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Protein / target

Melanocyte protein PMEL

Encoded byPMELP40967Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
1
Approved medicines
Open Targets target-level
View by indication →
10
Clinical trials
Antibody-tractable
Druggability
UniProt loc high conf

Protein at a glance

Biological role

Identical protein binding

Strongest disease association

Autoimmune Diseases

Via encoding gene PMEL · Genetic evidence · score 0.14

Therapeutic position

Established drug target

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

Forms physiological amyloids that play a central role in melanosome morphogenesis and pigmentation.

View complete UniProt function annotation

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.

Approved medicines with mapped indications

1 medicine · 3 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.

Melanoma1 medicine
Uveal Neoplasms1 medicine
Broader indication categories (1)
Neoplasms1 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.

tebentafusp
Narrow target profileApprovedBinding agent

Melanocyte protein PMEL binding agent

Indicated for Melanoma, Uveal Neoplasms, Neoplasms

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 PMEL

Gene-level evidence surfaced through the gene PMELthat 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.

Melanoma
0.61Moderately supported

Clinical evidence dominant · Open Targets 0.47

Neoplasms
0.53Moderately supported

Clinical evidence dominant · Open Targets 0.40

Stomach Neoplasms
0.46Limited support

Clinical evidence dominant · Open Targets 0.37

Retinitis Pigmentosa
0.24Preliminary

Animal model evidence dominant · Open Targets 0.07 · no direct causal or clinical evidence

Uncombable hair syndrome
0.24Preliminary

Animal model evidence dominant · Open Targets 0.07 · no direct causal or clinical evidence

View evidence synthesis (5)
MelanomaModerately supported
0.61
agreement 0.470.74
Clinical82%Literature16%RNA expression2%

Open Targets aggregate 0.47 · 3 independent evidence families

NeoplasmsModerately supported
0.53
agreement 0.380.69
Clinical76%Literature24%

Open Targets aggregate 0.40 · 2 independent evidence families

Stomach NeoplasmsLimited 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

Uncombable hair syndromePreliminary
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 per-type scores above show the calculation.

Show all associations
Melanoma0.47
Neoplasms0.40
Stomach Neoplasms0.37
Uveal Melanoma0.12
Cutaneous melanoma0.10
Autoimmune Diseases0.09
Sarcoma, Clear Cell0.08
Retinitis Pigmentosa0.07
Uncombable hair syndrome0.07

Drug development

2 compounds recorded · 1 approved · 1 in clinical development

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 (2)
TEBENTAFUSPApproval
IMC-GP100Early Phase 1

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

AntibodiesEmerging

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

Other modalitiesStrong

Approved Drug support this modality.

View underlying tractability evidence (4)
AB · UniProt loc high confAB · GO CC high confAB · UniProt SigP or TMHMMOC · Approved Drug

Raw Open Targets tractability assessment buckets, by modality.

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. Ranked by active status, then clinical phase and recency, and spread across the targeting drugs.

ClinicalTrials.gov via the drug-target graph.

What's happening now

3

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. 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.