🎯 Drug Targets

Browse 97 drug targets with druggability analysis, composite scores, and clinical context

97

Targets

0

High Druggability

0.51

Avg Score

15

Target Classes

Druggability Distribution

High: 0Medium: 0Low: 97Unknown: 0

Avg druggability score: 0.362

Clinical Pipeline

Approved: 25Phase III: 13Phase II: 37Phase I: 21Preclinical: 1

Total compounds: 197 · Approved: 22

Filtered by: druggability=Low — 97 results

P2RY1 P2Y purinoreceptor 1 Phase I

Gpcr Low Druggability

GPCR antagonists or agonists modulating purinergic signaling

HSP90AA1 Heat Shock Protein 90 Alpha Family Class A Member Phase III

Chaperone Low Druggability

Small molecule inhibitor binding to ATP-binding pocket of chaperone

TREM2 Triggering receptor expressed on myeloid cells 2 Phase II

Receptor Low Druggability

Agonist antibodies that enhance TREM2 signaling to promote microglial function

ULK1 Unc-51 like autophagy activating kinase 1 Phase I

Kinase Low Druggability

Small molecule activators or inhibitors of ULK1 kinase activity to modulate autophagy

LOX Lysyl oxidase Phase II

Enzyme Low Druggability

Small molecule inhibition of lysyl oxidase enzymatic activity

CX3CR1 C-X3-C Motif Chemokine Receptor 1 Phase II

Gpcr Low Druggability

GPCR antagonist modulating microglial activation and neuroinflammation

SIRT6 Sirtuin-6 Phase I

Epigenetic Regulator Low Druggability

Small molecule activation or inhibition of NAD+-dependent deacetylase activity

TFR1 Transferrin receptor protein 1 Approved

Receptor Low Druggability

Monoclonal antibodies targeting receptor or iron chelation affecting iron uptake

C1Q Complement C1q Approved

Signaling Protein Low Druggability

Complement cascade inhibitor or antibody-mediated neutralization

BDNF Brain Derived Neurotrophic Factor Phase II

Ligand Low Druggability

Protein replacement therapy or small molecule mimetic

TET2 Tet methylcytosine dioxygenase 2 Phase II

Epigenetic Regulator Low Druggability

Small molecule enhancers of TET2 enzymatic activity or cofactor supplementation

TLR4 Toll-like receptor 4 Phase III

Receptor Low Druggability

Small molecule antagonist or antibody blocking TLR4-mediated neuroinflammatory signaling

GJA1 Gap junction alpha-1 protein (Connexin 43) Phase II

Ion Channel Low Druggability

Small molecule modulator of gap junction channel activity

MIRO1 Mitochondrial Rho GTPase 1 Phase II

Signaling Protein Low Druggability

MIRO1-targeting drugs modulate GTPase activity to enhance mitochondrial transport along axons and improve mitochondrial quality control through selective autophagy. By regulating MIRO1's interaction with kinesin motor proteins and PINK1-mediated mitophagy, these compounds restore energy homeostasis and reduce neuronal stress in neurodegenerative diseases.

SOAT1 Sterol O-acyltransferase 1 Phase III

Enzyme Low Druggability

Small molecule inhibitor of acyl-CoA:cholesterol acyltransferase activity

PRMT1 Protein arginine methyltransferase 1 Phase I

Epigenetic Regulator Low Druggability

Small molecule inhibitor of protein arginine methyltransferase activity

PPARGC1A Peroxisome proliferator-activated receptor gamma c Approved

Transcription Factor Low Druggability

Small molecule modulator of transcriptional coactivator function

STING1 Stimulator of interferon genes protein 1 Phase II

Signaling Protein Low Druggability

Small molecule agonists or antagonists of cGAS-STING pathway activation

GPR37 G-protein coupled receptor 37 Approved

Gpcr Low Druggability

GPR37 agonists or modulators would activate the receptor to promote dopaminergic neuron survival and neuroprotection, counteracting the neurodegeneration characteristic of Parkinson's disease. Ligands binding to GPR37 would enhance G-protein signaling cascades that support neuronal viability and function.

