Drug pipeline
Full profile accessExplore the programs pursuing this target and their development progress.
- Drug candidates
- Developers
- Development stage
Target intelligence / Profile preview
Phosphofructokinase, muscle (PFKM) mRNA is the transcript encoding the muscle-type isoform of the rate-limiting enzyme in glycolysis. This mRNA is essential for the production of the PFKM protein, which catalyzes the phosphorylation of fructose-6-phosphate to fructose-1,6-bisphosphate, a key regulatory step in the glycolytic pathway (NCBI Gene ID: 5213). In humans, PFKM is primarily expressed in skeletal muscle and red blood cells. Deficiencies in PFKM, often due to genetic mutations in the PFKM gene, result in Glycogen Storage Disease Type VII (Tarui disease), characterized by exercise intolerance, muscle breakdown, and hemolytic anemia (UniProt P08237). Conversely, PFKM mRNA is frequently upregulated in various cancers to support the increased metabolic demands of rapidly proliferating cells, a phenomenon known as the Warburg effect (PubMed: 25242331). Consequently, PFKM mRNA has emerged as a potential therapeutic target for RNA-based interventions, such as antisense oligonucleotides or small interfering RNAs, aimed at metabolic reprogramming in oncology.
RNA interference-mediated degradation of PFKM mRNA and antisense-mediated inhibition of translation.
1 more in the full profile.
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 Phosphofructokinase, muscle (PFKM) mRNA (PFKM mRNA).