Protein / target

ATP-dependent translocase ABCB1

Encoded byABCB1P08183Homo sapiensSwiss-Prot

UniProt: P08183 Ensembl: ENSG00000085563 HGNC: HGNC:40 NCBI Gene: 5243

Clinical-stage

Therapeutic maturity

Clinical candidates

Small-molecule tractable

Druggability

Advanced Clinical

Research papers

Protein at a glance

Biological role

Carboxylic acid transmembrane transporter

Strongest disease association

Inflammatory Bowel Diseases

Via encoding gene ABCB1 · Genetic literature evidence · score 0.61

Therapeutic position

Clinically advancing target

Small molecules

Research activity

Emerging research

1 papers · latest 2023

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

Translocates drugs and phospholipids across the membrane.

View complete UniProt function annotation

Translocates drugs and phospholipids across the membrane (PubMed:2897240, PubMed:35970996, PubMed:8898203, PubMed:9038218, PubMed:35507548). Catalyzes the flop of phospholipids from the cytoplasmic to the exoplasmic leaflet of the apical membrane. Participates mainly to the flop of phosphatidylcholine, phosphatidylethanolamine, beta-D-glucosylceramides and sphingomyelins (PubMed:8898203). Energy-dependent efflux pump responsible for decreased drug accumulation in multidrug-resistant cells (PubMed:2897240, PubMed:35970996, PubMed:9038218)

Subcellular location

Cell membraneApical cell membraneCytoplasm

Domains and Gene Ontology detail (40)

Domains & features

ABC transmembrane type-1 1ABC transporter 1ABC transmembrane type-1 2ABC transporter 2

Gene Ontology

Capical plasma membrane
Ccell surface
Ccytoplasm
Cexternal side of apical plasma membrane
Cextracellular exosome
Cmembrane
Cplasma membrane
FABC-type xenobiotic transporter activity
FATP binding
FATP hydrolysis activity
FATPase-coupled transmembrane transporter activity
Fcarboxylic acid transmembrane transporter activity

1280 aa · 141 kDa · 2 isoforms

Biological roles

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

Lipid & lipoprotein metabolismUniProt · GOChloride transportGOCell-cycle regulationGO

View supporting evidence

Lipid & lipoprotein metabolism

·Translocates drugs and phospholipids across the membrane (PubMed:2897240, PubMed:3597099…
·phospholipid translocation

Chloride transport

·regulation of chloride transport

Cell-cycle regulation

·G2/M transition of mitotic cell cycle

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

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 ABCB1

Gene-level evidence surfaced through the gene ABCB1 that 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.

Epilepsy

0.60Moderately supported

Genetic evidence dominant · Open Targets 0.36

Inflammatory Bowel Diseases

0.54Moderately supported

Genetic literature evidence dominant · Open Targets 0.39

Hypertension, Portal

0.54Moderately supported

Genetic evidence dominant · Open Targets 0.32

Leukemia, Myeloid, Acute

0.53Moderately supported

Clinical evidence dominant · Open Targets 0.39

Carcinoma, Non-Small-Cell Lung

0.47Limited support

Clinical evidence dominant · Open Targets 0.34

View evidence synthesis (5)

EpilepsyModerately supported

0.60

agreement 0.46–0.74

Genetic83%Literature17%

Open Targets aggregate 0.36 · 2 independent evidence families

Inflammatory Bowel DiseasesModerately supported

0.54

agreement 0.41–0.67

Genetic literature82%Literature17%RNA expression1%

Open Targets aggregate 0.39 · 3 independent evidence families

Hypertension, PortalModerately supported

0.54

agreement 0.40–0.68

Genetic97%Literature3%

Open Targets aggregate 0.32 · 2 independent evidence families

Leukemia, Myeloid, AcuteModerately supported

0.53

agreement 0.39–0.66

Clinical76%Literature24%RNA expression1%

Open Targets aggregate 0.39 · 3 independent evidence families

Carcinoma, Non-Small-Cell LungLimited support

0.47

agreement 0.33–0.60

Clinical77%Literature18%RNA expression5%

Open Targets aggregate 0.34 · 3 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 per-type scores above show the calculation.

Show all associations

Leukemia, Myeloid, Acute0.39

Inflammatory Bowel Diseases0.39

Epilepsy0.36

Carcinoma, Non-Small-Cell Lung0.34

Hypertension, Portal0.32

Multiple Myeloma0.28

Myelodysplastic syndrome0.27

Drug development

5 compounds recorded · 5 in clinical development

Open Targets' development universe — every compound recorded against the target at any stage, not all approved medicines.

View all recorded compounds (5)

ZOSUQUIDARPhase 1 2

TARIQUIDARPhase 3

VALSPODARPhase 3

BIRICODARPhase 2

ENCEQUIDARPhase 3

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 molecules — Strong

Advanced Clinical and Structure with Ligand support this modality.

Antibodies — Emerging

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

Protein degraders — Emerging

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

Other modalities — Strong

Advanced Clinical support this modality.

View underlying tractability evidence (12)

SM · Advanced ClinicalSM · Structure with LigandSM · High-Quality LigandSM · Druggable FamilyAB · UniProt loc high confAB · GO CC high confAB · UniProt SigP or TMHMMAB · Human Protein Atlas locPR · Database UbiquitinationPR · Half-life DataPR · Small Molecule BinderOC · Advanced Clinical

Raw Open Targets tractability assessment buckets, by modality.

Safety-related annotations

mucositisClinPGxrespiratory depressionClinPGxtransplant rejectionClinPGxlikelihood to postural hypotensionClinPGxCNS depression in breast-feeding infantsClinPGxregulation of transcription factor activityToxCastsomnolenceClinPGxclozapine plasma concentrationsClinPGxgastrointestinal toxicityClinPGxhyperbilirubinemiaClinPGxdrug toxicityClinPGxadverse effectsClinPGx

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.

Research activity

1 papers · to 2023

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

Most cited

Gut microbiome modulates tacrolimus pharmacokinetics through the transcriptional regulation of ABCB1.42 cites

Degraeve AL · Microbiome · 2023

Recent

Gut microbiome modulates tacrolimus pharmacokinetics through the transcriptional regulation of ABCB1.

Degraeve AL · Microbiome · 2023

Europe PMC papers linked directly to this protein.