Protein / target

Melanocyte protein PMEL

PMELP40967Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
1
Approved medicines
10
Clinical trials
Antibody-tractable
Druggability
UniProt loc high conf

Protein at a glance

Biological role

Identical protein binding

Strongest disease association

autoimmune disease

Genetic evidence · score 0.14

Therapeutic maturity

Clinically validated target

1 approved medicine against this target

Druggability

Antibody

Open Targets tractability · UniProt loc high conf

Clinical development

1 approved · 1 in clinical development

10 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

Forms physiological amyloids that play a central role in melanosome morphogenesis and pigmentation. The maturation of unpigmented premelanosomes from stage I to II is marked by assembly of processed amyloidogenic fragments into parallel fibrillar sheets, which elongate the vesicle into a striated ellipsoidal shape. In pigmented stage III and IV melanosomes, the amyloid matrix serves as a platform where eumelanin precursors accumulate at high local concentrations for pigment formation. May prevent pigmentation-associated toxicity by sequestering toxic reaction intermediates of eumelanin biosynthesis pathway

Subcellular location

Endoplasmic reticulum membraneGolgi apparatus, cis-Golgi network membraneEndosome, multivesicular bodyMelanosomeExtracellular vesicleSecreted
Domains and Gene Ontology detail (15)

Domains & features

PKD

Gene Ontology

  • Ccis-Golgi network membrane
  • Cendoplasmic reticulum
  • Cendoplasmic reticulum membrane
  • Cextracellular exosome
  • CGolgi apparatus
  • Cmelanosome
  • Cmelanosome membrane
  • Cmultivesicular body membrane
  • Cmultivesicular body, internal vesicle
  • Cplasma membrane
  • Fidentical protein binding
  • Pmelanin biosynthetic process

661 aa · 70 kDa · 5 isoforms

Biological roles

Reactome v97

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

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.

MLANATYRTYRP1DCTCD8AHLA-AMAGEA3CTAG1BCD63GPR143PMEL

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.

tebentafusp
Narrow target profileApprovedBinding agent

Melanocyte protein PMEL binding agent

Appears in clinical studies involving gastric cancer, uveal cancer, neoplasm, melanoma

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.

autoimmune disease0.14

Genetic · overall 0.09

Highest-confidence therapeutic associations

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

uveal cancer0.76

Clinical · overall 0.46

melanoma0.74

Clinical · overall 0.47

neoplasm0.61

Clinical · overall 0.40

gastric cancer0.61

Clinical · overall 0.37

uveal melanoma0.19

Clinical · overall 0.12

Highest overall evidence

Remaining associations by Open Targets' aggregated evidence score.

cutaneous melanoma0.10

Literature

clear cell sarcoma0.08

Clinical

retinitis pigmentosa0.07

Animal model

uncombable hair syndrome0.07

Animal model

Show all associations
melanoma0.47
uveal cancer0.46
neoplasm0.40
gastric cancer0.37
uveal melanoma0.12
cutaneous melanoma0.10
autoimmune disease0.09
clear cell sarcoma0.08
retinitis pigmentosa0.07
uncombable hair syndrome0.07

Open Targets ranks 851 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 · 2 total

TEBENTAFUSPApproval

gastric cancer · uveal cancer · neoplasm

IMC-GP100Early Phase 1

melanoma

Tractability

AB · UniProt loc high confAB · GO CC high confAB · UniProt SigP or TMHMMOC · Approved Drug

Clinical trials

10

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.

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.

melanomaModerately supported
0.61
agreement 0.470.74
Clinical82%Literature16%RNA expression2%

Open Targets aggregate 0.47 · 3 independent evidence families

uveal cancerModerately supported
0.57
agreement 0.410.73
Clinical100%

Open Targets aggregate 0.46 · 1 independent evidence family

neoplasmModerately supported
0.53
agreement 0.380.69
Clinical76%Literature24%

Open Targets aggregate 0.40 · 2 independent evidence families

gastric cancerLimited support
0.46
agreement 0.300.61
Clinical99%Literature1%

Open Targets aggregate 0.37 · 2 independent evidence families

retinitis pigmentosaPreliminary
0.24
agreement 0.020.47
Animal model100%

Open Targets aggregate 0.07 · 1 independent evidence family · no direct causal or clinical evidence

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 publication2023-10-21
    Three-Year Overall Survival with Tebentafusp in Metastatic Uveal Melanoma.

    The New England journal of medicine · 2023 · 190 citations · Europe PMC · via tebentafusp

  2. Regulatory approval2022-04-01

    Approval: Kimmtrak (EMA)

    ema · regulatory · ema · via tebentafusp

  3. New publication2021-09-01
    Overall Survival Benefit with Tebentafusp in Metastatic Uveal Melanoma.

    The New England journal of medicine · 2021 · 685 citations · Europe PMC · via tebentafusp

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.