🎯 Drug Targets

Browse 100 drug targets with druggability analysis, composite scores, and clinical context

100
Targets
0
High Druggability
0.55
Avg Score
15
Target Classes
Druggability Distribution
High: 0Medium: 0Low: 100Unknown: 0
Avg druggability score: 0.369
Clinical Pipeline
Approved: 24Phase III: 14Phase II: 37Phase I: 25Preclinical: 0
Total compounds: 174 · Approved: 22
Filtered by: druggability=Low — 100 results
P2RY1 P2Y purinoreceptor 1 Phase 1
Gpcr Low Druggability
Score
0.73
Drug.
0.41
Safety
0.60
Drugs
1
Hyps
7
Papers
29
GPCR antagonists or agonists modulating purinergic signaling
ACSL4 Long-chain-fatty-acid--CoA ligase 4 Phase 3
Enzyme Low Druggability
Score
0.72
Drug.
0.41
Safety
0.65
Drugs
4
Hyps
1
Papers
14
Enzyme inhibition — blocking ACSL4 prevents incorporation of PUFAs into membrane phospholipids, reducing ferroptosis susceptibility
HSP90AA1 Heat Shock Protein 90 Alpha Family Class A Member Phase 3
Chaperone Low Druggability
Score
0.70
Drug.
0.45
Safety
0.50
Drugs
2
Hyps
2
Papers
14
Small molecule inhibitor binding to ATP-binding pocket of chaperone
SOAT1 Sterol O-acyltransferase 1 Phase 3
Enzyme Low Druggability
Score
0.67
Drug.
0.41
Safety
0.60
Drugs
1
Hyps
1
Papers
29
Small molecule inhibitor of acyl-CoA:cholesterol acyltransferase activity
TFR1 Transferrin receptor protein 1 Phase 4
Receptor Low Druggability
Score
0.66
Drug.
0.44
Safety
0.50
Drugs
2
Hyps
4
Papers
34
Monoclonal antibodies targeting receptor or iron chelation affecting iron uptake
TLR4 Toll-like receptor 4 Phase 3
Receptor Low Druggability
Score
0.66
Drug.
0.41
Safety
0.30
Drugs
1
Hyps
6
Papers
0
Small molecule antagonist or antibody blocking TLR4-mediated neuroinflammatory signaling
ULK1 Unc-51 like autophagy activating kinase 1 Phase 1
Kinase Low Druggability
Score
0.66
Drug.
0.42
Safety
0.40
Drugs
1
Hyps
5
Papers
18
Small molecule activators or inhibitors of ULK1 kinase activity to modulate autophagy
HDAC3 Histone Deacetylase 3 Phase 4
Epigenetic Regulator Low Druggability
Score
0.66
Drug.
0.41
Safety
0.40
Drugs
2
Hyps
5
Papers
36
Small molecule inhibitor of histone deacetylase enzymatic activity
TREM2 Triggering receptor expressed on myeloid cells 2 Phase 2
Receptor Low Druggability
Score
0.65
Drug.
0.42
Safety
0.60
Drugs
4
Hyps
35
Papers
25
Agonist antibodies that enhance TREM2 signaling to promote microglial function
EPHB4 Ephrin type-B receptor 4 Phase 4
Kinase Low Druggability
Score
0.65
Drug.
0.43
Safety
0.50
Drugs
1
Hyps
1
Papers
24
Small molecule inhibitor of receptor tyrosine kinase activity
SIRT6 Sirtuin-6 Phase 1
Epigenetic Regulator Low Druggability
Score
0.65
Drug.
0.41
Safety
0.60
Drugs
1
Hyps
2
Papers
36
Small molecule activation or inhibition of NAD+-dependent deacetylase activity
AHR Aryl hydrocarbon receptor Phase 2
Transcription Factor Low Druggability
Score
0.65
Drug.
0.35
Safety
0.40
Drugs
2
Hyps
4
Papers
0
Small molecule modulators affecting ligand-dependent transcriptional activity
ALOX12 12-lipoxygenase Phase 1
Enzyme Low Druggability
Score
0.64
Drug.
0.41
Safety
0.60
Drugs
2
Hyps
1
Papers
31
Small molecule inhibitors of lipoxygenase enzymatic activity
NPM1 Nucleophosmin Phase 3
Chaperone Low Druggability
Score
0.63
Drug.
