Transcriptional coactivator, Protein phosphatase (including protein tyrosine phosphatase activity), Enzyme, Developmental regulator (EYA protein family)
01
Overview
Eyes absent homolog 1 (EYA1) is a multifunctional protein with both **transcriptional coactivator** and **phosphatase** activities, encoded by the EYA1 gene. It is part of the eyes absent gene family and plays a pivotal role in developmental organogenesis, especially of the kidney, branchial arches, eye, and inner ear. EYA1 partners principally with SIX family transcription factors to regulate the expression of genes crucial for cell fate specification, proliferation, and survival during embryonic development. EYA1 acts as a protein tyrosine phosphatase that can also mediate serine/threonine phosphatase activity (likely through complex formation), with substrates including γ-H2AX (modulating DNA damage response), estrogen receptor β, and others. Mutations or dosage imbalances in EYA1 cause congenital developmental disorders such as branchiootorenal syndrome, and reactivation or overexpression—particularly in concert with SIX proteins—has been implicated in cancer progression and metastasis. As a result, EYA1 is recognized as a potential therapeutic target in oncology and genetic disease.
Experimental EYA1 inhibitors act primarily by inhibition of **protein tyrosine phosphatase** activity, purported to block the pro-tumor transcriptional and survival activities of EYA1, reducing cancer cell growth and metastasis.
03
Biological functions
Transcriptional activation (as a coactivator with SIX proteins)Protein tyrosine phosphatase activity (removes phosphate from tyrosine residues)Protein serine/threonine phosphatase activity (in context, via interactions)DNA damage repair (dephosphorylates γ-H2AX)Regulation of organogenesis (kidney, eye, ear, branchial arches, pharyngeal glands)Regulation of cell proliferation and survivalNeurogenesis of placodal derivatives
04
Disease associations
Cancer (oncogenesis, particularly breast and other solid tumors through overexpression and interaction with SIX1)Developmental disorders (Branchiootorenal syndrome, Branchiootic syndrome, congenital cataracts, ocular anterior segment anomalies, branchio-oto-renal spectrum disorders)Other: Sensory deficits, morphogenetic defects in kidney, ear, and facial tissues
05
Safety considerations
Potential effects on normal organogenesis and tissue function (since EYA1 is essential for developmental processes and cell survival, especially in kidney and ear tissues)Off-target effects of phosphatase inhibitors (risk for unintended effects on DNA repair and normal proliferative tissues)Dose sensitivity (EYA1 dosage reduction linked to severe syndromic outcomes in development)
06
Interacting drugs
No approved, marketed drugs currently target EYA1 directly. Some experimental small-molecule inhibitors (e.g., targeting EYA tyrosine phosphatase activity) have been developed for research and preclinical studies in cancer models, but none are currently in clinical use
07
Biomarkers
EYA1 overexpression (as a **biomarker** for certain cancers and for identifying tumors likely to benefit from EYA-targeted therapies in preclinical research)Genetic mutations in EYA1 (as diagnostic markers in BOR/BO syndrome and related developmental disorders)
Beyond the preview
Go deeper on Eyes absent homolog 1 (EYA1).
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 Eyes absent homolog 1 (EYA1).