🎯 Drug Targets

Browse 56 drug targets with druggability analysis, composite scores, and clinical context

56

Targets

0

High Druggability

0.56

Avg Score

15

Target Classes

Druggability Distribution

High: 0Medium: 56Low: 0Unknown: 0

Avg druggability score: 0.518

Clinical Pipeline

Approved: 31Phase III: 9Phase II: 9Phase I: 5Preclinical: 2

Total compounds: 145 · Approved: 63

Filtered by: druggability=Medium — 56 results

MTNR1A Melatonin receptor 1A Approved

Gpcr Medium Druggability

Small molecule agonists that activate G-protein coupled receptor signaling

PRKAA1 AMP-activated protein kinase catalytic subunit alp Approved

Kinase Medium Druggability

Small molecule activator of AMP-activated protein kinase

KCNK2 Potassium two pore domain channel subfamily K memb Approved

Ion Channel Medium Druggability

KCNK2 encodes a two-pore domain potassium channel (TREK-1) that regulates neuronal excitability, membrane potential, and cellular responsiveness to mechanical and chemical stimuli in the central nervous system. In neurodegeneration, TREK-1 modulation can influence neuronal survival, mitochondrial function, and potentially mitigate oxidative stress and excitotoxicity through precise potassium conductance regulation.

C3 Complement C3 Approved

Signaling Protein Medium Druggability

Small molecule inhibitor of complement activation or convertase activity

PARP1 Poly [ADP-ribose] polymerase 1 Approved

Enzyme Medium Druggability

Competitive inhibitors of PARP1 enzymatic activity blocking DNA repair

TH Tyrosine hydroxylase Approved

Enzyme Medium Druggability

Enzyme replacement therapy via L-DOPA supplementation to bypass reduced TH activity

IL1B Interleukin-1 beta Approved

Ligand Medium Druggability

IL-1 receptor antagonist or neutralizing monoclonal antibody

NLRP3 NACHT, LRR and PYD domains-containing protein 3 Approved

Signaling Protein Medium Druggability

Small molecule inhibitors targeting NLRP3 inflammasome assembly or activation

G3BP1 Ras GTPase-activating protein-binding protein 1 Phase II

Signaling Protein Medium Druggability

G3BP1 inhibitors would prevent stress granule assembly and stabilization by disrupting the interaction between G3BP1 and its binding partners, potentially reducing pathological aggregation of TDP-43 and FUS proteins. This mechanism could alleviate neurodegeneration in conditions characterized by aberrant stress granule formation and RNA metabolism dysfunction.

GLP1R Glucagon-like peptide-1 receptor Approved

Gpcr Medium Druggability

GLP-1 receptor agonist peptides and small molecule modulators

PDGFRB Platelet-derived growth factor receptor beta Approved

Kinase Medium Druggability

Small molecule inhibitor of receptor tyrosine kinase activity

CGAS Cyclic GMP-AMP synthase Phase III

Enzyme Medium Druggability

Small molecule inhibitor of cyclic dinucleotide synthesis

APOE Apolipoprotein E Phase II

Ligand Medium Druggability

Protein-protein interaction modulator or lipid metabolism enhancer

ADORA2A Adenosine A2A receptor Approved

Gpcr Medium Druggability

Small molecule antagonists blocking adenosine A2A receptor signaling

SLC7A11 Cystine/glutamate transporter Approved

Transporter Medium Druggability

Small molecule inhibition of cystine/glutamate exchange

ADRA2A Alpha-2A adrenergic receptor Approved

Gpcr Medium Druggability

Small molecule agonists and antagonists modulating noradrenergic signaling

HSPA1A Heat Shock Protein Family A Member 1A Phase III

Chaperone Medium Druggability

Small molecule activators or allosteric modulators of chaperone function

SNCA Synuclein alpha Phase II

Structural Protein Medium Druggability

Immunotherapy targeting alpha-synuclein aggregates or small molecules preventing aggregation