GPX4 Glutathione peroxidase 4 Phase II

Enzyme Low Druggability

Small molecule inhibitor or activator of peroxidase activity

EPHB4 Ephrin type-B receptor 4 Approved

Kinase Low Druggability

Small molecule inhibitor of receptor tyrosine kinase activity

SRPK1 SRSF protein kinase 1 Phase II

Kinase Low Druggability

SRPK1 inhibitors block phosphorylation of serine/arginine-rich splicing factors, modulating alternative splicing patterns implicated in neurodegeneration and cancer. By preventing aberrant splicing, these inhibitors can restore normal protein isoform expression and reduce pathogenic tau accumulation.

HDAC3 Histone Deacetylase 3 Approved

Epigenetic Regulator Low Druggability

Small molecule inhibitor of histone deacetylase enzymatic activity

ANGPT1 Angiopoietin-1 Approved

Ligand Low Druggability

Protein therapeutics or small molecule mimetics activating Tie2 signaling

FOXO3 Forkhead box protein O3 Approved

Transcription Factor Low Druggability

FOXO3-targeting drugs modulate the PI3K/Akt/FOXO3 signaling pathway to promote FOXO3 nuclear translocation and transcriptional activity, enhancing autophagy, stress resistance, and apoptosis in cancer cells. Indirect activators increase FOXO3 expression or prevent its degradation, thereby amplifying cellular protective responses.

ANXA1 Annexin A1 Approved

Signaling Protein Low Druggability

Calcium-dependent phospholipid binding protein modulator

TGFB1 Transforming growth factor beta-1 Phase III

Ligand Low Druggability

Monoclonal antibodies neutralizing TGF-β1 or small molecule inhibitors of TGF-β signaling

AQP1 Aquaporin-1 Approved

Ion Channel Low Druggability

AQP1 functions as a water channel protein facilitating transmembrane water flux, with emerging evidence suggesting its involvement in neuroinflammatory processes and potential contribution to neuronal cell volume regulation and edema formation in neurodegenerative conditions. In the context of neurodegeneration, AQP1 may modulate neuronal and glial cell responses to oxidative stress, inflammation, and cellular metabolic perturbations, making it a potential strategic target for therapeutic intervention.

LRP1 LDL receptor related protein 1 Phase III

Receptor Low Druggability

Modulation of receptor-mediated endocytosis and clearance pathways

HK2 Hexokinase 2 Phase III

Enzyme Low Druggability

Small molecule inhibitor of glucose phosphorylation

MCOLN1 Mucolipin-1 Phase I

Ion Channel Low Druggability

Small molecule agonists or modulators of lysosomal calcium channel activity

RAB27A Ras-related protein Rab-27A Phase II

Signaling Protein Low Druggability

RAB27A inhibitors would block GTP binding or GTPase activity, preventing the recruitment of effector proteins required for vesicular trafficking and exosome biogenesis. This would reduce the release of extracellular vesicles and potentially modulate intercellular communication in disease states involving excessive exosome-mediated signaling or pathological cargo transfer.

SDC1 Syndecan-1 Phase II

Receptor Low Druggability

Small molecule modulation of heparan sulfate interactions or shedding

CMKLR1 Chemokine-like Receptor 1 Phase II

Gpcr Low Druggability

GPCR antagonist or agonist modulating chemerin signaling

CLOCK Circadian Locomotor Output Cycles Kaput Approved

Transcription Factor Low Druggability

Modulation of circadian transcriptional activity through protein-protein interaction disruption

ALOX15 15-lipoxygenase Approved

Enzyme Low Druggability

Small molecule inhibitors targeting the active site iron and substrate binding

C1QA Complement C1q A Chain Phase II

Signaling Protein Low Druggability

Complement cascade inhibitor or antibody-mediated neutralization

CRH Corticotropin Releasing Hormone Approved

Ligand Low Druggability

CRH receptor antagonist blocking stress hormone signaling

NAMPT Nicotinamide phosphoribosyltransferase Phase II

Enzyme Low Druggability

Small molecule competitive inhibitors of NAD+ biosynthesis enzyme

BMAL1 Basic Helix-Loop-Helix ARNT Like 1 Phase I

Transcription Factor Low Druggability

Small molecule modulator of circadian transcription

AHR Aryl hydrocarbon receptor Phase II

Transcription Factor Low Druggability

Small molecule modulators affecting ligand-dependent transcriptional activity

LAMP1 Lysosomal associated membrane protein 1 Phase I

Structural Protein Low Druggability

Direct LAMP1 modulators enhance lysosomal membrane trafficking and autophagy flux, while indirect pathway drugs alter lysosomal pH and autophagosome-lysosome fusion dynamics to modulate LAMP1-mediated cellular clearance mechanisms.

