Drug pipeline
Full profile accessExplore the programs pursuing this target and their development progress.
- Drug candidates
- Developers
- Development stage
Target intelligence / Profile preview
ASXL transcriptional regulator 2 (ASXL2)
- Target
- ASXL2
- Molecular classification
- Epigenetic regulator, Chromatin-binding protein, Transcriptional regulator, Histone modification factors
01
Overview
ASXL transcriptional regulator 2 (ASXL2) is a chromatin-binding protein involved in epigenetic regulation, acting as a scaffold that assembles and modulates the activity of histone-modifying complexes. ASXL2 regulates key developmental, metabolic, and cellular pathways via transcriptional repression (via Polycomb group complexes) and activation (via interactions with nuclear hormone receptors and BAP1). It is essential for normal hematopoietic stem cell renewal and differentiation and acts as a context-dependent tumor suppressor in various malignancies, particularly in AML with the RUNX1-RUNX1T1 fusion. ASXL2 mutation or altered expression is implicated in several cancers, metabolic pathways, and rare genetic syndromes
- Other names
- ASXH2SHAPNSadditional sex combs like 2mKIAA1685ASXL transcriptional regulator 2
02
Mechanism of action
Modulation of epigenetic marks (e.g., via interaction with BAP1 for histone H2A deubiquitination); Enhancement or repression of transcription factor binding and chromatin remodeling
03
Biological functions
Regulation of chromatin accessibility and gene expression (via histone modification)Stem cell self-renewal (hematopoiesis)Adipocyte differentiationTranscriptional regulationProtein-protein scaffolding for complexes involved in histone deubiquitination and methylation
04
Disease associations
Cancer (e.g., myeloid leukemia, breast, bladder, pancreas, ovarian, prostate, blood)Shashi-Pena SyndromeAutism Spectrum DisorderMyelodysplastic syndrome-like disorders
05
Safety considerations
Tumor heterogeneity and context-dependent effects (ASXL2 can be tumor-promoting or suppressing in different cancers)Lack of direct targetable catalytic activity—may complicate therapeutic approachesPossible off-target epigenetic effects if modulated pharmacologically
06
Biomarkers
ASXL2 mutation status and expression patterns as prognostic biomarkers in acute myeloid leukemia (AML, especially t(8;21))ASXL2 mutation status and expression patterns as prognostic biomarkers in colorectal cancerASXL2 mutation status and expression patterns as prognostic biomarkers in other cancers
Beyond the preview
Go deeper on ASXL transcriptional regulator 2 (ASXL2).
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Clinical trials
Full profile accessFollow the clinical studies evaluating therapies directed at this target.
- Trial design
- Status
- Readouts
Competitive landscape
Full profile accessCompare approaches across drug candidates, modalities, and indications.
- Programs
- Modalities
- Indications
Literature & evidence
Full profile accessInvestigate the research and source evidence behind target biology and development.
- Publications
- Sources
- Analysis
Patents
Full profile accessExplore patent activity around therapies and technologies addressing this target.
- Patents
- Assignees
- Technologies
Research & analysis
Full profile accessConnect 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 ASXL transcriptional regulator 2 (ASXL2).