Table 3.
Drugs and compounds for identified drug targets.
| Gene description | Drugs/compounda | Status of drugs/compoundsa |
|---|---|---|
| Lanosterol synthase (LSS) | R0-48-8071 | Preclinical |
| 4-aminobutyrate aminotransferase (ABAT) | Vigabatrin, phenelzine, valproic acid | launched |
| Nicotinamide phosphoribosyltransferase (NAMPT) | FK866 | Phase 2 |
| Farnesyl pyrophosphate synthase (FDPS) | Pamidronic acid, Zoledronic acid, Alendronic acid, Ibandronate, Risedronic acid | Launched |
| Squalene monooxygenase (SQLE) | Ellagic-acid | Phase 2 |
| Farnesyl-diphosphate farnesyltransferase (FDFT1) | TAK-475 | Investigational |
| Bifunctional epoxide hydrolase 2 (EPHX2) | GSK2256294A | Phase 1 |
| 2-oxoglutarate dehydrogenase (OGDH) | Valproic acid | Launched |
| Catalase (CAT) | Fomepizole | Launched |
| Citrate synthase (CS) | Oxaloacetate | Phase 2/phase 3 |
| Pyruvate dehydrogenase E1 component subunit beta (PDHB) | 2-oxopropanoate | Preclinical |
| Catechol O-methyltransferase (COMT) | Entacapone, Nitecapone, Opicapone | Launched, Phase 2, phase 3 |
| 3-hydroxyisobutyryl-CoA hydrolase (HIBCH) | Quercetin | Launched |
| Superoxide dismutase [Mn], mitochondrial (SOD2) | Tetraethylenepentamine | Phase 2/ Phase 3 |
| Dihydroorotate dehydrogenase (DHODH) | Atovaquone, Leflunomide, Teriflunomide, Brequinar | Launched |
| Alpha-glucosidase (GAA) | Miglitol, Acarbose | Launched |
| Acetyl-CoA acetyltransferase, mitochondrial (ACAT1) | Sulfasalazine | Launched |
| Carnitine O-palmitoyltransferase 2 (CPT2) | Perhexiline | Launched |
| ATP synthase subunit alpha (ATP5F1A) | Quercetin | Launched, |
| ATP synthase subunit beta (ATP5F1B) | Quercetin | Launched |
| ATP synthase F1 subunit gamma (ATP5F1C) | Quercetin | Launched |
| Ornithine decarboxylase (ODC1) | MC-1, Putrescine, DFMO | Phase 3, Phase 2, launched |
| MP cyclohydrolase (ATIC) | Pemetrexed | Launched |
| ATP synthase subunit delta (ATP5F1D) | Sevoflurane, Enflurane, Methoxyflurane | Launched |
| ATP synthase F(0) complex subunit B1 (ATP5PB) | BTB06584 | Preclinical |
| Alcohol dehydrogenase class-3 (ADH5) | N6022 | Phase 1/Phase 2 |
| Dihydropyrimidine dehydrogenase (DPYD) | 5-fluorouracil, Gimeraci | Launched |
| Isocitrate dehydrogenase (IDH2) | AGI-6780 | Preclinical |
| Glutathione reductase (GSR) | Carmustine | Launched |
| 3-hydroxy-3-methylglutaryl-coenzyme A reductase (HMGCR) | Atorvastatin, Fluvastatin, Lovastatin, Meglutol, Pitavastatin, Pravastatin, Rosuvastatin, Simvastatin, Nadide | Launched |
| Dihydrofolate reductase (DHFR) | Aminopterin, Chlorproguanil, Methotrexate, Pralatrexate, Proguanil, Pyrimethamine, Sulfameter, Trimethoprim, Pemetrexed | Launched |
| Pyruvate kinase (PKM) | TEPP-46, 2-oxopropanoate | Preclinical |
| Phosphoribosylglycinamide formyltransferase (GART) | Pemetrexed | Launched |
| Fatty acid synthase (FASN) | Pyrazinamide, Cerulenin | Launched |
| Phosphoribosyl pyrophosphate amidotransferase (PPAT) | Azathioprine, Mercaptopurine | Launched |
| Purine nucleoside phosphorylase (PNP) | Acyclovir, Didanosine | Launched |
| ADP/ATP translocase 3 (SLC25A6) | Clodronic-acid | Launched |
| Carbamoyl-phosphate synthetase 2 (CAD) | Sparfosate | Phase 3 |
| Inosine triphosphate pyrophosphatase (ITPA) | Citric-acid | Preclinical |
| Adenosine kinase (ADK) | ABT-702 | Preclinical |
| Long-chain-fatty-acid–CoA ligase 3 (ACSL3) | Eicosapentaenoic acid | Launched |
| Thymidine phosphorylase (TYMP) | Tipiracil | Launched |
| S-adenosylmethionine decarboxylase proenzyme (AMD1) | Ademetionine, Putrescine | Launched, Phase 2 |
| Adenine phosphoribosyltransferase (APRT) | Citric acid | Preclinical |
| Beta-galactosidase (GLB1) | Fagomine | Phase 2 |
| Phosphatidylglycerophosphatase and protein-tyrosine phosphatase 1 (PTPMT1) | Alexidine | Preclinical |
| Adenosine deaminase (ADA) | Cladribine, Pentostatin, Dipyridamole, Fludarabine | Launched |
| Lactoylglutathione lyase (GLO1) | Indomethacin | Launched |
| Glucose-6-phosphate 1-dehydrogenase (G6PD) | RRx-001 | Phase 2 |
| Hypoxanthine-guanine phosphoribosyltransferase (HPRT1) | Azathioprine, Mercaptopurine | Launched |
| Solute carrier family 2, facilitated glucose transporter member 3 (SLC2A3) | 2-deoxyglucose | Phase 2 |
| Multidrug resistance-associated protein 1 (ABCC1) | Reversan, Ko143 | Preclinical |
| Thioredoxin reductase 1 (TXNRD1) | Fotemustine | Launched |
| Superoxide dismutase (SOD1) | Tetraethylenepentamine | Phase 2/Phase 3 |
| ATP-citrate synthase (ACLY) | ETC-1002 | Phase 3 |