Protein / target

von Willebrand factor

VWFP04275Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
3
Approved medicines
7
Clinical trials
Small-molecule tractable
Druggability
Med-Quality Pocket

Protein at a glance

Biological role

Extracellular matrix structural constituent

Primary system

Cardiovascular system

Strongest disease association

von Willebrand disease 2

Genetic evidence · score 0.98

Therapeutic maturity

Clinically validated target

3 approved medicines against this target

Druggability

Small molecule

Open Targets tractability · Med-Quality Pocket

Clinical development

3 approved · 1 in clinical development

7 linked trials

Research activity

Emerging research

2 papers · latest 2024

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

Important in the maintenance of hemostasis, it promotes adhesion of platelets to the sites of vascular injury by forming a molecular bridge between sub-endothelial collagen matrix and platelet-surface receptor complex GPIb-IX-V. Also acts as a chaperone for coagulation factor VIII, delivering it to the site of injury, stabilizing its heterodimeric structure and protecting it from premature clearance from plasma

Subcellular location

SecretedSecreted, extracellular space, extracellular matrix
Domains and Gene Ontology detail (37)

Domains & features

VWFD 1TIL 1VWFD 2TIL 2TIL 3VWFD 3TIL 4VWFA 1; binding site for platelet glycoprotein IbVWFA 2VWFA 3; main binding site for collagens type I and IIIVWFD 4VWFC 1VWFC 2VWFC 3CTCK

Gene Ontology

  • Cendoplasmic reticulum
  • Cextracellular exosome
  • Cextracellular matrix
  • Cextracellular region
  • Cextracellular space
  • Cplatelet alpha granule
  • Cplatelet alpha granule lumen
  • CWeibel-Palade body
  • Fcollagen binding
  • Fextracellular matrix structural constituent
  • Fidentical protein binding
  • Fimmunoglobulin binding

2813 aa · 309 kDa · 2 isoforms

Biological roles

Reactome v97

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

HaemostasisUniProt · GO · ReactomeCell adhesionUniProt · GOKinase signallingReactomeProteolysisGO
View supporting evidence

Haemostasis

  • ·Important in the maintenance of hemostasis, it promotes adhesion of platelets to the sit…
  • ·platelet alpha granule
  • ·platelet alpha granule lumen
  • ·blood coagulation

Cell adhesion

  • ·Secreted, extracellular space, extracellular matrix
  • ·extracellular matrix
  • ·extracellular matrix structural constituent
  • ·cell adhesion

Kinase signalling

  • ·Signaling by moderate kinase activity BRAF mutants
  • ·Signaling by high-kinase activity BRAF mutants

Proteolysis

  • ·protease binding
View underlying pathways (25)

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.

F8GP1BASELPITGA2BITGB3GP9FN1GP1BBADAMTS…GP6VWF

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.

caplacizumab
Narrow target profileApprovedInhibitor

von Willebrand factor inhibitor

Appears in clinical studies involving Recurrent thrombophlebitis, thrombotic thrombocytopenic purpura, acquired thrombotic thrombocytopenic purpura, Thrombocytopenia

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.

von Willebrand disease 20.98

Genetic · overall 0.77

hereditary von Willebrand disease0.96

Genetic · overall 0.67

Von Willebrand disease type 20.95

Genetic · overall 0.70

von Willebrand disease (hereditary or acquired)0.91

Genetic · overall 0.69

Von Willebrand disease0.91

Genetic literature · overall 0.83

Highest-confidence therapeutic associations

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

von Willebrand disease type 2B0.12

Clinical · overall 0.71

Highest overall evidence

Remaining associations by Open Targets' aggregated evidence score.

von Willebrand disease 30.80

Genetic

Von Willebrand disease type 10.73

Genetic

von Willebrand disease 10.69

Literature

von Willebrand disease type 2A0.67

Genetic

Show all associations
Von Willebrand disease0.83
von Willebrand disease 30.80
von Willebrand disease 20.77
Von Willebrand disease type 10.73
von Willebrand disease type 2B0.71
Von Willebrand disease type 20.70
von Willebrand disease 10.69
von Willebrand disease (hereditary or acquired)0.69
hereditary von Willebrand disease0.67
von Willebrand disease type 2A0.67

Open Targets ranks 1,575 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 · 4 total

EGAPTIVON PEGOLPhase 2

thrombotic disease · intracranial embolism · thrombotic thrombocytopenic purpura

CAPLACIZUMABApproval

Recurrent thrombophlebitis · thrombotic thrombocytopenic purpura · acquired thrombotic thrombocytopenic purpura

VON WILLEBRAND FACTOR HUMANApproval

hemorrhage · hemophilia · Von Willebrand disease

VONICOG ALFAApproval

Von Willebrand disease · von Willebrand disease (hereditary or acquired) · hemorrhage

Tractability

SM · Med-Quality PocketAB · Approved DrugAB · UniProt loc high confAB · GO CC high confAB · UniProt SigP or TMHMMPR · Database UbiquitinationOC · Approved Drug

Clinical trials

7

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

2 papers · to 2024

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.

von Willebrand disease 2Well supported
0.99
agreement 0.871.00
Genetic82%Literature10%Animal model9%Genetic literaturedup

Open Targets aggregate 0.77 · 3 independent evidence families · 1 not counted as duplicate

Von Willebrand diseaseWell supported
0.97
agreement 0.881.00
Genetic46%Clinical36%Animal model13%Literature5%Genetic literaturedup

Open Targets aggregate 0.83 · 4 independent evidence families · 1 not counted as duplicate

von Willebrand disease (hereditary or acquired)Well supported
0.97
agreement 0.861.00
Genetic55%Clinical36%Literature9%

Open Targets aggregate 0.69 · 3 independent evidence families

Von Willebrand disease type 2Well supported
0.96
agreement 0.841.00
Genetic87%Animal model10%Literature4%Genetic literaturedup

Open Targets aggregate 0.70 · 3 independent evidence families · 1 not counted as duplicate

hereditary von Willebrand diseaseWell supported
0.96
agreement 0.821.00
Genetic100%Literature0%Genetic literaturedup

Open Targets aggregate 0.67 · 2 independent evidence families · 1 not counted as duplicate

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

2

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 publication2020-02-01
    The Therapeutic Potential of Nanobodies.

    BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2020 · 535 citations · Europe PMC · via caplacizumab

  2. Regulatory approval2018-08-30

    Approval: Cablivi (EMA)

    ema · regulatory · ema · via caplacizumab

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.