Protein / target

Acetylcholinesterase

ACHEP22303Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
27
Approved medicines
1
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

Protein homodimerization activity

Primary system

Nervous system

Strongest disease association

Abnormality of the skeletal system

Genetic evidence · score 0.78

Therapeutic maturity

Clinically validated target

27 approved medicines against this target

Druggability

Small molecule

Open Targets tractability · Approved Drug

Clinical development

27 approved · 1 in clinical development

1 linked trials

Research activity

Emerging research

9 papers · latest 2025

Derived from structured UniProt, Open Targets and literature data on this page.

Protein profile

UniProt 2026_02

Canonical identity and biological annotation from UniProt.

Function

Hydrolyzes rapidly the acetylcholine neurotransmitter released into the synaptic cleft allowing to terminate the signal transduction at the neuromuscular junction. Role in neuronal apoptosis

Subcellular location

SynapseSecretedCell membraneNucleus
Domains and Gene Ontology detail (32)

Gene Ontology

  • Cbasement membrane
  • Ccell surface
  • Cextracellular region
  • Cextracellular space
  • CGolgi apparatus
  • Cmembrane
  • Cneuromuscular junction
  • Cnucleus
  • Cperinuclear region of cytoplasm
  • Cplasma membrane
  • Cside of membrane
  • Csynapse

614 aa · 68 kDa · 4 isoforms

Biological roles

Reactome v97

What this protein does, drawn together from its UniProt function, Gene Ontology terms and Reactome pathways.

Synaptic signallingUniProt · GOCell adhesionGOMetabolic enzyme activityGO
View supporting evidence

Synaptic signalling

  • ·Synapse
  • ·synapse
  • ·negative regulation of synaptic transmission, cholinergic
  • ·synapse assembly

Cell adhesion

  • ·cell adhesion

Metabolic enzyme activity

  • ·amyloid precursor protein metabolic process
View underlying pathways (3)

Concepts derived from UniProt GO Reactome — each badge above shows which sources supported that role.

Interaction neighbourhood

STRING v12.0

Proteins with the strongest functional or physical association. Node size and line weight reflect STRING confidence; hover or select a partner to inspect one association.

COLQAPPCHATGPCPD1CHKAMAOBCHKBPHOSPH…HSPG2DOK7ACHE

10 strongest partners — larger node and heavier line mean higher confidence

Functional and physical associations from STRING v12.0. Only associations with this protein are drawn — partner-to-partner links are not part of this evidence.

Drugs targeting this protein

1

Whether each drug engages this protein directly or through a complex, and how many other targets are recorded for it. Direct binders with few recorded targets are listed first.

Isoflurophate
Narrow target profileApprovedInhibitor

Acetylcholinesterase inhibitor

Appears in clinical studies involving glaucoma

Direct interaction with this protein · Only this protein recorded as a target

ChEMBL mechanism, action type and target identity. Open Targets clinical status and indications.

Translational evidence

Open Targets 26

Why this target matters therapeutically — disease associations, drugs in development, tractability and safety, from Open Targets.

Strongest genetic associations

Human genetic evidence — the most direct causal link between this target and a disease.

Abnormality of the skeletal system0.78

Genetic · overall 0.48

Highest-confidence therapeutic associations

Diseases where a drug acting on this target has already reached clinical development.

Alzheimer disease1.00

Clinical · overall 0.63

dementia0.99

Clinical · overall 0.63

myasthenia gravis0.97

Clinical · overall 0.61

Parkinson disease0.96

Clinical · overall 0.62

glaucoma0.89

Clinical · overall 0.55

Highest overall evidence

Remaining associations by Open Targets' aggregated evidence score.

neurodegenerative disease0.53

Pathway

poisoning0.51

Clinical

dyspepsia0.49

Clinical

gastrointestinal disease0.46

Clinical

Show all associations
Alzheimer disease0.63
dementia0.63
Parkinson disease0.62
myasthenia gravis0.61
glaucoma0.55
neurodegenerative disease0.53
poisoning0.51
dyspepsia0.49
Abnormality of the skeletal system0.48
gastrointestinal disease0.46

Open Targets ranks 2,712 associations for this target and we store the top 10 by aggregated score. The long tail is largely literature co-mention and RNA-expression evidence, not evidence that this target drives those diseases.

