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Devafidugene civaparvovec
go.drugbank.com/drugs/DB16790InvestigationalIt uses proprietary zinc finger nucleases (ZFN) that insert a functional copy of the alpha-L-iduronidase (IDUA) enzyme. … Devafidugene civaparvovec is an investigational _in vivo_ genome editing product.
CY-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 … It was investigated in cardiopulmonary bypass during surgical repair of congenital heart lesions.
NGN-401
go.drugbank.com/drugs/DB18594InvestigationalNGN-401 is a recombinant serotype 9 adeno-associated virus (AAV) encoding an intron encoding a miRNA generating sequence, complementary miRNA binding sites and a full-length human methyl cytosine binding … Developed by Neurogene Inc., it is being investigated for the treatment of Rett syndrome.
Synonyms: Recombinant serotype 9 adeno-associated virus (AAV) encoding an intron encoding a miRNA generating sequence, complementary miRNA binding sites and a full-length human methyl cytosine binding protein 2AZD2389
go.drugbank.com/drugs/DB22104InvestigationalAZD2389 is an investigational small molecule inhibitor of fibroblast activation protein (FAP).[L54978] It is currently undergoing safety, tolerability, and PK/PD profiling in clinical trials (e.g. … NCT06973005: A Study to Investigate How Multiple Oral Doses of AZD2389 Affect the Pharmacokinetics of Midazolam, Caffeine, and Bupropion in Healthy Participants).
WNY0824
go.drugbank.com/drugs/DB23606Experimental[A274253] It is a dual inhibitor of PLK-1 and BRET-4, which have both been found to be involved in CRPC via suppression of AR/MYC-mediated transcription. [A274258] … WNY0824 is a preclinical small molecule drug being investigated for the treatment of Castration-Resistant Prostrate Cancer (CRPC).
Ezatiostat
go.drugbank.com/drugs/DB05460InvestigationalIt might also be relevant as an adjunct therapy since a reduction in blood cell levels is also a common, toxic effect of many standard chemotherapeutic drugs. … Ezatiostat is a small molecule drug that is an analog inhibitor of glutathione S-transferase P1-1. It acts intracellularly on the MAPK signaling pathway by activating ERK2.
Epratuzumab
go.drugbank.com/drugs/DB04958InvestigationalEpratuzumab is a humanized monoclonal antibody derived from the murine IG2a monoclonal antibody, LL2 (EPB-2). … This agent may subsequently be well matched for use in oncology and the treatment of inflammatory autoimmune disorders, such as lupus.
Ularitide
go.drugbank.com/drugs/DB05034InvestigationalUlaritide is currently in Phase 2 development as a potential treatment for patients with acute decompensated heart failure (ADHF). … When injected into the blood, ularitide appears to cause diuresis (urine output) and natriuresis (sodium excretion), as well as vasodilation.
Butobarbital
go.drugbank.com/drugs/DB01353ApprovedIllicitButobarbital is a sedative and a hypnotic drug.
MIV-701
go.drugbank.com/drugs/DB05736ExperimentalMIV-701 is a selective, potent inhibitor of the protease Cathepsin K. It is developed for the treatment of osteoporosis.