stapled peptide PTBP1 inhibitor

This stapled peptide PTBP1 inhibitor is an experimental therapeutic designed to target polypyrimidine tract-binding protein 1 (PTBP1), a protein frequently upregulated in glioblastoma. PTBP1 drives the alternative splicing of Annexin A7 (ANXA7), favoring the oncogenic isoform I2 over the tumor-suppressive isoform I1. By inhibiting PTBP1, the peptide restores the expression of ANXA7 isoform I1, which interacts with the endocytic machinery to promote the degradation of oncogenic receptor tyrosine kinases (RTKs), thereby inhibiting tumor cell signaling and growth. Developed by researchers at the University of Miami, UT Southwestern, and UAB, the compound is currently undergoing preclinical evaluation for the treatment of glioblastoma.