HCRTR1 Hypocretin Receptor 1 Approved

Gpcr Medium Druggability

Small molecule antagonist blocking orexin receptor signaling

FCGRT Fc fragment of IgG receptor and transporter Approved

Transporter Medium Druggability

Monoclonal antibody blocking FcRn-mediated IgG recycling

HCRTR2 Hypocretin Receptor 2 Approved

Gpcr Medium Druggability

Small molecule antagonist blocking orexin receptor signaling

P2RY12 P2Y purinoreceptor 12 Approved

Gpcr Medium Druggability

P2Y12 receptor antagonists blocking ADP-mediated signaling

TGM2 Protein-glutamine gamma-glutamyltransferase 2 Phase II

Enzyme Medium Druggability

Small molecule inhibitors of transglutaminase enzymatic activity

CD38 ADP-ribosyl cyclase/cyclic ADP-ribose hydrolase 1 Approved

Enzyme Medium Druggability

Monoclonal antibody targeting or small molecule inhibitor of NAD+ consuming activity

FKBP5 FKBP prolyl isomerase 5 Phase I

Enzyme Medium Druggability

Small molecule inhibitor of prolyl isomerase activity and HSP90 co-chaperone function

MMP9 Matrix metalloproteinase-9 Phase III

Protease Medium Druggability

Small molecule zinc-chelating inhibitors or antibody-based inhibition

NR3C1 Glucocorticoid receptor Approved

Transcription Factor Medium Druggability

Selective glucocorticoid receptor modulators (SRMs) or antagonists

TUBB3 Tubulin beta-3 chain Approved

Structural Protein Medium Druggability

Small molecule stabilizers of microtubule structure or assembly

STX17 Syntaxin-17 Phase II

Structural Protein Medium Druggability

STX17-targeting drugs would enhance or modulate autophagosome-lysosome fusion by facilitating SNARE complex assembly, thereby improving autophagic clearance of misfolded proteins and damaged organelles. This mechanism is particularly relevant for neurodegenerative diseases characterized by protein aggregation.

TFRC Transferrin receptor protein 1 Approved

Receptor Medium Druggability

Monoclonal antibodies targeting receptor or iron chelation affecting iron uptake

FOXO1 Forkhead box protein O1 Phase I

Transcription Factor Medium Druggability

Small molecule modulation of protein-DNA interactions or cofactor binding

TNFA Tumor necrosis factor alpha Approved

Signaling Protein Medium Druggability

Monoclonal antibodies or soluble receptors that neutralize TNF-alpha activity

TFAM Transcription factor A, mitochondrial Approved

Transcription Factor Medium Druggability

Drugs targeting TFAM would enhance or modulate its transcriptional activity to increase mitochondrial DNA replication and gene expression, thereby boosting mitochondrial biogenesis and ATP production. Alternatively, indirect approaches activate upstream regulators like PGC-1α and SIRT1 to increase TFAM expression and activity.

HCN1 Hyperpolarization-activated cyclic nucleotide-gate Approved

Ion Channel Medium Druggability

HCN1 encodes a hyperpolarization-activated cyclic nucleotide-gated channel critical for neuronal pacemaking and synaptic integration, with dysregulation linked to hyperexcitability disorders and potential neurodegeneration mechanisms. Specifically, HCN1 channel dysfunction can alter neuronal excitability, synaptic plasticity, and potentially contribute to neuronal death or dysfunction in conditions like epilepsy and neurodegenerative diseases.

