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PRX-OTC
go.drugbank.com/drugs/DB17680ExperimentalPRX-OTC is an intracellular enzyme replacement therapy designed to replace missing or defective enzyme in patients with ornithine transcarbamylase deficiency.
SPL23-2
go.drugbank.com/drugs/DB17866ExperimentalSPL23-2 is an antisense oligonucleotide (ASO) targeting the W1282X nonsense mutation associated with a severe form of cystic fibrosis.[L46767]
TTX-MC138
go.drugbank.com/drugs/DB18628InvestigationalTTX-MC138 is an antisense oligonucleotide targeting miR-10b.[L48335] It is under investigation for the treatment of various metastatic cancers.
Rilvegostomig
go.drugbank.com/drugs/DB18991InvestigationalRilvegostomig is under investigation in clinical trial NCT06109779 (Rilvegostomig + Chemotherapy as Adjuvant Therapy for Biliary Tract Cancer After Resection (Artemide-biliary01)).
BL-1020
go.drugbank.com/drugs/DB05687InvestigationalBL-1020 is a first in class novel compound for the treatment of schizophrenia. It is being developed by BioLineRx (BioLine).
Lemzoparlimab
go.drugbank.com/drugs/DB17572InvestigationalLemzoparlimab is under investigation in clinical trial NCT04912063 (Study to Evaluate Adverse Events and Movement of Lemzoparlimab in Body When Used Intravenously (IV) With Azacitidine Subcutaneously or IV and Venetoclax Orally in Partic...
Velafermin
go.drugbank.com/drugs/DB06493InvestigationalVelafermin was a novel investigational protein therapeutic, also known as fibroblast growth factor-20 (FGF-20), which promotes both epithelial and mesenchymal cell proliferation in vitro and has demonstrated … Velafermin was investigated as a potential therapy for the prevention and treatment of oral mucositis by fostering the regeneration and repair of the cells lining the gastrointestinal tract.
Tasimelteon
go.drugbank.com/drugs/DB09071ApprovedInvestigationalTasimelteon is a selective dual melatonin receptor agonist indicated for the treatment of Non-24-Hour Sleep-Wake Disorder (N24HSWD). … In blind individuals, a lack of light stimulation causes an extension of the 24-hour circadian cycle and can lead to progressively delayed sleep onset.
Categories: Melatonin Receptor AgonistsLGD2941
go.drugbank.com/drugs/DB05234ExperimentalLGD2941 is a non-steroidal, selective androgen receptor modulator (SARM) developed jointly by Ligand and TAP.
AAV9/MFSD8
go.drugbank.com/drugs/DB18616ExperimentalAAV9/MFSD8 is a gene therapy developed by Neurogene Inc. … It is an adeno-associated virus serotype 9 (AAV9) vector with an engineered transgene encoding the human CLN7/MFSD8 gene for expression of active human major facilitator superfamily domain containing 8
Synonyms: An adeno-associated virus serotype 9 (AAV9) vector with engineered transgene encoding the human CLN7/MFSD8 gene for expression of active human major facilitator superfamily domain containing 8