Protein / target

Protein cereblon

CRBNQ96SW2Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
3
Approved medicines
30
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

Transmembrane transporter binding

Primary system

Nervous system

Strongest disease association

congenital sideroblastic anemia-B-cell immunodeficiency-periodic fever-developmental delay syndrome

Genetic evidence · score 0.94

Therapeutic maturity

Clinically validated target

3 approved medicines against this target

Druggability

Small molecule

Open Targets tractability · Approved Drug

Clinical development

3 approved · 1 in clinical development

30 linked trials

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

Substrate recognition component of a DCX (DDB1-CUL4-X-box) E3 protein ligase complex that mediates the ubiquitination and subsequent proteasomal degradation of target proteins, such as MEIS2, ILF2 or GLUL (PubMed:26990986, PubMed:33009960). Normal degradation of key regulatory proteins is required for normal limb outgrowth and expression of the fibroblast growth factor FGF8 (PubMed:20223979, PubMed:24328678, PubMed:25043012, PubMed:25108355). Maintains presynaptic glutamate release and consequently cognitive functions, such as memory and learning, by negatively regulating large-conductance calcium-activated potassium (BK) channels in excitatory neurons (PubMed:18414909, PubMed:29530986). Likely to function by regulating the assembly and neuronal surface expression of BK channels via its interaction with KCNT1 (PubMed:18414909). May also be involved in regulating anxiety-like behaviors via a BK channel-independent mechanism (By similarity). Plays a negative role in TLR4 signaling by interacting with TRAF6 and ECSIT, leading to inhibition of ECSIT ubiquitination, an important step of the signaling (PubMed:31620128)

Subcellular location

CytoplasmNucleusMembrane
Domains and Gene Ontology detail (18)

Domains & features

Lon N-terminalCULT

Gene Ontology

  • CCul4A-RING E3 ubiquitin ligase complex
  • Ccytoplasm
  • Ccytosol
  • Cmembrane
  • Cnucleus
  • Cperinuclear region of cytoplasm
  • Fmetal ion binding
  • Ftransmembrane transporter binding
  • Plimb development
  • Plocomotory exploration behavior
  • Pnegative regulation of monoatomic ion transmembrane transport
  • Pnegative regulation of protein-containing complex assembly

442 aa · 51 kDa · 2 isoforms

Biological roles

Reactome v97

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

Synaptic signallingUniProtIon channel gatingGOMotor controlGO
View supporting evidence

Synaptic signalling

  • ·Substrate recognition component of a DCX (DDB1-CUL4-X-box) E3 protein ligase complex tha…

Ion channel gating

  • ·negative regulation of monoatomic ion transmembrane transport

Motor control

  • ·locomotory exploration behavior
View underlying pathways (1)

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.

CUL4BDDB1RBX1IKZF1CUL4AIKZF3CSNK1A1GSPT1ZFP91BRD4CRBN

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

2

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.

Thalidomide
ApprovedInhibitor

CRL4(CRBN) E3 ubiquitin ligase inhibitor

Appears in clinical studies involving plasma cell myeloma, immune system disorder, prostate cancer, non-small cell lung carcinoma

Acts on a complex — shared with DDB1, CUL4A, RBX1 · 1 of 4 recorded protein targets

lenalidomide
ApprovedInhibitor

CRL4(CRBN) E3 ubiquitin ligase inhibitor

Appears in clinical studies involving anemia, mantle cell lymphoma, myelodysplastic syndrome, plasma cell myeloma

Acts on a complex — shared with DDB1, CUL4A, RBX1 · 1 of 4 recorded protein targets

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.

congenital sideroblastic anemia-B-cell immunodeficiency-periodic fever-developmental delay syndrome0.94

Genetic · overall 0.57

retinitis pigmentosa and erythrocytic microcytosis0.82

Genetic · overall 0.51

autosomal recessive non-syndromic intellectual disability0.79

Genetic · overall 0.60

hereditary disease0.77

Genetic · overall 0.47

Highest-confidence therapeutic associations

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

plasma cell myeloma0.99

Clinical · overall 0.68

follicular lymphoma0.93

Clinical · overall 0.57

myelodysplastic syndrome0.93

Clinical · overall 0.57

mantle cell lymphoma0.90

Clinical · overall 0.56

immune system disorder0.83

Clinical · overall 0.50

Highest overall evidence

Remaining associations by Open Targets' aggregated evidence score.