MMP2 Matrix metalloproteinase-2 Phase III

Protease Medium Druggability

Small molecule zinc-chelating inhibitors of metalloprotease activity

CASP1 Caspase-1 Phase II

Protease Medium Druggability

Small molecule covalent inhibitor of caspase-1 proteolytic activity

BACE1 Beta-secretase 1 Phase III

Protease Medium Druggability

Small molecule inhibitor of aspartyl protease activity

DNMT1 DNA methyltransferase 1 Approved

Epigenetic Regulator Medium Druggability

Small molecule inhibitor of DNA methyltransferase activity

ALOX5 5-lipoxygenase Approved

Enzyme Medium Druggability

Small molecule inhibitors blocking leukotriene synthesis

ABCA1 ATP-binding cassette transporter A1 Approved

Transporter Medium Druggability

Small molecule modulators to enhance cholesterol efflux and HDL formation

VCP Valosin containing protein Phase I

Enzyme Medium Druggability

Small molecule inhibitors of VCP ATPase activity affecting protein quality control

HCRT Hypocretin/Orexin Approved

Ligand Medium Druggability

Peptide hormone replacement or stabilization approaches

ABCB1 P-glycoprotein Phase III

Transporter Medium Druggability

Small molecule inhibitors to reduce efflux pump activity and enhance CNS drug penetration

CYP46A1 Cytochrome P450 Family 46 Subfamily A Member 1 Approved

Enzyme Medium Druggability

Small molecule activator of cholesterol 24-hydroxylase enhancing brain cholesterol turnover

SMPD1 Sphingomyelin phosphodiesterase 1 Approved

Enzyme Medium Druggability

Small molecule inhibitor or modulator of sphingomyelinase activity

NLGN1 Neuroligin-1 Phase II

Receptor Medium Druggability

Drugs targeting NLGN1 would modulate synaptic adhesion by either enhancing or blocking the neuroligin-neurexin interaction at the postsynaptic membrane, thereby regulating synapse formation, stabilization, and synaptic transmission. Indirect modulators may enhance GABAergic signaling or vasopressin-mediated pathways that influence neuroligin-dependent synaptic maturation and plasticity.

SIRT1 NAD-dependent protein deacetylase sirtuin-1 Phase III

Epigenetic Regulator Medium Druggability

SIRT1 activation via NAD+ precursors or allosteric activators deacetylates tau (reducing aggregation), enhances mitophagy, and restores circadian gene expression

CSGA CSGA Protein Phase I

Protein Medium Druggability

Prioritized from 1 SciDEX hypotheses, including: Targeting Bacterial Curli Fibrils to Prevent α-Synuclein Cross-Seeding

GFAP Glial fibrillary acidic protein Phase II

Structural Protein Medium Druggability

Biomarker readout — GFAP levels reflect astrocyte reactivity; modulating upstream pathways (e.g., NF-κB, JAK-STAT) reduces GFAP expression

AADC Aromatic L-amino acid decarboxylase Approved

Enzyme Medium Druggability

Small molecule inhibitor preventing peripheral conversion of levodopa to dopamine

SIRT3 NAD-dependent deacetylase sirtuin-3, mitochondrial Phase II

Enzyme Medium Druggability

Enzyme activation — enhancing SIRT3 deacetylase activity restores mitochondrial protein function and reduces oxidative stress

PIEZO1ANDKCNK2 PIEZO1ANDKCNK2

Signaling Protein Medium Druggability

Prioritized from 1 SciDEX hypotheses, including: Mechanosensitive Ion Channel Reprogramming

Structural Protein Medium Druggability

Tau aggregation inhibitors, anti-tau antibodies, tau phosphorylation modulators

DGAT1ANDSOAT1 DGAT1ANDSOAT1

Signaling Protein Medium Druggability

Prioritized from 1 SciDEX hypotheses, including: Lipid Droplet Dynamics as Phenotype Switches

SLC17A7 Vesicular glutamate transporter 1 (VGLUT1) Phase III

Transporter Medium Druggability

Expression restoration — upregulating SLC17A7 transcription or stabilizing VGLUT1 protein to preserve excitatory synaptic function

MITOCHONDRIALBIOGENESISGENES MITOCHONDRIALBIOGENESISGENES Phase I

Signaling Protein Medium Druggability

Prioritized from 1 SciDEX hypotheses, including: Metabolic Reprogramming via Coordinated Multi-Gene CRISPR Circuits