UTRxMYCN M1-14

UTRxMYCN M1-14 (also designated 3′UTRMYCN M1-14) is a novel RNA therapeutic being developed by UTR Therapeutics Inc. It utilizes the company's proprietary ultra-targeted 3′UTR engineering platform to target the oncogenic mRNA of MYCN (N-MYC), an oncogenic transcription factor historically considered 'undruggable'. The drug works by overwriting the endogenous oncogenic message of MYCN and triggering its degradation via the nonsense-mediated decay pathway, thereby eliminating diseased mRNA while sparing healthy mRNA. UTRxMYCN M1-14 is being developed for the treatment of soft tissue sarcoma, including rhabdomyosarcoma (RMS), with a particular focus on aggressive MYCN-driven and fusion-positive rhabdomyosarcoma. The U.S. FDA has granted it Orphan Drug Designation (ODD) for soft tissue sarcoma/RMS and Rare Pediatric Disease Designation (RPDD) for rhabdomyosarcoma.