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BT200
go.drugbank.com/drugs/DB18333InvestigationalBT200 (Rondoraptivon pegol) is a PEGylated anti-von Willebrand factor (VWF) aptamer targeted against the VWF A1 domain. … [A261491] It is under investigation for the treatment of von Willebrand disease and hemophilia A.[L48156]
Synonyms: PEGylated aptamer targeting Von Willebrand Factor A1 domainAVR-RD-04
go.drugbank.com/drugs/DB18644ExperimentalAVR-RD-04 is an investigational gene therapy for cystinosis. … It consists of autologous CD34+ enriched cells transduced with a lentiviral vector containing RNA resulting in codon-optimized cDNA encoding for functional human cystinosin.
Synonyms: autologous CD34+ enriched cells transduced with a lentiviral vector containing RNA resulting in codon-optimized cDNA encoding for functional human cystinosinZodasiran
go.drugbank.com/drugs/DB17626InvestigationalIt was granted an orphan drug designation for the treatment of homozygous familial hypercholesterolemia (HoFH).[L45869] … ARO-ANG3 is a subcutaneously administered RNA interference (RNAi)-based investigational medicine targeting angiopoietin-like protein 3 (ANGPTL3).
Carbenoxolone
go.drugbank.com/drugs/DB02329ExperimentalAn agent derived from licorice root. It is used for the treatment of digestive tract ulcers, especially in the stomach.
Clofutriben
go.drugbank.com/drugs/DB21636InvestigationalClofutriben is a small molecule drug. Clofutriben is under investigation in clinical trial NCT05307328 (SPI-62 as a Treatment for Adrenocorticotropic Hormone-dependent Cushing's Syndrome). … Clofutriben has a monoisotopic molecular weight of 424.09 Da.
AMP-201
go.drugbank.com/drugs/DB18663ExperimentalAMP-201 comprises an adeno-associated virus type 8 vector (AAV8) encoding the collagen Q gene (COLQ). It is under investigation for the treatment of collagen Q congenital myasthenia.
Benapenem
go.drugbank.com/drugs/DB18883Investigational(AP)). … Benapenem is under investigation in clinical trial NCT04505683 (A Study to Assess Efficacy and Safety Intravenous Benapenem in Patients With Complicated Urinary Tract Infection (Cuti) or Acute Pyelonephritis
Minzasolmin
go.drugbank.com/drugs/DB18961InvestigationalMinzasolmin is under investigation in clinical trial NCT05543252 (An Extension Study to Evaluate the Long-term Efficacy, Safety and Tolerability of Minzasolmin (UCB0599) in Study Participants With Parkinson's
Esaxerenone
go.drugbank.com/drugs/DB15207InvestigationalEsaxerenone is under investigation in clinical trial NCT02722265 (Long-term Study of CS-3150 as Monotherapy or in Combination With Other Antihypertensive Drug in Japanese Patients With Essential Hypertension
Nesolicaftor
go.drugbank.com/drugs/DB15239InvestigationalNesolicaftor (PTI-428) is under investigation in clinical trial NCT03258424 (Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis on KALYDECO® as Background