miR17-92-i-PT

miR17-92-i-PT is a Locked Nucleic Acid (LNA) gapmer antisense oligonucleotide designed to target the primary transcript of the miR-17-92 cluster (pri-miR-17-92). Developed by researchers at the University Magna Graecia of Catanzaro in collaboration with Exiqon, this agent triggers RNase H-mediated degradation of the polycistronic primary transcript, leading to the simultaneous downregulation of all six mature miRNAs in the cluster (miR-17, miR-18a, miR-19a, miR-20a, miR-19b-1, and miR-92a-1). The miR-17-92 cluster is a well-known oncogenic driver often upregulated by c-Myc in multiple myeloma (MM). By inhibiting this cluster, miR17-92-i-PT impairs cell cycle progression, DNA repair, and survival in MM cells, while upregulating Hexim1 (a BRD4 antagonist) and downregulating c-Myc through a feedback loop. Preclinical studies have demonstrated anti-tumor activity in human MM xenograft models and a favorable safety profile in non-human primates.