ALOX12 12-lipoxygenase Phase I

Enzyme Low Druggability

Small molecule inhibitors of lipoxygenase enzymatic activity

HNRNPA2B1 Heterogeneous Nuclear Ribonucleoprotein A2/B1 Phase II

Other Low Druggability

Therapeutic agents targeting HNRNPA2B1 would work by modulating its RNA-binding activity, preventing pathological protein aggregation, or reducing the formation of cytoplasmic inclusions associated with neurodegeneration. These drugs could utilize antisense oligonucleotides to reduce HNRNPA2B1 expression or small molecules to inhibit its protein-protein interactions and RNA-binding functions.

NTN1 Netrin-1 Phase I

Ligand Low Druggability

Modulation of axon guidance and neuronal survival pathways

IL10 Interleukin-10 Phase II

Ligand Low Druggability

Recombinant protein replacement or monoclonal antibody modulation

DNASE2 Deoxyribonuclease 2 lysosomal Phase I

Enzyme Low Druggability

Small molecule inhibitor or activator of nuclease activity

SST Somatostatin Approved

Ligand Low Druggability

Somatostatin is a peptide neurotransmitter that modulates neuronal signaling by inhibiting hormone release and neuronal excitability, with emerging evidence suggesting its potential neuroprotective role in neurodegenerative processes through regulation of inflammatory responses and synaptic plasticity. In neurodegeneration contexts, SST receptors may serve as modulators of neuroinflammatory cascades and potentially mitigate progressive neuronal damage through receptor-mediated signaling mechanisms.

DNAJB1 DnaJ heat shock protein family (Hsp40) member B1 Phase I

Chaperone Low Druggability

Allosteric modulation of protein-protein interactions with Hsp70

PIEZO1 Piezo-type mechanosensitive ion channel component Phase I

Ion Channel Low Druggability

Small molecule modulator of mechanosensitive ion channel activity

IGF2R Insulin-like growth factor 2 receptor Approved

Receptor Low Druggability

Drugs targeting IGF2R typically work by blocking IGF2 binding and signaling through the IGF1R/IGF2R axis, thereby inhibiting mitogenic and anti-apoptotic effects in cancer cells. Alternatively, agents may enhance IGF2 clearance through IGF2R-mediated endocytosis to reduce circulating IGF2 levels and downstream signaling.

MLCK Myosin light chain kinase Phase II

Kinase Low Druggability

MLCK inhibitors block the phosphorylation of myosin light chains, thereby reducing cytoskeletal contraction and stabilizing tight junctions at the blood-brain barrier and endothelial surfaces. This mechanism reduces vascular permeability and preserves barrier integrity in conditions characterized by excessive leakage or cytoskeletal dysfunction.

PLA2G4A Phospholipase A2 group IVA Phase II

Enzyme Low Druggability

Small molecule inhibitor of cytosolic phospholipase A2 enzymatic activity

KDM6A Lysine demethylase 6A Phase I

Epigenetic Regulator Low Druggability

Small molecule inhibition of histone demethylase activity

COX4I1 Cytochrome C Oxidase Subunit 4I1 Phase II

Enzyme Low Druggability

Drugs targeting COX4I1 pathway would enhance electron transport chain efficiency or stabilize complex IV assembly, improving ATP production and reducing oxidative stress in mitochondria. These agents work by either promoting mitochondrial bioenergetics, acting as electron donors/acceptors, or preventing complex IV degradation in diseases with impaired oxidative phosphorylation.

