Drug pipeline
Full profile accessExplore the programs pursuing this target and their development progress.
- Drug candidates
- Developers
- Development stage
Target intelligence / Profile preview
Protein VAC14 homolog (VAC14)
- Target
- VAC14
- Molecular classification
- Other (Scaffold protein), Enzyme complex component, Signaling complex
01
Overview
Protein VAC14 homolog (VAC14) is a highly conserved scaffold protein and a core component of the PIKfyve–VAC14–FIG4 complex, regulating the dynamic interconversion between phosphoinositide species in endosomal membranes. VAC14 nucleates the assembly of this complex by pentamerizing into a central structure that binds the lipid kinase PIKfyve and the lipid phosphatase FIG4, together mediating the phosphorylation of phosphatidylinositol-3-phosphate (PtdIns3P) to generate phosphatidylinositol-3,5-bisphosphate (PtdIns(3,5)P2), which is critical for endosome and lysosome maturation, vesicular trafficking, and autophagy[1][5][6]. Mutations or dysfunction in VAC14 and its associated complex can lead to profound cellular and neurological pathologies, making it a vital regulatory protein and a potential, though currently indirect, therapeutic target in diseases associated with phosphoinositide dysregulation[3][2].
- Other names
- VAC14TAX1BP2TRXArPIKfyveTax1-binding protein 2FLJ10305PIKFYVE complex componentprotein VAC14 homolog
02
Mechanism of action
Inhibition of the PIKfyve–VAC14–FIG4 complex (primarily via PIKfyve inhibitors) results in impaired synthesis of phosphatidylinositol-3,5-bisphosphate, leading to vacuolization, defective endosomal trafficking, and autophagy disruption[2][3].
03
Biological functions
Regulation of phosphoinositide metabolismEndosome and lysosome maturationMembrane traffickingAutophagy regulationVesicular transport organization
04
Disease associations
Neurodegenerative disease (e.g., Charcot–Marie–Tooth disease type 4J, Yunis–Varón syndrome, ALS)[3]Other (Cellular vacuolization, potential links to viral infections and cancer)[3]
05
Safety considerations
Disruption of VAC14 or its complex partners can lead to massive cytoplasmic vacuolization, impaired endosomal and lysosomal maturation, and autophagy defects[2][3].In animal models, knockout or mutations in VAC14 are associated with severe neurodegeneration, indicating essential roles and potential for toxicity if targeted therapeutically[3].
06
Interacting drugs
There are currently no approved drugs that target VAC14 directly; however, the PIKfyve inhibitor YM201636 is used experimentally to disrupt the VAC14 complex function in research[2].
07
Biomarkers
There are no established clinical biomarkers for patient selection or efficacy specifically tied to VAC14 at present.Altered levels of phosphatidylinositol-3,5-bisphosphate or endolysosomal/autophagy marker proteins (e.g., Rab7, CD63, LC3) may indicate complex dysfunction in research contexts[2].
Beyond the preview
Go deeper on Protein VAC14 homolog (VAC14).
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 Protein VAC14 homolog (VAC14).