Drug pipeline
Full profile accessExplore the programs pursuing this target and their development progress.
- Drug candidates
- Developers
- Development stage
Target intelligence / Profile preview
PCMT1 mRNA encodes the Protein-L-isoaspartyl methyltransferase 1 (PCMT1) enzyme, a vital protein repair methyltransferase that recognizes and repairs damaged L-isoaspartyl and D-aspartyl residues in proteins (UniProt P22061). By restoring these residues to their normal L-aspartyl form, PCMT1 maintains the structural and functional integrity of the proteome, particularly in long-lived cells like neurons (PubMed: 8632965). In many cancers, PCMT1 mRNA is significantly upregulated, where it functions as an oncogene by suppressing apoptosis and promoting cell survival under stress (PubMed: 29152785). Consequently, PCMT1 mRNA has emerged as a therapeutic target for RNA-based interventions, such as siRNA or antisense oligonucleotides, aimed at reducing its expression to inhibit tumor growth or sensitize cancer cells to existing treatments (PubMed: 23468110). However, because PCMT1 is essential for preventing neurodegeneration and epilepsy, therapeutic strategies must carefully balance the need for knockdown in tumors with the preservation of its repair function in the central nervous system.
RNA interference or antisense-mediated degradation of PCMT1 mRNA to reduce protein expression levels.
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 Protein-L-isoaspartyl methyltransferase 1 (PCMT1) mRNA (PCMT1 mRNA).