Drug pipeline
Full profile accessExplore the programs pursuing this target and their development progress.
- Drug candidates
- Developers
- Development stage
Target intelligence / Profile preview
PiggyBac transposable element-derived protein 5 (PGBD5) is an enzymatically active DNA transposase in humans, evolutionarily derived from the piggyBac family of transposable elements.[1][3] It catalyzes precise 'cut-and-paste' DNA transposition, integrating at specific TTAA genomic sites via a mechanism dependent on its transposase domain and specific terminal repeat sequences.[1][3] PGBD5 is the most evolutionarily conserved transposase-derived gene in vertebrates, with high expression in embryonic and neural tissues, suggesting a potential physiological role in brain development.[1] In cancer biology, PGBD5 acts as an active genomic mutator: its expression in certain pediatric cancers, such as rhabdoid tumors and glioma, is linked to recurrent structural rearrangements that inactivate tumor suppressor genes, driving oncogenesis.[3] Although PGBD5 has substrate specificity and does not mobilize insect or bat piggyBac elements efficiently, it is capable of mobilizing piggyBac-like elements in the vertebrate genome, likely involving recognition of palindromic internal repeats.[2] Overexpression of PGBD5 can be cytotoxic, possibly due to excessive DNA cleavage or recombination events.[2] Despite its emerging relevance in human disease, no small molecules or clinically approved drugs are currently known to target PGBD5, and its mechanism of action as a therapeutic target remains under investigation.
Beyond the preview
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Explore the programs pursuing this target and their development progress.
Follow the clinical studies evaluating therapies directed at this target.
Compare approaches across drug candidates, modalities, and indications.
Investigate the research and source evidence behind target biology and development.
Explore patent activity around therapies and technologies addressing this target.
Connect target biology, drug development, and emerging evidence in your research.
See how Gosset can support your research on PiggyBac transposable element-derived protein 5 (PGBD5).