ZO1 Zonula occludens-1 Approved

Structural Protein Low Druggability

Drugs targeting ZO1 typically work by stabilizing or modulating tight junction protein interactions, either by directly binding to ZO1 scaffolding domains or by regulating upstream signaling pathways that control ZO1 phosphorylation and localization. These approaches aim to restore blood-brain barrier integrity or modulate paracellular permeability depending on therapeutic context.

OCLN Occludin Approved

Structural Protein Low Druggability

Drugs targeting OCLN would stabilize or enhance tight junction protein interactions, reinforcing the structural integrity of the blood-brain barrier and reducing pathological barrier permeability. Therapeutic approaches would involve either direct protein stabilization or indirect modulation of occludin phosphorylation and trafficking to maintain barrier function.

TARDBP TAR DNA-binding protein 43 Phase III

Transcription Factor Low Druggability

RNA-binding and transcription regulation, challenging for small molecule targeting

NPM1 Nucleophosmin Phase III

Chaperone Low Druggability

Small molecule modulators of nucleolar function and protein interactions

CHMP2B Charged multivesicular body protein 2B Phase II

Other Low Druggability

Therapeutic agents targeting CHMP2B would modulate ESCRT-III complex function to enhance endosomal sorting and autophagy, potentially clearing protein aggregates associated with frontotemporal dementia or compensating for loss-of-function mutations. Strategies include antisense oligonucleotides to modulate protein expression, autophagy enhancers to promote cellular clearance pathways, or small molecules that stabilize ESCRT-III complex assembly and function.

CERS2 Ceramide synthase 2 Phase II

Enzyme Low Druggability

CERS2 inhibitors would reduce the enzymatic synthesis of very long-chain ceramides by blocking the condensation of serine and palmitoyl-CoA, thereby decreasing ceramide-mediated neuroinflammation and apoptosis implicated in neurodegenerative pathology. This modulation of the ceramide signaling cascade may provide neuroprotective effects in Alzheimer's disease and related neurodegenerative conditions.

PYCARD Apoptosis-associated speck-like protein containing Phase II

Signaling Protein Low Druggability

PYCARD acts as a critical adaptor protein in inflammasome activation, facilitating pro-inflammatory caspase-1 recruitment and subsequent IL-1β/IL-18 processing, which contributes to neuroinflammatory processes in neurodegenerative conditions like Alzheimer's and Parkinson's disease. Its protein-protein interaction domains mediate inflammatory signal transduction, potentially amplifying neuronal damage through excessive immune response activation.

ST6GAL1 ST6 beta-galactoside alpha-2,6-sialyltransferase 1 Approved

Enzyme Low Druggability

ST6GAL1 inhibitors would block the addition of α2,6-linked sialic acids to N-glycans on cell surface proteins, altering immune cell recognition and potentially enhancing anti-tumor immunity or modulating inflammatory responses. This glycosylation modification is crucial for immune evasion in cancer and autoimmune disease pathogenesis.

HSPG2 Heparan Sulfate Proteoglycan 2 Phase II

Structural Protein Low Druggability

Targeting protein-heparan sulfate interactions or enzymatic modification

DGAT1 Diacylglycerol O-Acyltransferase 1 Phase III

Enzyme Low Druggability

Small molecule inhibitor of triglyceride synthesis enzyme

SYNCRIP Heterogeneous nuclear ribonucleoprotein Q Phase II

Signaling Protein Low Druggability

Small molecule or antisense-based inhibitors would disrupt SYNCRIP's RNA-binding capability, reducing its ability to facilitate mRNA transport and local protein synthesis, potentially modulating disease pathways in neurological disorders or cancers where SYNCRIP dysfunction is implicated.

CHR2 Channelrhodopsin-2 Phase II

Ion Channel Low Druggability

CHR2 functions as a light-gated cation channel that opens in response to blue light (470nm), allowing depolarization through Na+ and Ca2+ influx. Therapeutic approaches would involve delivering CHR2 via gene therapy to degenerated neurons or photoreceptors, enabling light-dependent control of neural activity for vision restoration or neuromodulation applications.

