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Olenasufligene relduparvovec
go.drugbank.com/drugs/DB16264InvestigationalIt is an adeno-associated virus (AAV) vector that delivers a functional copy of the human SGSH gene.[L46671]
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]
EGT-301
go.drugbank.com/drugs/DB17649ExperimentalEGT-301 is a gene therapy being investigated for Hunter Syndrome. … It consists of an adeno-associated viral vector serotype 9 (AAV9) containing the human Iduronate-2-sulfatase (I2S) transgene designed to restore I2S functional deficiency in patients with Hunter syndrome
Coagulin-B
go.drugbank.com/drugs/DB17703ExperimentalCoagulin-B is an adeno-associated viral vector expressing human blood-coagulation Factor IX. … [A259038, A259043] It was previously investigated for the treatment of hemophilia; however, the manufacturing company discontinued the Phase I trial in 2004, halting its further development and investigation
ALGRX 1207
go.drugbank.com/drugs/DB06482ExperimentalALGRX 1207 is a topical local anesthetic that acts by binding to the fast sodium channel. … ALGRX 1207 entered clinical trials for cutaneous neuropathic pain, such as chemotherapy-induced neuropathy, in 2006. It was being developed by Anesiva, but trials have halted.
PTI-801
go.drugbank.com/drugs/DB05300InvestigationalIt is a combination of oxycodone with ultralow-dose naltrexone, an opioid antagonist. … PTI-801 represents a new class of drugs to treat pain. PTI-801 can minimize the opioid tolerance, dependence or addiction that is often associated with repeat use of oxycodone.
INGN 225
go.drugbank.com/drugs/DB05325InvestigationalINGN 225 is a therapeutic consisting of a cancer patient's own immune cells treated with an adenovector carrying the human p53 gene, Ad-p53.
L-tartaric acid
go.drugbank.com/drugs/DB09459Approved, and is a dihydroxyl derivative of succinic acid. … Tartaric acid is a white crystalline organic acid that occurs naturally in many plants, most notably in grapes.Tartaric is an alpha-hydroxy-carboxylic acid, is diprotic and aldaric in acid characteristics
Mebutamate
go.drugbank.com/drugs/DB06797ApprovedMebutamate is a sedative and anxiolytic drug with anti-hypertensive (blood pressure lowering) effects comparable to those of other barbiturates but is only around 1/3rd the potency of secobarbital as a
Elosulfase alfa
go.drugbank.com/drugs/DB09051ApprovedInvestigationalElosulfase alfa is a synthetic version of the enzyme N-acetylgalactosamine-6-sulfatase. It was approved by the FDA in 2014 for the treatment of Morquio syndrome. … The recommended dose is 2 mg per kg given intravenously over a minimum range of 3.5 to 4.5 hours, based on infusion volume, once every week.