem Cells
go.drugbank.com/drugs/DB15723InvestigationalCord blood transplants have been used to treat conditions from aplastic anemia, B-thalassemia, X-linked lymphoproliferative syndrome, Hunter’s syndrome, acute lymphoid leukemia, chronic myeloid leukemia
Nezastomig
go.drugbank.com/drugs/DB19061InvestigationalNezastomig is under investigation in clinical trial NCT06085664 (A Pilot Presurgical Trial of REGN5678 (Anti-psma X CD28) in Patients With High-risk, Localized Prostate Cancer Followed by Radical Prostatectomy
UCB7362
go.drugbank.com/drugs/DB17096ExperimentalUCB7362 is an orally active plasmepsin X (PMX) inhibitor currently investigated for the treatment of malaria.
L-3972-T
go.drugbank.com/drugs/DB17441ExperimentalL-3972-T is one of the components of the human papilloma virus (HPV) type 16 E6/E7 synthetic long peptide (SLP) vaccine ISA101b.
Synonyms: ISA101B component L-3972-T, Human Papilloma Virus (HPV) Type 16 E6/E7 Synthetic Long Peptide (SLP) Vaccine Component L-3972-TRed blood cell-encapsulated L-asparaginase
go.drugbank.com/drugs/DB17477InvestigationalRed blood cell-encapsulated L-asparaginase is [Asparaginase Escherichia coli] encapsulated inside donor-derived red blood cells, which target cancer cells capable of altering asparagine and glutamine metabolism
Synonyms: RBC-encapsulated l-asparaginase, Erythrocyte encapsulated l-asparaginase6-diazo-5-oxo-L-norleucine
go.drugbank.com/drugs/DB17537Investigational6-diazo-5-oxo-L-norleucine is a broad-acting glutamine antagonist.
Synonyms: 6-Diazo-5-oxo-L-norleucine