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INZ-701
go.drugbank.com/drugs/DB17629InvestigationalINZ-701 is an enzyme replacement therapy under development with the intention to be used for the treatment of calcification disorders of the circulatory system, bones, and kidneys.
mRNA-3705
go.drugbank.com/drugs/DB17668InvestigationalmRNA-3705 is an investigational mRNA encoding methylmalonic CoA mutase (MUT) enzyme. Developed by Moderna TX Inc., it is being investigated for the treatment of methylmalonic academia.[A258843]
Efdoralprin alfa
go.drugbank.com/drugs/DB18384InvestigationalEfdoralprin alfa is a recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein.[L54928] It is being investigated as a restorative therapy in adults with AATD emphysema.[L54928]
AB-201
go.drugbank.com/drugs/DB18629InvestigationalAB-201 is a CAR-NK cell therapy targeted against HER2.[L48340] It is under investigation for the treatment of HER2-positive cancers, including breast and gastric carcinomas.
Synonyms: Ex vivo expanded allogeneic cord blood-derived NK cells that have been genetically modified to express a HER2-directed CAR and secrete IL-15OTL-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 geneMitiperstat
go.drugbank.com/drugs/DB18963InvestigationalMitiperstat is under investigation in clinical trial NCT05492877 (An Efficacy and Safety Study of Mitiperstat (AZD4831) (MPO Inhibitor) vs Placebo in the Treatment of Moderate to Severe COPD.).
THR-184
go.drugbank.com/drugs/DB19097InvestigationalTHR-184 is under investigation in clinical trial NCT01830920 (A Study of THR-184 in Patients at Risk of Developing Cardiac Surgery Associated-acute Kidney Injury (CSA-AKI)).
Phosphonotyrosine
go.drugbank.com/drugs/DB01962ExperimentalAn amino acid that occurs in endogenous proteins. Tyrosine phosphorylation and dephosphorylation plays a role in cellular signal transduction and possibly in cell growth control and carcinogenesis.
Butyrylthiocholine
go.drugbank.com/drugs/DB04250ExperimentalA sulfur-containing analog of butyrylcholine which is hydrolyzed by butyrylcholinesterase to butyrate and thiocholine. It is used as a reagent in the determination of butyrylcholinesterase activity.
AMP-101
go.drugbank.com/drugs/DB18253ExperimentalAMP-101 is a gene therapy comprising an adeno-associated virus type 9 (AAV9) vector encoding the human Dok-7 protein. It is under investigation for the treatment of Dok-7-associated myasthenia.