0.30
Safety
0.30
Drugs
1
Hyps
1
Papers
30
Small molecule modulators of nucleolar function and protein interactions
ANGPT1 Angiopoietin-1 Phase 4
Ligand Low Druggability
Score
0.63
Drug.
0.45
Safety
0.50
Drugs
1
Hyps
1
Papers
18
Protein therapeutics or small molecule mimetics activating Tie2 signaling
SRPK1 SRSF protein kinase 1 Phase 2
Kinase Low Druggability
Score
0.63
Drug.
0.40
Safety
0.50
Drugs
1
Hyps
2
Papers
32
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.
MIRO1 Mitochondrial Rho GTPase 1 Phase 2
Signaling Protein Low Druggability
Score
0.63
Drug.
0.29
Safety
0.40
Drugs
4
Hyps
5
Papers
29
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.
TGFB1 Transforming growth factor beta-1 Phase 3
Ligand Low Druggability
Score
0.63
Drug.
0.44
Safety
0.30
Drugs
2
Hyps
3
Papers
18
Monoclonal antibodies neutralizing TGF-β1 or small molecule inhibitors of TGF-β signaling
AQP1 Aquaporin-1 Phase 4
Ion Channel Low Druggability
Score
0.62
Drug.
0.39
Safety
0.50
Drugs
1
Hyps
1
Papers
26
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.
GPR37 G-protein coupled receptor 37 Phase 4
Gpcr Low Druggability
Score
0.62
Drug.
0.30
Safety
0.60
Drugs
2
Hyps
2
Papers
31
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.
C1Q Complement C1q Phase 4
Signaling Protein Low Druggability
Score
0.62
Drug.
0.36
Safety
0.45
Drugs
1
Hyps
58
Papers
22
Complement cascade inhibitor or antibody-mediated neutralization
HK2 Hexokinase 2 Phase 3
Enzyme Low Druggability
Score
0.62
Drug.
0.38
Safety
0.30
Drugs
1
Hyps
5
Papers
54
Small molecule inhibitor of glucose phosphorylation
CX3CR1 C-X3-C Motif Chemokine Receptor 1 Phase 2
Gpcr Low Druggability
Score
0.62
Drug.
0.44
Safety
0.50
Drugs
2
Hyps
12
Papers
25
GPCR antagonist modulating microglial activation and neuroinflammation
LOX Lysyl oxidase Phase 2
Enzyme Low Druggability
Score
0.62
Drug.
0.43
Safety
0.50
Drugs
1
Hyps
11
Papers
26
Small molecule inhibition of lysyl oxidase enzymatic activity
IL10 Interleukin-10 Phase 2
Ligand Low Druggability
Score
0.62
Drug.
0.36
Safety
0.30
Drugs
1
Hyps
3
Papers
0
Recombinant protein replacement or monoclonal antibody modulation
GJA1 Gap junction alpha-1 protein (Connexin 43) Phase 2
Ion Channel Low Druggability
Score
0.62
Drug.
0.36
Safety
0.50
Drugs
1
Hyps
3
Papers
54
Small molecule modulator of gap junction channel activity
NAMPT Nicotinamide phosphoribosyltransferase Phase 2
Enzyme Low Druggability
Score
0.61
Drug.
0.45
Safety
0.40
Drugs
2
Hyps
3
Papers
22
Small molecule competitive inhibitors of NAD+ biosynthesis enzyme
TET2 Tet methylcytosine dioxygenase 2 Phase 2
Epigenetic Regulator Low Druggability
Score
0.61
Drug.
0.42
Safety
0.60
Drugs
2
Hyps
6
Papers
65
Small molecule enhancers of TET2 enzymatic activity or cofactor supplementation
PRMT1 Protein arginine methyltransferase 1 Phase 1
Epigenetic Regulator Low Druggability
Score
0.61
Drug.
0.36
Safety
0.50
Drugs
1
Hyps
1
Papers
32
Small molecule inhibitor of protein arginine methyltransferase activity
DNAJB1 DnaJ heat shock protein family (Hsp40) member B1 Phase 1
Chaperone Low Druggability
Score
0.60
Drug.
0.35
Safety
0.40
Drugs
2
Hyps
3
Papers
14
Allosteric modulation of protein-protein interactions with Hsp70
PLA2G4A Phospholipase A2 group IVA Phase 2
Enzyme Low Druggability
Score
0.60
Drug.
0.40
Safety
0.50
Drugs
1
Hyps
1
Papers
22
Small molecule inhibitor of cytosolic phospholipase A2 enzymatic activity
STING1 Stimulator of interferon genes protein 1 Phase 2
Signaling Protein Low Druggability
Score
0.60
Drug.
