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CDX-6512
go.drugbank.com/drugs/DB17841ExperimentalCDX-6512 is a modified, gastrointestinal-stable bacterial leucine decarboxylase. Developed by Codexis, it is being investigated for the treatment of homocystinuria And Maple Syrup Urine Disease.
Deuremidevir
go.drugbank.com/drugs/DB26606InvestigationalDeuremidevir is a small molecule drug. … Deuremidevir is under investigation in clinical trial NCT06328400 (A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Oral Deuremidevir Hydrobromide for Suspension).
VIR-5500
go.drugbank.com/drugs/DB28578InvestigationalVIR-5500 is a small molecule drug. VIR-5500 is under investigation in clinical trial NCT05997615 (Safety, Pharmacokinetics, and Preliminary Efficacy of VIR-5500 (AMX-500) in Prostate Cancer).
Temanogrel
go.drugbank.com/drugs/DB05227InvestigationalAPD791 is an oral anti-thrombotic drug candidate being evaluated in a Phase 1 clinical trial by Arena. … APD791 is intended to lower the risk of arterial thrombosis by reducing the amplification of platelet aggregation, arterial constriction and intimal hyperplasia mediated by serotonin.
N-Methylmesoporphyrin
go.drugbank.com/drugs/DB01911ExperimentalThis medication is known to target ferrochelatase. … N-methylmesoporphyrin is a solid. This compound belongs to the porphyrins.
RGX-501
go.drugbank.com/drugs/DB17639InvestigationalIt is being investigated for the treatment of homozygous familial hypercholesterolemia.[L46696] … RGX-501 is an adeno-associated virus vector 8 (AAV8)-mediated gene therapy that delivers a functional copy of the human low-density lipoprotein receptor (LDLR) gene to liver cells.
Dabocemagene autoficel
go.drugbank.com/drugs/DB17878InvestigationalDeveloped by Castle Creek Biosciences Inc., dabocemagene autoficel is being investigated for the treatment of dystrophic epidermolysis bullosa.[L46797] … Dabocemagene autoficel is an _ex vivo_, autologous cell-based gene therapy. It comprises autologous dermal fibroblasts genetically modified to express functional type VII collagen.
DYNE-251
go.drugbank.com/drugs/DB18130InvestigationalIt is being investigated for the treatment of Duchenne muscular dystrophy (DMD) in patients who are amenable to exon 51 skipping. … DYNE-251 is a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1).
Zibrofusidic acid
go.drugbank.com/drugs/DB20799InvestigationalZibrofusidic acid is a small molecule drug. … Zibrofusidic acid is under investigation in clinical trial NCT00626795 (Efficacy, Safety, and Tolerability of TD1414 2% Cream in Impetigo and Secondarily Infected Traumatic Lesions (SITL)).
NTLA-2002
go.drugbank.com/drugs/DB17597InvestigationalNTLA-2002 is a lipid nanoparticle encapsulating single guide RNA (G012267) targeting the human KLKB1 gene and messenger RNA (mRNA000042) encoding Cas9. … It is an investigational therapy designed to knock out the target gene kallikrein B1 (KLKB1) to reduce plasma kallikrein activity, thereby preventing hereditary angioedema attacks.[L45738]