FOXP3 Forkhead box protein P3 Phase I

Transcription Factor Low Druggability

Transcription factor modulation - no direct small molecule binding

SETX Senataxin Approved

Enzyme Low Druggability

Therapeutic agents targeting SETX would likely enhance RNA helicase activity or stabilize protein function to improve transcriptional regulation and DNA repair, or alternatively inhibit aberrant signaling pathways triggered by SETX dysfunction in neuronal tissues. Small molecule stabilizers or gene therapy approaches could restore deficient helicase activity in ataxia-causing mutations.

PLA2G6 Phospholipase A2 group VI Approved

Enzyme Low Druggability

Small molecule inhibitor of calcium-independent phospholipase A2 activity

SNAP25 Synaptosome associated protein 25 Approved

Structural Protein Low Druggability

Protein cleavage by botulinum toxin or small molecule modulators of SNARE complex formation

RELN Reelin Approved

Signaling Protein Low Druggability

Drugs targeting RELN would primarily work by enhancing reelin expression or signaling through its receptors (VLDLR and ApoER2) to promote neuronal migration, synaptic plasticity, and cognitive function. Alternatively, pathway modulators could enhance downstream signaling cascades (Dab1-mediated pathways) to compensate for reelin deficiency or dysfunction.

SGMS2 Sphingomyelin synthase 2 Phase III

Enzyme Low Druggability

SGMS2 inhibitors would block the enzymatic transfer of phosphocholine to ceramide, reducing sphingomyelin biosynthesis and altering membrane composition and cellular signaling. This modulation could reduce pathological lipid accumulation and restore normal cell membrane dynamics in diseases characterized by sphingolipid dysregulation.

MCU Mitochondrial calcium uniporter Phase III

Ion Channel Low Druggability

MCU inhibitors block the mitochondrial calcium uniporter channel, reducing excessive calcium accumulation in mitochondria that leads to oxidative stress, energy depletion, and cell death. This mechanism may protect against ischemic injury, neurodegeneration, and heart failure by preserving mitochondrial function and preventing apoptosis in calcium-sensitive tissues.

FUT8 Alpha-(1,6)-fucosyltransferase Phase II

Enzyme Low Druggability

FUT8 inhibitors would block the addition of core fucose to N-linked glycans on proteins, modulating antibody-dependent cellular cytotoxicity (ADCC) and potentially reducing pathological protein aggregation in neurodegenerative diseases. By preventing fucosylation, these drugs could enhance immune effector function or alter protein conformational stability and clearance.

SGMS1 Sphingomyelin synthase 1 Approved

Enzyme Low Druggability

SGMS1 inhibitors would block the transfer of phosphocholine from phosphatidylcholine to ceramide, reducing sphingomyelin synthesis and potentially decreasing membrane rigidity and inflammatory signaling. This mechanism could modulate membrane-dependent cellular processes and may have therapeutic effects in lipid storage disorders and metabolic diseases.

AQP4 Aquaporin-4 Phase I

Ion Channel Low Druggability

Water channel inhibitor or modulator

FLOT1 Flotillin 1 Phase II

Structural Protein Low Druggability

Drug candidates would modulate FLOT1-mediated lipid raft organization and signaling platform assembly, thereby disrupting pathological protein trafficking and reducing amyloid-beta or tau-related pathology in neurodegenerative diseases. FLOT1 inhibitors would interfere with membrane scaffold formation necessary for aberrant signaling in neuroinflammatory and proteinopathic cascades.

CAV1 Caveolin-1 Approved

Structural Protein Low Druggability

No established drugging mechanism for structural membrane protein

LOXL1-4 Lysyl oxidase-like 1-4 Phase II

Enzyme Low Druggability

Small molecule enzyme inhibitors targeting the catalytic domain

BRD4 Bromodomain-containing protein 4 Phase II

Epigenetic Regulator Low Druggability

BRD4 inhibition reduces neuroinflammatory super-enhancer activity while biased BRD4 modulators can selectively restore neuroprotective gene expression

ST8SIA1 ST8 alpha-N-acetyl-neuraminide alpha-2,8-sialyltra Phase II

Enzyme Low Druggability

ST8SIA1 inhibitors would block the synthesis of polysialic acid chains on NCAM, reducing neural cell plasticity and potentially modulating neuroinflammatory responses. This mechanism could be therapeutically relevant for neurodevelopmental disorders or neuroinflammatory conditions where excessive polysialylation contributes to pathology.