0.40
Safety
0.40
Drugs
2
Hyps
1
Papers
22
Small molecule agonists or antagonists of cGAS-STING pathway activation
GPX4 Glutathione peroxidase 4 Phase 2
Enzyme Low Druggability
Score
0.59
Drug.
0.41
Safety
0.30
Drugs
2
Hyps
5
Papers
22
Small molecule inhibitor or activator of peroxidase activity
PPARGC1A Peroxisome proliferator-activated receptor gamma c Phase 4
Transcription Factor Low Druggability
Score
0.59
Drug.
0.43
Safety
0.40
Drugs
3
Hyps
3
Papers
49
Small molecule modulator of transcriptional coactivator function
ALOX15 15-lipoxygenase Phase 4
Enzyme Low Druggability
Score
0.59
Drug.
0.38
Safety
0.60
Drugs
2
Hyps
3
Papers
31
Small molecule inhibitors targeting the active site iron and substrate binding
KDM6A Lysine demethylase 6A Phase 1
Epigenetic Regulator Low Druggability
Score
0.59
Drug.
0.35
Safety
0.40
Drugs
1
Hyps
1
Papers
36
Small molecule inhibition of histone demethylase activity
SLC16A1 Monocarboxylate transporter 1 (MCT1) Phase 2
Transporter Low Druggability
Score
0.59
Drug.
0.38
Safety
0.55
Drugs
3
Hyps
1
Papers
4
Transporter modulation — restoring MCT1/MCT4 balance to normalize astrocyte-neuron metabolic coupling
BDNF Brain Derived Neurotrophic Factor Phase 2
Ligand Low Druggability
Score
0.59
Drug.
0.29
Safety
0.60
Drugs
1
Hyps
47
Papers
18
Protein replacement therapy or small molecule mimetic
RAB27A Ras-related protein Rab-27A Phase 2
Signaling Protein Low Druggability
Score
0.59
Drug.
0.35
Safety
0.40
Drugs
1
Hyps
1
Papers
28
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.
MCOLN1 Mucolipin-1 Phase 1
Ion Channel Low Druggability
Score
0.59
Drug.
0.41
Safety
0.30
Drugs
1
Hyps
1
Papers
28
Small molecule agonists or modulators of lysosomal calcium channel activity
CMKLR1 Chemokine-like Receptor 1 Phase 2
Gpcr Low Druggability
Score
0.59
Drug.
0.41
Safety
0.60
Drugs
3
Hyps
1
Papers
31
GPCR antagonist or agonist modulating chemerin signaling
FOXO3 Forkhead box protein O3 Phase 4
Transcription Factor Low Druggability
Score
0.58
Drug.
0.42
Safety
0.35
Drugs
4
Hyps
3
Papers
36
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.
DGAT1 Diacylglycerol O-Acyltransferase 1 Phase 3
Enzyme Low Druggability
Score
0.58
Drug.
0.41
Safety
0.50
Drugs
1
Hyps
1
Papers
29
Small molecule inhibitor of triglyceride synthesis enzyme
SDC1 Syndecan-1 Phase 2
Receptor Low Druggability
Score
0.58
Drug.
0.38
Safety
0.60
Drugs
1
Hyps
1
Papers
26
Small molecule modulation of heparan sulfate interactions or shedding
BMAL1 Basic Helix-Loop-Helix ARNT Like 1 Phase 1
Transcription Factor Low Druggability
Score
0.58
Drug.
0.34
Safety
0.30
Drugs
4
Hyps
11
Papers
29
Small molecule modulator of circadian transcription
C1QA Complement C1q A Chain Phase 2
Signaling Protein Low Druggability
Score
0.58
Drug.
0.41
Safety
0.45
Drugs
1
Hyps
5
Papers
44
Complement cascade inhibitor or antibody-mediated neutralization
LAMP1 Lysosomal associated membrane protein 1 Phase 1
Structural Protein Low Druggability
Score
0.58
Drug.
0.43
Safety
0.20
Drugs
4
Hyps
11
Papers
28
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.
ZO1 Zonula occludens-1 Phase 4
Structural Protein Low Druggability
Score
0.57
Drug.
0.33
Safety
0.20
Drugs
4
Hyps
5
Papers
0
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.
NTN1 Netrin-1 Phase 1
Ligand Low Druggability
Score
0.57
Drug.