anemia0.49

Clinical

Show all associations
plasma cell myeloma0.68
autosomal recessive non-syndromic intellectual disability0.60
myelodysplastic syndrome0.57
congenital sideroblastic anemia-B-cell immunodeficiency-periodic fever-developmental delay syndrome0.57
follicular lymphoma0.57
mantle cell lymphoma0.56
retinitis pigmentosa and erythrocytic microcytosis0.51
immune system disorder0.50
anemia0.49
hereditary disease0.47

Open Targets ranks 926 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

LENALIDOMIDEApproval

anemia · mantle cell lymphoma · myelodysplastic syndrome

THALIDOMIDEApproval

plasma cell myeloma · immune system disorder · prostate cancer

POMALIDOMIDEApproval

plasma cell myeloma · systemic sclerosis · immune system disorder

IBERDOMIDEPhase 3

plasma cell myeloma · sarcoidosis · systemic lupus erythematosus

Tractability

SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandAB · UniProt loc med confPR · LiteraturePR · UniProt UbiquitinationPR · Database UbiquitinationPR · Half-life DataPR · Small Molecule Binder

Clinical trials

30

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

Show remaining trials (24)

UNKNOWN · via lenalidomide · NCT04025593

ClinicalTrials.gov via the drug-target graph.

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.

congenital sideroblastic anemia-B-cell immunodeficiency-periodic fever-developmental delay syndromeWell supported
0.94
agreement 0.821.00
Genetic100%

Open Targets aggregate 0.57 · 1 independent evidence family

plasma cell myelomaWell supported
0.86
agreement 0.730.99
Clinical60%Pathway29%Literature10%

Open Targets aggregate 0.68 · 3 independent evidence families

retinitis pigmentosa and erythrocytic microcytosisWell supported
0.84
agreement 0.720.97
Genetic88%Animal model13%

Open Targets aggregate 0.51 · 2 independent evidence families

autosomal recessive non-syndromic intellectual disabilityWell supported
0.80
agreement 0.660.94
Genetic96%Literature4%

Open Targets aggregate 0.60 · 2 independent evidence families

hereditary diseaseWell supported
0.77
agreement 0.650.89
Genetic100%

Open Targets aggregate 0.47 · 1 independent evidence family

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

60

Recent activity around this target, drawn from one canonical event stream. Every item is reached through 2 drugs that target this protein, so each event is news about that drug rather than about the protein directly.

  1. Label change2026-07-22

    Label change: LENALIDOMIDE (NDA021880)

    fda · regulatory · fda · via lenalidomide

  2. Industry development2026-06-29
    Advanced hierarchical ZnFe-LDH@MnO₂ nanostructures for sensitive voltammetric determination of lenalidomide

    Nature — Environmental Sciences · news · via lenalidomide

  3. Label change2026-06-15

    Label change: LENALIDOMIDE (ANDA201452)

    fda · regulatory · fda · via lenalidomide

  4. Label change2026-06-15

    Label change: LENALIDOMIDE (ANDA201452)

    fda · regulatory · fda · via lenalidomide

  5. Supplemental approval2026-04-27

    Supplemental approval: LENALIDOMIDE (ANDA201452)

    fda · regulatory · fda · via lenalidomide

  6. Supplemental approval2026-04-27

    Supplemental approval: LENALIDOMIDE (NDA021880)

    fda · regulatory · fda · via lenalidomide

  7. Supplemental approval2023-03-24

    Supplemental approval: LENALIDOMIDE (ANDA201452)

    fda · regulatory · fda · via lenalidomide

  8. Supplemental approval2023-03-24

    Supplemental approval: LENALIDOMIDE (NDA021880)

    fda · regulatory · fda · via lenalidomide

  9. Regulatory approval2023-03-06

    Approval: LENALIDOMIDE (ANDA201452)

    fda · regulatory · fda · via lenalidomide

  10. Regulatory approval2022-09-19

    Approval: Thalidomide Lipomed (EMA)

    ema · regulatory · ema · via Thalidomide

  11. Label change2022-05-24

    Label change: LENALIDOMIDE (NDA021880)

    fda · regulatory · fda · via lenalidomide

  12. Label change2021-08-31

    Label change: LENALIDOMIDE (NDA021880)

    fda · regulatory · fda · via lenalidomide

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.