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Lithium hydroxide
go.drugbank.com/drugs/DB14506ApprovedWithdrawnUsed for tryptophan determinations in proteins & foods.
Itepekimab
go.drugbank.com/drugs/DB16206InvestigationalItepekimab is under investigation in clinical trial NCT03546907 (Proof-of-concept Study to Assess the Efficacy, Safety and Tolerability of SAR440340 (Anti-il-33 Mab) in Patients With Moderate-to-severe
HMI-103
go.drugbank.com/drugs/DB17684InvestigationalHMI-102 is an investigational gene therapy designed to encode the phenylalanine-4-hydroxylase (PAH) gene, mutated in people with phenylketonuria.
OPB-111077
go.drugbank.com/drugs/DB16630InvestigationalOPB-111077 is a novel inhibitor of STAT3 and mitochondrial oxidative phosphorylation. It has demonstrated promising anticancer activity in preclinical models.
Tiazofurine
go.drugbank.com/drugs/DB13243ExperimentalTiazofurine has potential clinical use in cancer treatment as it is a potential inhibitor of Inosine- 5’-monophosphate (IMP) dehydrogenase [A174991].
RCT1100
go.drugbank.com/drugs/DB18830InvestigationalRCT1100 is an investigational mRNA-based therapeutic that aims to treat primary ciliary dyskinesia caused by pathogenic mutations in DNAI1.[L50788]
ABX1100
go.drugbank.com/drugs/DB17655InvestigationalABX1100 is an investigational Centyrin-siRNA conjugate that targets the Gys1 gene, which encodes glycogen synthase 1 (Gys1), an enzyme that is responsible for glycogen synthesis in the muscle. … It was first developed by Aro Biotherapeutics and is being investigated for the treatment of Pompe disease.[L45978]
AUM-601
go.drugbank.com/drugs/DB17382ExperimentalAUM-601 is a highly selective pan-TRK(tropomyosin receptor kinase). It is currently being investigated in clinical trials for the treatment of cancer.
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]