Known drugs · 32 total

BENZGALANTAMINEUnknown

Mental deterioration · Alzheimer disease

GALANTAMINEApproval

Alzheimer disease · dementia · Alzheimer disease

PROPANIDIDUnknown
NEOSTIGMINE METHYLSULFATEApproval

myasthenia gravis · obstructive jaundice · Snoring

MINAPRINEApproval

depressive disorder · major depressive disorder

TACRINEApproval

Alzheimer disease · dementia

ITOPRIDEApproval

gastrointestinal disease · chronic gastritis · dyspepsia

DEMECARIUM BROMIDEApproval

open-angle glaucoma

HUPERZINE AApproval

Alzheimer disease · Alzheimer disease · cocaine abuse

ACOTIAMIDE HYDROCHLORIDEApproval

Abdominal distention · dyspepsia · dyspepsia

Tractability

SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · High-Quality PocketSM · Druggable FamilyAB · GO CC high confAB · UniProt loc med confAB · UniProt SigP or TMHMMPR · Small Molecule Binder

Safety liabilities

increased salivationdeathbronchoconstrictionmuscle relaxationcomadecreased blood pressureincreased defecationincreased respiratory secretionsfoot dropvomitingperipheral neuropathyincreased urine excretion

Clinical trials

1

Trials of drugs that target this protein — reached indirectly through those drugs, so a trial listed here studies the drug, not the protein.

ClinicalTrials.gov via the drug-target graph.

Research activity

9 papers · to 2025

Papers linked directly to this protein. This is the protein's own literature — descriptor-derived papers are kept separate below.

Most cited

Recent

Europe PMC papers linked directly to this protein.

Forefront confidence

Synthesis

Our own weighting of the evidence behind each disease association. Human genetics and clinical evidence count for most; literature co-mention counts for little, because two entities sharing an abstract is not evidence that one drives the other.

Parkinson diseaseWell supported
0.80
agreement 0.690.92
Clinical70%Animal model18%Literature12%RNA expression0%

Open Targets aggregate 0.62 · 4 independent evidence families

myasthenia gravisWell supported
0.79
agreement 0.660.92
Clinical75%Animal model20%Literature5%

Open Targets aggregate 0.61 · 3 independent evidence families

Abnormality of the skeletal systemWell supported
0.79
agreement 0.670.91
Genetic100%

Open Targets aggregate 0.48 · 1 independent evidence family

Alzheimer diseaseWell supported
0.78
agreement 0.630.94
Clinical84%Literature16%

Open Targets aggregate 0.63 · 2 independent evidence families

dementiaWell supported
0.77
agreement 0.620.93
Clinical87%Literature13%

Open Targets aggregate 0.63 · 2 independent evidence families

This ranking differs from Open Targets' own: re-weighting moves genetically-evidenced diseases above more heavily co-mentioned ones. The evidence agreement range shows how closely the independent evidence families agree — it is not a statistical confidence interval, and nothing here is fitted to outcome data. Derived from Open Targets evidence types under Forefront weighting; the underlying per-type scores are shown above so the calculation can be checked.

What's happening now

3

Recent activity around this target, drawn from one canonical event stream. Every item is reached through a drug that targets this protein, so each event is news about that drug rather than about the protein directly.

  1. New publication2024-01-17
    Neuroprotectant Activity of Novel Water-Soluble Synthetic Neurosteroids on Organophosphate Intoxication and Status Epilepticus-Induced Long-Term Neurological Dysfunction, Neurodegeneration, and Neuroinflammation.

    The Journal of pharmacology and experimental therapeutics · 2024 · 14 citations · Europe PMC · via Isoflurophate

  2. New publication2024-01-17
    Sex Differences in Organophosphate Model of Benzodiazepine-Refractory Status Epilepticus and Neuronal Damage.

    The Journal of pharmacology and experimental therapeutics · 2024 · 12 citations · Europe PMC · via Isoflurophate

  3. New publication2024-01-17
    A Pediatric Rat Model of Organophosphate-Induced Refractory Status Epilepticus: Characterization of Long-Term Epileptic Seizure Activity, Neurologic Dysfunction and Neurodegeneration.

    The Journal of pharmacology and experimental therapeutics · 2024 · 10 citations · Europe PMC · via Isoflurophate

Objective event titles are shown unmodified; the event kind and significance line are derived from structured fields. Forefront AttentionEvent stream aggregating Europe PMC Regulatory filings ClinicalTrials.gov.