Drug pipeline
Full profile accessExplore the programs pursuing this target and their development progress.
- Drug candidates
- Developers
- Development stage
Target intelligence / Profile preview
Polymerase I and transcript release factor (PTRF), also known as Cavin-1, is a multifunctional protein essential for the biogenesis and structural integrity of caveolae, which are specialized invaginations of the plasma membrane (UniProt Q6NZI2; Hill et al., 2008, Cell). It functions by stabilizing caveolin-1 and organizing the caveolar coat, thereby playing a central role in lipid homeostasis, signal transduction, and mechanoprotection (Hayashi et al., 2009, Nature Genetics). Beyond its membrane-associated functions, PTRF is involved in the termination of RNA polymerase I transcription by facilitating the dissociation of the transcription complex from DNA (Jansa et al., 1998, EMBO J). Mutations in the PTRF gene are clinically linked to congenital generalized lipodystrophy type 4 (CGL4) and associated muscular dystrophies, highlighting its importance in metabolic and mechanical stability (Shastry et al., 2010, Am J Med Genet). In oncology, PTRF acts as a context-dependent modulator; it is frequently downregulated in prostate cancer, where it serves as a tumor suppressor, but upregulated in glioblastoma and pancreatic cancer, where it promotes tumor progression and chemoresistance (Nassar et al., 2013, Oncotarget; Huang et al., 2018, Cell Death & Disease). While PTRF is a significant target for research into membrane dynamics and disease mechanisms, there are currently no FDA-approved drugs that specifically target this protein (ClinicalTrials.gov).
Modulation of caveolae-mediated signaling and RNA polymerase I transcription termination
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 Polymerase I and transcript release factor (PTRF) (PTRF).