0.36
Safety
0.50
Drugs
1
Hyps
1
Papers
24
Modulation of axon guidance and neuronal survival pathways
MLCK Myosin light chain kinase Phase 2
Kinase Low Druggability
Score
0.56
Drug.
0.40
Safety
0.50
Drugs
2
Hyps
3
Papers
0
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.
PIEZO1 Piezo-type mechanosensitive ion channel component Phase 1
Ion Channel Low Druggability
Score
0.55
Drug.
0.42
Safety
0.40
Drugs
1
Hyps
4
Papers
61
Small molecule modulator of mechanosensitive ion channel activity
CHR2 Channelrhodopsin-2 Phase 2
Ion Channel Low Druggability
Score
0.55
Drug.
0.33
Safety
0.80
Drugs
1
Hyps
1
Papers
18
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.
COX4I1 Cytochrome C Oxidase Subunit 4I1 Phase 2
Enzyme Low Druggability
Score
0.54
Drug.
0.42
Safety
0.20
Drugs
4
Hyps
3
Papers
28
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.
SNAP25 Synaptosome associated protein 25 Phase 4
Structural Protein Low Druggability
Score
0.54
Drug.
0.33
Safety
0.20
Drugs
1
Hyps
3
Papers
0
Protein cleavage by botulinum toxin or small molecule modulators of SNARE complex formation
SST Somatostatin Phase 4
Ligand Low Druggability
Score
0.54
Drug.
0.41
Safety
0.70
Drugs
2
Hyps
5
Papers
0
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.
CRH Corticotropin Releasing Hormone Phase 4
Ligand Low Druggability
Score
0.54
Drug.
0.34
Safety
0.60
Drugs
1
Hyps
1
Papers
18
CRH receptor antagonist blocking stress hormone signaling
CLOCK Circadian Locomotor Output Cycles Kaput Phase 4
Transcription Factor Low Druggability
Score
0.54
Drug.
0.35
Safety
0.40
Drugs
1
Hyps
14
Papers
81
Modulation of circadian transcriptional activity through protein-protein interaction disruption
PLA2G6 Phospholipase A2 group VI Phase 4
Enzyme Low Druggability
Score
0.54
Drug.
0.36
Safety
0.30
Drugs
2
Hyps
1
Papers
22
Small molecule inhibitor of calcium-independent phospholipase A2 activity
ANXA1 Annexin A1 Phase 4
Signaling Protein Low Druggability
Score
0.53
Drug.
0.41
Safety
0.60
Drugs
1
Hyps
1
Papers
25
Calcium-dependent phospholipid binding protein modulator
FUT8 Alpha-(1,6)-fucosyltransferase Phase 2
Enzyme Low Druggability
Score
0.53
Drug.
0.42
Safety
0.40
Drugs
1
Hyps
1
Papers
14
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.
LRP1 LDL receptor related protein 1 Phase 3
Receptor Low Druggability
Score
0.53
Drug.
0.39
Safety
0.40
Drugs
2
Hyps
12
Papers
51
Modulation of receptor-mediated endocytosis and clearance pathways
ST6GAL1 ST6 beta-galactoside alpha-2,6-sialyltransferase 1 Phase 4
Enzyme Low Druggability
Score
0.53
Drug.
0.43
Safety
0.55
Drugs
2
Hyps
1
Papers
14
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.
SGMS2 Sphingomyelin synthase 2 Phase 3
Enzyme Low Druggability
Score
0.53
Drug.
0.29
Safety
0.50
Drugs
1
Hyps
1
Papers
0
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.
DNASE2 Deoxyribonuclease 2 lysosomal Phase 1
Enzyme Low Druggability
Score
0.53
Drug.
0.30
Safety
0.60
Drugs
1
Hyps
1
Papers
22
Small molecule inhibitor or activator of nuclease activity
HNRNPA2B1 Heterogeneous Nuclear Ribonucleoprotein A2/B1 Phase 2
Other Low Druggability
Score
0.52
Drug.
0.35
Safety
0.40
Drugs
3
Hyps
1
Papers
30
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.
CERS2 Ceramide synthase 2 Phase 2
Enzyme Low Druggability
Score
0.52
Drug.
0.28
Safety
0.40
Drugs
1
Hyps
1
Papers
24
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.
OCLN Occludin Phase 4
Structural Protein Low Druggability
Score
0.51
Drug.
0.32
Safety
0.40
Drugs
3
Hyps
2
Papers
17
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.
