UTR Therapeutics Inc, a New York-based biotech company, announced on Tuesday that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to 3'UTRMYCN M1-14 (UTRxMYCN M1-14) for the treatment of soft tissue sarcoma, including rhabdomyosarcoma (RMS), and Rare Pediatric Disease Designation (RPDD) for the treatment of rhabdomyosarcoma.

UTR's core technology of ultra-targeted 3'UTR engineering, invented by the company's CEO, Chidiebere U. Awah, M.D., Ph.D., targets the oncogenic mRNA of undruggable genes, overwrites their endogenous oncogenic mRNA messages, and triggers them for degradation by the nonsense-mediated decay pathway, thus eliminating the diseased mRNA while sparing the healthy mRNA.

In preclinical studies, UTRxMYCN M1-14 targeted and degraded the oncogenic MYCN in aggressive MYCN-driven rhabdomyosarcoma, including the deadly fusion-positive rhabdomyosarcoma. The company says that the drug achieved on-target specificity and therapeutic efficacy, demonstrating dose-dependent inhibition of MYCN, tumour inhibition, and inhibition of liver and lung metastasis while remaining safe.