SLC16A2 Monocarboxylate transporter 8 Phase I

Transporter Low Druggability

Substrate supplementation or transporter modulation

CHMP4B Charged multivesicular body protein 4B Phase II

Other Low Druggability

CHMP4B-targeting drugs would inhibit or modulate ESCRT-III complex assembly and membrane scission, disrupting cellular degradation pathways (autophagy, lysosomal trafficking) and potentially inhibiting viral budding or cancer cell survival. Therapeutics could involve blocking protein-protein interactions within the ESCRT complex or stabilizing/destabilizing CHMP4B conformational states.

CNO Cappuccino Phase III

Other Low Druggability

No established druggable mechanisms

MTOR Mechanistic Target of Rapamycin Phase I

Kinase Low Druggability

Prioritized from 1 SciDEX hypotheses, including: APOE-Dependent Autophagy Restoration

ST3GAL2 ST3 beta-galactoside alpha-2,3-sialyltransferase 2 Phase II

Enzyme Low Druggability

Inhibitors of ST3GAL2 would block the transfer of sialic acid (α-2,3 linkage) to galactose residues on glycoproteins and glycolipids, reducing cell surface sialylation and potentially enhancing immune recognition of cancer cells or modulating inflammatory responses. Alternatively, activators or substrate analogs could enhance sialylation for immune tolerance in inflammatory conditions.

GPR109A Hydroxycarboxylic Acid Receptor 2 (HCAR2)

Receptor Low Druggability

Prioritized from 1 SciDEX hypotheses, including: Targeted Butyrate Supplementation for Microglial Phenotype Modulation

CELL-TYPE-SPECIFICESSENTIALGENES CELL-TYPE-SPECIFICESSENTIALGENES Phase I

Signaling Protein Low Druggability

Prioritized from 1 SciDEX hypotheses, including: Context-Dependent CRISPR Activation in Specific Neuronal Subtypes

TRAK1_KIF5A Trafficking kinesin protein 1 / Kinesin family mem Phase II

Transporter Low Druggability

Therapeutic approaches targeting TRAK1_KIF5A aim to restore or enhance axonal mitochondrial transport by either stabilizing the KIF5A motor protein complex, reducing mutant protein expression through antisense oligonucleotides, or replacing defective protein via gene therapy. This restores cellular energy homeostasis and reduces neuronal degeneration in motor neurons.

PVALB Parvalbumin Phase II

Signaling Protein Low Druggability

No known druggable mechanism

ACSL4 Long-chain-fatty-acid--CoA ligase 4 Phase III

Enzyme Low Druggability

Enzyme inhibition — blocking ACSL4 prevents incorporation of PUFAs into membrane phospholipids, reducing ferroptosis susceptibility

TDC TDC Protein Approved

Protein Low Druggability

Prioritized from 1 SciDEX hypotheses, including: Restoring Neuroprotective Tryptophan Metabolism via Targeted Probiotic Engineering

SLC16A1 Monocarboxylate transporter 1 (MCT1) Phase II

Transporter Low Druggability

Transporter modulation — restoring MCT1/MCT4 balance to normalize astrocyte-neuron metabolic coupling

DISEASE-CAUSINGMUTATIONSWITHINTEGRATEDREPORTERS DISEASE-CAUSINGMUTATIONSWITHINTEGRATEDREPORTERS Phase I

Signaling Protein Low Druggability

Prioritized from 1 SciDEX hypotheses, including: Multi-Modal CRISPR Platform for Simultaneous Editing and Monitoring

SYNTHETICFUSIONPROTEINS SYNTHETICFUSIONPROTEINS Phase I

Signaling Protein Low Druggability

Prioritized from 1 SciDEX hypotheses, including: Synthetic Biology Approach: Designer Mitochondrial Export Systems

NEURONALIDENTITYTRANSCRIPTIONFACTORS NEURONALIDENTITYTRANSCRIPTIONFACTORS Phase I

Signaling Protein Low Druggability

Prioritized from 1 SciDEX hypotheses, including: Programmable Neuronal Circuit Repair via Epigenetic CRISPR