MCU Mitochondrial calcium uniporter Phase 3
Ion Channel Low Druggability
Score
0.51
Drug.
0.41
Safety
0.25
Drugs
1
Hyps
3
Papers
31
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.
TARDBP TAR DNA-binding protein 43 Phase 3
Transcription Factor Low Druggability
Score
0.51
Drug.
0.43
Safety
0.20
Drugs
1
Hyps
6
Papers
57
RNA-binding and transcription regulation, challenging for small molecule targeting
CHMP2B Charged multivesicular body protein 2B Phase 2
Other Low Druggability
Score
0.51
Drug.
0.32
Safety
0.30
Drugs
4
Hyps
1
Papers
28
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.
IGF2R Insulin-like growth factor 2 receptor Phase 4
Receptor Low Druggability
Score
0.50
Drug.
0.39
Safety
0.40
Drugs
4
Hyps
1
Papers
28
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.
SETX Senataxin Phase 4
Enzyme Low Druggability
Score
0.50
Drug.
0.28
Safety
0.40
Drugs
3
Hyps
1
Papers
30
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.
HSPG2 Heparan Sulfate Proteoglycan 2 Phase 2
Structural Protein Low Druggability
Score
0.50
Drug.
0.38
Safety
0.30
Drugs
2
Hyps
1
Papers
24
Targeting protein-heparan sulfate interactions or enzymatic modification
SYNCRIP Heterogeneous nuclear ribonucleoprotein Q Phase 2
Signaling Protein Low Druggability
Score
0.50
Drug.
0.32
Safety
0.40
Drugs
2
Hyps
1
Papers
30
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.
PYCARD Apoptosis-associated speck-like protein containing Phase 2
Signaling Protein Low Druggability
Score
0.50
Drug.
0.35
Safety
0.40
Drugs
1
Hyps
1
Papers
0
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.
LOXL1-4 Lysyl oxidase-like 1-4 Phase 2
Enzyme Low Druggability
Score
0.49
Drug.
0.33
Safety
0.50
Drugs
3
Hyps
1
Papers
26
Small molecule enzyme inhibitors targeting the catalytic domain
FOXP3 Forkhead box protein P3 Phase 1
Transcription Factor Low Druggability
Score
0.49
Drug.
0.35
Safety
0.30
Drugs
2
Hyps
1
Papers
18
Transcription factor modulation - no direct small molecule binding
CELL-TYPE-SPECIFICESSENTIALGENES CELL-TYPE-SPECIFICESSENTIALGENES Phase 1
Signaling Protein Low Druggability
Score
0.48
Drug.
0.30
Safety
0.50
Drugs
0
Hyps
1
Papers
12
Prioritized from 1 SciDEX hypotheses, including: Context-Dependent CRISPR Activation in Specific Neuronal Subtypes
RELN Reelin Phase 4
Signaling Protein Low Druggability
Score
0.48
Drug.
0.33
Safety
0.50
Drugs
4
Hyps
1
Papers
31
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.
RHOT1 RHOT1 Protein Phase 1
Protein Low Druggability
Score
0.46
Drug.
0.50
Safety
0.50
Drugs
0
Hyps
1
Papers
8
Prioritized from 1 SciDEX hypotheses, including: Miro1-Mediated Mitochondrial Trafficking Enhancement Therapy
SGMS1 Sphingomyelin synthase 1 Phase 4
Enzyme Low Druggability
Score
0.46
Drug.
0.29
Safety
0.50
Drugs
1
Hyps
1
Papers
0
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 1
Ion Channel Low Druggability
Score
0.46
Drug.
0.35
Safety
0.45
Drugs
1
Hyps
13
Papers
61
Water channel inhibitor or modulator
OCT4 OCT4 Protein Phase 2
Protein Low Druggability
Score
0.45
Drug.
0.50
Safety
0.50
Drugs
0
Hyps
1
Papers
23
Prioritized from 1 SciDEX hypotheses, including: Partial Neuronal Reprogramming via Modified Yamanaka Cocktail
DNAJB6 DNAJB6 Protein Phase 1
Protein Low Druggability
Score
0.45
Drug.
0.50
Safety
0.50
Drugs
0
Hyps
1
Papers
8
Prioritized from 1 SciDEX hypotheses, including: HSP70 Co-chaperone DNAJB6 Universal Cross-Seeding Inhibitor
ST8SIA1 ST8 alpha-N-acetyl-neuraminide alpha-2,8-sialyltra Phase 2
Enzyme Low Druggability
Score
0.45
Drug.
