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Protein / target

ATP-citrate synthase

Encoded byACLYP53396Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
1
Approved medicines
Open Targets target-level
View by indication →
25
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

ATP citrate synthase

Strongest disease association

Familial hypercholesterolemia

Via encoding gene ACLY · Clinical evidence · score 0.53

Therapeutic position

Established drug target

Small molecules

Research activity

Emerging research

1 papers · latest 2022

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 overview

Catalyzes the cleavage of citrate into oxaloacetate and acetyl-CoA, the latter serving as common substrate in multiple biochemical reactions in protein, carbohydrate and lipid metabolism

Subcellular location

Cytoplasm, cytosol
Domains and Gene Ontology detail (18)

Domains & features

ATP-grasp

Gene Ontology

  • Cazurophil granule lumen
  • Ccytosol
  • Cextracellular exosome
  • Cextracellular region
  • Cficolin-1-rich granule lumen
  • Cmembrane
  • FATP binding
  • FATP citrate synthase activity
  • Fmetal ion binding
  • Pacetyl-CoA biosynthetic process
  • Pcholesterol biosynthetic process
  • Pcitrate metabolic process

1101 aa · 121 kDa · 3 isoforms

Biological roles

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

Lipid & lipoprotein metabolismGO
View supporting evidence

Lipid & lipoprotein metabolism

  • ·cholesterol biosynthetic process
  • ·fatty acid biosynthetic process

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

Approved medicines with mapped indications

1 medicine · 6 areas

Approved therapies targeting this protein, grouped by what they are approved to treat — the disease-first view of the medicines below. Relationships come from the canonical approved-indication graph (ChEMBL phase-4), the same source as the drug cards.

Atherosclerosis1 medicine
Coronary Artery Disease1 medicine
Dyslipidemias1 medicine
Hypercholesterolemia1 medicine
Hyperlipoproteinemia Type II1 medicine
Broader indication categories (1)
Cardiovascular Diseases1 medicine

Broad umbrella indications (e.g. “Neoplasms”). Shown here because every medicine also appears under a more specific disease above — kept for completeness, de-emphasised for clarity.

Approved indications from ChEMBL (phase-4), via the canonical drug→disease graph.

Drugs targeting this protein

1

How approved and investigational drugs engage this protein — mechanism and action type, direct vs complex targeting, and how broadly each acts across other targets. A drug-first view (the section above groups the approved ones disease-first); direct binders with few recorded targets are listed first.

bempedoic acid
Narrow target profileApprovedInhibitor

ATP-citrate synthase inhibitor

Indicated for Atherosclerosis, Coronary Artery Disease, Dyslipidemias, Hypercholesterolemia

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

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

Translational evidence

Open Targets 26

Why this target matters therapeutically, strongest evidence first. Disease associations are gene-level (via the gene that encodes this protein) and open into the full confidence synthesis; the development universe, tractability and safety annotations are target-level, from Open Targets.

Strongest disease associations · via encoding gene ACLY

Gene-level evidence surfaced through the gene ACLYthat encodes this protein — not a direct protein–disease relationship. Ranked by Forefront's causal-directness weighting, so genetically- and clinically-evidenced diseases lead over ones that merely share the literature.

Familial hypercholesterolemia
0.66Moderately supported

Clinical evidence dominant · Open Targets 0.53

Atherosclerosis
0.59Moderately supported

Clinical evidence dominant · Open Targets 0.47

Inherited lipid metabolism disorder
0.58Moderately supported

Clinical evidence dominant · Open Targets 0.47

Cardiovascular Diseases
0.56Moderately supported

Clinical evidence dominant · Open Targets 0.45

Coronary Artery Disease
0.47Limited support

Clinical evidence dominant · Open Targets 0.37

View evidence synthesis (5)
Familial hypercholesterolemiaModerately supported
0.66
agreement 0.510.81
Clinical96%Literature5%

Open Targets aggregate 0.53 · 2 independent evidence families

AtherosclerosisModerately supported
0.59
agreement 0.430.74
Clinical94%Literature6%

Open Targets aggregate 0.47 · 2 independent evidence families

Inherited lipid metabolism disorderModerately supported
0.58
agreement 0.420.73
Clinical99%Literature1%

Open Targets aggregate 0.47 · 2 independent evidence families

Cardiovascular DiseasesModerately supported
0.56
agreement 0.400.71
Clinical97%Literature3%

Open Targets aggregate 0.45 · 2 independent evidence families

Coronary Artery DiseaseLimited support
0.47
agreement 0.320.63
Clinical95%Literature5%

Open Targets aggregate 0.37 · 2 independent evidence families

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 per-type scores above show the calculation.

Show all associations
Familial hypercholesterolemia0.53
Atherosclerosis0.47
Inherited lipid metabolism disorder0.47
Cardiovascular Diseases0.45
Coronary Artery Disease0.37
Hyperlipidemias0.36
Acute Coronary Syndrome0.26
Carcinoma, Hepatocellular0.11

Drug development

1 compounds recorded · 1 approved

Open Targets' development universe — every compound recorded against the target at any stage, not all approved medicines. Distinct from the 1 drug that targets this protein in Forefront's canonical graph (1 with a mapped approved indication, shown above): these count different sets and are not a subset relation.

View all recorded compounds (1)
BEMPEDOIC ACIDApproval

Open Targets known-drugs universe. Drug name and highest clinical stage only — the disease relationship is NOT read from this slice (it carries trial-context noise); approved indications come from the canonical graph above.

Tractability

Small moleculesStrong

Approved Drug and Structure with Ligand support this modality.

AntibodiesEmerging

Feasibility evidence (go cc high conf) — no clinical-stage drug of this modality recorded.

Protein degradersEmerging

Feasibility evidence (uniprot ubiquitination and database ubiquitination) — no clinical-stage drug of this modality recorded.

View underlying tractability evidence (10)
SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · High-Quality PocketSM · Druggable FamilyAB · GO CC high confPR · UniProt UbiquitinationPR · Database UbiquitinationPR · Half-life DataPR · Small Molecule Binder

Raw Open Targets tractability assessment buckets, by modality.

Safety-related annotations

regulation of transcription factor activityToxCast

Terms indexed against this target in Open Targets' safety data, with their datasource. These are annotations, not causal claims: the direction of effect (whether activation or inhibition is implicated), species and evidence strength are not captured here, so an entry does not mean that modulating this target is known to cause that condition.

Clinical trials

25

Trials of drugs that target this protein — reached indirectly through those drugs, so a trial listed here studies the drug, not the protein. Ranked by active status, then clinical phase and recency, and spread across the targeting drugs.

View all trials (21)

NOT_YET_RECRUITING · via bempedoic acid · NCT07282821

ClinicalTrials.gov via the drug-target graph.

What's happening now

1

Recent therapeutic activity around this target — regulatory actions, clinical trials and safety signals for a drug that targets this protein, plus publications where such a drug is a genuine subject. Every item is reached indirectly through the drug, not the protein itself; incidental mentions (a paper that merely measures a drug) and press items are excluded.

  1. Regulatory approval2020-04-01

    Approval: Nilemdo (EMA)

    ema · regulatory · ema · via bempedoic acid

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.

Research activity

1 papers · to 2022

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

Most cited

Batchuluun B · Nature reviews. Drug discovery · 2022

Recent

Lipogenesis inhibitors: therapeutic opportunities and challenges.

Batchuluun B · Nature reviews. Drug discovery · 2022

Europe PMC papers linked directly to this protein.