Target intelligence / Profile preview

Wilms' tumor protein 1 (WT1) (WT1)

Target
WT1
Molecular classification
Transcription factor, Zinc finger protein, Tumor-associated antigen
01

Overview

Wilms' tumor protein 1 (WT1) is a zinc-finger transcription factor that plays a critical role in the embryonic development of the urogenital system (UniProt P19544). While its expression is highly restricted in healthy adult tissues, it is significantly overexpressed in a wide range of hematological malignancies and solid tumors, leading the National Cancer Institute to rank it as a top priority cancer antigen (Cheever et al., 2009). The therapeutic strategy involves the processing of intracellular WT1 protein into peptides, which are then presented on the cell surface by Major Histocompatibility Complex (MHC) Class I and II molecules. This presentation allows for the specific recognition and elimination of tumor cells by CD8+ cytotoxic T-cells and the activation of CD4+ helper T-cells to sustain the immune response (PMID: 26154453). Current clinical developments include peptide-based vaccines like Galinpepimut-S and TCR-engineered T-cell therapies designed to target these specific WT1 peptide-MHC complexes (PMID: 19584355).

Other names
Wilms tumor proteinWT33GUDAWT1WAGRAmino-terminal truncated WT1
02

Mechanism of action

Induction of cytotoxic and helper T-cell responses through the recognition of WT1-derived peptides presented on MHC Class I and II molecules.

03

Biological functions

Transcription regulationCell differentiationUrogenital developmentApoptosis regulationImmune response
04

Disease associations

CancerLeukemiaWilms tumorMesotheliomaOvarian cancerGlioblastoma
05

Safety considerations

Potential renal toxicity due to low-level WT1 expression in podocytesHematological suppression (myelotoxicity) affecting hematopoietic stem cellsCytokine release syndrome (CRS) associated with TCR-T cell therapiesOff-target recognition of normal tissues expressing WT1
06

Interacting drugs

Galinpepimut-S (GPS)

3 more in the full profile.

07

Biomarkers

WT1 mRNA expressionWT1 protein expressionHLA-A*02:01 statusHLA-A*24:02 statusHLA-DRB1 status

Beyond the preview

Go deeper on Wilms' tumor protein 1 (WT1) (WT1).

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Drug pipeline

Full profile access

Explore the programs pursuing this target and their development progress.

  • Drug candidates
  • Developers
  • Development stage

Clinical trials

Full profile access

Follow the clinical studies evaluating therapies directed at this target.

  • Trial design
  • Status
  • Readouts

Competitive landscape

Full profile access

Compare approaches across drug candidates, modalities, and indications.

  • Programs
  • Modalities
  • Indications

Literature & evidence

Full profile access

Investigate the research and source evidence behind target biology and development.

  • Publications
  • Sources
  • Analysis

Patents

Full profile access

Explore patent activity around therapies and technologies addressing this target.

  • Patents
  • Assignees
  • Technologies

Research & analysis

Full profile access

Connect target biology, drug development, and emerging evidence in your research.

  • Biology
  • Development news
  • Analysis

Bring the full picture into focus.

See how Gosset can support your research on Wilms' tumor protein 1 (WT1) (WT1).

Explore the full profile

Gosset Free

Get started with Gosset.

Enter your work email and we’ll be in touch with next steps.

Work email preferred.

Book a call