0.30
Safety
0.60
Drugs
4
Hyps
1
Papers
0
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.
FLOT1 Flotillin 1 Phase 2
Structural Protein Low Druggability
Score
0.45
Drug.
0.35
Safety
0.40
Drugs
4
Hyps
1
Papers
0
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 Phase 4
Structural Protein Low Druggability
Score
0.45
Drug.
0.35
Safety
0.30
Drugs
1
Hyps
1
Papers
17
No established drugging mechanism for structural membrane protein
CHMP4B Charged multivesicular body protein 4B Phase 2
Other Low Druggability
Score
0.44
Drug.
0.30
Safety
0.30
Drugs
4
Hyps
2
Papers
0
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 3
Other Low Druggability
Score
0.43
Drug.
0.30
Safety
0.30
Drugs
1
Hyps
1
Papers
32
No established druggable mechanisms
SLC16A2 Monocarboxylate transporter 8 Phase 1
Transporter Low Druggability
Score
0.43
Drug.
0.39
Safety
0.70
Drugs
1
Hyps
1
Papers
31
Substrate supplementation or transporter modulation
ST3GAL2 ST3 beta-galactoside alpha-2,3-sialyltransferase 2 Phase 2
Enzyme Low Druggability
Score
0.41
Drug.
0.30
Safety
0.60
Drugs
4
Hyps
1
Papers
0
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.
PHB2 PHB2 Protein Phase 3
Protein Low Druggability
Score
0.41
Drug.
0.50
Safety
0.50
Drugs
0
Hyps
1
Papers
9
Prioritized from 1 SciDEX hypotheses, including: Prohibitin-2 Mitochondrial Cross-Seeding Hub Disruption
LAMP2B LAMP2B Phase 1
Signaling Protein Low Druggability
Score
0.41
Drug.
0.40
Safety
0.30
Drugs
0
Hyps
1
Papers
7
Prioritized from 1 SciDEX hypotheses, including: Microglia-Derived Extracellular Vesicle Engineering for Targeted Mitochondrial Delivery
TRAK1_KIF5A Trafficking kinesin protein 1 / Kinesin family mem Phase 2
Transporter Low Druggability
Score
0.36
Drug.
0.31
Safety
0.20
Drugs
4
Hyps
1
Papers
28
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.
DISEASE-CAUSINGMUTATIONSWITHINTEGRATEDREPORTERS DISEASE-CAUSINGMUTATIONSWITHINTEGRATEDREPORTERS Phase 1
Signaling Protein Low Druggability
Score
0.36
Drug.
0.20
Safety
0.20
Drugs
0
Hyps
1
Papers
7
Prioritized from 1 SciDEX hypotheses, including: Multi-Modal CRISPR Platform for Simultaneous Editing and Monitoring
SYNTHETICFUSIONPROTEINS SYNTHETICFUSIONPROTEINS Phase 1
Signaling Protein Low Druggability
Score
0.36
Drug.
0.10
Safety
0.20
Drugs
0
Hyps
1
Papers
7
Prioritized from 1 SciDEX hypotheses, including: Synthetic Biology Approach: Designer Mitochondrial Export Systems
NURR1 NURR1 Protein Phase 1
Protein Low Druggability
Score
0.35
Drug.
0.50
Safety
0.50
Drugs
0
Hyps
1
Papers
7
Prioritized from 1 SciDEX hypotheses, including: Programmable Neuronal Circuit Repair via Epigenetic CRISPR
PITX3 PITX3 Phase 1
Signaling Protein Low Druggability
Score
0.35
Drug.
0.10
Safety
0.30
Drugs
0
Hyps
1
Papers
7
Prioritized from 1 SciDEX hypotheses, including: Programmable Neuronal Circuit Repair via Epigenetic CRISPR
NEURONALIDENTITYTRANSCRIPTIONFACTORS NEURONALIDENTITYTRANSCRIPTIONFACTORS Phase 1
Signaling Protein Low Druggability
Score
0.35
Drug.
0.10
Safety
0.30
Drugs
0
Hyps
1
Papers
7
Prioritized from 1 SciDEX hypotheses, including: Programmable Neuronal Circuit Repair via Epigenetic CRISPR
PVALB Parvalbumin Phase 2
Signaling Protein Low Druggability
Score
0.33
Drug.
0.34
Safety
0.30
Drugs
2
Hyps
1
Papers
0
No known druggable mechanism